[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"leadSponsorName\":\"St. Jude Children's Research Hospital\",\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:":660},{"pageToken":4,"total":5,"offset":6,"count":7,"results":8},null,96,0,25,[9,44,75,99,125,150,180,205,224,289,311,338,364,386,410,432,463,487,504,527,552,572,594,616,642],{"id":10,"slug":11,"hasResults":12,"nctId":13,"briefTitle":14,"officialTitle":14,"acronym":4,"eligibilityCriteria":15,"healthyVolunteers":12,"sex":16,"minAge":4,"maxAge":4,"enrollmentInfo":17,"targetDuration":4,"studyType":20,"phases":21,"briefSummary":23,"conditions":24,"keywords":4,"overallStatus":32,"whyStopped":4,"lastUpdateSubmitDate":33,"lastUpdatePostDateStruct":34,"startDateStruct":37,"completionDateStruct":38,"leadSponsor":40,"locationsCount":43},"100632525","integration-of-adaptive-proton-therapy-in-pediatric-solid-tumors-and-hodgkins-lymphoma-100632525",false,"NCT07514819","Integration of Adaptive Proton Therapy in Pediatric Solid Tumors and Hodgkin's Lymphoma","Inclusion Criteria:\n\n* Participants diagnosed with solid tumors, including Rhabdomyosarcoma, Osteosarcoma, Ewing sarcoma, other sarcomas and carcinomas or also Hodgkin's lymphoma.\n* Participants who receive proton radiation therapy at St. Jude Children's Research Hospital.\n* Research participant or legal guardian\u002Frepresentative gives written informed consent.\n\nExclusion Criteria:\n\n* Participants who are not diagnosed with solid tumors or Hodgkin's lymphoma.\n* Participants who are diagnosed with Wilm's tumor or neuroblastoma\n* Participants who do not undergo proton therapy.\n* Participants who are prescribed equal or less than 5 fractions of proton therapy.\n* Participants with severe comorbid conditions that may impact imaging feasibility.\n* Inability to obtain written consent from research participant or legal guardian\u002Frepresentative.\n* Females of child-bearing potential cannot be pregnant or breast-feeding. Female participants \\>10 years of age or post-menarchal must have a negative serum or urine pregnancy test\n\nAll participants receiving proton therapy at St. Jude Children's Research Hospital will be screened for participation on this research protocol based on the Inclusion Criteria and the Exclusion Criteria. Qualified candidates will be selected during the consultation.","ALL",{"count":18,"type":19},100,"ESTIMATED","INTERVENTIONAL",[22],"NA","Pediatric patients receiving proton therapy for solid tumors or Hodgkin's lymphoma may experience anatomical changes during treatment that can affect proton therapy accuracy. This prospective single-arm study uses regular low-dose imaging to monitor these changes and adjust treatment plans as needed. Participants will receive weekly or every-other-week CT scans, with MRI when appropriate, to assess whether the original plan remains accurate. Treatment plans will be updated if tumor coverage decreases by more than 5% or if radiation dose to normal tissues increases by more than 10%; otherwise, the original plan will continue. The study aims to determine how often plan adjustments are needed and to identify which disease sites are most likely to experience significant anatomical changes during treatment.\n\nPrimary Objective:\n\n* Define the frequency of replanning necessary to ensure tumor coverage never falls below 95% (or 5% drop) of the prescribed daily dose in participants with intact (gross) tumors to keep the tumor control optimal throughout the multi-week treatment regimen.\n* Define the frequency of replanning necessary to ensure organs-at-risk (critical organs) do not deviate by more than 10% of the initially approved dose constraints to keep the normal tissue complication minimal throughout the multi-week treatment regimen.\n\nSecondary Objectives\n\n* Establish a cone beam CT (CBCT)-based framework for quantifying body surface changes throughout the treatment course. This goal will be achieved by developing a novel algorithm that detects and tracks external anatomical variations longitudinally, without requiring CBCT image enhancement, enabling precise assessment of daily participant setup consistency and anatomical stability.\n* Overcome daily CBCT quality limitations by generating synthetic CT images that accurately represent daily anatomy and support proton dose recalculation or verification planning. This goal will be achieved by developing a hybrid pipeline that integrates deep learning models with the deformable image registration algorithm, trained and validated on disease site-specific data. This will enable precise dose mapping and tissue density estimation, directly supporting adaptive planning decisions without the need of diagnostic- quality CT images.",[25,26,27,28,29,30,31],"Pediatric Solid Tumors","Rhabdomyosarcoma","Ewing Sarcoma","Osteosarcoma","Hodgkin Lymphoma","Bone Tumor","Soft Tissue Sarcoma","RECRUITING","2026-08-20",{"date":35,"type":36},"2026-08-21","ACTUAL",{"date":33,"type":36},{"date":39,"type":19},"2031-08",{"name":41,"class":42},"St. Jude Children's Research Hospital","OTHER",1,{"id":45,"slug":46,"hasResults":12,"nctId":47,"briefTitle":48,"officialTitle":49,"acronym":4,"eligibilityCriteria":50,"healthyVolunteers":51,"sex":16,"minAge":52,"maxAge":4,"enrollmentInfo":53,"targetDuration":4,"studyType":20,"phases":55,"briefSummary":56,"conditions":57,"keywords":60,"overallStatus":32,"whyStopped":4,"lastUpdateSubmitDate":67,"lastUpdatePostDateStruct":68,"startDateStruct":70,"completionDateStruct":72,"leadSponsor":74,"locationsCount":43},"100592458","revealing-information-genuinely--honestly-across-time---communication-preferences-visit-100592458","NCT06993688","Revealing Information Genuinely & Honestly Across Time - Communication Preferences Visit","Revealing Information Genuinely & Honestly Across Time - Communication Preferences Visit (RIGHTimeCPV) Pilot","Inclusion Criteria: Patients\n\n* Aged 12-25 years diagnosed with poor prognosis cancer (high risk or otherwise difficult to treat cancers), as defined by a pediatric oncologist estimating odds of overall survival as 50% or less\n* Anticipated by a pediatric oncologist to have one or more disease re-evaluation timepoints over the next six months\n* Not anticipated by a pediatric oncologist to approach end of life in the next three months\n\nInclusion Criteria: Parents\u002FCaregivers\n\n* Aged 18 years or older and\u002For legally emancipated\n* Parent or other self-identified caregiver of a patient of any age with poor prognosis cancer (as defined above)\n\nInclusion Criteria: Oncologists\n\n* Pediatric oncologists who treat eligible patients\u002Fcaregivers at the study site, St. Jude Children's Research Hospital (SJCRH) or its included affiliates:\n\n  * Peoria, IL: The Jim and Trudy Maloof St. Jude Midwest Affiliate Clinic\n  * Charlotte, NC: Novant Health Hemby Children's Hospital\n  * Shreveport, LA: Ochsner LSU Health-Feist-Weiller Cancer Center\n\nInclusion Criteria: Communication Preferences Companions (CPCs)\n\n* Multidisciplinary clinicians from a participant's psychosocial or nursing care team, identified by that participant to serve as their 'communication preferences companion' (CPC) during the pilot\n* Provides clinical care to pediatric cancer patients under the auspices of psychology, social work, spiritual care, child life, cultural navigation, quality of life\u002Fpalliative care, or nursing\n\nExclusion Criteria:\n\n* Does not meet the stated inclusion criteria",true,"12 Years",{"count":54,"type":19},85,[22],"The purpose of this research study is to obtain insights and feedback from patients and parents about a new approach to support conversations about how cancer may affect one's future life and quality of life (i.e., prognostic communication). This study involves creating a personalized approach to discussing prognosis.\n\nPrimary Objectives\n\n* To evaluate the feasibility of implementing the RIGHTimeCPV intervention among pediatric oncology patients, caregivers, and clinicians (referred to herein as \"shareholders\").\n* To assess the acceptability of the intervention across the shareholder groups.\n\nSecondary Objectives\n\n* To explore the potential impact of the RIGHTimeCPV intervention on communication quality, concordance in prognostic understanding, and therapeutic alliance between patients\u002Ffamilies and multidisciplinary clinicians.\n* To explore whether the practice of eliciting, sharing, and honoring individualized communication preferences is sustained by clinicians after participation in the RIGHTimeCPV intervention.",[58,59],"Cancer","Communication",[61,58,62,63,64,65,66],"Prognostic Communication","Poor Prognosis","Participants 12-25 years of age","Caregivers","Pediatric oncologists","Communication Preferences Companions","2026-08-14",{"date":69,"type":36},"2026-08-17",{"date":71,"type":36},"2025-06-16",{"date":73,"type":19},"2027-11-01",{"name":41,"class":42},{"id":76,"slug":77,"hasResults":12,"nctId":78,"briefTitle":79,"officialTitle":80,"acronym":4,"eligibilityCriteria":81,"healthyVolunteers":12,"sex":16,"minAge":82,"maxAge":83,"enrollmentInfo":84,"targetDuration":4,"studyType":20,"phases":86,"briefSummary":87,"conditions":88,"keywords":4,"overallStatus":32,"whyStopped":4,"lastUpdateSubmitDate":92,"lastUpdatePostDateStruct":93,"startDateStruct":94,"completionDateStruct":96,"leadSponsor":98,"locationsCount":43},"100607635","transcutaneous-auricular-vagus-nerve-stimulation-for-insomnia-in-survivors-of-childhood-acute-lymphoblastic-leukemia-100607635","NCT07191119","Transcutaneous Auricular Vagus Nerve Stimulation for Insomnia in Survivors of Childhood Acute Lymphoblastic Leukemia","Feasibility and Efficacy of Transcutaneous Auricular Vagus Nerve Stimulation for Insomnia in Survivors of Childhood Acute Lymphoblastic Leukemia","Inclusion Criteria:\n\n* Survivor of Acute Lymphoblastic Leukemia (ALL)\n* Enrolled on SJLIFE\n* Participant was less than 21 years of age at time of diagnosis.\n* Age 20-50 years at the time of enrollment\n* Insomnia Severity Index \\>=8 (Proxy \\>=8) confirmed prior to enrollment\n* Access to home Wi-Fi and Smartphone\n* Participant is able to speak and understand the English language\n* Participant is able and willing to give consent\n\nExclusion Criteria:\n\n* Unable to understand the details and requirements of the study (at the discretion of the PI)\n* Female participants who are pregnant or planning to become pregnant\n* Presence of implanted electrical medical devices (i.e. pacemaker)\n* Currently taking medication intended to treat neurocognitive impairment (i.e. stimulants) or medications prescribed for seizure management\n* History of skin irritation or other issues during stimulation of inner ear\n* Currently utilizing a technological intervention for a sleep disorder (e.g. CPAP)\n* Medications and behavioral practices (white noise, night-time yoga, etc) are acceptable as long as the insomnia is persistent.\n* History of a contraindicated health condition including:\n* Syncope (CTCAE \\>2)\n* Cardiac dysrhythmia (CTCAE \\>2)\n* Vascular Disease (CTCAE \\>2)\n* Coronary Artery Disease (CTCAE \\>2)\n* Active contraindicated heath condition including:\n* Cranial Nerve Disorder (CTCAE \\>2)\n* Cardiac tachycardia (CTCAE \\>2)\n* Neuropathy (Cranial Nerves) (CTCAE \\>2)\n* Neuralgia (Cranial Nerves) (CTCAE \\>2)\n* Overt Cerebrovascular Accident (CTCAE \\>2)\n* Seizures (Any in most recent 1 year\n* Currently enrolled or participating in any other neurostimulation or neuromodulation ancillary research studies","20 Years","50 Years",{"count":85,"type":19},40,[22],"This pilot study will assess the usefulness and potential effectiveness of using transcutaneous auricular vagus nerve stimulation (tVNS) for treating insomnia in adult survivors of childhood acute lymphoblastic leukemia (ALL). Participants will be randomized to receive either active (verum) or inactive (sham) nightly stimulation using a non-invasive earbud device over two time periods: 2 weeks and 8 weeks. The study will assess adherence to the intervention and estimate its effects on sleep quality, stress, and neurocognitive function.\n\nPrimary Objective:\n\nAim 1: To determine a) short-term and b) long-term feasibility of tVNS in terms of participation in ALL Survivors with moderate to severe insomnia.\n\nAim 2: To estimate the effect size of tVNS on sleep quality, stress, and neurocognitive outcomes in ALL survivors with insomnia.\n\nExploratory Objectives\n\nAim 1: To investigate the onset of tVNS effect via actigraphy measures over the intervention epoch.