[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"neuroblastoma\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:neuroblastoma":28},{"pageToken":4,"total":5,"offset":6,"count":7,"results":8},null,85,0,25,[9,42,69,93,126,156,186,211,238,262,290,315,341,369,388,415,441,463,499,521,543,575,601,674,695],{"id":10,"slug":11,"hasResults":12,"nctId":13,"briefTitle":14,"officialTitle":15,"acronym":4,"eligibilityCriteria":16,"healthyVolunteers":12,"sex":17,"minAge":18,"maxAge":19,"enrollmentInfo":20,"targetDuration":4,"studyType":23,"phases":24,"briefSummary":26,"conditions":27,"keywords":4,"overallStatus":29,"whyStopped":4,"lastUpdateSubmitDate":30,"lastUpdatePostDateStruct":31,"startDateStruct":34,"completionDateStruct":36,"leadSponsor":38,"locationsCount":41},"100613027","phase-2-nant-2021-02-randomized-mibg-with-vorinostatdinutuximabvorinostat--dinutuximab-100613027",false,"NCT07261241","NANT 2021-02: Randomized MIBG With Vorinostat\u002FDinutuximab\u002FVorinostat + Dinutuximab","NANT 2021-02: A Randomized Phase 2 Study of 131I-MIBG With Vorinostat VS. 131I-MIBG With Dinutuximab vs. 131I-MIBG With Dinutuximab and Vorinostat for Relapsed or Refractory Neuroblastoma","Inclusion Criteria\n\nAge Patients must be ≥ 1 year and ≤31 years of age at the time of enrollment on the study.\n\nDiagnosis Patients must have a diagnosis of neuroblastoma by histologic verification of neuroblastoma and\u002For demonstration of tumor cells in the bone marrow with increased urinary catecholamines.\n\nDisease Risk Group Patients must have high risk neuroblastoma according to COG risk classification at the time of study registration. Patients whose disease was initially considered low or intermediate risk but then reclassified as high risk neuroblastoma prior to enrollment also meet this criteria.\n\nResponse to Prior Therapy (using INRC definitions)\n\nPatients must have at least ONE of the following:\n\nRecurrent\u002Fprogressive disease after the diagnosis of high-risk neuroblastoma at any time prior to enrollment - regardless of response to frontline therapy. (Note that this excludes patients initially considered low or intermediate-risk that progressed to high-risk disease but have not progressed after the diagnosis of high-risk neuroblastoma).\n\nIf no prior history of recurrent\u002Fprogressive disease since the diagnosis of high-risk neuroblastoma,\n\nRefractory disease: A best overall response of no response\u002Fstable disease since diagnosis of high-risk neuroblastoma AND after at least 4 courses of induction therapy.\n\nPersistent disease: A best overall response of partial response since diagnosis of high-risk neuroblastoma AND after at least 4 courses of induction therapy:\n\ni. If a patient with persistent disease has 3 or more MIBG avid sites (including all soft tissue and\u002For bone lesions) OR a Curie Score of ≥ 3, then no biopsy is required for eligibility.\n\nii. If a patient with persistent disease has only 1 or 2 MIBG avid sites (including all soft tissue and\u002For bone lesions) then biopsy confirmation of neuroblastoma and\u002For ganglioneuroblastoma in at least one MIBG avid site (bone marrow, bone, or soft tissue) present at the time of registration is required. Bone and\u002For soft tissue lesions may be biopsied at any time point prior to study registration; bone marrow must be done at the time of study registration.\n\nSites of Disease: MIBG Uptake Patients must have evidence of MIBG uptake by planar imaging into tumor at ≥ 1 site (bone or soft tissue) within 21 days prior to study entry and subsequent to any intervening therapy. See exclusion criteria.\n\nAutologous peripheral blood stem cells (PBSC)\n\n* The minimum dose for peripheral blood stem cells is 1.5 x 10\\^6 viable CD34+ cells\u002Fkg. Patients who do not meet this minimum requirement for available PBSCs are not eligible.\n* For patients whose body weight exceeds ideal body weight (IBW) by more than 20%, adjusted body weight may be used for the calculation of PBSC dose.\n\nPerformance Status Patients must have a Lansky (≤ 16 years) or Karnofsky (\\> 16 years) score of ≥ 50.\n\nNote: Patients who are unable to walk because of paralysis, but who are up in a wheelchair, will be considered ambulatory for the purpose of assessing the performance score.\n\nPrior Therapy\n\nPatients must have fully recovered from the acute toxic effects and beyond the washout period of all prior chemotherapy, immunotherapy (monoclonal antibodies \\[MOAB\\], bi-\u002Ftri-specific antibody T-cell engagers, and cellular therapy) or radiotherapy prior to study registration.\n\nOrgan Function Requirements\n\nHematologic Function:\n\nPatients must meet the following hematologic criteria for enrollment regardless of bone marrow disease involvement:\n\n1. ANC ≥750\u002FuL (no short-acting granulocyte growth factors ≤ 7 days of blood draw documenting eligibility and no long-acting granulocyte growth factors ≤ 14 days of blood draw documenting eligibility); and\n2. Platelet count ≥ 50,0000\u002Fµl, transfusion independent (no platelet transfusions or platelet growth factors ≤ 7 days of blood draw documenting eligibility) Renal Function\n\na. Patients must have adequate renal function defined as age-adjusted serum creatinine ≤1.5 ULN for age:\n\nLiver Function\n\n1. Total bilirubin ≤ 1.5 x ULN for age; and,\n2. SGPT (ALT) ≤ 135 U\u002FL (≤ 3x ULN). Note that for ALT, the upper limit of normal for all sites is defined as 45 U\u002FL.\n\nCentral Nervous System (CNS) Function:\n\n1. Patients with a history of intraparenchymal or leptomeningeal based CNS disease must have no clinical or radiological evidence of active CNS disease at the time of study enrollment.\n2. Patients with skull-based tumors with direct intracranial extension are eligible as long as there are no neurologic signs or symptoms related to the lesion.\n\nCardiac Function\n\n1. Normal ejection fraction (≥ 55%) documented by either echocardiogram or radionuclide MUGA evaluation OR normal fractional shortening (≥ 27%) documented by echocardiogram.\n2. Corrected QT (QTcF) interval ≤ 480 msec.\n\nPulmonary Function No evidence of dyspnea at rest, no exercise intolerance, or oxygen requirement.\n\nReproductive Function\n\n1. All females of childbearing potential (female patients 10 and older without documented ovarian failure) must have a negative serum or urine beta-HCG ≤ 7 days prior to registration.\n2. Male and female subjects of reproductive age and childbearing potential must agree to use two acceptable methods of birth control (i.e., intra-uterine device, hormonal contraception, diaphragm with spermicide, condom with spermicide, or abstinence) or to abstain from heterosexual intercourse for the duration of their participation in the study, or for 3 months after last dose of protocol therapy, whichever is longer.\n\nExclusion Criteria\n\nPregnancy, breast feeding, or unwillingness to use effective contraception during the study will not be entered on this study due to risks of fetal and teratogenic adverse events.\n\nPatients who, in the opinion of the investigator, may not be able to comply with the safety monitoring or radiation isolation requirements of the study.\n\nPatients with disease of any major organ system that would compromise their ability to withstand therapy.\n\nPatients must not have received prior allogeneic stem cell transplant.\n\nPatients who have received prior solid organ transplantation.\n\nPatients must not have received prior total body irradiation.\n\nPatients who are on hemodialysis.\n\nPatients with an active or uncontrolled infection. Patients on prolonged antifungal therapy are still eligible if they are culture negative, afebrile, and meet other organ function criteria.\n\nKnown history of active human immunodeficiency virus (HIV) infection, hepatitis B, or hepatitis C. Testing is not required in the absence of clinical findings or suspicion.\n\nPatients with a history of having to permanently discontinue anti-GD2 antibody therapy, GM-CSF, or vorinostat due to toxicity are not eligible.\n\nPatients who have received prior MIBG in combination with anti-GD2 monoclonal antibody and\u002For histone deacetylase inhibitor\n\nThe maximum total allowable dose of 131I-MIBG that can be given per institutional guidelines must be at least 90% of the calculated or protocol maximum 131I-MIBG dose or the patient is not eligible.\n\nPatients with a history of deep venous thrombosis that was not associated with the presence of a central venous catheter.\n\nOnly for institutions that have not been granted a waiver by NANT:\n\n1. Patient declines participation in NANT 2004-05, Biology Study\n2. Patient declines participation in NANT 2023-03, MIGHTY Study\n\nPatients with evidence of active MIBG non-avid disease; patients with previously treated and stable disease that is not MIBG avid are still eligible.\n\nPatients whose best response post previous MIBG therapy was progressive disease.\n\nPatients with a cumulative lifetime dose of 131I-MIBG greater than 20 mCi\u002Fkg.","ALL","1 Year","30 Years",{"count":21,"type":22},118,"ESTIMATED","INTERVENTIONAL",[25],"PHASE2","Patients will then be randomized at study entry to one of three treatment arms. Patients on Arm A will receive a single treatment course with 131I-MIBG with vorinostat. Patients on Arm B will receive a single treatment course with 131I-MIBG and dinutuximab. Patients on Arm C will receive a single treatment course with 131I-MIBG with dinutuximab + vorinostat. After this course of treatment, we will check to see your response and then check to see how you are doing over time. All patients may choose to proceed to a second course of the same treatment if they and their physician feel healthy enough to do so. Approximately 118 patients will be receiving therapy on this trial.",[28],"Neuroblastoma","NOT_YET_RECRUITING","2026-08-19",{"date":32,"type":33},"2026-08-21","ACTUAL",{"date":35,"type":22},"2026-12-31",{"date":37,"type":22},"2031-12-31",{"name":39,"class":40},"New Approaches to Neuroblastoma Therapy Consortium","OTHER",13,{"id":43,"slug":44,"hasResults":12,"nctId":45,"briefTitle":46,"officialTitle":47,"acronym":4,"eligibilityCriteria":48,"healthyVolunteers":12,"sex":17,"minAge":49,"maxAge":4,"enrollmentInfo":50,"targetDuration":4,"studyType":23,"phases":52,"briefSummary":54,"conditions":55,"keywords":57,"overallStatus":59,"whyStopped":4,"lastUpdateSubmitDate":30,"lastUpdatePostDateStruct":60,"startDateStruct":61,"completionDateStruct":63,"leadSponsor":65,"locationsCount":68},"100493142","phase-1-apg-115-alone-or-in-combination-with-apg-2575-in-children-with-recurrent-or-refractory-neuroblastoma-or-solid-tumors-100493142","NCT05701306","APG-115 Alone or in Combination With APG-2575 in Recurrent or Refractory Neuroblastoma or Solid Tumors","A Phase Ib-ⅡClinical Study of APG-115 Alone or in Combination With APG-2575 in Recurrent or Refractory Neuroblastoma or Solid Tumors","Inclusion Criteria:\n\n1. Age ≥5 years for APG-115 monotherapy; age ≥12 years for APG-115 + APG-2575 combination.\n2. Relapsed or refractory neuroblastoma or solid tumors with no available or suitable standard therapy.\n3. Physical state score ≥ 50.\n4. Expected survival ≥ 3 months.\n5. There are target lesions (neuroblastoma) or measurable lesions (other solid tumors).\n6. Have adequate organ function.\n7. Fresh or archived tumor tissue samples should be provided prior to treatment. If none of these specimens are available, inclusion may be made after consultation with the sponsor.\n8. Fertile women (≥14 years of age or having menarche) must have a negative serum pregnancy test at the time of the screening visit and must not be breastfeeding or planning to become pregnant during the study period.\n9. A potentially fertile male subject (who has spermatoses) or female subject (ibid.) must agree to use effective contraception during the trial period and for 3 months after the trial ends (or is prematurely discontinued).\n10. Informed consent must be obtained before carrying out any study procedure specified in the test. For child subjects, the consent of the subject and one of the parent\u002Flegal guardian must be obtained.\n11. The ability to swallow research drugs.\n\nExclusion Criteria:\n\n1. Received biological therapy, chemotherapy, or other investigational drugs within 21 days prior to dosing.\n2. Received radiotherapy, minor surgery\u002Fminimally-invasive surgery; small-molecule targeted agents, short-half-life chemotherapeutic agents, or traditional Chinese medicines with anti-tumor indications within 14 days prior to study drug administration (or within 5 half-lives, whichever is shorter).\n3. Patients who, according to the investigators' judgment, did not recover sufficiently after surgical treatment. Patients who underwent major surgery within 21 days before receiving the study drug for the first time.\n4. Adverse events due to previous antitumor therapy (except grade 2 peripheral neurotoxicity and alopecia that the investigators judged to be of no safety risk) have not recovered (severity higher than grade 1 according to CTCAE version 5.0).\n5. Patients with active brain tumors or brain metastases.\n6. Active gastrointestinal diseases (e.g. Crohn's disease, ulcerative colitis, or short bowel syndrome) or other malabsorption syndromes that may affect drug absorption.\n7. A known hemorrhagic predisposition\u002Fdisease, such as a history of non-chemotherapy-induced thrombocytopenic bleeding within 1 year before first receiving the study drug; Have active immune thrombocytopenic purpura (ITP), active autoimmune hemolytic anemia (AIHA), or a history of platelet transfusion failure (within 1 year before first receiving the study drug); Severe gastrointestinal bleeding occurred within 3 months.\n8. Clinically significant cardiovascular disease, cardiomyopathy, myocardial infarction or history within 6 months prior to administration.\n9. Symptomatic active fungal, bacterial, and\u002For viral infections requiring systemic treatment.\n10. Unexplained fever \\> 38.5℃ within 2 weeks prior to initial administration (subjects with tumor-related fever, as determined by the investigator, could be enrolled).\n11. Received MDM2 inhibitors or BCL-2 inhibitors.\n12. Any other circumstances or conditions that the investigator considers the patient inappropriate for participation in the study.","5 Years",{"count":51,"type":22},100,[53,25],"PHASE1","An open, non-randomized Phase Ib-II trial of dose-escalation and cohorts expansion to evaluate the safety, pharmacokinetic profile and initial efficacy of APG-115 alone or in combination with APG-2575 in the treatment of recurrent or refractory neuroblastoma or solid tumors.",[28,56],"Solid Tumor",[28,56,58],"APG-115","RECRUITING",{"date":32,"type":33},{"date":62,"type":33},"2023-02-28",{"date":64,"type":22},"2027-12-31",{"name":66,"class":67},"Ascentage Pharma Group Inc.","INDUSTRY",3,{"id":70,"slug":71,"hasResults":12,"nctId":72,"briefTitle":73,"officialTitle":74,"acronym":4,"eligibilityCriteria":75,"healthyVolunteers":12,"sex":17,"minAge":4,"maxAge":19,"enrollmentInfo":76,"targetDuration":4,"studyType":23,"phases":78,"briefSummary":80,"conditions":81,"keywords":4,"overallStatus":59,"whyStopped":4,"lastUpdateSubmitDate":83,"lastUpdatePostDateStruct":84,"startDateStruct":85,"completionDateStruct":87,"leadSponsor":89,"locationsCount":92},"100529324","dinutuximab-with-chemotherapy-surgery-and-stem-cell-transplantation-for-the-treatment-of-children-with-newly-diagnosed-high-risk-neuroblastoma-100529324","NCT06172296","Dinutuximab With Chemotherapy, Surgery and Stem Cell Transplantation for the Treatment of Children With Newly Diagnosed High Risk Neuroblastoma","A Phase 3 Study of Dinutuximab Added to Intensive Multimodal Therapy for Children With Newly Diagnosed High-Risk Neuroblastoma","Inclusion Criteria:\n\n* Patients must be enrolled on APEC14B1 and have consented to testing through the Molecular Characterization Initiative (MCI), prior to enrollment on ANBL2131\n* ≤ 30 years at the time of initial diagnosis with high-risk disease\n* \\* Must have a diagnosis of neuroblastoma (NBL) or ganglioneuroblastoma (nodular) verified by tumor pathology analysis or demonstration of clumps of tumor cells in bone marrow with elevated urinary catecholamines\n\n  * Newly diagnosed, high risk neuroblastoma (HRNBL) defined as one of the following:\n\n    * Any age with International Neuroblastoma Risk Group (INRG) Stage L2, MS, or M and MYCN amplification\n    * Age ≥ 547 days and INRG stage M regardless of biologic features (clinical MYCN testing not required prior to enrollment)\n    * Any age initially diagnosed with INRG Stage L1 MYCN amplified NBL who have progressed to stage M without systemic chemotherapy\n    * Age ≥ 547 days of age initially diagnosed with INRG Stage L1, L2, or MS who have progressed to stage M without systemic chemotherapy (clinical MYCN testing not required prior to enrollment)\n* Patients must have a body surface area (BSA) ≥ 0.25 m\\^2\n* No prior anti-cancer therapy except as outlined below:\n\n  * Patients initially recognized to have high-risk disease treated with topotecan\u002Fcyclophosphamide initiated on an emergent basis and within allowed timing, and with consent\n  * Patients observed or treated with a single cycle of chemotherapy per a low or intermediate risk neuroblastoma regimen (e.g., as per ANBL0531, ANBL1232 or similar) for what initially appeared to be non-high-risk disease but subsequently found to meet the criteria\n  * Patients who received localized emergency radiation to sites of life threatening or function-threatening disease prior to or immediately after establishment of the definitive diagnosis\n* Human immunodeficiency virus (HIV) -infected patients on effective anti-retroviral therapy with undetectable viral load within 6 months are eligible for this trial\n* A serum creatinine based on age\u002Fsex as follows:\n\n  * 1 month to \\\u003C 6 months: Male 0.4 mg\u002FdL and female 0.4mg\u002FdL\n  * 6 months to \\\u003C 1 year: Male 0.5 mg\u002FdL and female 0.5 mg\u002FdL\n  * 1 to \\\u003C 2 years: Male 0.6 mg\u002FdL and female 0.6 mg\u002FdL\n  * 2 to \\\u003C 6 years: Male 0.8 mg\u002FdL and female 0.8 mg\u002FdL\n  * 6 to \\\u003C 10 years: Male 1 mg\u002FdL and female 1 mg\u002FdL\n  * 10 to \\\u003C 13 years: Male 1.2 mg\u002FdL and female 1.2 mg\u002FdL\n  * 13 to \\\u003C 16 years: Male 1.5 mg\u002FdL and female 1.4 mg\u002FdL\n  * ≥ 16 years: Male 1.7 mg\u002FdL and female 1.4 mg\u002FdL\n\n    * The threshold creatinine values were derived from the Schwartz formula for estimating glomerular filtration rate (GFR) utilizing child length and stature data published by the Centers for Disease Control (CDC)\n  * or a 24-hour urine creatinine clearance ≥ 70 mL\u002Fmin\u002F1.73 m\\^2 or\n  * or a GFR ≥ 70 mL\u002Fmin\u002F1.73 m\\^2. GFR must be performed using direct measurement with a nuclear blood sampling method or direct small molecule clearance method (iothalamate or other molecule per institutional standard)\n\n    * Note: Estimated GFR (eGFR) from serum creatinine, cystatin C or other estimates are not acceptable for determining eligibility\n* Total bilirubin ≤ 1.5 x upper limit of normal (ULN) for age\n* Serum glutamic pyruvic transaminase (SGPT) (Alanine aminotransferase \\[ALT\\]) ≤ 10 x ULN\\*\n\n  * Note: For the purpose of this study, the ULN for SGPT (ALT) has been set to the value of 45 U\u002FL\n* \\* Shortening fraction of ≥ 27% by echocardiogram, or\n\n  * Ejection fraction of ≥ 50% by echocardiogram or radionuclide angiogram\n* Ability to tolerate Peripheral Blood Stem Cell (PBSC) collection:\n\nNo known contraindication to PBSC collection. Examples of contraindications might be a weight or size less than the collecting institution finds feasible, or a physical condition that would limit the ability of the child to undergo apheresis catheter placement (if necessary) and\u002For the apheresis procedure\n\nExclusion Criteria:\n\n* Patients who are 365-546 days of age with INRG Stage M and MYCN non-amplified NBL, irrespective of additional biologic features\n* Patients ≥ 547 days of age with INRG Stage L2, MYCN non-amplified NBL, regardless of additional biologic features\n* Patients with known bone marrow failure syndromes\n* Patients on chronic immunosuppressive medications (e.g., tacrolimus, cyclosporine, corticosteroids) for reasons other than prevention\u002Ftreatment of allergic reactions and adrenal replacement therapy are not eligible. Topical and inhaled corticosteroids are acceptable\n* Patients with a primary immunodeficiency syndrome who require ongoing immune globulin replacement therapy\n* Female patients who are pregnant since fetal toxicities and teratogenic effects have been noted for several of the study drugs. A pregnancy test is required prior to enrollment for female patients of childbearing potential\n* Lactating females who plan to breastfeed their infants\n* Sexually active patients of reproductive potential who have not agreed to use an effective contraceptive method for the duration of their study participation\n* All patients and\u002For their parents or legal guardians must sign a written informed consent\n* All institutional, food and drug administration (FDA), and National Cancer Institute (NCI) requirements for human studies must be met",{"count":77,"type":22},478,[79],"PHASE3","This