\n\nAim 2: To estimate the effect size of genetic variants on sleep quality within verum tVNS.",[89,90,91],"Survivor of Childhood Cancer","Insomnia","Acute Lymphoblastic Leukemia (ALL)","2026-08-12",{"date":67,"type":36},{"date":95,"type":36},"2026-03-31",{"date":97,"type":19},"2029-12",{"name":41,"class":42},{"id":100,"slug":101,"hasResults":12,"nctId":102,"briefTitle":103,"officialTitle":103,"acronym":4,"eligibilityCriteria":104,"healthyVolunteers":12,"sex":16,"minAge":4,"maxAge":4,"enrollmentInfo":105,"targetDuration":4,"studyType":107,"phases":4,"briefSummary":108,"conditions":109,"keywords":111,"overallStatus":117,"whyStopped":4,"lastUpdateSubmitDate":92,"lastUpdatePostDateStruct":118,"startDateStruct":120,"completionDateStruct":122,"leadSponsor":124,"locationsCount":43},"100593560","real-world-asparaginase-therapy-toxicity-100593560","NCT07008027","Real World Asparaginase Therapy Toxicity","Inclusion Criteria:\n\n* Diagnosis of acute lymphoblastic leukemia, lymphoblastic lymphoma, or mixed phenotype acute leukemia\n* Enrolled on INITIALL and no more than 10 days after initiation of post-INITIALL therapy\n* Post-INITIALL therapy is:\n\n  * Standard of Care (SOC)\u002FNon Protocol Treatment Plan (NPTP) as per Total therapy or\n  * SJALL23T and not scheduled to receive venetoclax\n\nExclusion Criteria:\n\n* Inability or unwillingness of research participant or legal guardian\u002Frepresentative to give written informed consent.",{"count":106,"type":19},200,"OBSERVATIONAL","This research study is being done to learn more about the short term and long term side effects of treatment with asparaginase drugs, which are commonly used in acute lymphoblastic leukemia (ALL) or acute lymphoblastic lymphoma (LLy) therapy.",[110],"Drug Toxicity",[112,113,114,115,116],"Asparaginase drugs","Toxicity","Acute lymphoblastic leukemia","Acute lymphoblastic lymphoma","Mixed phenotype acute leukemia","NOT_YET_RECRUITING",{"date":119,"type":36},"2026-08-13",{"date":121,"type":19},"2026-09",{"date":123,"type":19},"2031-07",{"name":41,"class":42},{"id":126,"slug":127,"hasResults":12,"nctId":128,"briefTitle":129,"officialTitle":129,"acronym":4,"eligibilityCriteria":130,"healthyVolunteers":12,"sex":131,"minAge":132,"maxAge":133,"enrollmentInfo":134,"targetDuration":4,"studyType":107,"phases":4,"briefSummary":136,"conditions":137,"keywords":140,"overallStatus":117,"whyStopped":4,"lastUpdateSubmitDate":92,"lastUpdatePostDateStruct":145,"startDateStruct":146,"completionDateStruct":147,"leadSponsor":149,"locationsCount":43},"100510936","liver-biopsy-following-gene-therapy-for-hemophilia-100510936","NCT05932914","Liver Biopsy Following Gene Therapy For Hemophilia","Inclusion Criteria:\n\n* Age ≥18 to 80 years\n* Patients, who were enrolled and treated in one of the following clinical trials:\n\n  * AGT4HB (EudraCT number: 2005-005711-17; NCT00979238) - FIX AAV gene therapy trial (sponsor: St. Jude Children's Research Hospital)\n  * GO8 (EudraCT number:2014-003880-38; NCT02576795) - FVIII AAV gene therapy trial (sponsor: University College, London)\n* Able to give informed consent\n* Able to comply with study requirements\n\nExclusion Criteria (Do not apply to participants who will not undergo liver biopsy, and have leftover liver tissue from a previous biopsy procedure, because all exclusion criteria only cover the safety considerations for the biopsy procedure.):\n\n* Any condition that, in the opinion of the investigator or sponsor of the ongoing clinical trial in which the patient is participating in, would prevent the patient from fully complying with the requirements of the clinical trial and\u002For would influence or interfere with evaluation and interpretation of subject safety or efficacy result of that ongoing clinical trial\n* Platelet count \\\u003C140x10\\^9\u002FL\n* INR \\>1.5\n* Abnormal kidney function with estimated GFR \\\u003C50 mL\u002Fmin (calculated using the CKD-EPI equation)\n* Known allergy to iodine-based intravenous contrast agents\n* Known allergy to local or general anesthetics\n* Known allergic reaction to FVIII\u002FFIX concentrate infusions\n* Presence of FVIII inhibitor or FIX inhibitor (historical result can be used if done within 14 weeks of this liver biopsy)\n* Evidence of any bleeding disorder other than hemophilia A or B","MALE","18 Years","80 Years",{"count":135,"type":19},8,"This observational study will obtain liver biopsy samples and evaluate the long-term effect of adeno-associated virus (AAV)-mediated gene therapy on the liver tissue in adult patients with hemophilia A or hemophilia B who have previously been treated with a factor VIII or factor IX gene-containing AAV-vector for liver-targeted gene transfer. Participants are from a cohort of patients treated with AAV-mediated gene transfer and at least 6 months after vector infusion.",[138,139],"Hemophilia A","Hemophilia B",[141,142,143,138,139,144],"AAV-mediated factor VIII (FVIII) gene transfer","AAV-mediated factor IX (FIX) gene transfer","Gene Therapy","Transjugular liver biopsy",{"date":119,"type":36},{"date":121,"type":19},{"date":148,"type":19},"2030-01",{"name":41,"class":42},{"id":151,"slug":152,"hasResults":12,"nctId":153,"briefTitle":154,"officialTitle":155,"acronym":4,"eligibilityCriteria":156,"healthyVolunteers":12,"sex":16,"minAge":157,"maxAge":132,"enrollmentInfo":158,"targetDuration":4,"studyType":20,"phases":159,"briefSummary":161,"conditions":162,"keywords":166,"overallStatus":32,"whyStopped":4,"lastUpdateSubmitDate":171,"lastUpdatePostDateStruct":172,"startDateStruct":174,"completionDateStruct":176,"leadSponsor":178,"locationsCount":179},"100546083","adding-dasatinib-or-venetoclax-to-improve-responses-in-children-with-newly-diagnosed-t-cell-acute-lymphoblastic-leukemia-all-or-lymphoma-t-lly-or-mixed-phenotype-acute-leukemia-mpal-100546083","NCT06390319","Adding Dasatinib Or Venetoclax To Improve Responses In Children With Newly Diagnosed T-Cell Acute Lymphoblastic Leukemia (ALL) Or Lymphoma (T-LLY) Or Mixed Phenotype Acute Leukemia (MPAL)","SJALL23T: Adding Dasatinib Or Venetoclax To Improve Responses In Children With Newly Diagnosed T-Cell Acute Lymphoblastic Leukemia (ALL) Or Lymphoma (T-LLY) Or Mixed Phenotype Acute Leukemia (MPAL)","Inclusion Criteria:\n\n* Enrollment on INITIALL.\n* Age 1-18.99 years at the time of enrollment on INITIALL.\n* T-Acute lymphoblastic leukemia or lymphoblastic lymphoma or mixed phenotype acute leukemia\u002F lymphoma\n* No prior chemotherapy excluding therapy given on or allowed by INITIALL.\n* Patient has completed no more than 3 days of chemotherapy on INITIALL.\n* Direct bilirubin ≤ 1.5x the upper limit of normal for age\n* Alanine aminotransferase (ALT) ≤ 5x the upper limit of normal for age\n* Calculated glomerular filtration rate (GFR) ≥ 50 mL\u002Fmin\u002F1.73m\\^2 using the Bedside Schwartz equation OR creatinine below or equal to the maximum defined below:\n\n  * Age: 1 to \\\u003C 2 years - Maximum serum creatinine (mg\u002FdL): 0.6 (Male), 0.6 (Female)\n  * Age: 2 to \\\u003C 6 years - Maximum serum creatinine (mg\u002FdL): 0.8 (Male), 0.8 (Female)\n  * Age: 6 to \\\u003C 10 years - Maximum serum creatinine (mg\u002FdL): 1 (Male), 1 (Female)\n  * Age: 10 to \\\u003C 13 years - Maximum serum creatinine (mg\u002FdL): 1.2 (Male), 1.2 (Female)\n  * Age: 13 to \\\u003C 16 years - - Maximum serum creatinine (mg\u002FdL): 1.5 (Male), 1.4 (Female)\n  * Age: ≥ 16 years - Maximum serum creatinine (mg\u002FdL): 1.7 (Male), 1.4 (Female)\n\nExclusion Criteria:\n\n* Inability or unwillingness to give informed consent\u002F assent as applicable.\n* Patients with \\> Grade 2 neuropathy at the time of enrollment (participant with T-LLy only).\n* Documented malabsorption syndrome or any other condition that precludes receipt of oral medications.\n* Known HIV infection or active hepatitis B (defined as hepatitis B surface antigen-positive) or C (defined as hepatitis C antibody-positive).\n* Pregnant or lactating.\n* For patients of reproductive potential, unwillingness to use highly effective contraception for the duration of protocol therapy and for 90 days afterwards.\n* Receipt of a strong or moderate CYP3A4 inducer such as rifampin, carbamazepine, phenytoin, and St. John's wort within 7 days of the start of protocol treatment.\n* Consumption of grapefruit, grapefruit products, Seville oranges, or starfruit within 3 days of the start of protocol therapy.","1 Year",{"count":18,"type":19},[160],"PHASE2","This is a clinical trial testing whether the addition of one of two chemotherapy agents, dasatinib or venetoclax, can improve outcomes for children and young adults with newly diagnosed T-cell acute lymphoblastic leukemia and lymphoma or mixed phenotype acute leukemia.\n\nPrimary Objective\n\n* To evaluate if the end of induction MRD-negative rate is higher in patients with T-ALL treated with dasatinib compared to similar patients treated with 4-drug induction on AALL1231.\n* To evaluate if the end of induction MRD-negative rate is higher in patients with ETP or near-ETP ALL treated with venetoclax compared to similar patients treated with 4-drug induction on AALL1231.\n\nSecondary Objectives\n\n* To assess the event free and overall survival of patients treated with this therapy.\n* To compare grade 4 toxicities, event-free survival (EFS) and overall survival (OS) of patients treated with this therapy in induction and reinduction to toxicities of similar patients treated on TOT17.",[163,164,165],"T-cell Acute Lymphoblastic Leukemia","T-cell Lymphoma","Mixed Phenotype Acute Leukemia",[167,168,169,163,164,170],"Newly Diagnosed","Children","Young Adults","Mixed Phenotype Acute Leukemia (MPAL)","2026-08-07",{"date":173,"type":36},"2026-08-10",{"date":175,"type":36},"2024-12-27",{"date":177,"type":19},"2033-12",{"name":41,"class":42},4,{"id":181,"slug":182,"hasResults":12,"nctId":183,"briefTitle":184,"officialTitle":185,"acronym":4,"eligibilityCriteria":186,"healthyVolunteers":12,"sex":16,"minAge":4,"maxAge":187,"enrollmentInfo":188,"targetDuration":4,"studyType":20,"phases":190,"briefSummary":191,"conditions":192,"keywords":194,"overallStatus":32,"whyStopped":4,"lastUpdateSubmitDate":197,"lastUpdatePostDateStruct":198,"startDateStruct":199,"completionDateStruct":201,"leadSponsor":203,"locationsCount":204},"100599503","phase-2-a-phase-ii-study-with-a-safety-run-in-of-the-addition-of-n-803-to-a-chemoimmunotherapy-backbone-for-the-treatment-of-patients-with-relapsed-or-refractory-neuroblastoma-100599503","NCT07085338","A Phase II Study With a Safety Run-In of the Addition of N-803 to a Chemoimmunotherapy Backbone for the Treatment of Patients With Relapsed or Refractory Neuroblastoma","A Phase II Study With a Safety Run-In of the Addition of N-803, a Novel IL-15 Super-Agonist, to a Chemoimmunotherapy Backbone for the Treatment of Patients With Relapsed or Refractory Neuroblastoma","Inclusion Criteria:\n\nAge\n\n\\- Patients must be \\\u003C 30 at the time of enrollment on study.\n\nDiagnosis\n\n\\- Patients must have had histologic verification of neuroblastoma or demonstration of neuroblastoma cells in the bone marrow with elevated urinary or serum catecholamines \\[i.e., \\> 2 x upper limit of normal (ULN)\\], at the time of initial diagnosis.\n\nDisease Risk Group\n\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 were then reclassified as high-risk neuroblastoma prior to enrollment are also eligible.\n\nResponse to Prior Therapy (using INRC definitions)\n\n\\- Patients must have at least ONE (recurrent\u002Fprogressive, refractory, or persistent) of the following:\n\n* 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* Refractory disease: A best overall response of no response\u002Fstable disease since diagnosis of high-risk neuroblastoma AND after at least 4 cycles of induction therapy.\n* Persistent disease: A best overall response of partial response since diagnosis of high-risk neuroblastoma AND after at least 4 cycles of induction therapy\n\nSites of Disease\n\n\\- Patients must have at least ONE of the following (lesions may have received prior radiation therapy if they meet the other criteria listed below) based on institutional assessment:\n\nBone Sites\n\n• MIBG avid tumors: patients must meet one of the following criteria:\n\na. 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 lesions on SPECT\u002FCT. A biopsy is not required unless the above imaging criteria are not met\n\nb. Patients with persistent disease: i. If a patient has 3 or more MIBG avid bone lesions, then no biopsy is required.\n\nii. If a patient has only 1 or 2 MIBG avid bone lesion sites, 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* For MIBG non-avid tumors, patients must have at least an FDG-PET avid site and meet the following criteria:\n\n  1. Biopsy confirmation of neuroblastoma and\u002For ganglioneuroblastoma at any time prior to enrollment of at least one FDG-PET avid site.\n\n     Bone Marrow\n\n     \\- 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. NOTE: Patients with bone marrow disease only will be eligible if they have more than 5% disease involvement (documented neuroblastoma cells) in at least one sample from bilateral bone marrow biopsies.\n\n     Soft Tissue Sites\n\n     \\- At least one soft tissue lesion that meets criteria for a TARGET lesion as defined by:\n* 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* 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  a. For MIBG avid tumors: lesion must be MIBG avid and meet one of the following criteria: i. For patients with recurrent\u002Fprogressive or refractory disease: no biopsy is required ii. For patients with persistent disease:\n* If a patient has 3 or more MIBG avid soft tissue lesions, then no biopsy is required.\n* If a patient has only 1 or 2 MIBG avid soft tissue lesion sites) 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 at least one FDG avid site and meet the following criteria: i. Biopsy confirmation of neuroblastoma and\u002For ganglioneuroblastoma from a lesion at any time prior to enrollment of at least one FDG-PET avid site.\n\nii. 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\nPerformance Status\n\n\\- Patients must have 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\nPrior Therapy\n\n\\- Patients must have fully recovered from the acute toxic effects of all prior chemotherapy, immunotherapy, or radiotherapy prior to study registration. Patients must not have received the therapies indicated below within the specified time prior to registration on this study as follows:\n\nTable 2: Prior Therapies List Type of Therapy Specified Time Period Additional Comments Myelosuppressive Chemotherapy ≤ 14 days This includes cytotoxic agents given on a low dose metronomic regimen as well as retinoids.\n\nBiologic Antineoplastic (anti-neoplastic agents)1 ≤ 7 days Monoclonal Antibodies ≤ 7 days or 3 half-lives whichever is longer, but no longer than 30 days (with recovery of any associated toxicities).\n\nCellular Therapy (e.g., modified T cells, NK cells, dendritic cells, etc.) ≤ 21 days and with recovery of all associated toxicities Radiation Small port radiation ≤ 7 days Large field radiation therapy ≤ 12 weeks i.e., total body irradiation, craniospinal, whole abdominal, total lung, \\> 50% marrow space Other Substantial Bone Marrow Radiation ≤ 6 weeks 131I-MIBG therapy ≤ 6 weeks Hematopoietic Stem Cell Transplant Autologous Stem Cell Infusion Following Myeloablative Therapy ≤ 6 weeks Patients who have received an autologous stem cell infusion to support non-myeloablative therapy (such as 131I-MIBG) are eligible at any time as long as they meet the other criteria for eligibility.\n\nAny other investigational agents (covered under another IND) ≤ 14 days\n\n1. Anti-cancer agents not known to be myelosuppressive (e.g., not associated with reduced platelet or ANC counts)\n\n   Concomitant Therapy Restrictions\n\n   \\- Patients must not have received the concomitant medications indicated below within the specified time prior to study enrollment or planned treatment start date on this study as follows:\n\n   • No other anti-cancer agents or radiotherapy at the time of study registration or while on study.\n\n   • 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\n   • Patients must not have received 0.5 mg\u002Fkg\u002Fday (prednisone equivalent) doses of systemic steroids for at least 7 days prior to study enrollment.\n\n   • Inhaled steroids are permitted to treat reactive airways\n\n   • \\\u003C 2mg\u002Fkg of hydrocortisone or equivalent is permitted as blood product premedication to avoid allergic reactions.\n\n   • Physiologic hydrocortisone dosing is permitted for patients with known adrenal insufficiency.\n   * The use of dexamethasone as an antiemetic is not permitted.\n   * Irinotecan is a substrate for CYP3A4 (major) and CYP2B6 (major). Patients who have received drugs that are strong inducers or inhibitors of CYP3A4 within 7 days prior to study enrollment are not eligible. The use of strong inducers or inhibitors of CYP3A4 (Appendix II) and CYP2B6 (e.g., carbamazepine) should be avoided for the duration of protocol therapy. Consult drug information references for further information. In addition, concomitant use of BCRP inhibitors (cyclosporine, eltrombopag, gefitinib), and UGT1A1 inhibitors (diclofenac, ketoconazole, probenecid, silibinin, nilotinib, and atazanavir) should be avoided due to potential increased risk of irinotecan toxicity.\n\n     * Moderate inducers or inhibitors of CYP3A4 (Appendix II) should also be avoided during protocol therapy if reasonable alternatives exist.\n\n   Organ Function Requirements\n\n   Hematologic Function:\n\n   \\- Patients must meet the following hematologic criteria for enrollment regardless of bone marrow disease involvement:\n   1. ANC ≥750\u002FμL, (no short-acting hematopoietic growth factors ≤ 7 days of blood draw documenting eligibility and no long-acting hematopoietic growth factors ≤ 14 days of blood draw documenting eligibility); and\n   2. Platelet count ≥ 75,000\u002FμL, transfusion independent (no platelet transfusions ≤ 7 days of blood draw documenting eligibility).\n\n   Renal Function\n\n   a. Patients must have adequate renal function defined as age-adjusted serum creatinine ≤1.5 ULN for age (see below):\n\n   Table 3: Age-Adjusted Serum Creatinine Age Maximum Serum Creatine (mg\u002FdL) Male Female\n\n1 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 1.4 \\> 16 years 1.7 1.4\n\nLiver Function\n\n1. Total bilirubin ≤ 1.5 x ULN for age; and,\n2. SGPT (ALT) ≤ 225 U\u002FL (≤ 5x ULN). Note that for ALT, the upper limit of normal for all sites is defined as 45 U\u002FL.\n\nCardiac Function\n\n1. Shortening fraction of ≥ 27% by ECHO, or\n2. Ejection fraction of ≥ 50% by ECHO or gated radionuclide study.\n\nPulmonary Function\n\nNo evidence of dyspnea at rest, no exercise intolerance.\n\nAdequate Central Nervous System Function\n\n1. Patients with a history of CNS disease must have no clinical or radiological evidence of active CNS disease at the time of study enrollment\n2. Patients with seizure disorders may be enrolled if seizures are well controlled on anti-seizure medications\n3. CNS toxicity ≤ Grade 2\n\n   Reproductive Function\n\n   \\- All post-menarchal females must have a negative serum or urine beta-HCG ≤ 7 days prior to registration. 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, diaphragm with spermicide, condom with spermicide, or abstinence) or to abstain from heterosexual intercourse for the duration of their participation in the study.\n\n   Central Nervous System (CNS)\n\n   \\- 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\n   \\- Patients with skull-based tumors with direct intracranial extension are eligible if there are no neurologic signs or symptoms related to the lesion.\n\n   Exclusion Criteria:\n\n   \\- Pregnancy, 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. Females of childbearing potential must have a negative pregnancy test to be eligible for this study.\n\n   \\- Patients who, in the opinion of the investigator, may not be able to comply with the safety monitoring requirements of the study.\n\n   \\- Patients with disease of any major organ system that would compromise their ability to withstand therapy.\n\n   \\- Patients who have undergone a prior allogeneic stem cell or solid organ transplant.\n\n   \\- Patients who are on hemodialysis.\n\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\n   \\- Known history of human immunodeficiency virus (HIV) infection, hepatitis B, or hepatitis C. Testing is not required in the absence of clinical findings or suspicions.\n\n   \\- Patients who require or are likely to require pharmacologic doses of systemic corticosteroids while receiving treatment on this study are ineligible. The only exception is for 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 in order to avoid allergic transfusion reactions. The use of conventional doses of inhaled steroids for the treatment of reactive airway disease is permitted, as is the use of physiological doses of steroids for patients with known adrenal insufficiency.\n\n   \\- Patients on any other immunosuppressive medications (e.g., cyclosporine, tacrolimus) are not eligible.\n\n   \\- Patients must not have received enzyme-inducing anticonvulsants including phenytoin, phenobarbital, or carbamazepine for at least 7 days prior to study enrollment. Patients receiving non-enzyme inducing anticonvulsants such as gabapentin, valproic acid, or levetiracetam will be eligible.\n\n   \\- Patients who have received drugs that are strong inducers or inhibitors of CYP3A4 within 7 days prior to study enrollment are not eligible.\n\n   \\- Patients must not have been diagnosed with myelodysplastic syndrome or with any malignancy other than neuroblastoma.\n\n   \\- Patients with symptoms of congestive heart failure are not eligible.\n\n   \\- Patients must not have \\> Grade 2 diarrhea.\n\n   \\- Patients with a history of progressive disease while receiving therapy per ANBL1221 (irinotecan\u002Ftemozolomide\u002Fdinutuximab\u002FGMCSF).\n\n   \\- Patients with a history of Grade 4 allergic reactions to anti-GD2 antibodies or reactions that required permanent discontinuation of the anti-GD2 therapy are not eligible.\n\n   \\- Patients with elevated catecholamines (i.e., \\> 2 x ULN) only and no evidence of disease are NOT eligible for this study.","30 Years",{"count":189,"type":19},54,[160],"The study participant is being asked to take part in this research study because the participant has been diagnosed with neuroblastoma that did not fully respond to previous treatment (refractory), or it has returned after treatment (relapsed).\n\nPrimary Aims\n\n* To evaluate if the administration of N-803 in combination with irinotecan, temozolomide, hu14-18K322A, and GM-CSF in patients with relapsed\u002Frefractory neuroblastoma is feasible and tolerable\n* To determine if the response rate of N-803 with irinotecan, temozolomide, hu14.18K322A and GM-CSF in patients with relapsed\u002Frefractory neuroblastoma is superior to the combination of irinotecan, temozolomide, hu14.18K322A, and GM-CSF\n\nSecondary Aims\n\n* To describe the toxicity profile of N-803 administered with irinotecan, temozolomide, hu14.18K322A and GM-CSF\n* To evaluate and compare the progression free survival (PFS) and overall survival (OS) of and between patients receiving irinotecan, temozolomide, hu14.18K322A and GM-CSF with and without N-803",[193],"Neuroblastoma Recurrent",[195,196],"relapse","refractory","2026-08-06",{"date":171,"type":36},{"date":200,"type":36},"2025-11-10",{"date":202,"type":19},"2029-02-01",{"name":41,"class":42},5,{"id":206,"slug":207,"hasResults":12,"nctId":208,"briefTitle":209,"officialTitle":209,"acronym":4,"eligibilityCriteria":210,"healthyVolunteers":51,"sex":16,"minAge":211,"maxAge":4,"enrollmentInfo":212,"targetDuration":4,"studyType":107,"phases":4,"briefSummary":214,"conditions":215,"keywords":4,"overallStatus":32,"whyStopped":4,"lastUpdateSubmitDate":217,"lastUpdatePostDateStruct":218,"startDateStruct":219,"completionDateStruct":221,"leadSponsor":223,"locationsCount":43},"100501509","gene-therapy-communication-use-of-a-needs-assessment-to-drive-decision-aids-for-gene-therapy-for-rare-diseases-genetx-100501509","NCT05810181","Gene Therapy Communication: Use of a Needs Assessment to Drive Decision-AIDS for Gene Therapy for Rare Diseases (GENETX)","Inclusion Criteria:\n\n1. For Group 1 participants only (Undergone Gene Therapy):\n\n   * Parent\u002Fcaregiver whose child has undergone gene therapy. OR Parent\u002Fcaregiver of a child who died after receiving gene therapy at least 6 months prior to enrollment, but no more than 24 months prior to enrollment, to be contacted no sooner than 3 months after the death has occurred and no longer than 2 years. OR Patients age 8 and above who have undergone gene therapy.\n   * Willingness to participate in one-on-one video interview with a study team member using a personal mobile device or computer with working internet connection.\n   * Must be willing to provide verbal informed consent.\n   * Release of information form signed by participant providing our study team with permission to contact healthcare provider to verify their diagnosis and receipt of gene therapy (if received).\n   * Successful verification of diagnosis of rare genetic disease targeted for treatment using gene therapy.\n   * A positive confirmation on receipt of gene therapy and type received from their healthcare provider (only for those received gene therapy).\n2. For Group 2 participants only (Offered, but did not Undergo Gene Therapy):\n\n   * Parent\u002Fcaregiver of children (or patients 8 and above ) with a rare genetic disease who had been offered but were not eligible for a trial or decided against receiving gene therapy.\n   * Willingness to participate in one-on-one video interview with a study team member using a personal mobile device or computer with working internet connection.\n   * Must be willing to provide verbal informed consent.\n   * Signed release of information form providing GeneTx study team with permission to contact participant's healthcare provider to verify the diagnosis.\n   * Successful verification of diagnosis of rare genetic disease targeted for treatment using gene therapy.\n3. For Group 3 participants only (Provider Interviews):\n\n   * Healthcare worker who has provided care to ≥ 2 patients receiving gene therapy.\n   * Willingness to participate in one-on-one video (or in-person) interview with a study team member using a personal mobile device or computer with working internet connection.\n   * Informed consent from a study participant.\n4. For Group 4 participants only (Undergone Gene Therapy for Bone Marrow Failure Condition):\n\n   * Parent\u002Fcaregiver whose child has undergone gene therapy. OR Parent\u002Fcaregiver of a child who died after receiving gene therapy at least 6 months prior to enrollment, but no more than 24 months prior to enrollment, to be contacted no sooner than 3 months after the death has occurred and no longer than 2 years. OR Patients age 8 and above who have undergone gene therapy.\n   * Willingness to participate in one-on-one video interview with a study team member using a personal mobile device or computer with working internet connection.\n   * Must be willing to provide verbal informed consent.