phase III trial tests how well the addition of dinutuximab to Induction chemotherapy along with standard of care surgical resection of the primary tumor, radiation, stem cell transplantation, and immunotherapy works for treating children with newly diagnosed high-risk neuroblastoma. Dinutuximab is a monoclonal antibody that binds to a molecule called GD2, which is found on the surface of neuroblastoma cells, but is not present on many healthy or normal cells in the body. When dinutuximab binds to the neuroblastoma cells, it helps signal the immune system to kill the tumor cells. This helps the cells of the immune system kill the cancer cells, this is a type of immunotherapy. When chemotherapy and immunotherapy are given together, during the same treatment cycle, it is called chemoimmunotherapy. This clinical trial randomly assigns patients to receive either standard chemotherapy and surgery or chemoimmunotherapy (chemotherapy plus dinutuximab) and surgery during Induction therapy. Chemotherapy drugs administered during Induction include, cyclophosphamide, topotecan, cisplatin, etoposide, vincristine, and doxorubicin. These drugs work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing or by stopping them from spreading. Upon completion of 5 cycles of Induction therapy, a disease evaluation is completed to determine how well the treatment worked. If the tumor responds to therapy, patients receive a tandem transplantation with stem cell rescue. If the tumor has little improvement or worsens, patients receive chemoimmunotherapy on Extended Induction. During Extended Induction, dinutuximab is given with irinotecan, temozolomide. Patients with a good response to therapy move on to Consolidation therapy, when very high doses of chemotherapy are given at two separate points to kill any remaining cancer cells. Following, transplant, radiation therapy is given to the site where the cancer originated (primary site) and to any other areas that are still active at the end of Induction. The final stage of therapy is Post-Consolidation. During Post-Consolidation, dinutuximab is given with isotretinoin, with the goal of maintaining the response achieved with the previous therapy. Adding dinutuximab to Induction chemotherapy along with standard of care surgical resection of the primary tumor, radiation, stem cell transplantation, and immunotherapy may be better at treating children with newly diagnosed high-risk neuroblastoma.",[82,28],"Ganglioneuroblastoma, Nodular","2026-08-18",{"date":30,"type":33},{"date":86,"type":33},"2024-04-19",{"date":88,"type":22},"2029-12-31",{"name":90,"class":91},"National Cancer Institute (NCI)","NIH",179,{"id":94,"slug":95,"hasResults":12,"nctId":96,"briefTitle":97,"officialTitle":97,"acronym":4,"eligibilityCriteria":98,"healthyVolunteers":12,"sex":17,"minAge":99,"maxAge":4,"enrollmentInfo":100,"targetDuration":4,"studyType":102,"phases":4,"briefSummary":103,"conditions":104,"keywords":109,"overallStatus":59,"whyStopped":4,"lastUpdateSubmitDate":119,"lastUpdatePostDateStruct":120,"startDateStruct":122,"completionDateStruct":4,"leadSponsor":124,"locationsCount":125},"100133389","prospective-comprehensive-molecular-analysis-of-endocrine-neoplasms-100133389","NCT01005654","Prospective Comprehensive Molecular Analysis of Endocrine Neoplasms","* INCLUSION CRITERIA:\n* Participants who have an endocrine neoplasm based on radiographic and biochemical testing, or histologically\u002Fcytologically proven lesions of the thyroid, parathyroid, adrenal, extra-adrenal endocrine nests, paragangliomas, neuroblastomas, gastrointestinal\n\ntrack and pancreas or participants with a described pre or potentially malignant condition that requires surgery or biopsy as a part of the standard of care treatment and\u002For follow up.\n\n* Participants referred to the Endocrine Consult Service on other protocols for surgical evaluation of endocrine disorders based on radiographic and biochemical testing, or histologically\u002Fcytologically proven lesions of the thyroid, parathyroid, adrenal, extra-adrenal endocrine nests, paragangliomas, neuroblastomas and pancreas.\n* Participants must have an ECOG performance score of 0-2.\n* Participants must have physical examination parameters within acceptable limits by standard of practice guidelines prior to biopsy or surgery.\n* Participants must be planning to undergo surgery or biopsy as part of their treatment plan. Note: Participants will not be enrolled exclusively for the procurement of tissue samples.\n* Age \\>= 4 years of age.\n\nEXCLUSION CRITERIA:\n\nNone.","4 Years",{"count":101,"type":22},2415,"OBSERVATIONAL","Background:\n\n* Endocrine neoplasms (tumors) are among the fastest growing tumors in incidence in the United States. Furthermore, it is often difficult to distinguish between benign or malignant tumors in cancers of the thyroid, parathyroid, adrenal gland, and pancreas. More research is needed to improve detection and treatment options for patients who develop these kinds of cancer.\n* Researchers are interested in studying the molecular changes that are involved in endocrine cancer development and growth. To collect a sample of tumor specimens and healthy tissue for further study, researchers are specifically looking for samples from participants who are scheduled for surgery or biopsy on endocrine tumors.\n\nObjectives:\n\n\\- To collect samples of precancerous, cancerous, and healthy tissue from individuals who are scheduled for surgery or biopsy of endocrine system tumors.\n\nEligibility:\n\n\\- Individuals who have a tumor in or around their thyroid, parathyroid, adrenal gland, pancreas, or any neuroendocrine tissue, and are scheduled for surgery at the National Institutes of Health Clinical Center.\n\nDesign:\n\n* Participants in this study will provide blood and urine samples prior to surgery.\n* During the surgery or biopsy, pieces of the tumor or precancerous growth and pieces of normal tissue near to the tumor will be removed for ongoing and future research. The rest of the tumor or growth will be sent for analysis.\n* After surgery, participants will receive routine care until discharge, and doctors will discuss possible treatment options. If there is an appropriate NIH protocol, participants may choose to be treated at the NIH.\n* After discharge, participants will return to the clinic for a routine postoperative check about 6 weeks following the operation, and then may be followed yearly at the Clinical Center or by phone....",[105,106,107,108,28],"Endocrine Tumors","Thyroid Neoplasms","Parathyroid Neoplasms","Adrenal Neoplasm",[110,111,112,113,114,115,116,117,118],"Gene Expression","Epigenetic (methylation)","Tissues Histological Evaluations","Establishment of Tumor Cell Lines","Metabolite and Protein Expression","Natural History","Adrenal Cancer","Endocrine Tumor","Thyroid Cancer","2026-08-14",{"date":121,"type":33},"2026-08-17",{"date":123,"type":33},"2009-10-07",{"name":90,"class":91},1,{"id":127,"slug":128,"hasResults":12,"nctId":129,"briefTitle":130,"officialTitle":131,"acronym":132,"eligibilityCriteria":133,"healthyVolunteers":12,"sex":17,"minAge":18,"maxAge":134,"enrollmentInfo":135,"targetDuration":4,"studyType":23,"phases":137,"briefSummary":138,"conditions":139,"keywords":143,"overallStatus":59,"whyStopped":4,"lastUpdateSubmitDate":146,"lastUpdatePostDateStruct":147,"startDateStruct":149,"completionDateStruct":151,"leadSponsor":153,"locationsCount":155},"100606238","phase-1-selective-antigen-specific-t-cells-and-car-t-cells-in-subjects-with-relapsedrefractory-embryonal-tumors-sabre-100606238","NCT07172958","Selective Antigen Specific T Cells and CAR T Cells in Subjects With Relapsed\u002FRefractory Embryonal Tumors (SABRE)","Selective Antigen Specific dTβRII-expressing T Cells and B7-H3 CAR T Cells in Subjects With Relapsed\u002FRefractory Embryonal Tumors (SABRE)","SABRE","Inclusion Criteria:\n\nRecipient Inclusion Criteria for Procurement:\n\n* Diagnosis of relapsed\u002Frefractory rhabdomyosarcoma, Ewing sarcoma, neuroblastoma, or Wilms tumor\n* Refractory disease, residual detectable disease or relapsed disease following available standard of care therapies with known clinical benefit for their specific tumor type, or unable to receive such therapies due to unacceptable toxicity or contraindication\n* Measurable or evaluable disease by imaging, as determined following most recent therapy\n* Age ≥ 1 year and \\\u003C 24 years\n* Weight ≥ 10 kg\n* No systemic steroid exposure within 1 week of procurement\n* Karnofsky\u002FLansky score of ≥ 60 (See Appendix 3)\n* Participants of childbearing potential or capable of fathering a child must agree to use effective contraceptive measure\u002Fs (as described in Appendix 5) during study protocol participation through 6 months following the administration of the CAR-TA T cells\n* ANC \\> 500\u002FµL\n* ALC \\> 1000\u002FµL\n* Platelet count \\> 50,000\u002FuL (level can be achieved with transfusion)\n* Bilirubin ≤ 2.5 mg\u002FdL\n* Aspartate aminotransferase (AST)\u002FAlanine transaminase (ALT) ≤ 5x the upper limit of normal for age\n* Serum creatinine Maximum serum creatinine (mg\u002FdL) Age Male Female\n\n  1. to \\\u003C 2 years 0.6 0.6\n  2. to \\\u003C 6 years 0.8 0.8\n\n  6 to \\\u003C 10 years 1 1 10 to \\\u003C 13 years 1.2 1.2 13 to \\\u003C 16 years 1.5 1.2\n\n  ≥ 16 years 1.7 1.4 OR Creatinine clearance or glomerular filtration rate (GFR) ≥ 70 mL\u002Fmin\u002F1.73 m for patients with levels above\n* For FOCBP: Negative pregnancy test\n* Pulse oximetry of \\> 90% on room air\n* Adequate cardiac function defined as:\n\n  * Shortening fraction of ≥ 27% by echocardiogram, or\n  * Ejection fraction of \\> 50% by echocardiogram or radionuclide angiogram (i.e., MUGA).\n* No acute neurological toxicity \\> grade 1 (with the exception of peripheral sensory neuropathy or controlled seizure disorder on anti-epileptics).\n* The following time frames must have elapsed between prior therapy completion and apheresis cell collection:\n\n  * Myelosuppressive chemotherapy\u002Fimmunomodulatory medications: At least 3 weeks, or 6 weeks if prior nitrosourea.\n  * Hematopoietic growth factors: At least 7 days since the completion of therapy with a growth factor. At least 14 days after receiving pegfilgrastim.\n  * Biological agent, tyrosine kinase inhibitor, targeted agent, metronomic chemotherapy: At least 7 days since the completion of therapy with a biologic agent, tyrosine kinase inhibitor, targeted agent, or metronomic non-myelosuppressive regimen.\n  * Monoclonal antibodies and checkpoint inhibitors: At least 3 weeks or 5 half-lives (whichever is shorter) since the last dose of a monoclonal antibody or checkpoint inhibitor.\n  * Radiotherapy (XRT): At least 3 weeks since XRT, and at least 6 weeks if radiation involved the CNS or lung fields. Exception: There is no time restriction for palliative radiation with minimal bone marrow involvement and the patient has measurable\u002Fevaluable disease outside the radiation port or the site of radiation has documented progression.\n  * Autologous stem cell transplant\u002Finfusion: At least 6 weeks from their infusion after an autologous stem cell infusion following myeloablative therapy. Patients who received an autologous stem cell infusion following non-myeloablative therapy do not have a wash-out period; they are eligible once they meet all other eligibility requirements, including recovery from acute side effects.\n  * Investigational agent: at least 28 days since receiving an investigational agent.\n* Adult participant or the legally authorized representative (LAR) of a minor (defined as \\\u003C18 years of age) must be capable of providing informed consent. When appropriate, pediatric participants ≥7 years of age will participate in an age-appropriate discussion and provide assent, unless an IRB-approved waiver of assent applies and documentation of the participant's eligibility for the waiver is maintained.\n\nRecipient Inclusion Criteria for CAR-TA T cell product Infusion:\n\n* No systemic steroid exposure within 1 week prior to protocol therapy initiation\n* Karnofsky\u002FLansky score of ≥ 60\n* ANC \\> 750\u002FuL\n* Platelet count \\> 75,000\u002FuL\n* Bilirubin ≤ 2.5 mg\u002FdL\n* AST\u002FALT ≤ 5x the upper limit of normal for age\n* Serum creatinine Maximum serum creatinine (mg\u002FdL) Age Male Female\n\n  1 to \\\u003C 2 years 0.6 0.6 2 to \\\u003C 6 years 0.8 0.8 6 to \\\u003C 10 years 1 1 10 to \\\u003C 13 years 1.2 1.2 13 to \\\u003C 16 years 1.5 1.2\n\n  ≥ 16 years 1.7 1.4 OR Creatinine clearance or glomerular filtration rate (GFR) ≥ 70 mL\u002Fmin\u002F1.73 m for patients with levels above\n* For FOCBP: Negative pregnancy test\n* Participants of childbearing potential or capable of fathering a child must agree to use effective contraceptive measure\u002Fs through 6 months following the administration of the CAR-TA T cells\n* Adequate respiratory function defined as oxygen saturation 90% or higher on room air\n* For participants who underwent prior mediastinum-directed therapies (e.g., post radiation to chest): resolution of any respiratory symptoms\n* Adequate respiratory rate, defined as \\\u003C30 breaths per minute for patients aged \\\u003C18 years, and \\\u003C25 breaths per minute for patients aged ≥18 years (respiratory rate may be repeated if initial value is thought to be temporarily abnormal; if repeated, 2 consecutive readings obtained ≥30 minutes apart must be adequate to be eligible)\n* No acute neurological toxicity \\> grade 1 (with the exception of peripheral sensory neuropathy or controlled seizure disorder on anti-epileptics).\n* Adequate cardiac function defined as:\n\n  * Shortening fraction of ≥ 27% by echocardiogram, or\n  * Ejection fraction of \\> 50% by echocardiogram or radionuclide angiogram\n* The following time frames must have elapsed between completion of prior therapy and the initiation of SABRE protocol therapy:\n\n  * Myelosuppressive chemotherapy: At least 2 weeks from last dose of chemotherapy.\n  * Hematopoietic growth factors: At least 7 days since the completion of therapy with a growth factor. At least 14 days after receiving pegfilgrastim.\n  * Biological agent, tyrosine kinase inhibitor, targeted agent, metronomic chemotherapy: At least 7 days since the completion of therapy with a biologic agent, tyrosine kinase inhibitor, targeted agent, or metronomic non-myelosuppressive regimen.\n  * Monoclonal antibodies and checkpoint inhibitors: At least 3 weeks or 5 half-lives (whichever is shorter) since the last dose of a monoclonal antibody or checkpoint inhibitor.\n  * Radiotherapy (XRT): At least 3 weeks since XRT, and at least 6 weeks if radiation involved CNS or lung fields. Exception: There is no time restriction for palliative radiation with minimal bone marrow involvement and the patient has measurable\u002Fevaluable disease outside the radiation port or the site of radiation has documented progression.\n  * Investigational agent: At least 28 days since receiving an investigational agent.\n* Adult participant or the legally authorized representative (LAR) of a minor (defined as \\\u003C18 years of age) must be capable of providing informed consent. When appropriate, pediatric participants ≥7 years of age will participate in an age-appropriate discussion and provide assent, unless an IRB-approved waiver of assent applies and documentation of the participant's eligibility for the waiver is maintained.\n\nExclusion Criteria:\n\nRecipient Procurement Exclusion Criteria:\n\n* Patients with known CNS disease.\n* Patients with uncontrolled infection\u002Fs or known HIV infection\n* Pregnant or lactating females.\n* Patients who have undergone previous allogeneic stem cell transplant.\n* Inability to tolerate leukapheresis (including any contraindication to the use of Anticoagulant Citrate Dextrose solution).\n\nRecipient Exclusion Criteria for CAR-TA T cell product Infusions:\n\n* Patients with uncontrolled infections or known HIV infection.\n* Pregnant or lactating females\n* Whole lung\u002Fmediastinal radiation within 12 weeks\n* Clinically significant systemic illness or medical condition likely to interfere with assessment of safety or efficacy\n* Patients who have received any live vaccine in the six weeks prior to planned initiation of lymphodepletion\n* Any contraindication to lymphodepletion or to the use of Cyclophosphamide or Fludarabine, as per the clinical judgment of the PI or treating Sub-I\n* History of allergy or hypersensitivity to study product excipients (e.g., DMSO)","23 Years",{"count":136,"type":22},18,[53],"This is a phase I dose-escalation study to determine the safety and feasibility of autologous CAR-TA T cells (B7-H3 CAR+ T cells administered with DNR-PRAME Tumor Antigen-specific T cells) following lymphodepleting chemotherapy in participants with relapsed\u002Frefractory rhabdomyosarcoma, Ewing sarcoma, neuroblastoma and Wilms tumor.\n\nPatients will be enrolled to one of three planned dose levels with B7-H3 CAR T cell dose determined based on the percentage of B7-H3 transduced cells (B7-H3+ population of cells), and dTBRII-transduced PRAME TA-specific T cell dose based on the total cell population. Both doses will be based on the recipient's body weight and combined in a 1:1 ratio.\n\nThe safety of the CAR-TA T cell product will be evaluated and the maximum tolerated dose (MTD) will be determined. The safety endpoint will be assessed by monitoring for dose limiting toxicities for 28 days following CAR-TA T cell administration.",[140,141,28,142],"Rhabdomyosarcoma","Ewing Sarcoma","Wilms Tumor",[144,145],"CAR T Therapy for Embryonal tumors","T cell Therapy for Embryonal tumors","2026-08-11",{"date":148,"type":33},"2026-08-13",{"date":150,"type":33},"2026-01-27",{"date":152,"type":22},"2044-12",{"name":154,"class":40},"Children's National Research Institute",2,{"id":157,"slug":158,"hasResults":12,"nctId":159,"briefTitle":160,"officialTitle":161,"acronym":4,"eligibilityCriteria":162,"healthyVolunteers":12,"sex":17,"minAge":4,"maxAge":163,"enrollmentInfo":164,"targetDuration":4,"studyType":23,"phases":166,"briefSummary":167,"conditions":168,"keywords":174,"overallStatus":59,"whyStopped":4,"lastUpdateSubmitDate":176,"lastUpdatePostDateStruct":177,"startDateStruct":179,"completionDateStruct":181,"leadSponsor":183,"locationsCount":185},"100551831","phase-1-eflornithine-dfmo-and-amxt-1501-for-neuroblastoma-cns-tumors-and-sarcomas-100551831","NCT06465199","Eflornithine (DFMO) and AMXT 1501 for Neuroblastoma, CNS Tumors, and Sarcomas","A Phase I\u002FII Study Using Eflornithine (DFMO) and AMXT 1501 for Relapsed and Refractory Neuroblastoma, CNS Tumors, and Sarcomas","Inclusion Criteria:\n\n1. Age:\n\n   All participants : Must be a maximum of 26 years of age at diagnosis\n\n   Age at enrollment by Phase:\n   1. Safety Run-in (Dose level 1)-The first three (3) participants enrolled will be ≥ 12 years of age at enrollment. Once evaluated for safety by DSMB, we will move on to the next three (3) participants enrolled who will be ≥6 years of age at enrollment. Once evaluated for safety by DSMB, we will move on to the Phase I.\n   2. Phase I and II: ≤ 26 years of age at diagnosis.\n2. Pathology\n\n   All participants must have a confirmed pathologic diagnosis of tumor type (except for DIPG):\n   * Relapsed\u002Frefractory Neuroblastoma (NB)\n   * Relapsed\u002Frefractory Embryonal tumor with multilayer rosettes (ETMR)\n   * Relapsed\u002Frefractory Atypical teratoid rhabdoid tumor (ATRT)\n   * Newly diagnosed Diffuse Intrinsic Pontine Glioma (DIPG)- radiologic diagnosis acceptable\n   * Relapsed\u002Frefractory Ewing Sarcoma (EWS)\n   * Relapsed\u002Frefractory Osteosarcoma (OST)\n3. Tumor assessment:\n\n   Disease staging must be performed at baseline during the 28 day screening period prior to first dose of study drug.\n4. Disease Status:\n\n   Relapsed or Refractory Neuroblastoma Relapsed disease defined as: High-risk neuroblastoma that was previously in remission after standard therapy (at least 4 cycles of aggressive multi-drug induction chemotherapy, with or without radiation, surgery, and immunotherapy, or according to a standard high-risk treatment\u002Fneuroblastoma protocol).\n\n   Refractory disease defined as: High-risk neuroblastoma that 1) failed to achieve CR after at least 4 cycles of aggressive multi-drug induction chemotherapy with or without radiation and surgery, followed by immunotherapy, or according to a standard high-risk treatment\u002Fneuroblastoma protocol, or 2) progression during upfront therapy or 3) with disease remaining after standard immunotherapy.\n\n   Eligible NB participants may have active disease or no active disease.\n\n   NB participants with no active disease need to meet the following criteria:\n\n   Timing from prior therapy: Enrollment (first dose of study drug) no later than 60 days from most recent therapy.\n\n   NB participants with active disease need to meet the following criteria:\n   * Received at least one recent treatment for their relapse\u002Frefractory disease and is stable (SD) or better on this treatment.\n   * Participants must not have disease in any organs (including lungs, liver, or brain).\n\n   Relapsed or refractory ETMR\u002FATRT Participants that have relapsed following standard of care therapy or having progressed during standard of care therapy and non-responsive\u002Fprogressive to accepted curative therapy, including up-front chemotherapy and radiation and\u002For high-dose chemotherapy with stem cell rescue.