\n   * Release of information form signed by participant providing our study team with permission to contact healthcare provider to verify their diagnosis and receipt of gene therapy (if received).\n   * Successful verification of diagnosis of rare genetic disease targeted for treatment using gene therapy.\n   * A positive confirmation on receipt of gene therapy and type received from their healthcare provider (only for those received gene therapy).\n5. For Group 5 participants only (Offered, but did not Undergo Gene Therapy for Bone Marrow Failure Condition ):\n\n   * Parent\u002Fcaregiver of children (or patients 8 and above ) with a bone marrow failure disease who had been offered but were not eligible for a trial or decided against receiving gene therapy.\n   * Willingness to participate in one-on-one video interview with a study team member using a personal mobile device or computer with working internet connection.\n   * Must be willing to provide verbal informed consent.\n   * Signed release of information form providing GeneTx study team with permission to contact participant's healthcare provider to verify the diagnosis.\n   * Successful verification of diagnosis of rare genetic disease targeted for treatment using gene therapy.\n6. For Group 6 participants only (Never offered gene therapy for Bone Marrow Failure Condition):\n\n   * Parent\u002Fcaregiver of children (or patients 8 and above ) with a bone marrow failure disease who had not been offered gene therapy.\n   * Willingness to participate in one-on-one video interview with a study team member using a personal mobile device or computer with working internet connection.\n   * Must be willing to provide verbal informed consent.\n   * Signed release of information form providing GeneTx study team with permission to contact participant's healthcare provider to verify the diagnosis.\n   * Successful verification of diagnosis of rare genetic disease targeted for treatment using gene therapy.\n7. For Group 7 participants only (Provider Interviews for Bone Marrow Failure Condition):\n\n   * Healthcare worker who has provided care to ≥ 2 patients receiving gene therapy.\n   * Willingness to participate in one-on-one video (or in-person) interview with a study team member using a personal mobile device or computer with working internet connection.\n   * Informed consent from a study participant.\n\nExclusion Criteria (for all 7 groups):\n\n* Participants who are unable to converse fluently in English will be excluded.\n* Inability or unwillingness of research participant to give verbal informed consent.\n* Participants who lack access to a computer or mobile device that supports video communications will be excluded.\n* Condition or chronic illness, which in the opinion of the PI\u002FCo-I, makes participation unsafe or untenable (i.e., cognitive impairment, concurrent acute morbidity).","8 Years",{"count":213,"type":19},145,"This prospective mixed-method interview study aims to qualitatively describe the beliefs, attitudes, and informational needs around gene therapy for rare pediatric diseases among patients and parents of children with a rare disease targeted for treatment using gene therapy techniques. Using learned insights, the team will develop an online platform providing educational content and patient decision aids for patients and their families.",[216],"Sickle Cell Disease","2026-08-05",{"date":173,"type":36},{"date":220,"type":36},"2023-06-01",{"date":222,"type":19},"2027-12",{"name":41,"class":42},{"id":225,"slug":226,"hasResults":12,"nctId":227,"briefTitle":228,"officialTitle":228,"acronym":4,"eligibilityCriteria":229,"healthyVolunteers":12,"sex":16,"minAge":4,"maxAge":187,"enrollmentInfo":230,"targetDuration":4,"studyType":20,"phases":232,"briefSummary":234,"conditions":235,"keywords":278,"overallStatus":32,"whyStopped":4,"lastUpdateSubmitDate":280,"lastUpdatePostDateStruct":281,"startDateStruct":283,"completionDateStruct":285,"leadSponsor":287,"locationsCount":288},"100534472","phase-1-nrsts2021-a-risk-adapted-study-evaluating-maintenance-pazopanib-limited-margin-dose-escalated-radiation-therapy-and-selinexor-in-non-rhabdomyosarcoma-soft-tissue-sarcoma-nrsts-100534472","NCT06239272","NRSTS2021, A Risk Adapted Study Evaluating Maintenance Pazopanib, Limited Margin, Dose-Escalated Radiation Therapy and Selinexor in Non-Rhabdomyosarcoma Soft Tissue Sarcoma (NRSTS)","Inclusion Criteria:\n\nInclusion Criteria - All Patients\n\n* Patients must be 1-30 years at the time of the biopsy that established the diagnosis of NRSTS.\n* Surgical Resection: Patients who had an upfront resection prior to enrollment will be eligible if they are able to begin therapy within 28 days of resection assuming other eligibility criteria are met. Delayed resection is preferred for all patients with intermediate and high-risk disease.\n* Lansky performance status score ≥ 60 for patients ≤ 16 years of age. Karnofsky performance status score ≥ 60 for patients \\>16 years of age. Note 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\nDiagnosis\n\n• Patients with CIC-DUX 4 rearranged sarcomas will be enrolled on the high-risk stratum only, regardless of presence of metastasis, size, or resection status.\n\nPatient has low-risk disease if the patient has a:\n\n* Low-grade tumor of any size where R0 or R1 surgical margins are anticipated or achieved.\n* High-grade tumors that are \\\u003C 5 cm where R0 or R1 resection margins are anticipated or achieved.\n\nPatient must have adequate organ function in the organs that will be within the radiotherapy field.\n\nAdequate renal function defined as:\n\n* Creatinine clearance or radioisotope GFR \\> 70 mL\u002Fmin\u002F1.73 m2, or\n* A normal serum creatinine based on age\u002Fgender as follows\n\nAge Maximum Serum Creatinine (mg\u002FdL) Male Female 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 1.4 \\> 16 years 1.5 1.4\n\nAdequate liver function defined as:\n\n* Total bilirubin \\\u003C 1.5 x upper limit of normal (ULN) for age\n* SGOT (AST) or SGPT (ALT) \\\u003C 2.5 x ULN for age\n\nAdequate cardiac function defined as:\n\n* Ejection fraction of \\> 55% by echocardiogram or cardiac MRI\n* QTc \\\u003C 480 msec\n\nAdequate pulmonary function defined as:\n\n* No evidence of dyspnea at rest, no exercise intolerance, and a resting pulse oximetry reading \\> 94% on room air if there is clinical indication for determination.\n\nInclusion Criteria - Intermediate and High Risk Participants\n\nPatient has intermediate-risk if the patient has a:\n\n* Low-grade non-metastatic initially unresectable disease at study enrollment where delayed resection is planned.\n* High-grade \\\u003C 5 cm non-metastatic initially unresectable disease at study enrollment where delayed resection is planned. Of note, patients enrolled on the low-risk arm who were unable to achieve gross total resection where delayed re-resection is planned are eligible for this arm.\n* High-grade tumor \\> 5 cm that is potentially resectable.\n\nPatient high-risk if the patient has:\n\n* Metastatic disease at presentation\n* Unresectable disease at study enrollment where delayed resection is not anticipated. Of note, patients enrolled on the low-risk arm who were unable to achieve gross total resection where delayed re-resection is not-planned are eligible for this arm.\n* CIC-DUX4 rearranged sarcoma\n\nOrgan Function\n\nAdequate bone marrow function defined as:\n\n* Absolute neutrophil count \\> 1000\u002FµL\n* Platelet count \\> 100,000\u002FµL\n* Hemoglobin \\> 8 g\u002FdL for patients \\\u003C 16 years of age\n* Hemoglobin \\> 9 g\u002FdL for patients \\> 16 years of age\n\nNote: No transfusions are permitted 7 days prior to laboratory studies to determine eligibility.\n\nAdequate renal function defined as:\n\n* Creatinine clearance or radioisotope GFR \\> 70 mL\u002Fmin\u002F1.73 m2, or\n* A normal serum creatinine based on age\u002Fgender as follows\n\nAge Maximum Serum Creatinine (mg\u002FdL) Male Female 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 1.4 \\> 16 years 1.5 1.4\n\nAdequate liver function defined as:\n\n* Total bilirubin \\\u003C 1.5 x upper limit of normal (ULN) for age\n* SGOT (AST) or SGPT (ALT) \\\u003C 2.5 x ULN for age\n\nAdequate cardiac function defined as:\n\n* Ejection fraction of \\> 55% by echocardiogram or cardiac MRI\n* QTc \\\u003C 480 msec\n\nAdequate pulmonary function defined as:\n\n* No evidence of dyspnea at rest, no exercise intolerance, and a resting pulse oximetry reading \\> 94% on room air if there is clinical indication for determination.\n\nAnticoagulation\n\nPatients on low molecular weight heparin, warfarin (with a stable INR), or direct oral anticoagulants (DOAC) who have been on a stable dose of are eligible. Patients being treated for a pulmonary embolism or deep venous thrombosis (DVT) must have been treated for a minimum of 6 weeks prior to starting therapy treatment.\n\nLife Expectancy\n\nPatient must have a life expectancy of at least 3 months with appropriate therapy.\n\nExclusion Criteria:\n\n* Patients with known primary CNS sarcoma or CNS metastases are not eligible. Note: Brain imaging is not an eligibility requirement. Tumors with intracranial extension will be allowed.\n* Patients with the following histologic diagnosis are not eligible: intermediate locally aggressive tumors as defined by WHO, malignant rhabdoid tumor, alveolar soft part sarcoma, infantile fibrosarcoma, unresectable\u002Fmetastatic dermatofibrosarcoma protuberans, inflammatory myofibroblastic tumor, desmoid fibromatosis, rhabdomyosarcoma, desmoplastic small round cell tumor, BCOR-CCNB3 fusion positive sarcoma.\n* Bleeding diathesis: Patients with evidence of active bleeding or bleeding diathesis will be excluded (Note: Patients aged \\> 17 years with excess of 2.5 mL of hemoptysis are not eligible).\n* Uncontrolled hypertension: Patients with uncontrolled hypertension (CTCAE v5 Grade ≥ 2) are ineligible. Hypertension must be well controlled on stable doses of medication for at least two weeks.\n\nPrior Therapy\n\n* Patients must have had no prior systemic therapy for the treatment of the NRSTS\n* Patients must have had no prior anthracycline or ifosfamide chemotherapy\n* Patients must have had no prior use of pazopanib or similar multi-targeted TKI.\n\nPatients must have had no prior radiotherapy to tumor-involved sites.\n\nNote: Patients previously treated for a non-NRSTS cancer are eligible provided they meet the prior therapy requirements. Patients who have had chemotherapy or radiotherapy within 4 weeks (6 weeks for nitrosoureas or mitomycin C) prior to entering the study or those who have not recovered from adverse events due to agents administered more than 4 weeks earlier are excluded.\n\n* CYP3A4 Substrates WITH Narrow Therapeutic Indices: Patients chronically receiving medications known to be metabolized by CYP3A4 and with narrow therapeutic indices within 7 days prior to study enrollment, including but not limited to pimozide, aripiprazole, triazolam, ergotamine and halofantrine are not eligible. Note: the use of fentanyl is permitted.\n* CYP3A4 Inhibitors: Patients chronically receiving drugs that are known potent CYP3A4 inhibitors within 7 days prior to study enrollment, including but not limited to itraconazole, clarithromycin, erythromycin, many NNRTIs, diltiazem, verapamil, and grapefruit juice are not eligible.\n* CYP3A4 Inducers: Patients chronically receiving drugs that are known potent CYP3A4 inducers within 14 days prior to study enrollment, including but not limited to carbamazepine, phenobarbital, phenytoin, rifampin, and St. John's wort are not eligible (with the exception of glucocorticoids).\n* Certain medications that are associated with a risk for QTc prolongation and\u002For Torsade's de Pointes, although not prohibited, should be avoided or replaced with medications that do not carry these risks, if possible.\n* Subjects with any condition that may impair the ability to absorb oral medications\u002Finvestigational product including:\n\n  * prior surgical procedures affecting absorption including, but not limited to major resection of stomach or small bowel\n  * active peptic ulcer disease\n  * malabsorption syndrome\n\n3.4.12 Thyroid Replacement Therapy: Patients who require thyroid replacement therapy are not eligible if they have not been receiving a stable replacement dose for at least 4 weeks prior to study enrollment.\n\n3.4.13 Subjects with any condition that may increase the risk of gastrointestinal bleeding or gastrointestinal perforation, including:\n\n* active peptic ulcer disease\n* known intraluminal metastatic lesions\n* inflammatory bowel disease (e.g., ulcerative colitis, Crohn's disease) or other gastrointestinal conditions which increase the risk of perforation\n* history of abdominal fistula, gastrointestinal perforation or intra- abdominal abscess within 28 days prior to beginning study treatment.\n\n3.4.14 Pulmonary embolism or DVT. Patients must not have experienced:\n\n* An untreated pulmonary embolism or DVT in last 6 months or\n* treated pulmonary embolism or DVT which has been treated with therapeutic anticoagulation for less than 6 weeks\n* arterial thrombosis in last 12 months\n\nHistory of serious or non-healing wound, ulcer, or bone fracture.