\n\n   ETMR\u002FATRT participants with no active disease need to meet the following criteria:\n\n   Timing from prior therapy: Enrollment (first dose of study drug) no later than 60 days from most recent therapy.\n\n   ETMR\u002FATRT participants with active disease need to meet the following criteria:\n\n   • Received at least one recent treatment for their relapse\u002Frefractory disease and is stable (SD) or better on this treatment.\n\n   Newly Diagnosed Diffuse Intrinsic Pontine Glioma (DIPG) Participants with DIPG to start greater than 30 days, and no longer than 60 days, after standard of care radiation therapy.\n\n   Participants with newly-diagnosed typical DIPG, defined as tumors with a pontine epicenter and diffuse involvement of the pons on at least 1 axial T2-weighted image, are eligible. No histologic confirmation is required. Participants with metastatic disease are not eligible. Participants with a biopsy and no evidence of H3K27m mutations are eligible as long as they meet radiographic criteria. Participants with H3K27m altered DMG outside of the brainstem are not eligible. Participants with progression or recurrence after initial standard of care radiation are ineligible.\n\n   Relapsed or refractory Ewing sarcoma and osteosarcoma Participants that have relapsed following standard of care therapy or having progressed during standard of care therapy. Standard of care therapy for Ewing sarcoma and osteosarcoma includes multi-agent chemotherapy with local control consisting of either surgery or radiation therapy.\n\n   EWS\u002FOST Participants with no active disease need to meet the following criteria:\n\n   Timing from prior therapy: Enrollment (first dose of study drug) no later than 60 days from most recent therapy.\n\n   EWS\u002FOST Participants with active disease need to meet the following criteria:\n\n   • Received at least one recent treatment for their relapse\u002Frefractory disease and is stable (SD) or better on this treatment.\n5. Participants must be able to swallow capsules.\n6. Participants with CNS disease currently taking steroids must have been on a stable dose of steroids for at least one week and must not have progressive hydrocephalus at enrollment.\n7. Participants must have fully recovered from the acute toxic effects of all prior anti- cancer chemotherapy and be within the following timelines:\n\n   1. Myelosuppressive chemotherapy: Must not have received within 2 weeks of enrollment onto this study (6 weeks if prior nitrosourea).\n   2. Small Molecule Inhibitor (anti-neoplastic agent): At least 7 days since the completion of therapy with a small molecule inhibitor. For agents that have known adverse events occurring beyond 7 days after administration, this period must be extended beyond the time during which adverse events are known to occur. The duration of this interval must be discussed with the Study Chair.\n   3. Immunotherapy: At least 4 weeks since the completion of any type of immunotherapy, e.g. tumor vaccines, CAR-T cells except for anti-GD2 Monoclonal antibodies (ex. naxitamab, dinutuximab, etc.) which should be at least 2 weeks since prior treatment with a monoclonal antibody.\n   4. XRT: At least 14 days since the last treatment except for radiation delivered with palliative intent to a non-target site.\n\n      Note: Participants with DIPG will be required to have had up front standard of care radiation. As above, participants with DIPG must be between 30-60 days post initial up- front radiation therapy.\n   5. Stem Cell Transplant:\n\n      1. Allogeneic: No evidence of active graft vs. host disease\n      2. Allo\u002FAuto: ≥ 45 days must have elapsed since transplant.\n   6. MIBG Therapy: At least 6 weeks since treatment with MIBG therapy.\n8. Participants must have a Lansky or Karnofsky Performance Scale score of \\>\u002F= 60\n9. Participants must have adequate organ function at the time of enrollment:\n\n   * Hematological: Hematological recovery as defined by ANC ≥750\u002FμL (unsupported- \\>24 hrs off G-CSF and 7 days off neulasta)\n   * Liver: Adequate liver function as defined by AST and ALT \\\u003C10x upper limit of normal\n   * Cardiac: all participants must have:\n\n     1. Normal serum Cardiac Troponin Concentration\n     2. Normal BNP (B-type natriuretic peptide) Level\n     3. A QTcF ≤ 470 msec (or EKG with no significant findings)\n     4. Normal ECHO defined as:\n\n     i. Shortening fraction of ≥ 27% by echocardiogram, or ii. Ejection fraction of ≥ 50% by echocardiogram or radionuclide angiogram\n   * Renal: Participants must have adequate renal function defined as:\n\n     1. For participants \\\u003C 17 years old: estimated Glomerular Filtration rate (eGFR) as calculated from the Bedside Schwartz equation (in units of mL\u002Fmin\u002F1.73 m2) or via radioisotope GFR of ≥ 70 mL\u002Fmin\u002F1.73 m2. The Bedside Schwartz equation is: \\[(0.413) X (Height in cm)\\] \u002F SCr\n     2. For participants ≥17 years old: estimated Glomerular Filtration rate (eGFR) as calculated from the Cockcroft and Gault formula (in units of mL\u002Fmin\u002F1.73 m2) or via radioisotope GFR of ≥ 70 mL\u002Fmin\u002F1.73 m2. The Cockcroft and Gault formula is: \\[(140-age) x (Wt in kg) x (0.85 if female)\\] \u002F (72 x SCr)\n10. Participants of childbearing potential must have a negative pregnancy test. Participants of childbearing potential must agree to use an effective birth control method. Participants who are lactating must agree to stop breast-feeding.\n11. Written informed consent in accordance with institutional and FDA guidelines must be obtained from all participants (or participants' legal representative).\n\nExclusion Criteria:\n\n1. BSA of \\\u003C0.25 m2\n2. Investigational Drugs: Participants who are currently receiving another investigational drug are excluded from participation.\n3. Anti-cancer Agents: Participants who are currently receiving other anticancer agents are not eligible. Participants must have fully recovered from the hematological and bone marrow suppression effects of prior chemotherapy.\n4. Infection: Participants who have an uncontrolled infection are not eligible until the infection is judged to be well controlled in the opinion of the investigator.\n5. Participants who, in the opinion of the investigator, may not be able to comply with the safety monitoring requirements of the study, or in whom compliance is likely to be suboptimal, should be excluded.","26 Years",{"count":165,"type":22},289,[53,25],"The purpose of this study is to evaluate the investigational oral drug AMXT 1501 in combination with oral eflornithine (DFMO). An investigational drug is one that has not been approved by the U.S. Food \\& Drug Administration (FDA), or any other regulatory authorities around the world for use alone or in combination with any drug, for the condition or illness it is being used to treat.\n\nThe goals of this part of the study are:\n\n* Establish a recommended dose of AMXT 1501 in combination with DFMO\n* Test the safety and tolerability of AMXT 1501 in combination with DFMO\n* To determine the activity of study treatments chosen based on:\n* How each subject responds to the study treatment\n* How long a subject lives without their disease returning\u002Fprogressing",[169,170,141,171,172,28,173],"Atypical Teratoid\u002FRhabdoid Tumor","Embryonal Tumor With Multilayered Rosettes","Diffuse Intrinsic Pontine Glioma","Osteosarcoma","DIPG Brain Tumor",[175],"DFMO","2026-08-10",{"date":178,"type":33},"2026-08-12",{"date":180,"type":33},"2026-05-13",{"date":182,"type":22},"2035-05",{"name":184,"class":40},"Milton S. Hershey Medical Center",14,{"id":187,"slug":188,"hasResults":12,"nctId":189,"briefTitle":190,"officialTitle":191,"acronym":4,"eligibilityCriteria":192,"healthyVolunteers":12,"sex":17,"minAge":193,"maxAge":194,"enrollmentInfo":195,"targetDuration":4,"studyType":23,"phases":197,"briefSummary":198,"conditions":199,"keywords":200,"overallStatus":59,"whyStopped":4,"lastUpdateSubmitDate":176,"lastUpdatePostDateStruct":203,"startDateStruct":204,"completionDateStruct":206,"leadSponsor":208,"locationsCount":210},"100476895","phase-2-naxitamab-added-to-induction-for-newly-diagnosed-high-risk-neuroblastoma-100476895","NCT05489887","Naxitamab Added to Induction for Newly Diagnosed High-Risk Neuroblastoma","A Phase II Study of Naxitamab Added to Induction Therapy for Subjects With Newly Diagnosed High-Risk Neuroblastoma","Inclusion Criteria:\n\n1. Diagnosis: Subjects must have a diagnosis of neuroblastoma or ganglioneuroblastoma (nodular or intermixed) verified by histology or demonstration of clumps of tumor cells in bone marrow with elevated urinary catecholamine metabolites. Subjects with the following disease stages at diagnosis are eligible, if they meet the other specified criteria:\n2. Subjects with newly diagnosed neuroblastoma with INRGSS Stage M disease with either of the following features:\n\n   1. MYCN amplification (\\> 4-fold increase in MYCN signals as compared to reference signals), regardless of additional biologic features; OR\n   2. 365 days to ≥ 547 days of age without MYCN amplification, but unfavorable biologic features such as unfavorable histology (INPC) or diploid tumor (DNA index=1) or the presence of any segmental chromosome aberration (SCA) (somatic copy number loss at 1p, 3p, 4p, or 11q or somatic copy number gain at 1q, 2p, or 17q); OR\n   3. Age \\> 547 days of age regardless of biologic features\n\n   Subjects with newly diagnosed neuroblastoma with INRGSS Stage MS disease with either of the following:\n   1. MYCN amplification (\\> 4-fold increase in MYCN signals as compared to reference signals); OR\n   2. 365 days to ≥ 547 days (18 months) of age without MYCN amplification, but unfavorable biologic features such as unfavorable histology (INPC) or diploid tumor (DNA index=1) or SCA as above\n\n   Subjects with newly diagnosed neuroblastoma INRGSS Stage L2 disease with either of the following:\n   1. MYCN amplification (\\> 4-fold increase in MYCN signals as compared to reference signals); OR\n   2. 18 months to \\\u003C5 years of age without MYCN amplification, but with unfavorable histology (INPC); OR\n   3. ≥5 years of age without MYCN amplification, but with undifferentiated or poorly differentiated INPC Subjects with newly diagnosed neuroblastoma INRGSS Stage L1 disease that is incompletely resected with MYCN amplification.\n\n   Subjects \\> 547 days of age initially diagnosed with INRGSS Stage L1, L2 or MS disease who progressed to Stage M without prior chemotherapy may enroll within 4 weeks of progression to Stage M.\n\n   Subjects ≥ 365 days of age initially diagnosed with MYCN amplified INRGSS Stage L1 disease who progress to Stage M without systemic therapy may enroll within 4 weeks of progression to Stage M.\n3. Subjects must be age ≤ 21 years at initial diagnosis.\n4. Subjects must be \\>12 months of age at enrollment.\n5. Adequate cardiac function defined as:\n\n   1. Shortening fraction of ≥ 27% by echocardiogram, or\n   2. Ejection fraction of ≥ 50% by radionuclide evaluation or echocardiogram.\n6. Adequate liver function must be demonstrated, defined as:\n\n   1. Total bilirubin ≤ 1.5 x upper limit of normal (ULN) for age AND\n   2. ALT (SGPT) \\\u003C 5 x upper limit of normal (ULN) for age\n7. 1\\. Subjects must have adequate renal function defined as:\n\n   * For subjects \\\u003C 17 years old: estimated Glomerular Filtration rate (eGFR) as calculated from the Bedside Schwartz equation (in units of mL\u002Fmin\u002F1.73 m2) or via radioisotope GFR of ≥ 70 mL\u002Fmin\u002F1.73 m2. The Bedside Schwartz equation is: \\[(0.413) X (Height in cm)\\] \u002F SCr\n   * For subjects ≥17 years old: estimated Glomerular Filtration rate (eGFR) as calculated from the Cockcroft and Gault formula (in units of mL\u002Fmin\u002F1.73 m2) or via radioisotope GFR of ≥ 70 mL\u002Fmin\u002F1.73 m2. The Cockcroft and Gault formula is: \\[(140-age) x (Wt in kg) x (0.85 if female)\\] \u002F (72 x SCr)\n   * OR a 24 hour urine Creatinine clearance ≥ 70 mL\u002Fmin\u002F1.73 m2\n8. A negative serum pregnancy test is required for subjects of childbearing potential (≥13 years of age or after onset of menses)\n9. Both male and female post-pubertal study subjects must be willing to use a highly effective contraceptive method (i.e., achieves a failure rate of \\\u003C1% per year when used consistently and correctly) from the time of informed consent (and assent, as applicable) until 6 months after study treatment discontinuation. Such methods include: combined (estrogen and progestogen containing) hormonal contraception associated with inhibition of ovulation (oral, intravaginal, transdermal), progestogen-only hormonal contraception associated with inhibition of ovulation (oral, injectable, implantable), intrauterine device (IUD), intrauterine hormone-releasing system (IUS), bilateral tubal occlusion, vasectomized partner, sexual abstinence.\n10. Informed Consent: All subjects and\u002For legal guardians must sign informed written consent. Assent, when appropriate, will be obtained according to institutional guidelines and applicable local regulations.\n\nExclusion Criteria:\n\n1. Subjects who are less than 1 year of age\n2. Subjects who are 12-18 months of age with INRGSS Stage M and all stage L2 subjects with favorable biologic features (i.e., nonamplified MYCN, favorable pathology, and DNA index \\> 1) are not eligible.\n3. Subjects who have had prior systemic therapy except for localized emergency radiation to sites of life-threatening or function-threatening disease and\u002For no more than 1 cycle of chemotherapy.\n4. Treatment with immunosuppressive treatment (topical, inhaled and short-term emergency steroids excluded) within 4 weeks prior to enrollment\n5. Inadequate pulmonary function defined as evidence of dyspnea at rest, exercise intolerance, and\u002For chronic oxygen requirement. In addition, room air pulse oximetry \\\u003C 94% and\u002For abnormal pulmonary function tests if these assessments are clinically indicated.\n6. Pregnant or breastfeeding (NOTE: breast milk cannot be stored for future use while the mother is being treated on study.)\n7. Subjects receiving any investigational drug concurrently.\n8. Subjects with any other medical condition, including but not limited to malabsorption syndromes, mental illness or substance abuse, deemed by the Investigator to be likely to interfere with the interpretation of the results or which would interfere with a subject's ability to sign or the legal guardian's ability to sign the informed consent, and subject's ability to cooperate and participate in the study\n9. Subjects with a significant intercurrent illness (any ongoing serious medical problem unrelated to cancer or its treatment) that is not covered by the detailed exclusion criteria and that is expected to interfere with the action of investigational medicinal products (IMPs) or to significantly increase the severity of the toxicities experienced from trial treatment.","12 Months","21 Years",{"count":196,"type":22},93,[25],"This is a prospective, multicenter clinical trial in subjects with newly diagnosed high-risk neuroblastoma to evaluate the efficacy and safety of administering naxitamab with standard induction therapy. The initial chemotherapy will include 5 cycles of multi-agent chemotherapy. Naxitamab will be added to all 5 Induction cycles. We hypothesize that the addition of anti-GD2 therapy to induction chemotherapy will result in improved end of induction responses and improved survival.",[28],[201,202],"naxitimab","induction",{"date":178,"type":33},{"date":205,"type":33},"2022-09-14",{"date":207,"type":22},"2036-09",{"name":209,"class":40},"Giselle Sholler",26,{"id":212,"slug":213,"hasResults":12,"nctId":214,"briefTitle":215,"officialTitle":216,"acronym":4,"eligibilityCriteria":217,"healthyVolunteers":218,"sex":17,"minAge":4,"maxAge":4,"enrollmentInfo":219,"targetDuration":4,"studyType":23,"phases":221,"briefSummary":222,"conditions":223,"keywords":224,"overallStatus":59,"whyStopped":4,"lastUpdateSubmitDate":176,"lastUpdatePostDateStruct":232,"startDateStruct":233,"completionDateStruct":235,"leadSponsor":237,"locationsCount":125},"100434392","phase-2-a-study-of-a-vaccine-in-combination-with--glucan-and-gm-csf-in-people-with-neuroblastoma-100434392","NCT04936529","A Study of a Vaccine in Combination With β-glucan and GM-CSF in People With Neuroblastoma","Phase II Trial of a Bivalent Vaccine With the Immunological Adjuvant OPT-821 (QS-21), in Combination With Oral β-glucan and Randomization of GM-CSF, for High-risk Neuroblastoma","Inclusion Criteria:\n\n* Diagnosis of NB as defined by international criteria, i.e., histopathology (confirmed by the MSK Department of Pathology) or BM metastases plus high urine catecholamine levels or positivity in MIBG scan\n* HR-NB as defined by risk-related treatment guidelines and international criteria,i.e., metastatic\u002Fnon-localized disease with MYCN amplification (any age), MYCN-non-amplified metastatic disease \\>18 months old, MYCNamplified localized disease (any age), or disease resistant to standard chemotherapy.\n* HR-NB (as defined above) and in 1) first CR at ≥ 6 months from initiation of immunotherapy using anti-GD2 antibody, or 2) second or subsequent CR (achieved after treatment for PD). CR is defined according to the International Neuroblastoma Response Criteria.Patients with positive MIBG scan but negative FDG-PET scan, and CR in BM, are eligible. For those patients with a positive MIBG scan, the negative FDG-PET scan can be per PI discretion to repeat within the 45 day window period prior to enrollment.\n* Patients with grade 3 toxicities or less using the Common Toxicity Criteria (Version 5.0) developed by the National Cancer Institute of the USA (CTCAE v5.0) related to hematologic, cardiac, neurological, pulmonary, renal, hepatic or gastrointestinal function as determined by blood tests or physical exam.\n* Hematologic Function\n\n  * Absolute neutrophil count (ANC) ≥ 500\u002Fmcl\n  * Absolute lymphocyte count ≥ 500\u002Fmcl\n  * Hemaglobin (Hgb) ≥ 8 g\u002FdL\n  * Platelet count ≥ 50,000 mm\\^3\n* Renal Function o Serum creatinine ≤ 3.0 x ULN\n\nor\n\n* eGFR \\>60 mL\u002Fmin\u002F1.73 m\\^2\n\n  \\- Hepatic Function\n* Serum bilirubin ≤ 3.0 × ULN\n* Aspartate transaminase (AST) ≤ 5.0 × ULN\n* Alanine aminotransferase (ALT) ≤ 5.0 × ULN\n\n  * Prior treatment with other immunotherapy, including mAbs or vaccine, is allowed but must be completed ≥ 21 days before the 1st vaccination.\n\nNote: Prior treatment with an investigational therapy must be completed ≥ 28 days before the 1st vaccination.\n\n* ≥ 21 and ≤ 180 days between completion of systemic therapy and 1st vaccination.\n* Patients have recovered from any toxicities grade 3 or higher caused by prior therapies.\n* Patients previously enrolled on this trial are eligible for repeat enrollment if they did not complete all vaccine injections during the first time on protocol but they will be assigned to Group 3 and will not be included in the primary biostatistical analyses.\n* A negative pregnancy test is required for patients w ith child-bearing capability.\n* Signed informed consent indicating awareness of the investigational nature of this program.\n\nExclusion Criteria:\n\n* Patients w ith significant (grade \\>4) hematologic, cardiac, neurological, pulmonary, renal, hepatic or gastrointestinal function as determined by blood tests or physical exam, using the Common Toxicity Criteria (Version 5.0) developed by the National Cancer Institute of the USA (CTCAE v5.0)\n* History of allergy to KLH, QS-21, OPT-821, or glucan.\n* Active life-threatening infection requiring systemic therapy.\n* Inability to comply with protocol requirements.\n* Patients with history of allergy to GM-CSF or who are unable to obtain GM-CSF because of insurance issues are ineligible",true,{"count":220,"type":22},286,[25],"The purpose of the study is to explore the combination of a bivalent vaccine, a sugar called beta-glucan (β-glucan), and a protein called granulocyte-macrophage colony stimulating factor (GM-CSF) as an effective treatment for people with high-risk neuroblastoma that is in complete remission. The combination may be effective because the different parts of the treatment work to strengthen the immune system's response against cancer cells in different ways.",[28],[28,225,226,227,228,229,230,231],"High-risk Neuroblastoma","OPT-821","QS-21","β-glucan","GM-CSF","Memorial Sloan Kettering Cancer Center","21-206",{"date":146,"type":33},{"date":234,"type":33},"2021-08-02",{"date":236,"type":22},"2027-06-15",{"name":230,"class":40},{"id":239,"slug":240,"hasResults":12,"nctId":241,"briefTitle":242,"officialTitle":242,"acronym":4,"eligibilityCriteria":243,"healthyVolunteers":12,"sex":17,"minAge":244,"maxAge":245,"enrollmentInfo":4,"targetDuration":4,"studyType":246,"phases":4,"briefSummary":247,"conditions":248,"keywords":250,"overallStatus":256,"whyStopped":4,"lastUpdateSubmitDate":257,"lastUpdatePostDateStruct":258,"startDateStruct":4,"completionDateStruct":4,"leadSponsor":260,"locationsCount":125},"100286991","a-compassionate-useexpanded-access-protocol-using-131i-mibg-therapy-for-patients-with-refractory-neuroblastoma-and-metastatic-pheochromocytoma-100286991","NCT03015844","A Compassionate Use\u002FExpanded Access Protocol Using 131I-MIBG Therapy for Patients With Refractory Neuroblastoma and Metastatic Pheochromocytoma","Inclusion Criteria:\n\n* Diagnosis: Relapsed\u002FRefractory neuroblastoma, with an original diagnosis made histologically or from elevated urine catecholamines with abnormal tumor cells in bone marrow OR relapsed\u002Frefractory pheochromocytoma.\n* Disease status: Progressive disease at any time (defined as any new lesion or an increase in size by \\>25% of pre-existing disease), or a failure to respond to standard therapy. Patients must have evidence of MIBG uptake into tumor at ≥ one site within 6 weeks prior to entry on study and subsequent to any intervening therapy\n* Prior therapy: A minimum of two weeks since should have elapsed since any chemotherapy causing myelosuppression. It must be a minimum of three months since receiving radiation to any of the following fields: craniospinal, total abdominal, whole lung, total body irradiation. For any other sites of radiation, at least 2 weeks should have elapsed. For patients who received radiation to the only site of MIBG-avid disease within two months of study entry, biopsy confirmation of residual active disease is required, with positive bone marrow being sufficient. At least 7 days should have elapsed since completion of therapy with a biologic agent and at least 3 half-lives should have elapsed since therapy with a monoclonal antibody. No cytokine therapy may be given within 24 hours of receiving 131I-MIBG. Patients may have received prior MIBG therapy, provided they demonstrated a response or stable disease initially, with progressive disease occurring at least 35 days following treatment.\n* Organ function: ANC \\>500\u002FuL, platelets \\>20,000\u002FuL with transfusion allowed. Bilirubin ≤2x ULN, AST\u002FALT ≤10x ULN.Serum Creatinine ≤2x ULN OR 24-hour creatinine clearance OR GFR ≥60ml\u002Fmin\u002F1.73m2. Normal lung function demonstrated by no dyspnea, exercise intolerance or oxygen requirement. Oxygen saturation ≥94% on room air. No clinically significant cardiac dysfunction and ejection fraction ≥45% on echocardiogram.\n* Stem cells: Patients must have a minimum of 2.0 x106\u002Fkg viable CD34+ peripheral blood stem cells for re-infusion following 131I-MIBG. An additional back-up of 2.0x106\u002Fkg CD34+ cells is recommended but not required.\n* Life expectancy longer than 8 weeks,Karnofsky or Lansky performance status of ≥ 50%\n\nExclusion Criteria:\n\n* Pregnant or lactating patients\n* Disease of any organ system that would compromise the patient's ability to participate in the study, including hemodialysis. Significant organ impairment should be discussed with the Principal Investigator prior to study entry\n* Patients with active grade 3-4 infection, as defined by the NCI CTCAE V4.0.\n* Patients with known MBIG-avid brain parenchymal disease (leptomeningeal or skull based metastases are eligible).\n* In patients with metastatic pheochromocytoma, a urinalysis must be preformed prior to study enrollment. If proteinuria is present, a 24 hour urine must be collected and total protein determined. If the 24-hour urine protein is above the institutional upper limit of normal, the patient is excluded.","365 Days","29 Years","EXPANDED_ACCESS","This is an expanded access protocol\u002Fcompassionate use single institution study designed to determine the palliative benefit and toxicity of 131I-MIBG in patients with progressive neuroblastoma and metastatic pheochromocytoma who are not eligible for therapies of higher priority. Response rate, toxicity, and time to progression and death will be evaluated.",[28,249],"Pheochromocytoma",[251,252,253,254,255],"MIBG","131I-MIBG","relapsed","neuroblastoma","pheochromocytoma","AVAILABLE","2026-08-04",{"date":259,"type":33},"2026-08-06",{"name":261,"class":40},"Northwell Health",{"id":263,"slug":264,"hasResults":12,"nctId":265,"briefTitle":266,"officialTitle":266,"acronym":4,"eligibilityCriteria":267,"healthyVolunteers":218,"sex":17,"minAge":268,"maxAge":4,"enrollmentInfo":269,"targetDuration":4,"studyType":102,"phases":4,"briefSummary":271,"conditions":272,"keywords":276,"overallStatus":59,"whyStopped":4,"lastUpdateSubmitDate":284,"lastUpdatePostDateStruct":285,"startDateStruct":286,"completionDateStruct":4,"leadSponsor":288,"locationsCount":289},"100141241","comprehensive-omics-analysis-of-pediatric-and-adult-solid-tumors-and-establishment-of-a-repository-for-related-biological-studies-100141241","NCT01109394","Comprehensive Omics Analysis of Pediatric and Adult Solid Tumors and Establishment of a Repository for Related Biological Studies","* SUBJECT INCLUSION CRITERIA:\n\nPediatric or adult subjects with one of the following:\n\n* Diagnosis of any tumor, malignancy, pre-malignant disorder, or suspected premalignant familial syndromes, regardless, of patient age;\n* Biological relatives of any patient with a tumor, malignancy, pre-malignant disorder, or suspected familial pre-malignant syndrome, regardless of patient age or the diagnosis of an adult malignancy or pre-malignant disorder;\n* Healthy Volunteer without history of malignancy nor a family member currently being treated for cancer who are undergoing surgery, treatment or during well visits;\n* Biospecimens can be collected with minimal additional risk to the subject during sampling or procedures required for routine patient care.\n* Human samples, specimens and data collected on IRB approved protocols that are now closed\n* Ability of subject, Legally Authorized Representative (LAR), or parent\u002Flegal guardian of children \\\u003C=18 to understand and be willing to sign an IRB-approved informed consent document that permits the use of the tumor and other samples for genomic-based molecular characterization projects.\n\nInclusion Criteria for Social and Behavioral Outcome Interviews:\n\n* Parent\u002Fcaregiver of a participating pediatric or adult patient who is being treated for, or who has previously been treated for any form of pediatric cancer.\n* Must be able to give consent and sign the informed consent document.\n* Able to understand the English language.\n\nEXCLUSION CRITERIA:\n\nNone","4 Weeks",{"count":270,"type":22},6035,"Background:\n\n\\- Laboratory investigators who are studying common childhood cancers are interested in developing a tissue repository to collect and store blood, serum, tissue, urine, or tumors of children who have cancer or adults who have common childhood cancers. To develop this repository, additional samples will be collected from children and adults who have been diagnosed with common childhood cancers such as leukemia and tumors of the central nervous system.\n\nObjectives:\n\n\\- To collect and store blood, serum, tissue, urine, or tumor samples of children who have cancer or adults who have common childhood cancers.\n\nEligibility:\n\n* Individuals who have been diagnosed with a common childhood cancer (e.g., leukemia) regardless of patient age.\n* Children, adolescents, and adults who have been diagnosed with a type of cancer more commonly found in adults.\n\nDesign:\n\n* Extra blood, serum (the liquid part of blood), tissue, urine, or tumor samples will be collected from participants at a time when sampling is required for medical care or as part of a research study.\n* No additional procedures will be performed for the sole purpose of obtaining additional tumor tissue, aside from what is required for clinical care.",[273,105,28,274,275],"Sarcoma","Retinoblastoma","Renal Cancer",[277,278,279,280,281,115,282,56,273,283,28],"Genomics","Proteomics","Tissue Repository","Omics","Cell Lines","Pediatric Cancer","Kidney Cancer","2026-08-01",{"date":257,"type":33},{"date":287,"type":33},"2010-04-21",{"name":90,"class":91},5,{"id":291,"slug":292,"hasResults":12,"nctId":293,"briefTitle":294,"officialTitle":295,"acronym":4,"eligibilityCriteria":296,"healthyVolunteers":12,"sex":17,"minAge":4,"maxAge":4,"enrollmentInfo":297,"targetDuration":4,"studyType":23,"phases":299,"briefSummary":300,"conditions":301,"keywords":303,"overallStatus":59,"whyStopped":4,"lastUpdateSubmitDate":306,"lastUpdatePostDateStruct":307,"startDateStruct":309,"completionDateStruct":311,"leadSponsor":313,"locationsCount":314},"100520544","phase-2-a-study-of-a-vaccine-in-combination-with-beta-glucan-in-people-with-neuroblastoma-100520544","NCT06057948","A Study of a Vaccine in Combination With Beta-glucan in People With Neuroblastoma","Phase II Trial of a Bivalent Vaccine With the Immunological Adjuvant OPT-821 (QS-21), in Combination With Randomization of Oral β-glucan, for High-Risk Neuroblastoma","Inclusion Criteria:\n\n* Diagnosis of NB as defined by international criteria,102 i.e., histopathology (confirmed by the MSK Department of Pathology) or BM metastases plus high urine catecholamine levels, or positivity in MIBG scan.\n* HR-NB as defined by risk-related treatment guidelines and international criteria,102 i.e., metastatic\u002Fnon-localized disease with MYCN amplification (any age), metastatic disease \\>18 months old, MYCN-amplified localized disease (any age), or disease resistant to standard chemotherapy.\n* HR-NB (as defined above) and in 1) first CR at ≥ 6 months from initiation of immunotherapy using anti-GD2 antibody, or 2) second or subsequent CR (achieved after treatment for PD). CR is defined according to the International Neuroblastoma Response Criteria. Patients with positive MIBG scan but negative FDG-PET scan, and CR in BM, are eligible. For those patients with a positive MIBG scan, the negative FDG-PET scan can be per PI discretion to repeat within the 45 day window period prior to enrollment.\n* Patients with grade 3 toxicities or less using the Common Toxicity Criteria (Version 5.0) developed by the National Cancer Institute of the USA (CTCAE v5.0) related to hematologic, cardiac, neurological, pulmonary, renal, hepatic or gastrointestinal function as determined by blood tests or physical exam. Plus:\n\n  * Absolute neutrophil count (ANC) ≥ 500\u002Fmcl\n  * Absolute lymphocyte count ≥ 500\u002Fmcl\n* \\>21 and \\\u003C180 days between completion of systemic therapy and 1st vaccination.\n* A negative pregnancy test is required for patients with child-bearing capability\n* Signed informed consent indicating awareness of the investigational nature of this program.\n\nExclusion Criteria:\n\n* Patients with grade 4 hematologic, cardiac, neurological, pulmonary, renal, hepatic or gastrointestinal function as determined by blood tests or physical exam, using the Common Toxicity Criteria (Version 5.0) developed by the National Cancer Institute of the USA.\n* History of allergy to KLH, QS-21, OPT-821, or glucan\n* Prior treatment with this vaccine.\n* Active life-threatening infection requiring systemic therapy.\n* Inability to comply with protocol requirements.",{"count":298,"type":22},94,[25],"The purpose of this study is to test which treatment schedule of β-glucan with bivalent vaccine is more effective for participants with high-risk neuroblastoma that is in complete remission.",[28,225,302],"Metastatic Neuroblastoma",[28,225,302,304,230,305],"HR-NB","23-198","2026-07-27",{"date":308,"type":33},"2026-07-28",{"date":310,"type":33},"2023-09-21",{"date":312,"type":22},"2027-03-21",{"name":230,"class":40},7,{"id":316,"slug":317,"hasResults":12,"nctId":318,"briefTitle":319,"officialTitle":320,"acronym":4,"eligibilityCriteria":321,"healthyVolunteers":12,"sex":17,"minAge":322,"maxAge":323,"enrollmentInfo":324,"targetDuration":4,"studyType":23,"phases":326,"briefSummary":327,"conditions":328,"keywords":4,"overallStatus":59,"whyStopped":4,"lastUpdateSubmitDate":332,"lastUpdatePostDateStruct":333,"startDateStruct":334,"completionDateStruct":336,"leadSponsor":338,"locationsCount":340},"100546451","phase-1-substudy-01a-zilovertamab-vedotin-in-pediatric-and-young-adult-participants-with-hematologic-malignancies-or-solid-tumors-mk-9999-01alightbeam-u01-100546451","NCT06395103","Substudy 01A: Zilovertamab Vedotin in Pediatric and Young Adult Participants With Hematologic Malignancies or Solid Tumors (MK-9999-01A\u002FLIGHTBEAM-U01)","LIGHTBEAM-U01 Substudy 01A: A Phase 1\u002F2 Substudy to Evaluate the Safety and Efficacy of Zilovertamab Vedotin in Pediatric and Young Adult Participants With Hematologic Malignancies or Solid Tumors","The main inclusion and exclusion criteria include but are not limited to the following:\n\nInclusion Criteria:\n\n* For hematological malignancies: Confirmed diagnosis of B-precursor B-ALL or DLBCL\u002FBurkitt lymphoma according to World Health Organization (WHO) classification of neoplasms of the lymphoid tissues.\n* For solid tumor malignancies: Histologically confirmed diagnosis of neuroblastoma or Ewing sarcoma.\n\nExclusion Criteria:\n\n* History of solid organ transplant.\n* Clinically significant (ie, active) cardiovascular disease.\n* Known history of liver cirrhosis.\n* Ongoing Grade \\>1 peripheral neuropathy.\n* Demyelinating form of Charcot-Marie-Tooth disease.\n* Diagnosed with Down syndrome.\n* Ongoing graft-versus-host disease (GVHD) of any grade or receiving systemic GVHD treatment or prophylaxis.\n* History of human immunodeficiency virus (HIV) infection.\n* Contraindication or hypersensitivity to any of the study intervention components.\n* Received prior radiotherapy within 4 weeks of start of study intervention. Participants must have recovered from all radiation-related toxicities.\n* Ongoing, chronic corticosteroid therapy (exceeding 10 mg daily of prednisone equivalent). Prednisone equivalent dosing must have been stable for at least 4 weeks before Cycle 1 Day 1 (C1D1).\n* Received a strong cytochrome P450 3A4 (CYP3A4) inhibitor within 7 days or a strong CYP3A4 inducer within 14 days before the start of study intervention or expected requirement for chronic use of a strong CYP3A4 inhibitor or inducer during the study intervention period and for 30 days after the last dose of study intervention\n* Received prior systemic anticancer therapy including investigational agents within 4 weeks before the first dose of study intervention (except for prophylactic intrathecal chemotherapy and\u002For cytoreductive therapy with steroids\u002Fhydroxyurea.\n* Received a live or live-attenuated vaccine within 30 days before the first dose of study intervention. Administration of killed vaccines is allowed.\n* Has received an investigational agent or has used an investigational device within 4 weeks prior to study intervention administration.\n* Known additional malignancy that is progressing or has required active treatment within the past 1 year.\n* Active infection requiring systemic therapy.\n* Known history of Hepatitis B or known active Hepatitis C virus infection.\n* Participants who have not adequately recovered from major surgery or have ongoing surgical complications.","6 Months","25 Years",{"count":325,"type":22},90,[53,25],"Substudy 01A is part of a platform study. The purpose of this study is to assess the efficacy and safety of zilovertamab vedotin in pediatric participants with relapsed or refractory B-cell acute lymphoblastic leukemia (B-ALL), diffuse large B-cell lymphoma (DLBCL)\u002FBurkitt lymphoma, or neuroblastoma and in pediatric and young adult participants with Ewing sarcoma.",[329,330,331,28,141],"B-cell Acute Lymphoblastic Leukemia","Diffuse Large B-cell Lymphoma","Burkitt Lymphoma","2026-07-24",{"date":306,"type":33},{"date":335,"type":33},"2024-08-16",{"date":337,"type":22},"2029-03-31",{"name":339,"class":67},"Merck Sharp & Dohme LLC",71,{"id":342,"slug":343,"hasResults":12,"nctId":344,"briefTitle":345,"officialTitle":346,"acronym":4,"eligibilityCriteria":347,"healthyVolunteers":12,"sex":17,"minAge":348,"maxAge":349,"enrollmentInfo":350,"targetDuration":4,"studyType":23,"phases":352,"briefSummary":353,"conditions":354,"keywords":356,"overallStatus":59,"whyStopped":4,"lastUpdateSubmitDate":361,"lastUpdatePostDateStruct":362,"startDateStruct":364,"completionDateStruct":366,"leadSponsor":368,"locationsCount":125},"100556699","phase-2-n10-a-study-of-reduced-chemotherapy-and-monoclonal-antibody-mab-based-therapy-in-children-with-neuroblastoma-100556699","NCT06528496","N10: A Study of Reduced Chemotherapy and Monoclonal Antibody (mAb)-Based Therapy in Children With Neuroblastoma","N10: Reduced Therapy for High-Risk Neuroblastoma","Inclusion Criteria:\n\n* Diagnosis of NB as defined by histopathology, BM metastases plus high urine catecholamine levels, or positivity in MIBG scan.\n* HR-NB, defined as MYCN-amplified stage L2\u002FM\u002FMS at any age and stage M in patients \\>18 months old.\n* No more than one prior cycle of HR-NB chemotherapy\n* Age \\\u003C19 years.\n* Signed informed consent indicating awareness of the investigational nature of this treatment.\n\nExclusion Criteria:\n\n* Severe dysfunction of major organs, i.e., renal, cardiac, hepatic, neurologic, pulmonary, hematologic, or gastrointestinal toxicity \\>\u002F= to grade 3\n\n  °Organ dysfunction due to direct effects of primary tumor or metastatic disease, or due to paraneoplastic syndromes associated with neuroblastoma, will not be considered in exclusion criteria. Similarly, complications of interventions to control above direct effects (e.g., steroid- induced hyperglycemia or hypertension) will also not be considered in exclusion criteria\n* Inability to comply with protocol requirements\n* Pregnancy","18 Months","19 Years",{"count":351,"type":22},45,[25],"The purpose of this study is to find out whether N10 chemotherapy is a safe and effective treatment for children with high-risk neuroblastoma.",[225,28,355],"Childhood Neuroblastoma",[357,254,358,359,230,360],"High-risk neuroblastoma","childhood neuroblastoma","N10","24-144","2026-07-21",{"date":363,"type":33},"2026-07-22",{"date":365,"type":33},"2024-07-22",{"date":367,"type":22},"2029-07-22",{"name":230,"class":40},{"id":370,"slug":371,"hasResults":12,"nctId":372,"briefTitle":373,"officialTitle":373,"acronym":4,"eligibilityCriteria":374,"healthyVolunteers":12,"sex":17,"minAge":18,"maxAge":4,"enrollmentInfo":4,"targetDuration":4,"studyType":246,"phases":4,"briefSummary":375,"conditions":376,"keywords":377,"overallStatus":256,"whyStopped":4,"lastUpdateSubmitDate":361,"lastUpdatePostDateStruct":384,"startDateStruct":4,"completionDateStruct":4,"leadSponsor":386,"locationsCount":125},"100161136","131i-labeled-mibg-for-refractory-neuroblastoma-a-compassionate-use-protocol-100161136","NCT01370330","131I-Labeled MIBG for Refractory Neuroblastoma: A Compassionate Use Protocol","Inclusion Criteria:\n\n* Diagnosis: Refractory or relapsed neuroblastoma with original diagnosis based on tumor histopathology or elevated urine catecholamines with typical tumor cells in the bone marrow. Refractory, progressive or metastatic pheochromocytoma\u002Fparaganglioma or related tumor.\n* MIBG uptake: Tumors must be shown to be MIBG avid within 6 weeks prior to enrollment\n* Age \\> 1 year and able to cooperate with radiation safety restrictions during therapy period. Patients with pheochromocytoma\u002Fparaganglioma and related tumors must be between 1 and 12 years of age.\n* Life Expectancy: greater than 6 weeks.\n* Lansky and Karnofsky Performance Status: 60% or higher.\n* Disease status: Failure to respond to standard therapy (usually combination chemotherapy with or without radiation and surgery) or development of progressive disease at any time (any new lesion or an increase in size of \\>25% of a pre-existing lesion). Disease evaluable by MIBG scan must be present within 6 weeks of study entry and subsequent to any intervening therapy.\n* Stem cells: Patients must have an autologous hematopoietic stem cell product available for re-infusion after MIBG treatment at doses of \\>12 mCi\u002Fkg if needed. The minimum quantity for purged or unpurged peripheral blood stem cells is 1.0 x 10\\^6 cluster of differentiation 34 (CD34)+ cells\u002Fkg (optimum \\> 2 x 10\\^6 CD34+ cells\u002Fkg). The minimum dose for bone marrow is 1.0 x 10\\^8 mononuclear cells\u002Fkg (optimum \\> 2.0 x 10\\^8 mononuclear cells\u002Fkg). If no stem cells are available, then the dose of 131I-MIBG should be \\\u003C12 mCi\u002Fkg .\n* Prior Therapy: Patients may enter this study with or without re-induction therapy for recurrent tumor. Patients must have fully recovered from the toxic effects of any prior therapy. At least 2 weeks should have elapsed since any anti-tumor therapy and the patient must meet hematologic criteria below. Three months should have elapsed in the case of completing radiation to any of the following fields: craniospinal, total abdominal, whole lung, total body irradiation). Cytokine therapy \\[eg granulocyte-colony stimulating factor (G-CSF), granulocyte-macrophage colony-stimulating factor (GM-CSF), interleukin-6 (IL-6), erythropoietin\\] must be discontinued a minimum or 24 hours prior to MIBG therapy. Prior 131I-MIBG therapy is allowed if \\> 6 months previous and if the patient has adequate hematopoietic stem cells available and if cumulative 131I-MIBG dose will not exceed 60 mCi\u002Fkg.\n* Organ Function\n* Liver function: bilirubin \\\u003C2x normal and aspartate aminotransferase (AST)\u002FAlanine aminotransferase (ALT) \\\u003C 10x normal.