\n\nUncontrolled intercurrent illness including, but not limited to, ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness\u002Fsocial situations that would limit compliance with study requirements.\n\nHIV-positive subjects on combination antiretroviral therapy are ineligible because of the potential for pharmacokinetic interactions with pazopanib. In addition, these subjects are at increased risk of lethal infections when treated with marrow-suppressive therapy.\n\nPatients who are receiving any other investigational agent(s).\n\nPregnancy and Breast Feeding\n\n* Pregnancy and Breast Feeding Female patients who are pregnant are ineligible due to risks of fetal and teratogenic adverse events as seen in animal\u002Fhuman studies.\n* Lactating females are not eligible unless they have agreed not to breastfeed their infants during treatment and for a period of 1 month following completion of treatment.\n* Female patients of childbearing potential are not eligible unless a negative pregnancy test result has been obtained.\n* Unwillingness to use an effective contraceptive method for the duration of their study participation and for at least 1 month after treatment is completed if sexually active with reproductive potential.",{"count":231,"type":19},139,[233,160],"PHASE1","The study participant has been diagnosed with non-rhabdomyosarcoma (NRSTS).\n\nPrimary Objectives\n\nIntermediate-Risk\n\n* To estimate the 3-year event-free survival for intermediate-risk patients treated with ifosfamide, doxorubicin, pazopanib, surgery, and maintenance pazopanib, with or without RT.\n* To characterize the pharmacokinetics of pazopanib and doxorubicin in combination with ifosfamide in intermediate-risk participants, to assess potential covariates to explain the inter- and intra-individual pharmacokinetic variability, and to explore associations between clinical effects and pazopanib and doxorubicin pharmacokinetics.\n\nHigh-Risk\n\n* To estimate the maximum tolerated dose (MTD) and\u002For the recommended phase 2 dosage (RP2D) of selinexor in combination with ifosfamide, doxorubicin, pazopanib, and maintenance pazopanib in high-risk participants.\n* To characterize the pharmacokinetics of selinexor, pazopanib and doxorubicin in combination with ifosfamide in high-risk participants, to assess potential covariates to explain the inter- and intra-individual pharmacokinetic variability, and to explore associations between clinical effects and selinexor, pazopanib and doxorubicin pharmacokinetics.\n\nSecondary Objectives\n\n* To estimate the cumulative incidence of primary site local failure and distant metastasis-free, disease-free, event-free, and overall survival in participants treated on the risk-based treatment strategy defined in this protocol.\n* To define and describe the CTCAE Grade 3 or higher toxicities, and specific grade 1-2 toxicities, in low- and intermediate-risk participants.\n* To study the association between radiation dosimetry in participants receiving radiation therapy and the incidence and type of dosimetric local failure, normal adjacent tissue exposure, and musculoskeletal toxicity.\n* To evaluate the objective response rate (complete and partial response) after 3 cycles for high-risk patients receiving the combination of selinexor with ifosfamide, doxorubicin, pazopanib, and maintenance pazopanib.\n* To assess the relationship between the pharmacogenetic variation in drug-metabolizing enzymes or drug transporters and the pharmacokinetics of selinexor, pazopanib, and doxorubicin in intermediate- or high-risk patients.\n\nExploratory Objectives\n\n* To explore the correlation between radiographic response, pathologic response, survival, and toxicity, and tumor molecular characteristics, as assessed through next-generation sequencing (NGS), including whole genome sequencing (WGS), whole exome sequencing (WES), and RNA sequencing (RNAseq).\n* To explore the feasibility of determining DNA mutational signatures and homologous repair deficiency status in primary tumor samples and to explore the correlation between these molecular findings and the radiographic response, survival, and toxicity of patients treated on this protocol.\n* To explore the feasibility of obtaining DNA methylation profiling on pretreatment, post-induction chemotherapy, and recurrent (if possible) tumor material, and to assess the correlation with this and pathologic diagnosis, tumor control, and survival outcomes where feasible.\n* To explore the feasibility of obtaining high resolution single-cell RNA sequencing of pretreatment, post-induction chemotherapy, and recurrent (if possible) tumor material, and to characterize the longitudinal changes in tumor heterogeneity and tumor microenvironment.\n* To explore the feasibility of identifying characteristic alterations in non-rhabdomyosarcoma soft tissue sarcoma in cell-free DNA (cfDNA) in blood as a non-invasive method of detecting and tracking changes during therapy, and to assess the correlation of cfDNA and mutations in tumor samples.\n* To describe cardiovascular and musculoskeletal health, cardiopulmonary fitness among children and young adults with NRSTS treated on this protocol.\n* To investigate the potential prognostic value of serum cardiac biomarkers (high-sensitivity cardiac troponin I (hs-cTnI), N-terminal pro B-type natriuretic peptide (NT-Pro-BNP), serial electrocardiograms (EKGs), and serial echocardiograms in patients receiving ifosfamide, doxorubicin, and pazopanib, with or without selinexor.\n* To define the rates of near-complete pathologic response (\\>90% necrosis) and change in FDG PET maximum standard uptake value (SUVmax) from baseline to week 13 in intermediate risk patients with initially unresectable tumors treated with induction pazopanib, ifosfamide, and doxorubicin, and to correlate this change with tumor control and survival outcomes.\n* To determine the number of high-risk patients initially judged unresectable at diagnosis that are able to undergo primary tumor resection after treatment with ifosfamide, doxorubicin, selinexor, and pazopanib.\n* To identify the frequency with which assessment of volumes of interest (VOIs) of target lesions would alter RECIST response assessment compared with standard linear measurements.",[236,237,238,239,240,241,242,243,244,245,246,247,248,249,250,251,252,253,254,255,256,257,258,259,260,261,262,263,264,265,266,267,268,269,270,271,272,273,274,275,276,277],"Adipocytic Neoplasm","Liposarcoma","Atypical Fibroxanthoma","Angiomatoid Fibrous Histiocytoma","Fibrosarcoma NOS","Myxofibrosarcoma","Angiosarcoma","Osteosarcoma, Extraskeletal","Dedifferentiated Liposarcoma","Myxoid Liposarcoma","Pleomorphic Liposarcoma","Myxoid Pleomorphic Liposarcoma","Low Grade Fibromyxoid Sarcoma","Sclerosing Epithelioid Fibrosarcoma","Malignant Tenosynovial Giant Cell Tumor of Soft Tissue","Epithelioid Hemangioendothelioma","Glomus Tumor","Inflammatory Leiomyosarcoma","Leiomyosarcoma","Ossifying Fibromyxoid Tumor, Malignant","Myoepithelioma","Synovial Sarcoma","Epithelioid Sarcoma","Perineurioma, Malignant","Clear Cell Sarcoma","Extraskeletal Myxoid Chondrosarcoma","Granular Cell Tumor, Malignant","Melanotic Malignant Nerve Sheath Tumor","Malignant Peripheral Nerve Sheath Tumor","Perivascular Epithelioid Tumor, Malignant","Intimal Sarcoma","Myoepithelial Carcinoma","Undifferentiated Sarcoma","Pleomorphic Sarcoma, Undifferentiated","Round Cell Sarcoma, Undifferentiated","NTRK-Rearranged Spindle Cell Neoplasm","Phosphaturic Mesenchymal Tumor, Malignant","Round Cell Sarcoma","Well Differentiated Liposarcoma","Giant Cell Tumor of Soft Parts NOS","Low Grade Myofibroblastic Sarcoma","Dermatofibrosarcoma Protuberans, Fibrosarcomatous",[279],"Proton therapy","2026-07-31",{"date":282,"type":36},"2026-08-03",{"date":284,"type":36},"2024-03-27",{"date":286,"type":19},"2037-06",{"name":41,"class":42},7,{"id":290,"slug":291,"hasResults":12,"nctId":292,"briefTitle":293,"officialTitle":294,"acronym":4,"eligibilityCriteria":295,"healthyVolunteers":12,"sex":16,"minAge":157,"maxAge":187,"enrollmentInfo":296,"targetDuration":4,"studyType":20,"phases":298,"briefSummary":299,"conditions":300,"keywords":4,"overallStatus":32,"whyStopped":4,"lastUpdateSubmitDate":280,"lastUpdatePostDateStruct":304,"startDateStruct":305,"completionDateStruct":307,"leadSponsor":309,"locationsCount":310},"100529691","phase-1-study-of-revumenib-azacitidine-and-venetoclax-in-pediatric-and-young-adult-patients-with-refractory-or-relapsed-acute-myeloid-leukemia-100529691","NCT06177067","Study of Revumenib, Azacitidine, and Venetoclax in Pediatric and Young Adult Patients With Refractory or Relapsed Acute Myeloid Leukemia","A Phase 1 Study of Revumenib, Azacitidine, and Venetoclax in Pediatric and Young Adult Patients With Refractory or Relapsed Acute Myeloid Leukemia","Inclusion Criteria: Participants must have a diagnosis of AML or ALAL and meet the criteria below:\n\n* Refractory leukemia, defined as persistent leukemia after at least two courses of induction chemotherapy (one course for secondary AML), or relapsed leukemia, defined as the re-appearance of leukemia after the achievement of remission. Patients must have ≥5% blasts in the bone marrow as assessed by morphology or ≥1% blasts flow cytometry.\n\nHowever, if an adequate bone marrow sample cannot be obtained (e.g., in a patient with acute megakaryoblastic leukemia with marrow fibrosis), patients may be enrolled if there is unequivocal evidence of leukemia with ≥5% blasts by morphology or ≥1% blasts flow cytometry in the blood.\n\n* Presence of KMT2A rearrangement (KMT2Ar), NUP98 rearrangement (NUP98r), NPM1 mutation or fusion, PICALM::MLLT10, DEK::NUP214, UBTF-TD, KAT6A rearrangement (KAT6Ar), or SET::NUP214\n* Adequate organ function, defined as total bilirubin \\\u003C 1.5 × institutional upper limit of normal for age or normal conjugated bilirubin (for patients with known Gilbert's syndrome, total bilirubin \\\u003C3 × the ULN) unless attributed to leukemia, calculated creatinine clearance ≥60 mL\u002Fmin\u002F1.73 m\\^2, and left ventricular ejection fraction ≥ 40%\n* QTcF \\\u003C 480 msec (average of triplicate)\n* Age ≥ 1 year and ≤ 30 years. The upper age limit may be defined by each institution, but may not exceed 30 years.\n* Lansky ≥ 60 for patients who are \\\u003C 16 years old and Karnofsky ≥ 60% for patients who are \\> 16 years old.\n* At least 14 days or 5 half-lives (whichever is longer) must have elapsed since the completion of myelosuppressive therapy, with the exception of low-dose therapy used for cytoreduction according to institutional standards, such as hydroxyurea or low-dose cytarabine (up to 200 mg\u002Fm\\^2\u002Fday). In addition, all toxicities must have resolved to grade 1 or less.\n* Patients must have a leukocyte count \\\u003C25,000 cells\u002FuL. Low-dose therapy, such as hydroxyurea or cytarabine as described above, to achieve this limit is acceptable.\n* For patients who have received prior HCT, there can be no evidence of GVHD and greater than 60 days must have elapsed since the HCT, and patients should be off calcineurin inhibitors for at least 28 days prior to the start of protocol therapy. Physiologic prednisone for the treatment of adrenal insufficiency is acceptable..\n* Patients must be taking posaconazole or voriconazole, which must be started at least 24 hours prior to the start of therapy.\n* Patients of reproductive potential must agree to use effective contraception for the duration of study participation.\n\nPatients who meet the criteria listed above are eligible for enrollment and treatment on the trial. However, patients in first relapse who are suitable for and willing to receive intensive remission induction therapy should be offered such therapy if deemed appropriate by the treating physician.\n\nExclusion Criteria:\n\n* Patients who are pregnant or breastfeeding are not eligible.\n* Patients with Down syndrome, acute promyelocytic leukemia, juvenile myelomonocytic leukemia, or bone marrow failure syndromes are not eligible.\n* Patients with uncontrolled infection are not eligible. Patients with infections that are controlled on concurrent anti-microbial agents are eligible.",{"count":297,"type":19},24,[233],"This is a research study to find out if adding a new study drug called revumenib to commonly used chemotherapy drugs is safe and if they have beneficial effects in treating patients with acute myeloid leukemia (AML) or acute leukemia of ambiguous lineage (ALAL) that did not go into remission after treatment (refractory) or has come back after treatment (relapsed), and to determine the total dose of the 3-drug combination of revumenib, azacitidine and venetoclax that can be given safely in participants also taking an anti-fungal drug.\n\nPrimary Objective\n\n* To determine the safety and tolerability of revumenib + azacitidine + venetoclax in pediatric patients with relapsed or refractory AML or ALAL.