\n* Kidney function: Creatinine less than or equal to 2\n* Hematopoietic Criteria Patients must have adequate hematopoietic function (without transfusion): absolute neutrophil count (ANC) \\>.750 x 10E9\u002FL; Platelets \\>50 x 10E9\u002FL if stem cells are not available; if stem cells are available, the patient should be independent of platelet transfusions with a platelet count of at least 20 x 10E9\u002FL. Hemoglobin \\>10g\u002Fdl at time of treatment (transfusion allowed). Patients with granulocytopenia and\u002For thrombocytopenia due to tumor metastatic to the bone marrow may be eligible after discussion with study chair or designee.\n* Normal lung function as manifested by no dyspnea at rest or exercise intolerance, no oxygen requirement.\n* No clinically significant cardiac dysfunction\n* Signed informed consent: The patient and\u002For the patient's legally authorized guardian must acknowledge in writing that consent to become a study subject has been obtained, in accordance with institutional policies approved by the U.S. Department of Health and Human Services.\n\nExclusion Criteria:\n\n* Patients with disease of any major organ system that would compromise their ability to withstand therapy. Any significant organ impairment should be discussed with the Study Chair or Vice Chair prior to patient entry.\n* Because of the teratogenic potential of the study medications, no patients who are pregnant or lactating will be allowed. Patients of childbearing potential must practice an effective method of birth control while participating on this study, to avoid possible pregnancy.\n* Patients who are on hemodialysis.\n* Patients with active infections that meet grade 3-4 toxicity criteria.\n* Patients with pheochromocytoma or paraganglioma who have any proteinuria on urinalysis must have a 24-hr urine collection for protein. If there is proteinuria above the reference range on a 24-hour urine collection, they are excluded due to increased risk of respiratory complications.","This is a compassionate use protocol to allow patients with advanced neuroblastoma palliative access to 131I-metaiodobenzylguanidine (131I-MIBG).",[28],[28,251,252,378,379,380,381,382,383],"Resistant","Relapsed","Treatment","University of California, San Francisco","Pediatric","Oncology",{"date":385,"type":33},"2026-07-23",{"name":387,"class":40},"Kieuhoa Vo",{"id":389,"slug":390,"hasResults":12,"nctId":391,"briefTitle":392,"officialTitle":392,"acronym":4,"eligibilityCriteria":393,"healthyVolunteers":12,"sex":17,"minAge":394,"maxAge":395,"enrollmentInfo":396,"targetDuration":4,"studyType":23,"phases":398,"briefSummary":399,"conditions":400,"keywords":401,"overallStatus":59,"whyStopped":4,"lastUpdateSubmitDate":407,"lastUpdatePostDateStruct":408,"startDateStruct":410,"completionDateStruct":412,"leadSponsor":414,"locationsCount":125},"100592626","phase-1-phase-i-trial-of-rhil-15-plus-dinutuximab-plus-irinotecantemozolomide-for-children-and-young-adults-with-relapsedrefractory-neuroblastoma-100592626","NCT06995872","Phase I Trial of rhIL-15 Plus Dinutuximab Plus Irinotecan\u002FTemozolomide for Children and Young Adults With Relapsed\u002FRefractory Neuroblastoma","* ELIGIBILITY CRITERIA:\n* Disease Requirements:\n\n  * Histologic diagnosis: Participants must have pathology-confirmed neuroblastoma (no time limit). Old reports and tissue blocks can be used if available. Confirmation of disease will be based on the review of pathology at the NIH any time before starting and is based on one of the following: (1) A confirmed pathological diagnosis made from tumor tissue by light microscopy (with or without immunohistology or electron microscopy), (2) the combination of bilateral bone marrow aspirate and trephine biopsy containing confirmed tumor cells (e.g., syncytia or immunocytologically positive clumps of cells) and increased levels of urinary catecholamine metabolites.\n  * Active disease status: Participants must have relapsed and\u002For refractory disease after receiving frontline chemotherapy and at least one salvage treatment (can include dinutuximab, temozolomide, and\u002For irinotecan), with no alternative curative options.\n  * Documentation of disease: Participants must have evaluable disease according to the International Neuroblastoma Response Criteria (INRC). This means they must have an area of active disease that can be identified by MIBG, PET, MRI\u002FCT, or bone marrow studies. Participants must have at least ONE of the following at the time of enrollment:\n\n    * Measurable tumors on magnetic resonance imaging (MRI) or computed tomography (CT) scan. Measurable is defined as \\>=10 mm in at least one dimension.\n    * Either 123MIBG-avid lesion detected on 123MIBG scan with positive uptake at a minimum of one site or FDG-PET-avid lesion detected on FDG-PET scan with positive uptake at a minimum of one site. This site must represent disease recurrence after completion of therapy, progressive disease on therapy, or refractory disease during induction.\n    * Participants with resistant\u002Frefractory soft tissue disease that is not 123MIBG avid or does not demonstrate increased FDG uptake on PET scan must undergo a biopsy to document the presence of viable neuroblastoma. Biopsy is not required for participants who have a new site of soft tissue disease (radiographic evidence of disease progression) regardless of whether progression occurs while receiving therapy or after completion of therapy.\n    * Participants with bone marrow disease in at least one sample from bilateral bone marrow biopsies will be eligible (documented neuroblastoma cells).\n    * Participants with a history of CNS disease must have no clinical or radiological evidence of active CNS disease at the time of study enrollment.\n* Prior Therapy:\n\nPotential trial participants should have recovered from clinically significant adverse events of their most recent therapy\u002Fintervention prior to enrollment.\n\n* Chemo-immunotherapy: Participants are eligible if they have received prior therapy with dinutuximab\u002Ftemozolomide\u002Firinotecan, even if they may not have responded previously\n* Myelosuppressive chemotherapy: Potential trial participants should have recovered from clinically significant myelosuppression.\n* Non-chemotherapy anti-neoplastic agents: With anti-cancer agents not known to be myelosuppressive (e.g., not associated with reduced platelet or ANC counts), sufficient time needs to have passed for the drug to have cleared the body. This is to prevent overlapping non-hematological toxicity.\n* Anti-GD2 antibody: toxicity related to prior antibody therapy must be recovered to grade \\\u003C=1.\n* Radiation therapy: Given the possible negative effect of radiation on bone marrow cells and count recovery after chemotherapy, potential trial participants should have recovered from clinically significant radiation-induced myelosuppression. Palliative radiation while on study is not permitted.\n* Stem cell transplants (SCT): After autologous stem cell transplants or stem cell infusions (including stem cell infusions given as supportive care following 131 I-MIBG therapy), all hematologic and other eligibility criteria must have been met.\n* 131I-MIBG therapy: After therapeutic 131 I-MIBG, all hematologic and other eligibility criteria must have been met.\n* Participants who have received drugs that are strong inducers or inhibitors of CYP3A4 within 7 days before study enrollment are not eligible.\n\n  -Age Requirement:\n* Age \\>= 3 years and \\\u003C= 35 years at the time of enrollment.\n\n  -Clinical Performance Status:\n* Participants \\>= 16 years of age: Karnofsky \\>= 50 percent; Participants \\\u003C 16 years of age: Lansky scale \\>= 50 percent. Participants who are unable to walk because of paralysis, but who are upright in a wheelchair will be considered ambulatory to calculate the performance score.\n\n  -Adequate Organ and Marrow Function as Defined Below:\n* Absolute neutrophil count: \\>= 750\u002FmcL (must be off of G-CSF for at least 1 week)\n* Platelets: \\>= 75,000\u002FmcL (transfusion-independent, unless patient has bone marrow disease in which case transfusions are permitted to reach this threshold)\n* Total bilirubin: \\\u003C=2 X ULN (except in the case of participants with documented Gilbert s disease \\\u003C 3x ULN)\n* AST(SGOT)\u002FALT(SGPT): \\\u003C=3 X institutional upper limit of normal\n* Creatinine: \\\u003C= the maximum for age listed in the table below OR\n* Measured creatinine clearance: \\>= 60 mL\u002Fmin\u002F1.73 m\\^2 for participants with creatinine levels above the max listed below per age:\n\n  * Age (Years) \\\u003C=5, Maximum Serum Creatinine \\\u003C= 0.8 (mg\u002FdL)\n  * Age (Years) 6 to \\\u003C= 10, Maximum Serum Creatinine \\\u003C=1.0 (mg\u002FdL)\n  * Age (Years) \\>10, Maximum Serum Creatinine \\\u003C= 1.2 (mg\u002FdL)\n* Cardiac function: Left ventricular ejection fraction \\>= 52 percent.\n* Pulmonary Function\n\n  * Baseline oxygen saturation \\>92 percent on room air at rest\n  * Participants with respiratory symptoms clinically concerning for decreased pulmonary function must have a DLCO\u002Fadjusted \\> 45 percent. For children who are unable to cooperate for PFTs, they must not have dyspnea at rest or a known requirement for supplemental oxygen.\n\n    * Given the teratogenic effects of the therapy, participants of childbearing or child fathering potential must agree to be abstinent or use highly effective contraception (hormonal; intrauterine device; surgical sterilization). This restriction will be from the time of enrollment on this study and for four months (in persons who can father children) or six months (in participants who can bear children) after completing therapy. Participants may also confirm with their partners that they are using a highly effective method.\n    * Participants who are nursing or plan to nurse must agree to discontinue\u002Fpostpone nursing while on study therapy since it cannot be ruled out with certainty that any of the investigational drugs can be transmitted via breast milk.\n    * Ability of participant or parents\u002Flegal guardian to understand and sign a written informed consent document.\n\nEXCLUSION CRITERIA:\n\n* Presence of pericardial effusion.\n* Chronic immunosuppression (physiologic steroid supplementation is allowed but any chronic immunosuppressant like steroids or other modulators are not allowed)\n* Major surgery within 4 weeks prior to initiation of study therapy\n* Current\u002Factive human immunodeficiency virus (HIV) infection, as measured by seropositivity for HIV antibody.\n* Current\u002Factive HBV\u002FHCV infection as measured by seropositivity for Hepatitis C or positive for Hepatitis B surface antigen (HBsAg).\n* History of severe, immediate hypersensitivity reaction attributed to compounds of similar chemical or biological composition to irinotecan and temozolomide used in study.\n* Participants with uncontrolled intercurrent illness or any other significant condition(s) that would make participation in this protocol unreasonably hazardous.\n* Positive serum or urine beta-HCG pregnancy test performed at screening due to the teratogenic effects of chemotherapy.\n* Participants with a prior or concurrent malignancy whose natural history or treatment with the safety or efficacy assessment of the investigational regimen.\n* Participants with \\>= grade 2 diarrhea at the time of entry.\n* Participants who are unable to tolerate oral\u002Fnasogastric\u002Fgastrostomy medications. Additionally, participants with significant malabsorption will not be eligible for this trial.\n* Participants with uncontrolled infection.\n* Participants with a history of Grade 4 allergic reactions to anti-GD2 antibodies or reactions that required permanent discontinuation of the anti-GD2 therapy.","3 Years","35 Years",{"count":397,"type":22},40,[53],"Background:\n\nNeuroblastoma is a type of cancer that causes tumors in nerves. It affects mainly infants and toddlers, and it causes about 15 percent of cancer-related deaths in children.\n\nObjective:\n\nTo test a new drug (rhIL-15), combined with 3 standard cancer drugs, in people with neuroblastoma.\n\nEligibility:\n\nPeople aged 3 to 35 years with neuroblastoma that did not respond or returned after standard treatment.\n\nDesign:\n\nParticipants will be screened. They will have a physical exam with blood and urine tests. They will have imaging scans and tests of their heart and lungs. They will have a bone marrow biopsy: A sample of tissue and fluid from inside a bone will be removed with a large needle.\n\nParticipants will be treated in 21-day cycles. They may have up to 4 treatment cycles.\n\nrhIL-15 is given through a needle into a vein over 5 to 7 days during the first week of each cycle. Participants will stay in the hospital while they are receiving the rhIL-15.\n\nStarting in the second week of the second cycle, participants will receive other drugs for treating cancer. They will have no study treatments during the third week of each cycle.\n\nParticipants will visit the clinic at least 2 times a week throughout all 4 treatment cycles. They will have a physical exam and blood tests during these visits. Imaging scans, bone marrow biopsy, and other tests will be repeated at the end of cycles 2 and 4.\n\nParticipants will have a follow-up visit 6 months after treatment ends. This visit will include a physical exam with blood and urine tests.",[28],[402,403,404,405,28,406],"Irinotecan","Temozolomide","ch14.18","IL-15","Dinituximab","2026-07-17",{"date":409,"type":33},"2026-07-20",{"date":411,"type":33},"2025-10-21",{"date":413,"type":22},"2029-06-01",{"name":90,"class":91},{"id":416,"slug":417,"hasResults":12,"nctId":418,"briefTitle":419,"officialTitle":420,"acronym":4,"eligibilityCriteria":421,"healthyVolunteers":12,"sex":17,"minAge":18,"maxAge":422,"enrollmentInfo":423,"targetDuration":4,"studyType":23,"phases":425,"briefSummary":426,"conditions":427,"keywords":428,"overallStatus":59,"whyStopped":4,"lastUpdateSubmitDate":434,"lastUpdatePostDateStruct":435,"startDateStruct":436,"completionDateStruct":438,"leadSponsor":440,"locationsCount":41},"100550666","phase-2-nant-2021-01-phase-ii-sting-sequential-temozolomide-irinotecan-nk-cells-and-gd2-mab-trial-100550666","NCT06450041","NANT 2021-01 Phase II STING (Sequential Temozolomide, Irinotecan, NK Cells and GD2 mAb) Trial","Phase II Study of Ex-Vivo Expanded Allogeneic Universal Donor TGFβi NK Cell Infusions in Combination With Temozolomide, Irinotecan, Dinutuximab, and Sargramostim in Patients With Relapsed or Refractory Neuroblastoma The STING (Sequential Temozolomide, Irinotecan, NK Cells and GD2 mAb) Trial","Inclusion Criteria:\n\n* Patients must be ≥ 1 year and ≤31 years of age at the time of enrollment on the study.\n* Patients must have a diagnosis of neuroblastoma either by histologic verification of neuroblastoma and\u002For demonstration of tumor cells in the bone marrow with increased urinary catecholamines.\n* Patients must have high-risk neuroblastoma according to COG risk classification at the time of study registration. Patients whose disease was initially considered low or intermediate risk but then reclassified as high-risk neuroblastoma prior to enrollment also meet this criteria.\n* Patients must have at least ONE of the following:\n\n  1\\) Recurrent\u002Fprogressive disease after the diagnosis of high risk neuroblastoma at any time prior to enrollment - regardless of response to frontline therapy. (Note that this excludes patients initially considered low or intermediate risk that progressed to high risk disease but have not progressed after the diagnosis of high risk neuroblastoma).\n\n  2\\) If no prior history of recurrent\u002Fprogressive disease since the diagnosis of high-risk neuroblastoma,\n\n  2a) Refractory disease: A best overall response of no response\u002Fstable disease since diagnosis of high-risk neuroblastoma AND after at least 4 courses of induction therapy.\n\n  2b) Persistent disease: A best overall response of partial response since diagnosis of high-risk neuroblastoma AND after at least 4 courses of induction therapy\n* Patients must have at least ONE of the following (lesions may have received prior radiation therapy as long as they meet the other criteria listed below) based on institutional assessment:\n\n  1\\) Bone Sites\n  1. a) MIBG avid tumors: patients must meet one of the following criteria:\n\n     a. Patients with recurrent\u002Fprogressive or refractory disease: i. Must have at least one MIBG avid bone site on planar imaging OR ii. Must have \\> 2 avid bone lesions on SPECT. iii. A biopsy is not required unless the above imaging criteria are not met. b. Patients with persistent disease: i. If a patient has 3 or more MIBG avid sites by planar or SPECT imaging (including soft tissue and\u002For bone), then no biopsy is required.\n\n     ii. If a patient has only 1 or 2 MIBG avid sites by planar or SPECT imaging (including soft tissue and\u002For bone) then biopsy confirmation of neuroblastoma and\u002For ganglioneuroblastoma in at least one MIBG avid site present at the time of enrollment is required. Bone lesions may be biopsied at any time point prior to enrollment.\n\n  1b) For MIBG non-avid tumors, patients must have biopsy confirmation of neuroblastoma and\u002For ganglioneuroblastoma from a lesion at any time prior to enrollment of at least one site (with or without FDG-PET uptake).\n\n  2\\) Bone Marrow Any amount of tumor cells in the bone marrow (including neuroblasts, mature and maturing ganglion cells) done at the time of study enrollment based on routine morphology and\u002For immunohistochemistry in at least one sample from bilateral aspirates and biopsies.\n\n  3\\) Soft Tissue Sites\n\n  3a) At least one soft tissue lesion that meets criteria for a TARGET lesion as defined by:\n  1. SIZE: Lesion can be accurately measured in at least one dimension with a longest diameter ≥ 10 mm, or for discrete lymph nodes ≥ 15mm on short axis. Lesions meeting size criteria will be considered measurable.\n  2. In addition to size, a lesion needs to meet ONE of the following criteria except for patients with parenchymal CNS lesions which will only need to meet size criteria:\n\n     1. For MIBG avid tumors: lesion must be MIBG avid and meet one of the following criteria:\n\n  \u003C!-- -->\n\n  1. For patients with recurrent\u002Fprogressive or refractory disease:\n\n     i. No biopsy is required\n  2. For patients with persistent disease:\n\n     i. If a patient has 3 or more MIBG avid sites by planar or SPECT imaging (including soft tissue and\u002For bone), then no biopsy is required.\n\n     ii. If a patient has only 1 or 2 MIBG avid sites by planar or SPECT imaging (including soft tissue and\u002For bone), then biopsy confirmation of neuroblastoma and\u002For ganglioneuroblastoma in at least one MIBG avid site present at the time of enrollment is required. Soft tissue lesions may be biopsied at any time point prior to enrollment.\n\n     b. For MIBG non-avid tumors, patient must have biopsy confirmation of neuroblastoma and\u002For ganglioneuroblastoma at any time prior to enrollment from soft tissue lesion (with or without FDG uptake) present at time of enrollment.\n\n  3b) At least one non-target soft tissue lesion that is not measurable, but had a biopsy positive for neuroblastoma and\u002For ganglioneuroblastoma at any time prior to enrollment OR is MIBG avid on planar imaging.\n* Patients must have a Lansky (≤ 16 years) or Karnofsky (\\> 16 years) score of ≥ 50 (Appendix I).\n\nNote: Patients who are unable to walk because of paralysis, but who are up in a wheelchair, will be considered ambulatory for the purpose of assessing the performance score.\n\n* Patients must have fully recovered from the acute toxic effects of all prior chemotherapy, immunotherapy, or radiotherapy prior to study registration.\n* Patients must not have received the therapies indicated below after disease evaluation or within the specified time period prior to registration on this study as follows:\n\n  1. Myelosuppressive chemotherapy: must not have received within 2 weeks prior to registration.\n  2. Biologic anti-neoplastics- agents not known to be associated with reduced platelet or ANC counts (including retinoids): must not have received within 7 days prior to registration.\n  3. Monoclonal antibodies: must not have received last dose within 14 days of registration and resolution of all toxicities.\n  4. Cellular Therapy (e.g. modified T cells, NK cells, dentritic cells etc.): must not have received within 3 weeks and resolution of all toxicities.\n  5. Radiation: must not have received small port radiation within 7 days prior to registration, large field radiation within 12 weeks, and 131I-MIBG therapy or other radiopharmaceutical within 6 weeks.\n  6. Hematopoietic Stem Cell Transplant- none following myeloblative therapy within 6 weeks\n  7. Any other investigational agents (covered under another IND within 14 days\n  8. Strong inducers or inhibitors of CYP3A4\n* Hematologic Function:\n\nNOTE: No short acting hematopoietic growth factors within 7 days of blood draw documenting eligibility and no long-acting hematopoietic growth factors within 14 days of blood draw documenting eligibility\n\n1. Absolute Neutrophil count ≥750\u002FµL\n2. Platelet count ≥ 75,000\u002FµL, transfusion independent (no platelet transfusions within 7 days of blood draw documenting eligibility)\n\nPatients with known bone marrow metastatic disease will be eligible for study as long as they meet hematologic function criteria above.\n\n* Renal Function Patients must have adequate renal function defined as age-adjusted serum creatinine ≤1.5 ULN for age\n* Liver Function\n\n  1. Total bilirubin ≤ 1.5 x ULN for age; and,\n  2. SGPT (ALT) ≤ 135 U\u002FL (≤ 3x ULN). Note that for ALT, the upper limit of normal for all sites is defined as 45 U\u002FL.\n* Cardiac Function\n\n  1. Normal ejection fraction (≥ 55%) documented by either echocardiogram OR\n  2. Normal fractional shortening (≥ 27%) documented by echocardiogram\n* Pulmonary Function No evidence of dyspnea at rest\n* Reproductive Function All females ≥ Tanner stage 2 and post-menarchal of childbearing potential must have a negative beta-HCG within 7 days prior to study registration. Males and females of reproductive age and childbearing potential must commit to using effective contraception for the duration of their participation.\n* Central Nervous System (CNS) Patients with a history of intraparenchymal or leptomeningeal based CNS disease must have no clinical or radiological evidence of active CNS disease at the time of study enrollment.\n\nPatients with skull-based tumors with direct intracranial extension are eligible as long as there are no neurologic signs or symptoms related to the lesion.\n\nExclusion Criteria:\n\n* Patients who are pregnant, breast feeding, or unwilling to use effective contraception during the study\n* Patients who, in the opinion of the investigator, may not be able to comply with the safety monitoring requirements of the study.\n* Patients with disease of any major organ system that would compromise their ability to withstand therapy.\n* Patients with \\> Grade 2 diarrhea.\n* Patients who have undergone a prior allogeneic stem cell or solid organ transplant.\n* Patients who are on hemodialysis.\n* Patients with an active or uncontrolled infection. Patients on prolonged antifungal therapy are still eligible if they are culture negative, afebrile, and meet other organ function criteria.\n* Patients with known history of human immunodeficiency virus (HIV) infection, hepatitis B, or hepatitis C. Testing is not required in the absence of clinical findings or suspicion.\n* Patients must not have been diagnosed with any other malignancy.\n* Patients with history of Grade 4 Allergic reactions to anti-GD2 antibody therapy or reactions that caused permanent discontinuation of therapy.\n* Patients with history of progressive disease while receiving therapy per ANBL1221.\n* Patient declines participation in the NANT biology study and the site has not been granted a waiver from participation.\n* Systemic Steroids and Immunosuppressive Medications\n* Patients who have received pharmacologic doses of systemic steroids 7 days prior to study registration or likely to require them after study registration.\n\nNote: Exceptions are the following:\n\n1. Patients known to require 2 mg\u002Fkg or less of hydrocortisone (or an equivalent dose of an alternative corticosteroid) as premedication for blood product administration.\n2. The use of conventional doses of inhaled steroids for the treatment of asthma\n3. The use of physiologic doses of steroids for patients with known adrenal insufficiency.\n\n   * Patients on any other immunosuppressive medications (e.g., cyclosporine, tacrolimus) at the time of study registration.","31 Years",{"count":424,"type":22},62,[25],"This is a phase II study looking at patient response to treatment with the combination dinutuximab, temozolomide, irinotecan, and GM-CSF.",[28],[429,430,431,432,433],"Relapsed Neuroblastoma","Refractory Neuroblastoma","Cellular Therapy","Immunotherapy","Chemoimmunotherapy","2026-07-16",{"date":409,"type":33},{"date":437,"type":33},"2024-12-16",{"date":439,"type":22},"2038-12",{"name":39,"class":40},{"id":442,"slug":443,"hasResults":12,"nctId":444,"briefTitle":445,"officialTitle":445,"acronym":4,"eligibilityCriteria":446,"healthyVolunteers":12,"sex":17,"minAge":18,"maxAge":19,"enrollmentInfo":447,"targetDuration":4,"studyType":23,"phases":448,"briefSummary":449,"conditions":450,"keywords":452,"overallStatus":59,"whyStopped":4,"lastUpdateSubmitDate":454,"lastUpdatePostDateStruct":455,"startDateStruct":457,"completionDateStruct":459,"leadSponsor":461,"locationsCount":155},"100595076","phase-1-feasibility-study-of-prolonged-administration-of-naxitamab-irinotecan-and-temozolomide-for-patients-with-relapsed-or-refractory-neuroblastoma-100595076","NCT07027748","Feasibility Study of Prolonged Administration of Naxitamab, Irinotecan, and Temozolomide for Patients With Relapsed or Refractory Neuroblastoma","Inclusion Criteria:\n\n* Histologic Diagnosis: Patients must have had histologic verification of neuroblastoma or ganglioneuroblastoma or demonstration of neuroblastoma cells in the bone marrow with elevated urinary catecholamines \\[i.e. \\> 2 x upper limit of normal (ULN)\\], at the time of initial diagnosis.\n* Relapsed or Refractory Disease Patients must have ONE of the following:\n\n  * 1\\) Any prior episode of recurrent high-risk disease following completion of frontline high-risk therapy. Patients may have received other lines of therapy for treatment of recurrent disease prior to enrolling to this trial.\n  * 2\\) Prior progressive high-risk disease during frontline high-risk therapy. Patients may have received other lines of therapy for treatment of progressive disease prior to enrolling to this trial.\n  * 3\\) Primary resistant\u002Frefractory disease (less than partial response by INRC) detected after the conclusion of at least 4 cycles of aggressive multidrug induction chemotherapy on or according to a high-risk neuroblastoma protocol (examples include ANBL0532, ANBL09P1, ANBL12P1, ANBL1531, ANBL2131) that was treated with additional therapy with the goal of improving remission status prior to enrolling to this trial.\n* Documentation of Disease: Patients must have at least ONE of the following at the time of enrollment:\n\n  * 1\\) Measurable tumor on MRI or CT scan. Measurable is defined as ≥ 10 mm in at least one dimension (or 15 mm in short axis for lymph node) on spiral\u002Fhelical CT or MRI that is MIBG avid or demonstrates increased FDG uptake on PET scan.\n  * 2\\) MIBG-avid lesion detected on MIBG scan with positive uptake at a minimum of one site. This site must represent disease recurrence or known refractory disease at a site not previously radiated.\n  * 3\\) In patients with known MIBG non-avid disease, FDG-avid lesion detected on FDG- PET scan with positive uptake at a minimum of one site. This site must represent disease recurrence or known refractory disease at a site not previously radiated.\n  * Of note, patients with isolated bone marrow only disease are NOT eligible for this trial.\n* Prior Therapy: Prior lines of anticancer therapy allowed as described in eligibility section above by disease status. Washout periods from prior therapy are as follows:\n\n  * Myelosuppressive chemotherapy: Last dose given 14 days prior to enrollment.\n  * Small molecule targeted therapies (anti-neoplastic agents including retinoids): Last dose given 7 days prior to enrollment.\n  * Monoclonal antibodies: Last given at least 7 days or 3 half-lives, whichever is longer, prior to enrollment.\n  * Radiation:\n\n    * Craniospinal irradiation: Last fraction received minimum of six weeks prior to enrollment\n    * All other radiation: Last fraction received minimum of 14 days prior to enrollment\n  * Hematopoietic stem cell transplant: Date of autologous stem cell infusion following myeloablative chemotherapy must have been a minimum of 12 weeks prior to enrollment. Patients are not eligible post allogeneic stem cell transplant.\n  * Cellular therapies (including CAR-T cells, NK cells, other related cellular therapies): 21 days from the last cellular therapy infusion prior to enrollment and recovery from all associated toxicities\n  * 131I-MIBG therapy: Last therapy received a minimum of 6 weeks prior to enrollment.\n* Age: Patients 1 - 30 years of age at the time of enrollment are eligible for this study.\n* Performance level: Patients must demonstrate adequate performance level as measured by Karnofsky ≥ 70% for patients aged 16 years or older, OR Lansky ≥ 70% for patients younger than 16 years. Please see Appendix A for performance score measurement.\n* Participants must meet the following organ and marrow function as defined below:\n* Adequate bone marrow function as defined as BOTH of the following:\n\n  * Peripheral absolute neutrophil count (ANC) ≥ 750\u002FuL. Must be more than 14 days from last administration of long-acting myeloid stimulating factor (e.g. pegfilgrastim) or 7 days from last administration of short- acting myeloid stimulating factor (e.g. filgrastim or sargramostim)\n  * Peripheral platelet count ≥ 75,000\u002FuL. Must be without support, defined as at least 7 days from last platelet transfusion and\u002For platelet stimulating agent.\n* Adequate renal function as defined as EITHER of the following:\n\n  * Radioisotope GFR ≥ 70ml\u002Fmin\u002F1.73 m2\n  * Serum creatinine based on age\u002Fsex as follows:\n  * Age Maximum Serum Creatinine (mg\u002FdL)\n\n    * 1 to \\\u003C 2 years Male 0.6 Female 0.6\n    * 2 to \\\u003C 6 years Male 0.8 Female 0.8\n    * 6 to \\\u003C 12 years Male 1.0 Female 1.0\n    * 12 to \\\u003C 13 years Male 1.2 Female 1.2\n    * 13 to \\\u003C 16 years Male 1.5 Female 1.4\n\n      ---≥ 16 years Male 1.7 Female 1.4\n    * The threshold creatinine values in this Table were derived from the Schwartz formula for estimating GFR.\n* Adequate liver function defined as ALL of the following:\n\n  * Total bilirubin ≤ 1.5 x ULN for age\\*\n  * ALT ≤ 3.0 x ULN for age (≤ 135 U\u002FL). For the purpose of this study, the ULN for ALT is 45 U\u002FL\n  * Albumin \\> 3 g\u002FdL --\\*If patient has known Gilbert syndrome, direct bilirubin should be used to measure liver function instead of total bilirubin. Direct bilirubin must be within normal limits for age for these patients.\n* Adequate cardiac function measured by echocardiogram as defined as EITHER of the following:\n\n  * Shortening fraction of ≥ 27%\n  * Ejection fraction of ≥ 50%\n* Adequate blood pressure as defined by BOTH of the following:\n\n  * Patients must have \\\u003C Grade 2 hypertension AND\n  * Be on no more than one standing antihypertensive\n* Adequate pulmonary function: Patients must have adequate pulmonary function, defined as:\n\n  * No dyspnea at rest\n  * No exercise intolerance\n  * Room air O2 saturation \\>94%\n  * Not on chronic oxygen therapy\n* Adequate pancreatic function, defined as lipase \\\u003C 1.5 x ULN\n* Able to comply with protocol requirements\n* Adequate contraception: The effects of naxitamab on the developing human fetus are unknown. For this reason and because other therapeutic agents used in this trial are known to be teratogenic, participants with potential to become pregnant or to impregnate a partner must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to study entry and for the duration of study participation. Should a participant become pregnant or suspect they are pregnant while they or their partner is participating in this study, they should inform the treating physician immediately. Patients treated or enrolled on this protocol must also agree to use adequate contraception prior to the study, for the duration of study participation, and 4 months after completion of protocol therapy.\n* Ability to understand and\u002For the willingness of their parent or legally authorized representative to sign a written informed consent document.\n\nExclusion Criteria:\n\n* Chronic (more than 2 weeks duration) diarrhea \\> grade 1\n* Prior receipt of naxitamab\n* Untreated central nervous system (CNS) metastatic disease\n* Pregnant or currently breast feeding: Pregnant participants are excluded from this study because protocol therapy has the potential for teratogenic or abortifacient effects. Because there is an unknown but potential risk for adverse events in nursing infants secondary to treatment of the parent with protocol therapy, participants planning to continue breastfeeding are excluded from the study.\n* Clinically significant arrhythmias, i.e. those that cause clinical symptoms or require medications for control of symptoms\n* Prior allergic reaction to irinotecan or temozolomide\n* Discontinuation of prior irinotecan or temozolomide due to unacceptable toxicity\n* Discontinuation of prior GD2 directed immunotherapy due to unacceptable toxicity other than allergic reaction\n* Serious intercurrent illness\n* Active uncontrolled infection\n* Existing major organ dysfunction CTCAE \\>Grade 2, except for hearing loss and hematological status, kidney, and liver function as described in eligibility criteria\n* Concomitant Medication Restrictions:\n\n  * Patients may not be receiving immunosuppressive medications including pharmacologic doses of glucocorticoids or immunomodulatory agents due to concern for inhibition of antibody effect. Local and inhaled steroid agents are permitted.\n  * Patients may not be receiving concurrent anti-cancer agents or radiotherapy.\n  * Patients may not have received valproic acid within 14 days prior to enrollment.\n  * Patients may not have received strong CYP3A4 inducers, strong CYP3A4 inhibitors, or strong UGT1A1 inhibitors within 14 days prior to enrollment.\n\n    * Because the lists of these agents are constantly changing, it is important to regularly consult a frequently-updated medical reference. As part of the enrollment\u002Finformed consent procedures, the participant will be counseled on the risk of interactions with other agents, and what to do if new medications need to be prescribed or if the participant is considering a new over-the-counter medicine or herbal product.",{"count":136,"type":22},[53],"This research is being done to investigate a treatment regimen of Irinotecan, Temozolomide, and Sargramostin, and an immunotherapy called Naxitamab and whether giving Naxitamab more slowly reduces the side effects for participants with relapsed or refractory neuroblastoma.\n\nThe name of the study drugs involved in this study are:\n\n* Naxitamab (A type of monoclonal antibody)\n* Irinotecan (A standard of care chemotherapy)\n* Temozolomide (A standard of care chemotherapy)\n* Sargramostim (A standard of care, granulocyte-macrophage colony stimulating factor)",[28,451,429,430],"Neuroblastoma Recurrent",[28,451,429,430,453],"Progressive Neuroblastoma","2026-07-13",{"date":456,"type":33},"2026-07-14",{"date":458,"type":33},"2025-06-27",{"date":460,"type":22},"2027-12-01",{"name":462,"class":40},"Steven DuBois, MD",{"id":464,"slug":465,"hasResults":12,"nctId":466,"briefTitle":467,"officialTitle":468,"acronym":4,"eligibilityCriteria":469,"healthyVolunteers":12,"sex":17,"minAge":4,"maxAge":470,"enrollmentInfo":471,"targetDuration":4,"studyType":23,"phases":473,"briefSummary":475,"conditions":476,"keywords":489,"overallStatus":59,"whyStopped":4,"lastUpdateSubmitDate":454,"lastUpdatePostDateStruct":491,"startDateStruct":492,"completionDateStruct":494,"leadSponsor":496,"locationsCount":498},"100588487","early-phase-1-pilot-study-of-it-topotecan-and-maintenance-chemotherapy-for-hr-ebts-in-children--6-years-post-consolidation-100588487","NCT06942039","Pilot Study of IT Topotecan and Maintenance Chemotherapy for HR-EBTs in Children \u003C 6 Years, Post Consolidation","A Pilot Study of Intrathecal Topotecan and Maintenance Chemotherapy in the Post-consolidation Setting for the Treatment of High-risk Embryonal Central Nervous System Tumours in Children Less Than 6 Years of Age","Inclusion Criteria:\n\n1. Tumor Tissue Sample\n2. Age: Patient must be aged ≥ 0 years to ≤ 6 years at the time of definitive confirmation of histologic diagnosis of eligible CNS tumor.\n3. Diagnoses. Participants must have Central nervous system (CNS) HR-EBT including atypical teratoid rhabdoid tumour (ATRT), group 3 and group 4 medulloblastoma (MB), pineoblastoma, CNS neuroblastoma, embryonal tumor with multi-layered rosettes (ETMR including embryonal tumor with abundant neuropil and true rosettes (ETANTR), ependymoblastoma and ETMR not otherwise specified), medulloepithelioma, CNS embryonal tumor with rhabdoid features (INI-1 intact) and CNS embryonal tumor, not otherwise specified. Metastatic disease included. Any extent of resection included.\n4. Cranial and Spine MRI. A baseline MRI brain and spine with and without contrast is required for all patients. cranial MRI (with and without gadolinium) must be done pre-operatively. Post-operatively, cranial MRI (with and without gadolinium) must be done.\n5. Lumbar Puncture (LP) CSF for cytopathology (strongly recommended but not mandatory; if medically feasible). A baseline LP CSF cytology either pre-operatively or post-operatively at least 10 days after definitive surgery for all patients if medically feasible (This is not mandatory and will not make the patient ineligible).\n6. Life expectancy: Patients must have a life expectancy of greater than 8 weeks from diagnosis.\n7. Performance level: Patients must have a performance status corresponding of a Lansky score ≥ 50.\n8. Organ Function Requirements: Participants must have normal organ and marrow function as defined below:\n\nAdequate renal function defined as:\n\n\\- Creatinine clearance (12-24-hour urine collection) or radioisotope glomerular filtration rate (GFR) ≥ 60 ml\u002Fmin\u002F1.73m2\n\nAdequate cardiac function defined as:\n\n* Shortening fraction of ≥ 27% by echocardiogram, or\n* Ejection fraction of ≥ 47% by radionuclide angiogram.\n\nAdequate pulmonary function defined as:\n\n\\- No evidence of dyspnea at rest and a pulse oximetry \\> 94% on room air.\n\nAdequate Bone Marrow Function defined as:\n\n* Peripheral absolute neutrophil count (ANC) \\> 1000\u002FμL\n* Platelet Count \\> 100,000\u002FμL (without transfusion for 3 days)\n* Hemoglobin greater than 8 gm\u002FdL (may have received red blood cell (RBC) transfusions)\n\nAdequate liver function defined as:\n\n* Total bilirubin ≤ 1.5X upper limit of normal (ULN) within normal institutional limits for age (patients with documented Gilbert's Disease may be enrolled with Study Chair approval and total bilirubin ≤ 2.0 × ULN)\n* Alanine Aminotransferase (ALT) and aspartate aminotransferase (AST) ≤ 100 U\u002FL\n\nExclusion criteria:\n\n1. Patients who are receiving any other conventional anti-cancer agents or investigational agents.\n2. Patients who received previous therapy including radiotherapy or chemotherapy other than corticosteroids.\n3. Presence of another malignancy, except if the other primary malignancy is neither currently clinically significant nor requiring active intervention.\n4. Concomitant medications restrictions: Concurrent use of enzyme inducing anticonvulsants (e.g. phenytoin, phenobarbital, and carbamazepine), selected strong inhibitors of cytochrome P450 3A4 include azole antifungals, such as fluconazole, voriconazole, itraconazole, ketoconazole, and strong inducers include drugs such as rifampin, phenytoin, phenobarbitol, carbamazepine, and St. John's wort or CYP450 3A4 stimulators or inhibitors.\n5. Other uncontrollable medical disease: Patient has a severe and uncontrollable medical disease (i.e., uncontrolled diabetes, hyperglycemia, chronic renal disease or active uncontrolled infection), has chronic liver disease (i.e., chronic active hepatitis and cirrhosis), hypercholesterolemia (serum cholesterol \\>300 mg\u002FdL), intercurrent illness including, but not limited to, ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, active hyperparathyroidism, or psychiatric illness\u002Fsocial situations that would limit compliance with study requirements.\n6. Patients who have a known diagnosis of human immunodeficiency virus (HIV) infection, hepatitis B or C.\n7. Ineligible diagnoses for study entry by neuropathology: This includes sonic hedgehog (SHH) and wingless (WNT) MBs, all ependymomas, all choroid plexus carcinomas, all high grade glial and glio-neuronal tumors, all diffuse midline gliomas, all primary CNS germ cell tumors, all primary CNS sarcomas, all primary or metastatic CNS lymphomas and solid leukemic lesions (chloromas, granulocytic sarcomas).\n8. The participant or parent(s)\u002Fguardian(s) cannot comply with the study visit schedule and other protocol requirements, in the investigator's opinion.