\n\nSecondary Objectives\n\n* Describe the rates of complete remission (CR), complete remission with incomplete count recovery (CRi), and overall survival for patients treated with revumenib + azacitidine + venetoclax at the recommended phase 2 dose (RP2D).",[301,302,303],"Refractory Acute Myeloid Leukemia","Relapsed Acute Myeloid Leukemia","Acute Leukemia of Ambiguous Lineage",{"date":282,"type":36},{"date":306,"type":36},"2024-04-19",{"date":308,"type":19},"2027-04",{"name":41,"class":42},10,{"id":312,"slug":313,"hasResults":12,"nctId":314,"briefTitle":315,"officialTitle":316,"acronym":4,"eligibilityCriteria":317,"healthyVolunteers":51,"sex":16,"minAge":318,"maxAge":319,"enrollmentInfo":320,"targetDuration":4,"studyType":107,"phases":4,"briefSummary":322,"conditions":323,"keywords":325,"overallStatus":32,"whyStopped":4,"lastUpdateSubmitDate":331,"lastUpdatePostDateStruct":332,"startDateStruct":333,"completionDateStruct":335,"leadSponsor":337,"locationsCount":43},"100645728","functional-evaluation-in-patients-with-urea-cycle-disorders-ucd-during-a-driving-task-100645728","NCT07701694","Functional Evaluation in Patients With Urea Cycle Disorders (UCD) During a Driving Task","Cognitive and Motor Function Examination in Patients With Urea Cycle Disorders (UCD) During a Driving Task Using Functional Near Infrared Spectroscopy (fNIRS)","Inclusion Criteria:\n\n* Individuals 16 to 40 years with molecular or biochemical confirmation of a urea cycle disorder.\n* Healthy individuals will serve as an age and sex matched comparison group. Active driver's license for at least 6 months\n* Read and understand English. as the tasks have not been validated in other languages\n* IQ \\>=70.\n\nExclusion Criteria:\n\n* Skin disease that affects the scalp\n* Past or present vascular disease\n* Head trauma with loss of consciousness in the last year or evidence of cognitive impairment due to and persisting after head trauma\n* Motor movement disorder which may cause excessive movement or impede use of the steering wheel and\u002For pedal\n* Premature birth or birth before 32 weeks of gestation\n* The participant is taking a stimulant or antidepressant\n* Allergy to any component of the device, although expected to be rare\n* History of psychosis\n* Self-reported motion or cyber sickness\n* Self-reported illicit substance use\n* Vision disorder","16 Years","40 Years",{"count":321,"type":19},60,"This study is being done to understand how the brain works while people with urea cycle disorder (UCD) perform driving tasks that range from easy to difficult and to look at how that compares to traditional tests of thinking and attention.",[324],"Urea Cycle Disorder",[326,327,328,329,330],"Participants with Urea Cycle Disorder","Healthy Volunteers","Cognitive function examination","Motor function examination","Simulated driving task with functional near-infrared spectroscopy monitoring","2026-07-29",{"date":280,"type":36},{"date":334,"type":36},"2026-07-20",{"date":336,"type":19},"2032-08",{"name":41,"class":42},{"id":339,"slug":340,"hasResults":12,"nctId":341,"briefTitle":342,"officialTitle":343,"acronym":4,"eligibilityCriteria":344,"healthyVolunteers":51,"sex":16,"minAge":132,"maxAge":4,"enrollmentInfo":345,"targetDuration":4,"studyType":20,"phases":347,"briefSummary":348,"conditions":349,"keywords":351,"overallStatus":32,"whyStopped":4,"lastUpdateSubmitDate":357,"lastUpdatePostDateStruct":358,"startDateStruct":359,"completionDateStruct":361,"leadSponsor":363,"locationsCount":43},"100644955","bereaved-parent-conversations-on-hope-100644955","NCT07675083","Bereaved Parent Conversations on Hope","Bereaved Parent Conversations on Hope (BeHope)","Inclusion Criteria\n\nIntervention Participants must be:\n\n* Students, observers, trainees, and newly hired clinicians,\n* ≥18 years old,\n* participating in or observing clinical care at St. Jude Children's Research Hospital, and\n* willing to participate in the virtual intervention and associated study activities as learners\n\nIntervention Facilitators must be:\n\n* Bereaved parents,\n* ≥ 18 years old,\n* screened and cleared to volunteer as advisers with St. Jude Children's Research Hospital through Family, Guest and Volunteer Services, and\n* willing to participate in the virtual intervention and associated study activities as a facilitator\n\nExclusion Criteria\n\nIntervention Participants:\n\n* Age \\\u003C 18 years, decline participation, or unable to speak\u002Fwrite in conversational English.\n\nIntervention Facilitators:\n\n* Age \\\u003C 18 years, decline participation, or unable to speak\u002Fwrite in conversational English.",{"count":346,"type":19},75,[22],"This study looks at whether it is possible and helpful to have video call conversations about how hopes can change through the cancer journey between bereaved parents and learners at a children's cancer center.",[350],"Hope",[352,353,354,355,356],"Cancer Journey","St. Jude Children's Research Hospital (SJCRH)","SJCRH Bereaved Parent Advisors","SJCRH Learners","Virtual Intervention","2026-07-27",{"date":331,"type":36},{"date":360,"type":36},"2026-07-24",{"date":362,"type":19},"2028-10",{"name":41,"class":42},{"id":365,"slug":366,"hasResults":12,"nctId":367,"briefTitle":368,"officialTitle":369,"acronym":4,"eligibilityCriteria":370,"healthyVolunteers":12,"sex":16,"minAge":132,"maxAge":4,"enrollmentInfo":371,"targetDuration":4,"studyType":107,"phases":4,"briefSummary":373,"conditions":374,"keywords":375,"overallStatus":32,"whyStopped":4,"lastUpdateSubmitDate":357,"lastUpdatePostDateStruct":379,"startDateStruct":381,"completionDateStruct":383,"leadSponsor":385,"locationsCount":43},"100644398","insights-from-bereaved-parents-and-oncologists-100644398","NCT07663539","Insights From Bereaved Parents and Oncologists","Foundations of Communication in Uncertainty Study (FOCUS): Insights From Bereaved Parents and Oncologists","Inclusion Criteria:\n\n* All participants must be ≥ 18 years of age or legally emancipated\n* Parent Participants must have a child who:\n\n  * Received cancer care from a clinician at St. Jude, as documented in the electronic medical record, AND\n  * Died at least 6 months prior to enrollment, but no more than 24 months prior to enrollment.\n* Oncologist participants at St. Jude must:\n\n  * Be listed as the primary oncologist as documented in the electronic medical record, AND\n  * Have the respective patient's parent agreement to participate in the study.\n\nExclusion Criteria:\n\n* Declining, refusal, or unwillingness to participate\n* Inability or unwillingness of research participant to give informed consent.",{"count":372,"type":19},50,"Investigators want to find better ways for doctors and families to talk about cancer and how uncertainty may affect a child's life.",[59],[376,377,378],"Prognostic Uncertainty","Bereaved Parents","Oncologist",{"date":380,"type":36},"2026-07-28",{"date":382,"type":36},"2026-07-09",{"date":384,"type":19},"2026-12",{"name":41,"class":42},{"id":387,"slug":388,"hasResults":12,"nctId":389,"briefTitle":390,"officialTitle":390,"acronym":4,"eligibilityCriteria":391,"healthyVolunteers":51,"sex":16,"minAge":132,"maxAge":4,"enrollmentInfo":392,"targetDuration":4,"studyType":107,"phases":4,"briefSummary":394,"conditions":395,"keywords":399,"overallStatus":117,"whyStopped":4,"lastUpdateSubmitDate":360,"lastUpdatePostDateStruct":404,"startDateStruct":405,"completionDateStruct":407,"leadSponsor":409,"locationsCount":43},"100633808","muscle-aging-phenotypes-in-childhood-cancer-survivors-100633808","NCT07531498","Muscle Aging Phenotypes in Childhood Cancer Survivors","Inclusion Criteria:\n\n* Age 18 years old or older at time of consent and enrolled in SJLIFE.\n* Participant (100 per group for a total of 400) is\u002Fhas:\n* Group 1: No cancer history\n* Group 2: Age and sex specific relative lean mass z-score of less than -0.5 OR age and sex specific hand grip or isokinetic (60 degrees\u002Fsec) quadriceps strength z-score of \\\u003C-0.5 AND exposure to a peripheral neurotoxin.\n* Group 3: Age and sex specific relative lean mass z-score of less than -0.5 OR age and sex specific hand grip or isokinetic (60 degrees\u002Fsec) quadriceps strength z-score of \\\u003C-0.5 AND NOT exposed to a peripheral neurotoxin.\n* Group 4: Age and sex specific relative lean mass z-score of less than -0.5 AND age and sex specific hand grip strength or isokinetic (60 degrees\u002Fsec) quadriceps strength z-score of \\\u003C-0.5 REGARDLESS of exposure status.\n* Participant or legal guardian is able and willing to give informed consent.\n\nExclusion Criteria:\n\n* Presence of implanted medical devices or metal that would interfere with MRI or MRS.\n* Female Participant is pregnant.\n* Body weight exceeding 300 pounds, due to MRI restrictions.\n* Inability to lie flat on his\u002Fher back for 90 minutes or longer for MRI.\n* Inability or unwillingness of research participant or legal guardian\u002Frepresentative to give written informed consent.\n* Presence of known radiation-induced nerve injury.\n* Prescence of pre-existing neurologic (non-cancer related) or who develop chronic neurologic disorders (i.e. Charcot Marie Tooth Disease, Downs, congenital brain injury).\n* Participation on a lifestyle or medication clinical trial within the past 1 year.",{"count":393,"type":19},533,"Childhood cancer survivors experience premature declines in muscle mass, strength, and physical function that contribute to morbidity and early mortality. The biological mechanisms driving these impairments are heterogeneous and poorly understood. This observational study aims to characterize distinct muscle health endotypes in adult survivors of childhood cancer using advanced imaging, neuromuscular testing, and functional assessment. Survivors with reduced muscle health and community controls will undergo multimodal magnetic resonance imaging and spectroscopy, nerve conduction studies, surface electromyography, body composition assessment, and physical performance testing during a single study visit integrated into an ongoing cohort evaluation. Identifying mechanistic endotypes of impaired muscle health will support development of targeted interventions to preserve function and improve long-term outcomes in childhood cancer survivors.\n\nPrimary Objective:\n\n\\- Characterize reduced muscle health endotypes in childhood cancer survivors.\n\nSecondary Objective:\n\n\\- Identify specific treatment and lifestyle related risk factors for each reduced muscle health endotype.\n\nExploratory Objective:\n\n\\- Host germline genetics will be associated with specific muscle endotypes.",[396,397,398],"Muscle Weakness","Low Muscle Mass","Sarcopenia",[400,401,402,403],"Childhood Cancer Survivors","Adult Survivors of Childhood Cancer","Neuromuscular Function","Muscle Health",{"date":380,"type":36},{"date":406,"type":19},"2026-10-01",{"date":408,"type":19},"2031-05",{"name":41,"class":42},{"id":411,"slug":412,"hasResults":12,"nctId":413,"briefTitle":414,"officialTitle":414,"acronym":4,"eligibilityCriteria":415,"healthyVolunteers":12,"sex":16,"minAge":4,"maxAge":416,"enrollmentInfo":417,"targetDuration":4,"studyType":20,"phases":419,"briefSummary":420,"conditions":421,"keywords":422,"overallStatus":32,"whyStopped":4,"lastUpdateSubmitDate":425,"lastUpdatePostDateStruct":426,"startDateStruct":428,"completionDateStruct":429,"leadSponsor":431,"locationsCount":43},"100639208","phase-2-haploidentical-donor-hematopoietic-cell-transplant-for-sickle-cell-disease-100639208","NCT07616154","Haploidentical Donor Hematopoietic Cell Transplant for Sickle Cell Disease","Inclusion Criteria:\n\nTransplant Recipient\n\n* Age less than or equal to 22 years.\n* Patients without a suitable HLA-matched sibling donor but with a suitable single haplotype matched (≥ 3 of 6) family member donor. Potential donors do not need to undergo eligibility determination prior to the recipients enrolling on the study. As long as a potential donor is identified and willing to donate hematopoietic progenitor cells, recipients can enroll on the study.\n* Patients with SCD (any genotype) who meet any ONE of the following criteria:\n* History of an abnormal transcranial Doppler measurement defined as TCD velocity ≥200 cm\u002Fsec by the non-imaging technique (or ≥185 cm\u002Fsec by the imaging technique) measured at a minimum of two separate occasions.\n* History of cerebral infarction on brain MRI (overt stroke, or silent cerebral infarct).\n* History of two or more episodes of acute chest syndrome (ACS) in the 2-years period preceding enrollment.\n* History of two or more SCD related pain events requiring treatment with parenteral analgesics in the last 12 months.\n* History of two or more episodes of priapism (erection lasting ≥4 hours or requiring emergent medical care).\n* Administration of regular RBC transfusions (≥8 transfusions in the previous 12 months).\n* Evidence of progressive end organ damage (eg. cardiomyopathy, nephropathy, pulmonary hypertension etc) that in the opinion of the treating hematologist is not responsive to medical management and may benefit from an HCT. Such a determination must be made in writing by at least two independent hematologists and documented in the patient's electronic medical record prior to enrollment.\n\nDonor\n\n* An at least single haplotype matched (≥ 3 of 6) family member.\n* HIV negative\n* Not pregnant, as confirmed by negative serum or urine pregnancy test within 14 days prior to enrollment (if female).\n* Not breast feeding.\n* Donor should not have clinically significant hemoglobinopathy. Donors with sickle cell trait are acceptable.