\n\nAll patients must meet inclusion\u002Fexclusion criteria prior to starting any protocol therapy.","6 Years",{"count":472,"type":22},15,[474],"EARLY_PHASE1","Pilot study to determine feasibility of adding intrathecal chemotherapy and maintenance therapy after high dose chemotherapy for treatment of newly diagnosed HR-EBTs in patients less than 6 years of age.",[477,478,479,480,481,482,483,28,170,484,485,486,487,488],"CNS Embryonal Tumor","CNS, Medulloblastoma","Atypical Teratoid Rhabdoid Tumor","Medulloblastoma, Childhood","Medulloblastoma, Group 3","Medulloblastoma, Group 4","Pineoblastoma","Embryonal Tumor With Abundant Neuropil and True Rosettes","Ependymoblastoma","Medulloepithelioma","CNS Embryonal Tumor With Rhabdoid Features","CNS Embryonal Tumor, Nos",[490],"HR-EBT",{"date":456,"type":33},{"date":493,"type":33},"2025-09-23",{"date":495,"type":22},"2032-12-31",{"name":497,"class":40},"C17 Council",12,{"id":500,"slug":501,"hasResults":12,"nctId":502,"briefTitle":503,"officialTitle":504,"acronym":4,"eligibilityCriteria":505,"healthyVolunteers":12,"sex":17,"minAge":4,"maxAge":19,"enrollmentInfo":506,"targetDuration":4,"studyType":23,"phases":508,"briefSummary":509,"conditions":510,"keywords":4,"overallStatus":59,"whyStopped":4,"lastUpdateSubmitDate":512,"lastUpdatePostDateStruct":513,"startDateStruct":515,"completionDateStruct":517,"leadSponsor":519,"locationsCount":520},"100557680","phase-1-silmitasertib-cx-4945-in-combination-with-chemotherapy-for-relapsed-refractory-solid-tumors-100557680","NCT06541262","Silmitasertib (CX-4945) in Combination With Chemotherapy for Relapsed Refractory Solid Tumors","Phase I\u002FII Study of Silmitasertib (CX-4945) in Combination With Chemotherapy in Children and Young Adults With Relapsed Refractory Solid Tumors","Inclusion Criteria:\n\n1. Age: Less than 30 years old at initial diagnosis\n2. Pathology All subjects must have a confirmed diagnosis of tumor type. Phase I: Relapsed\u002Frefractory solid tumors: Neuroblastoma, Ewing Sarcoma, Osteosarcoma, Rhabdomyosarcoma, Liposarcoma\n\n   Phase II:\n   * Relapsed\u002Frefractory Neuroblastoma\n   * Relapsed\u002Frefractory Ewing sarcoma\n3. Tumor assessment:\n\n   Disease assessment is required for eligibility and must be done after last dose of previous therapy and prior to first dose of study drug.\n4. Disease Status:\n\n   Relapsed\u002FRefractory Neuroblastoma Relapsed disease defined as neuroblastoma that was previously in remission after standard therapy (at least 4 cycles of aggressive multi-drug induction chemotherapy, with or without radiation and surgery, followed by immunotherapy, or according to a standard high-risk treatment\u002Fneuroblastoma protocol) and has now relapsed and is in any number of relapses.\n\n   Refractory disease defined as High-risk neuroblastoma (as defined by INRG) that failed to achieve CR after at least 4 cycles of aggressive multi-drug induction chemotherapy, progression during upfront therapy or with disease remaining after standard immunotherapy.\n\n   International Neuroblastoma Risk Group Staging System (INRG) High Risk NB defined as one of the following:\n   1. Any age with International Neuroblastoma Risk Group (INRG) Stage L2, MS, or M with MYCN amplification\n   2. Age ≥ 547 days and INRG Stage M regardless of biologic features\n   3. Any age initially diagnosed with INRG Stage L1 MYCN amplified NBL who have progressed to Stage M without systemic chemotherapy\n   4. Age ≥ 547 days of age initially diagnosed with INRG Stage L1, L2, or MS who have progressed to Stage M without systemic chemotherapy\n\n   Relapsed\u002Frefractory Sarcoma Subjects that have relapsed following standard of care therapy or having progressed during standard of care therapy. Standard of care therapy for sarcoma includes multi-agent chemotherapy with local control consisting of either surgery or radiation therapy.\n5. Measurable or evaluable disease, including at least one of the following:\n\n   * Measurable tumor by CT or MRI\n   * MIBG or PET that is positive for disease\n   * Bone Marrow biopsy\u002Faspirate that is positive for disease\n6. Timing from prior therapy:\n\n   Subjects must have fully recovered from the acute toxic effects of all prior anti- cancer therapy and be within the following timelines:\n   1. Myelosuppressive chemotherapy: Must not have received within 2 weeks of enrollment onto this study.\n   2. Small Molecule Inhibitors (anti-neoplastic agent): At least 2 weeks from the completion of therapy with a small molecule inhibitor.\n   3. Immunotherapy: At least 4 weeks since the completion of any type of immunotherapy, e.g. tumor vaccines, CAR-T cells, anti-GD2 Monoclonal antibodies (ex. naxitamab, dinutuximab, etc.).\n   4. Radiotherapy: At least 30 days since the last treatment except for radiation delivered with palliative intent to a non-target site.\n   5. Stem Cell Transplant:\n\n      * Allogeneic: No evidence of active graft vs. host disease\n      * Allogeneic\u002FAutologous: ≥ 2 months must have elapsed since transplant.\n   6. MIBG Therapy: At least 6 weeks since treatment with MIBG therapy.\n7. Subjects must have a Lansky or Karnofsky Performance Scale score of \\>\u002F= 50.\n8. Subjects must have adequate organ function at the time of enrollment:\n\n   * Cardiac: Subjects must have a QTcF ≤ 480 msc.\n   * Hematological: Hematological recovery as defined by ANC ≥750\u002FμL\n   * Liver: Adequate liver function as defined by AST and ALT \\\u003C5x upper limit of normal\n   * Renal: Subjects must have adequate renal function defined as:\n   * estimated Glomerular Filtration rate (eGFR) as calculated from the Bedside Schwartz equation (for subjects \\\u003C 17 years old) (in units of mL\u002Fmin\u002F1.73 m2) or via radioisotope GFR of ≥ 70 mL\u002Fmin\u002F1.73 m2. The Bedside Schwartz equation is: \\[(0.413) X (Height in cm)\\] \u002F SCr\n   * estimated Glomerular Filtration rate (eGFR) as calculated from the Cockcroft and Gault formula (for subjects ≥17 years old (in units of mL\u002Fmin\u002F1.73 m2) or via radioisotope GFR of ≥ 70 mL\u002Fmin\u002F1.73 m2. The Cockcroft and Gault formula is: \\[(140-age) x (Wt in kg) x (0.85 if female)\\] \u002F (72 x SCr)\n   * OR a 24 hour urine Creatinine clearance ≥ 70 mL\u002Fmin\u002F1.73 m2\n9. Subjects of childbearing potential must have a negative serum pregnancy test. Subjects of childbearing potential must agree to use effective measures to avoid pregnancy.\n10. Written informed consent in accordance with institutional and FDA guidelines must be obtained from all subjects (or subjects' legal representative).\n\nExclusion Criteria:\n\n1. Investigational Drugs: Subjects who are currently receiving another investigational drug are excluded from participation.\n2. Anti-cancer Agents: Subjects who are currently receiving other anticancer agents are not eligible. Subjects must have fully recovered from the hematological and bone marrow suppression effects of prior therapy.\n3. Subjects who are currently receiving Vitamin K antagonists (warfarin).\n4. Subjects who are currently receiving the class of lipid-lowering medications HMG-CoA reductase inhibitors (statins).\n5. Infection: Subjects who have an uncontrolled infection are not eligible until the infection is judged to be well controlled in the opinion of the investigator.\n6. Subjects who, in the opinion of the investigator, may not be able to comply with the safety monitoring requirements of the study, or in whom compliance is likely to be suboptimal, should be excluded.\n7. Subjects with any clinically significant unrelated systemic illness (serious infections or significant cardiac, pulmonary, hepatic or other organ dysfunction), that in the opinion of the investigator would compromise the subject's ability to tolerate protocol therapy, put them at additional risk for toxicity or would interfere with the study procedures or results.\n8. Subjects with any of the following gastrointestinal disorders:\n\n   1. Active malabsorption (e.g. short gut) syndrome.\n   2. Uncontrolled diarrhea (excess of 4 stools\u002Fday)\n   3. Gastritis, ulcerative colitis, Chron's disease or hemorrhagic coloproctitis\n   4. History of gastric or small bowel surgery involving any extent of gastric or small bowel resection\n9. Lactating subjects are not eligible unless they have agreed to not breastfeed their infants. There is an unknown but potential risk for adverse events in nursing infants secondary to treatment of the nursing subject with silmitasertib. (NOTE: breast milk cannot be stored for future use while the nursing subject is being treated on study.)\n10. Subjects with a history of any other malignancy.",{"count":507,"type":22},104,[53,25],"The purpose of this study is to evaluate the investigational drug, silmitasertib (a pill taken by mouth), in combination with FDA approved drugs for solid tumors. An investigational drug is one that has not been approved by the U.S. Food \\& Drug Administration (FDA), or any other regulatory authorities around the world for use alone or in combination with any drug, for the condition or illness it is being used to treat.\n\nThe goals of this part of the study are:\n\n* Establish a recommended dose of silmitasertib in combination with chemotherapy\n* Test the safety and tolerability of silmitasertib in combination with chemotherapy in subjects with cancer\n* To determine the activity of study treatments chosen based on:\n* How each subject responds to the study treatment\n* How long a subject lives without their disease returning\u002Fprogressing",[28,141,172,140,511],"Liposarcoma","2026-06-30",{"date":514,"type":33},"2026-07-02",{"date":516,"type":33},"2024-10-30",{"date":518,"type":22},"2035-11-01",{"name":184,"class":40},22,{"id":522,"slug":523,"hasResults":12,"nctId":524,"briefTitle":525,"officialTitle":526,"acronym":527,"eligibilityCriteria":528,"healthyVolunteers":12,"sex":17,"minAge":18,"maxAge":19,"enrollmentInfo":529,"targetDuration":4,"studyType":23,"phases":531,"briefSummary":532,"conditions":533,"keywords":4,"overallStatus":59,"whyStopped":4,"lastUpdateSubmitDate":534,"lastUpdatePostDateStruct":535,"startDateStruct":537,"completionDateStruct":539,"leadSponsor":541,"locationsCount":68},"100426787","phase-1-peach-trial--precision-medicine-and-adoptive-cellular-therapy-100426787","NCT04837547","PEACH TRIAL- Precision Medicine and Adoptive Cellular Therapy","PEACH TRIAL- Precision mEdicine and Adoptive Cellular tHerapy for the Treatment of Recurrent Neuroblastoma and Newly Diagnosed Diffuse Intrinsic Pontine Glioma (DIPG)","PEACH","Inclusion Criteria:\n\n* Subjects must have proven pediatric cancer with confirmation at diagnosis or at the time of recurrence\u002Fprogression and clinical determination of disease for which there is no known effective curative therapy or disease that is refractory to established proven therapies fitting into one of the following categories:\n* Disease Status:\n\nHigh Risk Neuroblastoma-\n\n1. Patients that have relapsed following standard of care therapy or having progressed during standard of care therapy and non-responsive\u002Fprogressive to accepted curative chemotherapy.\n2. Neuroblastoma must be age \\>12 months at enrollment\n\nDiffuse Intrinsic Pontine (or other brain stem) Glioma\n\n1. Newly-diagnosed patients willing to undergo biopsy\n2. Must be within 2 months of diagnosis and prior to starting radiation\n3. DIPG must be ≥ 3 years of age at enrollment\n\n   * All subjects must be age ≤ 30 years at enrollment\n   * Patient and\u002For parents\u002Fguardian willing to consent to biopsy for obtaining tumor material for confirmatory diagnosis and\u002For tumor RNA extraction and amplification.\n   * Subjects must have measurable disease as defined Per section 8 at the time of biopsy and tumor or bone marrow must be accessible for biopsy. Tumor or bone marrow samples submitted for analysis must contain \\>20% viable tumor tissue to qualify. Note: Subjects with NB who are expected to have no evidence of disease after surgical removal of their tumor are still eligible for this trial if their disease would normally require adjuvant chemotherapy treatment after surgery despite NED status.\n   * Current disease state must be one for which there is currently no known effective therapy\n   * Specimens will be obtained only in a non-significant risk manner and not solely for the purpose of investigational testing.\n   * Lansky or Karnofsky Score must be ≥ 60\n   * Bone Marrow:\n\n     1. ANC (Absolute neutrophil count) ≥ 1000\u002Fµl (unsupported- \\>24 hrs off G-CSF and 7 days off neulasta)\n     2. Platelets ≥ 100,000\u002Fµl (can be transfused)\n     3. Hemoglobin \\> 8 g\u002FdL (can be transfused)\n   * Renal: Serum creatinine ≤ upper limit of institutional normal.\n   * Adequate liver function must be demonstrated, defined as:\n\n     1. Total bilirubin ≤ 1.5 x upper limit of normal (ULN) for age AND\n     2. ALT (SGPT) ≤ 3 times upper limit of normal (ULN) for age\n     3. AST (SGOT) ≤ 3 times upper limit of normal (ULN) for age.\n   * Subjects with CNS disease currently taking steroids must have been on a stable dose of steroids for at least one week prior to their biopsy and must not have progressive hydrocephalus at enrollment.\n   * A negative serum pregnancy test is required for female participants of childbearing potential (≥13 years of age or after onset of menses)\n   * Both male and female post-pubertal study subjects need to agree to use one of the more effective birth control methods during treatment and for six months after treatment is stopped. These methods include total abstinence (no sex), oral contraceptives (\"the pill\"), an intrauterine device (IUD), levonorgestrel implants (Norplant), or medroxyprogesterone acetate injections (Depo-provera shots). If one of these cannot be used, contraceptive foam with a condom is recommended.\n   * Informed Consent: All subjects and\u002For legal guardians must sign informed written consent. Assent, when appropriate, will be obtained according to institutional guidelines\n   * Post-Biopsy: Patients with post-biopsy neurological deficits should have deficits that are stable for a minimum of 1 week prior to registration.\n\nExclusion Criteria:\n\n* Absence of tumor on biopsy specimen or a diagnosis other than NBL or glioma on biopsy\n* Known autoimmune or immunosuppressive disease or human immunodeficiency virus infection.\n* Subjects with significant renal, cardiac, pulmonary, hepatic or other organ dysfunction.\n* Prior allergic reaction to GM-CSF or Td.\n* Subjects who have received any cytotoxic chemotherapy within the last 7 days prior to biopsy or focal radiotherapy in the case of patients with diffuse intrinsic pontine (or other brain stem) gliomas\n* Subjects with NBL who have received any radiotherapy to the primary sample site within the last 14 days (radiation may be included in treatment decision after biopsy).\n* Subjects receiving any investigational drug concurrently.\n* Subjects with uncontrolled serious infections or a life-threatening illness (unrelated to tumor)\n* Subjects with any other medical condition, including malabsorption syndromes, mental illness or substance abuse, deemed by the Investigator to be likely to interfere with the interpretation of the results or which would interfere with a subject's ability to sign or the legal guardian's ability to sign the informed consent, and subject's ability to cooperate and participate in the study",{"count":530,"type":22},24,[53],"A Phase I open-label, multicenter study, to evaluate the safety, feasibility, and maximum tolerated dose (MTD) of treating children with newly diagnosed DIPG or recurrent neuroblastoma with molecular targeted therapy in combination with adoptive cell therapy (Total tumor mRNA-pulsed autologous Dendritic Cells (DCs) (TTRNA-DCs), Tumor-specific ex vivo expanded autologous lymphocyte transfer (TTRNA-xALT) and Autologous G-CSF mobilized Hematopoietic Stem Cells (HSCs)).",[28,171],"2026-06-29",{"date":536,"type":33},"2026-07-01",{"date":538,"type":33},"2021-09-20",{"date":540,"type":22},"2032-09",{"name":542,"class":40},"University of Florida",{"id":544,"slug":545,"hasResults":12,"nctId":546,"briefTitle":547,"officialTitle":548,"acronym":4,"eligibilityCriteria":549,"healthyVolunteers":12,"sex":17,"minAge":322,"maxAge":323,"enrollmentInfo":550,"targetDuration":4,"studyType":23,"phases":552,"briefSummary":553,"conditions":554,"keywords":564,"overallStatus":59,"whyStopped":4,"lastUpdateSubmitDate":568,"lastUpdatePostDateStruct":569,"startDateStruct":570,"completionDateStruct":572,"leadSponsor":574,"locationsCount":125},"100564132","phase-1-alphabeta-t-and-b-cell-depletion-with-zoledronic-acid-for-solid-tumors-100564132","NCT06625190","Alpha\u002FBeta T and B Cell Depletion With Zoledronic Acid for Solid Tumors","Allogeneic Stem Cell Transplantation Utilizing Alpha\u002FBeta T Cell and CD19+ B Cell Depletion With Zoledronic Acid in Combination to Treat Pediatric, Adolescent, and Young Adult Patients With Relapsed\u002FRefractory Solid Tumors","Inclusion Criteria:\n\n* Patients 6 months to ≤ 25 years old\n* Relapsed\u002FRefractory Solid Tumor whom failed or deemed ineligible to receive autologous transplant or if autologous transplant did not offer \\>20% chance of cure with the following diseases:\n\n  1. neuroblastoma (high risk with relapsed or refractory disease),\n  2. relapsed\u002Frefractory rhabdomyosarcoma,\n  3. relapsed\u002Frefractory non-rhabdomyosarcoma soft tissue sarcoma (NRSTS): synovial sarcoma, malignant peripheral nerve sheath tumors (MPNST),\n  4. High risk adult type NRSTS: clear cell sarcoma, alveolar soft part sarcoma,\n  5. Other high-risk extracranial solid tumors: desmoplastic small round cell tumors, chordoma, malignant rhabdoid tumor, epithelioid sarcoma, myoepithelial tumor\n  6. relapsed\u002Frefractory bone tumors: osteosarcoma and Ewing sarcoma\u002FPNET, or\n  7. Wilm's tumor or other high-risk solid tumors with \\\u003C10% expected survival with conventional treatment.\n* Subjects must not have more than one active malignancy at the time of enrollment. (Subjects with a prior or concurrent malignancy whose natural history or treatment does not have the potential to interfere with the safety or efficacy assessment of the investigational regimen \\[as determined by the treating physician and approved by the PI\\] may be included.)\n* Haplo-identical related donor (at least one full haplotype must be matched).\n* Karnofsky or Lansky score ≥60% at the time of enrollment. Karnofsky scores must be used for patients \\>16 years of age and Lansky scores for patients ≤16 years of age\n* Adequate organ function (within 4 weeks of initiation of preparative regimen), defined as:\n\n  1. Pulmonary: FEV1, FVC, and corrected DLCO must all be ≥ 50% of predicted by pulmonary function tests (PFTs). For children who are unable to perform for PFTs due to age, the criteria are: no evidence of dyspnea at rest and no need for supplemental oxygen.\n  2. Renal: Creatinine clearance or radioisotope GFR ≥60 mL\u002Fmin\u002F1.73 m2 or a serum creatinine based on age\u002Fgender\n  3. Cardiac: Ejection fraction of ≥ 40% by echocardiogram or radionuclide scan (MUGA).\n* Written informed consent obtained from the subject and the subject agrees to comply with all the study-related procedures\n* Individuals of childbearing potential (IOCBP) must be using an adequate method of contraception to avoid pregnancy throughout the study and for one year following transplantation to minimize the risk of pregnancy. Prior to study enrollment, individuals of childbearing potential must be advised of the importance of avoiding pregnancy during trial participation and the potential risk factor for an unintentional pregnancy.\n* Subjects with female partners of child-bearing potential must agree to use physician-approved contraceptive methods (e.g., abstinence, condoms, vasectomy) throughout the study and should avoid conceiving children for one year following stem cell transplantation.\n\nExclusion Criteria:\n\n* Patients with documented uncontrolled infection at the time of study entry are not eligible.\n\n  a. Uncontrolled infection is patient without treatment antimicrobials and\u002For demonstrating progression despite antimicrobials\n* Patients with progressive solid tumor disease after relapsed\u002Frefractory treatment.\n* Demonstrated lack of compliance with medical care, as determined by the treating physician.\n* Patients who have received an allogeneic HSCT within 6 months.\n* Patients who do not have an eligible allogeneic donor available.\n* Patients with a life expectancy \\\u003C3 months\n* Patients not meeting inclusion criteria for organ function.\n* Females or males of childbearing potential who are unwilling or unable to use an acceptable method to avoid pregnancy for the entire study period and for at least one year after transplantation.\n* Females who are known to be pregnant or breastfeeding.\n* History of any other disease, metabolic dysfunction, clinical examination finding, or clinical laboratory finding giving reasonable suspicion of a disease or condition that contraindicates the use of protocol therapy or that might affect the interpretation of the results of the study or that puts the subject at high risk for treatment complications, in the opinion of the treating physician.\n* Prisoners or subjects who are involuntarily incarcerated, or subjects who are compulsorily detained for treatment of either a psychiatric or physical illness.",{"count":551,"type":22},27,[53,25],"Hematopoietic stem cell transplantation can cure patients with blood cancer and other underlying diseases. αβ-T cell and B cell depletion has been introduced to decrease GVHD and PTLD and has demonstrated effectiveness for hematologic malignancies and non-malignant diseases additionally increasing the donor pool as to allow for haploidentical transplant to safely occur.\n\nWhile solid tumors can be highly chemotherapy sensitive, many remain resistant and require multimodalities of treatment. Immunotherapy has been developed to harness the immune system in fighting solid tumors, though not all have targeted effects. Some solid tumors are treated with autologous transplants; however, they do not always demonstrate an improved event free survival or overall survival. There has been evidence of the use of allogeneic stem cell transplants to provide a graft versus tumor effect, though studies remain limited.\n\nBy utilizing αβ-T cell and B cell depletion for stem cell transplants and combining with zoledronic acid, the immune system may potentially be harnessed and enhanced to provide an improved graft versus tumor effect in relapsed\u002Frefractory solid tumors and promote an improved event-free survival and overall survival.\n\nThis study will investigate the safety of treatment with a stem cell graft depleted of αβ-T cell and CD19+ B cells in combination with zoledronic acid in pediatric and young adult patients with select solid tumors, as well as whether this treatment improves survival rates in these patients.",[28,140,555,556,557,558,559,560,561,562,563,172,141,142],"Synovial Sarcoma","Peripheral Nerve Sheath Tumors","Clear Cell Sarcoma","Alveolar Soft Part Sarcoma","Desmoplastic Small Round Cell Tumor","Chordoma","Rhabdoid Tumor","Epithelioid Sarcoma","Myoepithelial Tumor",[565,566,567],"pediatric solid tumors","stem cell transplantation","graft manipulation","2026-06-25",{"date":512,"type":33},{"date":571,"type":33},"2026-02-11",{"date":573,"type":22},"2030-02",{"name":542,"class":40},{"id":576,"slug":577,"hasResults":12,"nctId":578,"briefTitle":579,"officialTitle":580,"acronym":4,"eligibilityCriteria":581,"healthyVolunteers":12,"sex":17,"minAge":18,"maxAge":582,"enrollmentInfo":583,"targetDuration":4,"studyType":23,"phases":584,"briefSummary":585,"conditions":586,"keywords":587,"overallStatus":59,"whyStopped":4,"lastUpdateSubmitDate":592,"lastUpdatePostDateStruct":593,"startDateStruct":595,"completionDateStruct":597,"leadSponsor":599,"locationsCount":125},"100411436","phase-1-4scar-t-therapy-targeting-gd2-psma-and-cd276-for-treating-neuroblastoma-100411436","NCT04637503","4SCAR-T Therapy Targeting GD2, PSMA and CD276 for Treating Neuroblastoma","Multi-center Phase I\u002FII Clinical Trial of 4SCAR-T Therapy Targeting GD2, PSMA and CD276 for Treating Neuroblastoma","Inclusion Criteria:\n\n* Patients with tumors have received standard first-line therapy and been judged to be non-resectable, metastatic, progressive or recurrent.\n* The expression status of GD2, PSMA and CD276 antigens of the tumor will be determined for eligibility. Positive expression is defined by GD2, PMSA and CD276 antibody staining results based on immunohistochemistry or flow cytometry analyses.\n* Body weight greater than or equal to 10 kg.\n* Age: ≥1 year and ≤ 65 years of age at the time of enrollment.\n* Life expectancy: at least 8 weeks.\n* Prior Therapy:\n\n  1. There is no limit to the number of prior treatment regimens. Any grade 3 or 4 non-hematologic toxicity of any previous therapy must have resolved to grade 2 or less.\n  2. Participant must not have received hematopoietic growth factors for at least 1 week prior to mononuclear cells collection.\n  3. At least 7 days must have elapsed since the completion of therapy with a biologic agent, targeted agent, tyrosine kinase inhibitor or a metronomic non-myelosuppressive regimen.\n  4. At least 4 weeks must have elapsed since prior therapy that included a monoclonal antibody.\n  5. At least 1 week since any radiation therapy at the time of study entry.\n* Karnofsky\u002Fjansky score of 60% or greater.\n* Cardiac function: Left ventricular ejection fraction greater than or equal to 40\u002F55 percent.\n* Pulse Ox greater than or equal to 90% on room air.\n* Liver function: defined as alanine transaminase (ALT) \\\u003C3x upper limit of normal (ULN), aspartate aminotransferase (AST) \\\u003C3x ULN; serum bilirubin and alkaline phosphatase \\\u003C2x ULN.\n* Renal function: Patients must have serum creatinine less than 3 times upper limit of normal.\n* Marrow function: White blood cell count ≥1000\u002Ful, Absolute neutrophil count ≥500\u002Ful, Absolute lymphocyte count ≥500\u002Ful, Platelet count ≥25,000\u002Ful (not achieved by transfusion).\n* Patients with known bone marrow metastatic disease will be eligible for study as long as they meet hematologic function criteria, and the marrow disease not evaluable for hematologic toxicity.\n* For all patients enrolled in this study, their parents or legal guardians must sign an informed consent and assent.\n\nExclusion Criteria:\n\n* Existing severe illness (e.g. significant cardiac, pulmonary, hepatic diseases, etc.) or major organ dysfunction, with the exception of grade 3 hematologic toxicity.\n* Untreated central nervous system (CNS) metastasis: Patients with previous CNS tumor involvement that has been treated and is stable for at least 6 weeks following completion of therapy are eligible.\n* Previous treatment with other genetically engineered GD2, PSMA and CD276 CART cells.\n* Active HIV, Hepatitis B virus (HBV), Hepatitis C virus (HCV) infection or uncontrolled infection.\n* Patients who require systemic corticosteroid or other immunosuppressive therapy.\n* Evidence of tumor potentially causing airway obstruction.\n* Inability to comply with protocol requirements.\n* Insufficient CART cells availability.","65 Years",{"count":51,"type":22},[53,25],"The purpose of this clinical trial is to assess the feasibility, safety and efficacy of multiple 4SCAR-T cell therapy which targets GD2, PSMA and CD276 surface antigens in patients with relapsed and refractory neuroblastoma (NB). Another goal of the study is to understand the function of the multi-CAR-T cells and their persistency in the patients.",[28],[588,589,590,591],"CART","chimeric antigen receptor","adoptive T cell transfer","GD2, PSMA, CD276, B7-H3","2026-06-18",{"date":594,"type":33},"2026-06-22",{"date":596,"type":33},"2026-06-01",{"date":598,"type":22},"2030-12-31",{"name":600,"class":40},"Shenzhen Geno-Immune Medical Institute",{"id":602,"slug":603,"hasResults":12,"nctId":604,"briefTitle":605,"officialTitle":605,"acronym":606,"eligibilityCriteria":607,"healthyVolunteers":12,"sex":17,"minAge":4,"maxAge":4,"enrollmentInfo":608,"targetDuration":4,"studyType":102,"phases":4,"briefSummary":610,"conditions":611,"keywords":655,"overallStatus":59,"whyStopped":4,"lastUpdateSubmitDate":665,"lastUpdatePostDateStruct":666,"startDateStruct":668,"completionDateStruct":670,"leadSponsor":672,"locationsCount":125},"100289631","familial-investigations-of-childhood-cancer-predisposition-100289631","NCT03050268","Familial Investigations of Childhood Cancer Predisposition","SJFAMILY","NOTE: This is a research study and is not meant to be a substitute for clinical genetic testing. Families may never receive results from the study or may receive results many years from the time they enroll. If you are interested in clinical testing please consider seeing a local genetic counselor or other genetics professional. If you have already had clinical genetic testing and meet eligibility criteria for this study as shown below, you may enroll regardless of the results of your clinical genetic testing.\n\nDEFINITION OF FAMILIAR CANCER FOR THIS PROTOCOL:\n\nIn this protocol, the definition of \"Familial Cancer\" is met if any of the following is present:\n\n* An individual with a history of cancer diagnosed under 26 years of age who has at least one first, second or third degree relative with a history of cancer diagnosed under 51 years of age; OR\n* An individual who has been diagnosed with more than one cancer, at least one of which was diagnosed under 26 years of age; OR\n* An individual with a clinical or molecular diagnosis of a known cancer predisposition syndrome; OR\n* An individual with a congenital cancer diagnosed before 6 months of age; OR\n* An individual with a rare pediatric cancer or tumor diagnosed before 26 years of age\n\nº Excluding human papilloma virus-associated cervical cancer and non-melanoma skin cancer occurring in adults.\n\nINCLUSION CRITERIA:\n\n* An individual who meets this protocol's definition of \"Familial Cancer,\" as above.\n* Biologic relatives of an individual meeting this protocol's definition of \"Familial Cancer,\" who are either affected or unaffected by cancer.\n\nEXCLUSION CRITERIA:\n\n* An inability or unwillingness of the research participant or his\u002Fher legally authorized representative (LAR) to provide written informed consent.\n* The participant has received allogeneic bone marrow transplantation and has NO pre-transplant germline (cancer-unaffected) DNA available AND is unwilling to provide a skin sample.",{"count":609,"type":22},1500,"NOTE: This is a research study and is not meant to be a substitute for clinical genetic testing. Families may never receive results from the study or may receive results many years from the time they enroll. If you are interested in clinical testing please consider seeing a local genetic counselor or other genetics professional. If you have already had clinical genetic testing and meet eligibility criteria for this study as shown in the Eligibility Section, you may enroll regardless of the results of your clinical genetic testing.\n\nWhile it is well recognized that hereditary factors contribute to the development of a subset of human cancers, the cause for many cancers remains unknown. The application of next generation sequencing (NGS) technologies has expanded knowledge in the field of hereditary cancer predisposition. Currently, more than 100 cancer predisposing genes have been identified, and it is now estimated that approximately 10% of all cancer patients have an underlying genetic predisposition.\n\nThe purpose of this protocol is to identify novel cancer predisposing genes and\u002For genetic variants. For this study, the investigators will establish a Data Registry linked to a Repository of biological samples. Health information, blood samples and occasionally leftover tumor samples will be collected from individuals with familial cancer. The investigators will use NGS approaches to find changes in genes that may be important in the development of familial cancer. The information gained from this study may provide new and better ways to diagnose and care for people with hereditary cancer.\n\nPRIMARY OBJECTIVE:\n\n* Establish a registry of families with clustering of cancer in which clinical data are linked to a repository of cryopreserved blood cells, germline DNA, and tumor tissues from the proband and other family members.\n\nSECONDARY OBJECTIVE:\n\n* Identify novel cancer predisposing genes and\u002For genetic variants in families with clustering of cancer for which the underlying genetic basis is unknown.",[612,613,614,615,616,617,618,619,620,621,622,623,624,625,626,627,628,629,630,631,632,633,634,635,636,637,638,639,640,28,641,642,643,644,645,646,647,648,649,650,274,651,140,652,653,654],"Acute Leukemia","Adenomatous Polyposis","Adrenocortical Carcinoma","AML","BAP1 Tumor Predisposition Syndrome","Carney Complex","Choroid Plexus Carcinoma","Constitutional Mismatch Repair Deficiency Syndrome","Diamond-Blackfan Anemia","DICER1 Syndrome","Dyskeratosis Congenita","Emberger Syndrome","Familial Acute Myeloid Leukemia","Familial Adenomatous Polyposis","Fanconi Anemia","Familial Cancer","Familial Wilms Tumor","Familial Neuroblastoma","GIST","Hereditary Breast and Ovarian Cancer","Hereditary Paraganglioma-Pheochromocytoma Syndrome","Hodgkin Lymphoma","Juvenile Polyposis","Li-Fraumeni Syndrome","Lynch Syndrome","MDS","Melanoma Syndrome","Multiple Endocrine Neoplasia Type 1","Multiple Endocrine Neoplasia Type 2","Neurofibromatosis Type 1","Neurofibromatosis Type II","Nevoid Basal Cell Carcinoma Syndrome","Non Hodgkin Lymphoma","Noonan Syndrome and Other Rasopathy","Overgrowth Syndromes","Pancreatic Cancer","Peutz-Jeghers Syndrome","Pheochromocytoma\u002FParaganglioma","PTEN Hamartoma Tumor Syndrome","Rhabdoid Tumor Predisposition Syndrome","Rothmund-Thomson Syndrome","Tuberous Sclerosis","Von Hippel-Lindau Disease",[656,657,658,659,660,661,662,663,664],"Familial cancer","Genetic predisposition","Heritable disease","Cancer risk","Genome analysis","Genetic modifiers","Next generation sequencing (NGS)","Genetic counseling","DNA","2026-06-15",{"date":667,"type":33},"2026-06-17",{"date":669,"type":33},"2017-04-06",{"date":671,"type":22},"2037-03-31",{"name":673,"class":40},"St. Jude Children's Research Hospital",{"id":675,"slug":676,"hasResults":12,"nctId":677,"briefTitle":678,"officialTitle":678,"acronym":4,"eligibilityCriteria":679,"healthyVolunteers":12,"sex":17,"minAge":18,"maxAge":680,"enrollmentInfo":681,"targetDuration":4,"studyType":23,"phases":683,"briefSummary":684,"conditions":685,"keywords":4,"overallStatus":59,"whyStopped":4,"lastUpdateSubmitDate":686,"lastUpdatePostDateStruct":687,"startDateStruct":689,"completionDateStruct":691,"leadSponsor":693,"locationsCount":125},"100643832","phase-2-haploidentical-donor-cytokine-induced-memory-like-natural-killer-cells-ciml-nk-for-relapsed--refractory-neuroblastoma-100643832","NCT07635056","Haploidentical Donor Cytokine-Induced Memory-Like Natural Killer Cells (CIML-NK) for Relapsed & Refractory Neuroblastoma","Inclusion Criteria:\n\n* Age 1-39 years at the time of study enrollment\n* With diagnosis of neuroblastoma with histologic verification\n* Classified as high-risk neuroblastoma as defined by Children's Oncology Group (COG) risk classification, including patients initially classified as low or intermediate risk at diagnosis with subsequent reclassification as high-risk disease\n* With relapsed or refractory disease, including at least one of the following:\n\n  1. Recurrent disease at any time after completion of frontline therapy\n  2. Progressive disease at any time following standard induction therapy\n  3. Primary resistant or refractory disease defined by failure to achieve a complete response by International Neuroblastoma Response Criteria (INRC) after at least four cycles of standard, multidrug induction chemotherapy on or according to a high-risk neuroblastoma protocol\n* Patients must have evaluable disease documented within four weeks of study enrollment. Evaluable disease must include at least one of the following:\n\n  1. Measurable tumor (\\>10 mm in at least one dimension) on MRI or CT scan that is either MIBG, FDG or 68Ga-DOTATATE avid\n  2. One or more MIBG, FDG, or 68Ga-DOTATATE avid bone lesion\n  3. Microscopic marrow metastasis based on routine morphology and\u002For immunohistochemistry in at least one sample from bilateral aspirates and biopsies at the time of study enrollment.\n* With performance level of \\>50% on Lansky (\\\u003C16 years) or Karnofsky (\\>16 years) scales. Patients who are wheelchair bound due to paralysis will be considered ambulatory when assessing their performance score.\n* Adequate baseline cardiac and pulmonary function including a left ventricular ejection fraction (LVEF) \\>50% by echocardiogram and pulse oximetry \\>92% on room air documented within four weeks of study enrollment.\n* Adequate baseline hematologic function: peripheral absolute neutrophil count (ANC) ≥500\u002FµL, with no receipt of long-acting myeloid growth factors within 14 days or short-acting myeloid growth factors within 7 days of study entry, and a platelet count ≥50,000\u002FµL, with patients required to be transfusion independent for at least 7 days, unless cytopenias are related to marrow metastasis as defined above.\n* With available haploidentical related donors.\n\nExclusion Criteria:\n\n* Infectious disease: Active, uncontrolled infection or received a live vaccine within 30 days prior to study enrollment.\n* Cardiac function: LVEF \\\u003C50% by echocardiogram, serious uncontrolled cardiac arrhythmias, or history of myocarditis or congestive heart failure (New York Heart Association Functional Classification III or IV)\n* Pulmonary function: Active interstitial lung disease (ILD)\u002Fpneumonitis or history of ILD\u002Fpneumonitis requiring systemic corticosteroid treatment.\n* Renal function: Glomerular Function Rate (GFR) \\\u003C50 mL\u002Fmin\u002F1.73 m2 as measured by cystatin C or NM GFR\n* Hepatic function: Total bilirubin \\>5 mg\u002FdL, AST and ALT \\>10 times the upper limit of normal\n* Concomitant medications: receiving \\>0.5 mg\u002Fkg prednisone equivalent daily\n* Receipt of any concomitant investigational treatments within 30 days at the time of the infusion of the IP. These investigational treatments include drugs, biologics, or devices that are still under investigation in clinical trials or research settings. The use of such agents may confound study results or pose additional safety risks\n* Known allergy or hypersensitivity reaction to IL-2 injections\n* Pregnant or breastfeeding women","39 Years",{"count":682,"type":22},20,[25],"The goal of this study is to demonstrate that cytokine-induced memory-like natural killer cells (CIML-NK cells) can be generated from donor cells and infused safely into patients with relapsed or refractory neuroblastoma during dinutuximab-based therapy.",[28],"2026-06-04",{"date":688,"type":33},"2026-06-09",{"date":690,"type":22},"2026-07",{"date":692,"type":22},"2031-06",{"name":694,"class":40},"Children's Hospital Medical Center, Cincinnati",{"id":696,"slug":697,"hasResults":12,"nctId":698,"briefTitle":699,"officialTitle":700,"acronym":4,"eligibilityCriteria":701,"healthyVolunteers":12,"sex":17,"minAge":18,"maxAge":4,"enrollmentInfo":702,"targetDuration":4,"studyType":23,"phases":704,"briefSummary":705,"conditions":706,"keywords":707,"overallStatus":59,"whyStopped":4,"lastUpdateSubmitDate":713,"lastUpdatePostDateStruct":714,"startDateStruct":716,"completionDateStruct":718,"leadSponsor":720,"locationsCount":125},"100065755","phase-2-iodine-i-131-metaiodobenzylguanidine-in-treating-patients-with-recurrent-progressive-or-refractory-neuroblastoma-or-malignant-pheochromocytoma-or-paraganglioma-100065755","NCT00107289","Iodine I 131 Metaiodobenzylguanidine in Treating Patients With Recurrent, Progressive, or Refractory Neuroblastoma or Malignant Pheochromocytoma or Paraganglioma","Open Access Protocol of Targeted Radiotherapy With I-metaiodobenzylguanidine (I-MIBG) in Patients With Resistant Neuroblastoma or Malignant Chromaffin Cell Tumors","Inclusion criteria for NB:\n\n* Patients must have the diagnosis of NB in accordance with the International Criteria, i.e., either histopathology (confirmed by the MSKCC Department of Pathology) or BM involvement plus elevated urinary catecholamines.\n* Must have a history of tumor progression or recurrence or failure to achieve complete response with standard therapy.\n* Patients must have MIBG-avid NB and evaluable disease on MIBG scan at time of enrollment on protocol\n* Prior Therapy: At least 2 weeks should have elapsed since any biologic therapy. Three weeks should have elapsed since last dose of chemotherapy.\n* Age \\>1 year\n* Determination that radiation safety restrictions during therapy period can be implemented.\n* Stem cells: Patients for high does must have an autologous hematopoietic stem cell product cryopreserved and available for re-infusion after MIBG treatment. Patients for low dose do not require cryopreserved autologous hematopoietic stem cell product available. The minimum dose for peripheral blood stem cells is 2 X106 CD34+ cells\u002Fkg.\n* Minimum life expectancy of eight weeks\n* Signed informed consent indicating awareness of the investigational nature of this program.\n\nInclusion criteria for malignant CCT:\n\n* Patients must have the diagnosis of malignant CCT i.e. malignant pheochromocytoma or malignant paraganglioma\n* Patients must have MIBG-avid malignant CCT and evaluable disease on MIBG scan at time of enrollment on protocol\n* Prior Therapy: At least 2 weeks should have elapsed since any biologic therapy. Three weeks should have elapsed since last dose of chemotherapy.\n* Age between 1 and 21 years and able to cooperate with radiation safety restrictions during therapy period\n* Stem cells: Patients must have an autologous hematopoietic stem cell product cryopreserved and available for re-infusion after MIBG treatment. The minimum dose for peripheral blood stem cells is 2 X106 CD34+ cells\u002Fkg.\n* Minimum life expectancy of eight weeks.\n* Signed informed consent indicating awareness of the investigational nature of this program.\n\nExclusion Criteria:\n\n* Severe major organ toxicity. Specifically, renal, cardiac, hepatic, pulmonary, gastrointestinal and neurologic toxicity should all be grade 2 or less. A grade 3 hearing deficit is acceptable.\n* Active serious infections not controlled by antibiotics.\n* Pregnant women are excluded for fear of danger to the fetus. Therefore negative pregnancy test is required for all women of child-bearing age, and appropriate contraception is used during the study period.\n* Inability or unwillingness to comply with radiation safety procedures or protocol requirements.",{"count":703,"type":22},200,[25],"The purpose of this research study is to find how active and safe 131 I-MIBG is in patients with resistant neuroblastoma, malignant pheochromocytoma and malignant paraganglioma.",[28,249],[708,709,710,711,712],"metastatic pheochromocytoma","recurrent pheochromocytoma","regional pheochromocytoma","recurrent neuroblastoma","04-148","2026-05-28",{"date":715,"type":33},"2026-05-29",{"date":717,"type":33},"2006-05",{"date":719,"type":22},"2027-05",{"name":230,"class":40}]