\n* Regarding donation eligibility, is identified as either:\n* Completed the process of donor eligibility determination as outlined in 21 CFR 1271 and agency guidance; OR.\n* Does not meet 21 CFR 1271 eligibility requirements but has a declaration of urgent medical need completed by the principal investigator or physician sub-investigator per 21 CFR 1271.\n\nExclusion Criteria:\n\nTransplant Recipient\n\n* Karnofsky or Lansky performance score \\\u003C60.\n* Pregnant, as confirmed by positive serum or urine pregnancy test within 14 days prior to enrollment (if female).\n* Breast feeding.\n* Uncontrolled bacterial, viral or fungal infections (undergoing appropriate treatment and with progression of clinical symptoms) within 1 month prior to conditioning. Patients with febrile illness or suspected minor infection should await clinical resolution prior to starting conditioning. Patients with confirmed seropositivity or positive NAAT for HIV are excluded.\n* Serum conjugated (direct) bilirubin \\>3x upper limit of normal for age as per local laboratory. Participants with hyperbilirubinemia as the result of hyperhemolysis, or a severe drop in hemoglobin post blood transfusion, are not excluded as long as it downtrends and return to acceptable limits subsequently.\n* Left ventricular shortening fraction \\\u003C25% or ejection fraction \\\u003C40% by echocardiogram.\n* Estimated creatinine clearance less than 50 mL\u002Fmin\u002F1.73m2.\n* Diffusion capacity of carbon monoxide (DLCO) \\\u003C35% (adjusted for hemoglobin) OR baseline oxygen saturation \\\u003C85% or PaO2 \\\u003C70.\n* Presence of anti-donor specific HLA antibodies unresponsive to desensitization.","22 Years",{"count":418,"type":19},45,[160],"The purpose of this study it to evaluate a reduced toxicity conditioning regimen for haploidentical donor HCT followed by a GVHD prophylaxis regimen comprising of post-transplant cyclophosphamide, sirolimus and abatacept with the goal to improve the GVHD-free rejection-free survival (GRFS) to greater than 90% after haploidentical donor HCT in children and young adults with SCD.\n\nPrimary Objective:\n\n\\- To assess the GVHD-free and rejection free survival (GRFS) after haploidentical donor HCT in children and young adults with SCD.\n\nSecondary Objectives:\n\n* Assess the overall survival (OS) and disease-free survival (DFS) after haploidentical donor HCT for SCD.\n* Estimate incidence and severity of acute and chronic GVHD after haploidentical donor HCT for SCD.\n* Assess the neutrophil and platelet engraftment kinetics after haploidentical donor HCT for SCD.",[216],[423,424],"Sickle Cell","Hematopoietic Cell Transplant","2026-07-21",{"date":427,"type":36},"2026-07-23",{"date":121,"type":19},{"date":430,"type":19},"2035-09",{"name":41,"class":42},{"id":433,"slug":434,"hasResults":12,"nctId":435,"briefTitle":436,"officialTitle":437,"acronym":4,"eligibilityCriteria":438,"healthyVolunteers":12,"sex":16,"minAge":4,"maxAge":4,"enrollmentInfo":439,"targetDuration":4,"studyType":107,"phases":4,"briefSummary":441,"conditions":442,"keywords":444,"overallStatus":32,"whyStopped":4,"lastUpdateSubmitDate":425,"lastUpdatePostDateStruct":456,"startDateStruct":458,"completionDateStruct":460,"leadSponsor":462,"locationsCount":43},"100249818","next-generation-sequencing-of-normal-tissues-prospectively-in-pediatric-oncology-patients-100249818","NCT02530658","Next Generation Sequencing of Normal Tissues Prospectively in Pediatric Oncology Patients","Genomes for Kids (G4K)","Inclusion Criteria:\n\n* St. Jude patients prospectively identified at the time of study activation with a diagnosed solid or liquid tumor (benign or malignant).\n* Adequate tissue must be available (e.g. sufficient germline and\u002For tumor tissue, from which \\>1 µg DNA and \\>0.1 µg RNA must be isolated). Patients who have no tumor tissue available may enroll using only germline sample.\n\nExclusion Criteria:\n\n* Past history of hematopoietic stem cell transplantation (or other condition that would result in hematopoietic cell DNA failing to match host tissue DNA).\n* Tumor or germline tissue not meeting the criteria listed above.\n* Inability or unwillingness of research participant or legal guardian\u002Frepresentative to give written informed consent.\n* Participants who are unable to read, write or converse fluently in English or Spanish will be excluded from Prespecified Objectives 3 and 4.",{"count":440,"type":19},2500,"The development of next generation sequencing (NGS) techniques, including whole genome (WGS), exome (WES) and RNA sequencing has revolutionized the ability of investigators to query the molecular mechanisms underlying tumor formation. Through the Pediatric Cancer Genome Project (PCGP), investigators at St. Jude Children's Research Hospital (SJCRH) have successfully used NGS approaches to evaluate more than 1,000 pediatric cancers ranging from hematologic malignancies to central nervous system (CNS) and non-CNS solid tumors. From these and related studies, it has become clear that genomic approaches can accurately classify tumors into distinct pathologic and prognostic subtypes and detect alterations in cellular pathways that may serve as novel therapeutic targets. Collectively, these studies suggest that by characterizing the genomic make-up of individual tumors, investigators will be able to develop personalized and potentially more effective cancer treatments and\u002For preventive measures.\n\nThis protocol was initially enacted to usher NGS approaches into routine clinical care. During the initial phase of the G4K protocol, 310 participants were recruited and enrolled onto the study. Tumor and\u002For germline sequencing was completed on all 310 patients, with 253 somatic reports generated (representing 96% of the 263 participants for whom tumor tissue was available and analyzed) and 301 germline reports generated (100% of the 301 participants who agreed to the receipt of germline results). Analyses of the study data are ongoing with plans to prepare initial manuscripts within the next several months. Due to the successful initial execution of the G4K protocol, clinical genomic sequencing of tumor and germline samples is now offered as part of standard clinical care for pediatric oncology patients at St. Jude.\n\nThe G4K protocol has now been revised. With the revision, the study team will record, store and analyze germline and tumor genomic information. Through the collection of these data, we will examine how germline mutations in 150 cancer predisposition genes influence clinical presentation, tumor histology, tumor genomic findings, response to therapy and long-term outcomes. The overall goals of this research are to further define the prevalence, spectrum and heritability of germline variants in these genes and to decipher how germline mutations influence the phenotypes of an expanding array of cancer predisposition syndromes. These studies allow us to provide more accurate genetic counseling and management strategies to future children harboring mutations in these genes.\n\nThis remains a non-therapeutic study. Investigators anticipate a sample size of approximately 2500 patients who will be recruited over the next 7 years.",[443],"Solid, Liquid, Central Nervous System Tumors",[445,446,447,448,449,450,451,452,453,454,455],"Genetic Predisposition","Pediatric","Genomic Analysis","Heritable Disease","Cancer Risk","Genetic Counseling","DNA","Clinical Genomics","Clinical Decision-Making","Treatment Planning","Germline",{"date":457,"type":36},"2026-07-22",{"date":459,"type":36},"2015-08-28",{"date":461,"type":19},"2053-12-31",{"name":41,"class":42},{"id":464,"slug":465,"hasResults":12,"nctId":466,"briefTitle":467,"officialTitle":467,"acronym":4,"eligibilityCriteria":468,"healthyVolunteers":51,"sex":16,"minAge":4,"maxAge":4,"enrollmentInfo":469,"targetDuration":4,"studyType":20,"phases":471,"briefSummary":472,"conditions":473,"keywords":474,"overallStatus":117,"whyStopped":4,"lastUpdateSubmitDate":481,"lastUpdatePostDateStruct":482,"startDateStruct":483,"completionDateStruct":485,"leadSponsor":486,"locationsCount":4},"100648386","universal-newborn-screening-for-sickle-cell-disease-in-mozambique-100648386","NCT07719972","Universal Newborn Screening For Sickle Cell Disease In Mozambique","Inclusion Criteria:\n\n* Children participants: All infants between birth and 6.0 months of age who are born or receive care at secondary-level facilities involved in the UNIQUE study.\n* Children participants will fall into one of two categories:\n\n  * Patient participants: All infants between birth and 6.0 months of age who were screened through the UNIQUE study and tested positive for SCD (HbSS, HbSC, or other form of SCD) or had indeterminate results.\n  * Healthy control participants: Infants between birth and 6.0 months of age who screen negative for SCD (HbAA) through the UNIQUE study or tested positive for sickle cell trait (HbAS).\n* Health facility staff participants: Healthcare staff ages ≥18 years working at secondary-level facilities involved in the UNIQUE study.\n* Supply chain expert participants: Administrative professionals with experience working in or around the national supply chain systems in Mozambique to support procurement, importation, customs clearance, storage, and in-country distribution of medical products.\n* National public health system expert participants: Administrative professionals with experience working in the national public health system (e.g., Ministry of Health, MISAU) who oversee the delivery of health services to infants in-country, such as neonatal testing and vaccination programs.\n\nExclusion Criteria:\n\n* Children participants:\n\n  * Stillbirths.\n  * Children who received an erythrocyte (blood) transfusion within 3 months of testing. Exogenous (transfused) HbA could artificially lower the sickle hemoglobin concentration, thus causing false-negative results.\n* Patient participants: none\n* Healthy control participants:\n\n  * Stillbirths.\n  * Children who received an erythrocyte (blood) transfusion within 3 months of testing. Exogenous (transfused) HbA could artificially lower the sickle hemoglobin concentration, thus causing false-negative results.\n* Health facility staff participants: none.\n* Supply chain expert participants: none.\n* National public health system expert participants: none.",{"count":470,"type":19},6750,[22],"The overarching goal of this study is to evaluate the feasibility of a new methodology that combines three multi-level implementation strategies to optimize the population-level uptake of essential evidence-based, standard of care treatments for infants with sickle cell disease (SCD) in low-resource settings. The study will be done in Mozambique.",[216],[475,476,477,478,479,480],"Mozambique","Patient participants","Healthy control participants","Health facility staff participants","Supply chain expert participants","National public health system expert participants","2026-07-17",{"date":457,"type":36},{"date":484,"type":19},"2026-08",{"date":39,"type":19},{"name":41,"class":42},{"id":488,"slug":489,"hasResults":12,"nctId":490,"briefTitle":491,"officialTitle":491,"acronym":4,"eligibilityCriteria":492,"healthyVolunteers":12,"sex":16,"minAge":4,"maxAge":416,"enrollmentInfo":493,"targetDuration":4,"studyType":20,"phases":494,"briefSummary":495,"conditions":496,"keywords":4,"overallStatus":32,"whyStopped":4,"lastUpdateSubmitDate":481,"lastUpdatePostDateStruct":498,"startDateStruct":499,"completionDateStruct":501,"leadSponsor":503,"locationsCount":43},"100490283","phase-1-feasibility-safety-and-potential-efficacy-of-fecal-microbiota-transplantation-fmt-for-gastrointestinal-dysfunction-in-children-following-hematopoietic-cell-transplant-hct-100490283","NCT05664113","Feasibility, Safety, and Potential Efficacy of Fecal Microbiota Transplantation (FMT) for Gastrointestinal Dysfunction in Children Following Hematopoietic Cell Transplant (HCT).","Inclusion Criteria:\n\n* Age \\\u003C 22 years old.\n* Received an allogeneic HCT greater than or equal to 30 days prior to enrollment\n* Diagnosed with one of the following conditions:\n\n  1. Steroid-resistant gut a GvHD (defined as GI symptoms that do not improve within 5 days after initial steroid therapy, \\>\u002F= 1mg\u002Fkg of prednisolone) OR\n  2. Steroid-dependent gut a GvHD (defined as the presence of a response to methylprednisolone 2 mg\u002Fkg\u002Fday but relapsing when an attempt was made to taper steroid treatment).\n\n     OR\n  3. Current or prolonged GI dysfunction following HCT, defined as having diarrhea or loose stools \\>\u002F= 4 weeks with at least one of the following:\n\n     1. Requiring NG or G-tube feeds\n     2. Requiring TPN or IVF for more than 4 weeks\n     3. Diagnosis of gastroparesis by GI specialist documented in the medical record\n* Willing and able to provide informed assent\u002Fconsent\n\nExclusion Criteria:\n\n* Participant is at risk for aspiration pneumonia\n* History of anaphylactic allergy to foods that are not excluded from the stool donor diet\n* Female participant who is pregnant or nursing\n* History of previous FMT\n* Intra-abdominal surgery within 4 weeks of enrollment\n* At increased risk for peritonitis: presence of intra-abdominal devices (G-or GJ-tubes are acceptable), receiving peritoneal dialysis, or ascites\n* Concurrent abdominal radiation therapy\n* Any acute or chronic illness\u002Fcondition as well as medication that in the opinion of the investigator puts the subject at greater risk from FMT or may confound the study results.",{"count":310,"type":19},[233],"The study participant is being asked to take part in this clinical trial, a type of research study, because the participant has Gastrointestinal (GI) symptoms following a Hematopoietic Cell Transplant (HCT).\n\nPrimary Objective\n\n* To determine the safety and feasibility of FMT for treating a GvHD of the gut following HCT.\n* To determine the safety and feasibility of FMT for treating HCT induced gut dysfunction.\n\nSecondary Objectives\n\n* To assess the potential efficacy of FMT for treating a GvHD of the gut following HCT.\n* To assess the potential efficacy of FMT for treating HCT induced gut dysfunction.",[497],"Gastro-Intestinal Disorder",{"date":334,"type":36},{"date":500,"type":36},"2025-07-07",{"date":502,"type":19},"2028-12-31",{"name":41,"class":42},{"id":505,"slug":506,"hasResults":12,"nctId":507,"briefTitle":508,"officialTitle":508,"acronym":4,"eligibilityCriteria":509,"healthyVolunteers":12,"sex":16,"minAge":4,"maxAge":510,"enrollmentInfo":511,"targetDuration":4,"studyType":20,"phases":513,"briefSummary":514,"conditions":515,"keywords":519,"overallStatus":32,"whyStopped":4,"lastUpdateSubmitDate":521,"lastUpdatePostDateStruct":522,"startDateStruct":523,"completionDateStruct":524,"leadSponsor":526,"locationsCount":43},"100612733","phase-1-cd45ra-depleted-cd19-car-t-cell-consolidation-after-tcrcd19-b-cell-depleted-haploidentical-hematopoietic-cell-transplantation-for-relapsedrefractory-cd19-all-and-lymphoma-100612733","NCT07257419","CD45RA-depleted CD19-CAR T Cell Consolidation After TCRαβ+\u002FCD19 B Cell-depleted Haploidentical Hematopoietic Cell Transplantation for Relapsed\u002FRefractory CD19+ ALL and Lymphoma","Inclusion Criteria:\n\nRecipient\n\n* Age less than or equal to 21 years\n* High risk hematologic malignancy where allogeneic transplantation is the current standard of care. This includes (but is not limited to):\n\n  * High risk CD19+ B cell ALL in CR1 or CR2\n  * Any CD19+ B-cell ALL in CR3 or subsequent\n* If prior CNS leukemia, it must be treated and in CNS CR\n* Left ventricular ejection fraction \\> 40%, or shortening fraction ≥ 25%\n* Creatinine clearance (CrCl) or glomerular filtration rate (GFR) ≥ 50 ml\u002Fmin\u002F1.73m2\n* Forced vital capacity (FVC) ≥ 50% of predicted value; or pulse oximetry ≥ 92% on room air if patient is unable to perform pulmonary function testing\n* Karnofsky or Lansky (age dependent) performance score ≥ 50 (See APPENDIX A)\n* Bilirubin ≤ 3 times the upper limit of normal for age\n* Alanine aminotransferase (ALT) or Aspartate aminotransferase (AST) ≤ 5 times the upper limit of normal for age\n\nDonor\n\n* At least single haplotype matched (≥ 4 of 8) family member\n* At least 18 years of age\n* HIV negative\n* If sexually active, agreement to use birth control until 2 weeks after completion of the mobilization and apheresis procedure\n* Regarding donation eligibility, is identified as either:\n\n  * Completed the process of donor eligibility determination as outlined in 21 CFR 1271 and agency guidance; OR\n  * Does not meet 21 CFR 1271 eligibility requirements, but has a declaration of urgent medical need completed by the principal investigator or physician sub-investigator per 21 CFR 1271\n\nExclusion Criteria:\n\nRecipient\n\n* Has a suitable HLA-identical sibling or suitable 12\u002F12 (HLA-A, B, C, DRB1, DQB1, and DPB1) HLA-matched unrelated donor available in an appropriate time frame\n* Any other active malignancy other than the one for which this HCT is indicated\n* Received a prior allogeneic HCT at any time\n* Pregnant, if female is of childbearing potential, negative test must be confirmed by serum or urine pregnancy test within 14 days prior to enrollment\n* If sexually active, agreement to use birth control until 6 months after T cell infusion\n* Breast feeding\n* Any severe current uncontrolled bacterial, fungal or viral infection\n\nDonor\n\n* Pregnant, negative test must be confirmed by serum or urine pregnancy test within 14 days prior to enrollment if female\n* If female, breast feeding","21 Years",{"count":512,"type":19},70,[233],"The purpose of this study is to learn more about newer methods of transplanting blood cells donated by a partially matched family member to children with high-risk CD19 positive leukemia ALL.\n\nPrimary Objective:\n\n\\- To assess the safety and feasibility of combining CD19-CAR(Mem) T cells after TCRαβ+\u002FCD19 depleted haploidentical donor transplantation for pediatric patients with relapsed\u002Frefractory CD19+ B-cell malignancies.\n\nSecondary Objectives:\n\n* To estimate 1-year post-transplant overall survival, event-free survival, and GVHD-free relapse-free survival (GRFS).\n* To estimate cumulative incidence of engraftment, acute and chronic GVHD, and immune-related adverse events, including CRS and ICANS.",[516,517,518],"Relapsed Pediatric ALL","Hematopoietic Cell Transplantation","Hematologic Malignancy",[520],"Relapsed\u002FRefractory ALL","2026-07-16",{"date":481,"type":36},{"date":282,"type":19},{"date":525,"type":19},"2035-12",{"name":41,"class":42},{"id":528,"slug":529,"hasResults":12,"nctId":530,"briefTitle":531,"officialTitle":532,"acronym":4,"eligibilityCriteria":533,"healthyVolunteers":12,"sex":16,"minAge":157,"maxAge":132,"enrollmentInfo":534,"targetDuration":4,"studyType":20,"phases":536,"briefSummary":537,"conditions":538,"keywords":541,"overallStatus":32,"whyStopped":4,"lastUpdateSubmitDate":544,"lastUpdatePostDateStruct":545,"startDateStruct":546,"completionDateStruct":548,"leadSponsor":550,"locationsCount":551},"100557102","phase-2-therapy-for-newly-diagnosed-patients-with-b-cell-precursor-acute-lymphoblastic-leukemia-and-lymphoma-100557102","NCT06533748","Therapy for Newly Diagnosed Patients With B-Cell Precursor Acute Lymphoblastic Leukemia and Lymphoma","SJALL23H: Combination Antigen-Directed Induction Therapy for Newly Diagnosed Patients With B-Cell Precursor Acute Lymphoblastic Leukemia and Lymphoma","Inclusion Criteria:\n\n* Enrollment on INITIALL.\n* Age 1-18.99 years at the time of enrollment on INITIALL.\n* B-Acute lymphoblastic leukemia or lymphoblastic lymphoma.\n* No prior chemotherapy excluding therapy given on or allowed by INITIALL.\n* NCI high-risk (age 10 years or greater or presenting WBC count ≥50,000 cells\u002FmicroL) or NCI standard-risk and a HR clinical feature as listed below:\n\n  * CNS3 disease (≥5 WBC\u002FmicroL CSF with blasts present)\n  * Testicular involvement of leukemia\n  * Steroid pretreatment defined as \\>24 hours of therapy in the 14 days prior to enrollment on INITIALL if a preceding WBC to define NCI risk is unavailable\n* For lymphoblastic lymphoma, Stage 3-4 disease OR Stage 1-2 disease in patient ages ≥10 years OR HR clinical feature as defined above.\n* Adequate liver function defined as:\n\n  * Total bilirubin ≤ 1.5x the upper limit of normal for age and alanine transaminase (ALT) ≤ 5x the upper limit of normal for age. Patients with an elevated total bilirubin due to hemolysis are eligible if they have a direct bilirubin \\\u003C1.5x the upper limit of normal.\n* Adequate renal function defined as:\n\n  * Calculated glomerular filtration rate (GFR) ≥ 50 mL\u002Fmin\u002F1.73m\\^2 using the Bedside Schwartz equation OR creatinine below or equal to the maximum defined below:\n\n    * Age: 1 to \\\u003C2 years; maximum serum creatinine (mg\u002FdL): 0.6 (male and female)\n    * Age: 2 to \\\u003C6 years; maximum serum creatinine (mg\u002FdL): 0.8 (male and female)\n    * Age: 6 to \\\u003C10 years; maximum serum creatinine (mg\u002FdL): 1.0 (male and female)\n    * Age: 10 to \\\u003C13 years; maximum serum creatinine (mg\u002FdL): 1.2 (male and female)\n    * Age: 13 to \\\u003C16 years; maximum serum creatinine (mg\u002FdL): 1.5 (male); 1.4 (female)\n    * Age: ≥16 years; maximum serum creatinine (mg\u002FdL): 1.7 (male); 1.4 (female)\n* Eligibility for inclusion post-induction requires meeting the first 4 Inclusion criteria above AND:\n\n  * Treatment on SJALL23H for Induction OR\n  * Lymphoblastic lymphoma, initially treated on an SJALL protocol OR standard (non-protocol) therapy, without a complete response at the end of induction OR\n  * NCI-SR ALL at diagnosis and treated with an SJALL protocol OR standard (non-protocol) therapy who have\n\n    * Slow response to therapy (≥0.1% MRD at end of induction for patients with hyperdiploid ALL or ≥0.01% MRD at end of induction for others with ALL) OR\n    * HR genetics as defined in the protocol.\n    * These patients may receive no more than 2 weeks of post-induction therapy and should be transitioned to SJALL23H post-induction as soon as the qualifying genetic or MRD result is available.\n\nExclusion Criteria:\n\n* Presence of ETV6::RUNX1 fusion unless also having a HR clinical feature OR slow response to induction therapy.\n* History or presence of clinically relevant central nervous system (CNS) pathology or event such as epilepsy, childhood or adult non-febrile seizure, paresis, aphasia, stroke, severe brain injuries, dementia, Parkinson's disease, cerebellar disease, organic brain syndrome, or psychosis. History of simple febrile seizure during childhood and presence of CNS leukemia at diagnosis are not exclusions to participation.\n* Active uncontrolled infection.\n* Current active autoimmune disease or history of autoimmune disease with the potential for CNS involvement.\n* History of venoocclusive disease\u002F sinusoidal obstructive syndrome.\n* Unstable cardiac disease including QTc \\>500msec.\n* Inability or unwillingness to give informed consent\u002F assent as applicable.\n* Pregnant or lactating.\n* For patients of reproductive potential, unwillingness to use effective contraception for the duration of protocol therapy.",{"count":535,"type":19},128,[160],"This is a Phase II clinical trial testing the use of two antigen-directed therapies, inotuzumab and blinatumomab, as part of induction therapy for children and young adults with newly diagnosed B-cell precursor acute lymphoblastic leukemia and lymphoma.\n\nPrimary Objective\n\n* To assess if the flow-cytometry assessed MRD-negative remission rate following an immunotherapy-based Induction in NCI-high risk patients without favorable genetic features is higher than the results of similar patients treated on AALL1131.\n\nSecondary Objectives\n\n* To compare flow-cytometry assessed MRD-negative rates at the end of Induction for patients treated with this therapy compared to similar patients treated on TOT17.\n* To compare the rate of significant toxicities in patients treated with this therapy to those treated with standard-risk therapy on TOT17.\n* To assess the event free and overall survival of patients treated with this therapy.",[539,540],"Acute Lymphoblastic Leukemia","Lymphoblastic Lymphoma",[539,540,167,542,543,168,169],"Antigen-directed therapies","Induction therapy","2026-07-15",{"date":521,"type":36},{"date":547,"type":36},"2025-01-23",{"date":549,"type":19},"2034-05",{"name":41,"class":42},3,{"id":553,"slug":554,"hasResults":12,"nctId":555,"briefTitle":556,"officialTitle":557,"acronym":4,"eligibilityCriteria":558,"healthyVolunteers":12,"sex":16,"minAge":132,"maxAge":4,"enrollmentInfo":559,"targetDuration":4,"studyType":107,"phases":4,"briefSummary":561,"conditions":562,"keywords":564,"overallStatus":117,"whyStopped":4,"lastUpdateSubmitDate":566,"lastUpdatePostDateStruct":567,"startDateStruct":568,"completionDateStruct":569,"leadSponsor":571,"locationsCount":43},"100640608","clinical-assessment-and-targeted-imaging-to-characterize-high-risk-atherosclerotic-cardiovascular-disease-of-survivors-in-sjlife-100640608","NCT07594392","Clinical Assessment and Targeted Imaging to Characterize High-Risk Atherosclerotic Cardiovascular Disease of Survivors in SJLIFE","Clinical Assessment and Targeted Imaging to Characterize High-Risk Atherosclerotic Cardiovascular Disease of Survivors in the St. Jude Lifetime Cohort (SJLIFE)","Inclusion Criteria:\n\n\\- SJLIFE Survivor or Control\n\n* Age ≥18 years\n* Enrolled in the St. Jude Lifetime Cohort (SJLIFE)\n* Participant or legal guardian\u002Frepresentative is able and willing to give informed consent.\n\nSJLIFE Survivor only\n\n* Participant has been treated with at least one of the following cancer therapies:\n\nplatinum chemotherapy, neck radiation exposure of at least 1000 centigray (cGy), or chest radiation exposure of at least 1000 cGy (with potential exposure to the heart).\n\nExclusion Criteria:\n\nSJLIFE Survivor or Control\n\n* Previous stroke or intervention for severe carotid arterial disease including carotid endarterectomy or carotid angioplasty and stent placement.\n* Previous myocardial infarction or intervention for severe cardiovascular disease including Coronary Artery Bypass Graft (CABG), coronary stent placement or coronary angioplasty.\n* History of aortic valve replacement\n* Inability or unwillingness of research participant or legal guardian\u002Frepresentative to give written informed consent.\n\nControl Only\n\n* First degree relative of SJLIFE Survivor\n* History of cancer",{"count":560,"type":19},650,"Childhood cancer survivors are at increased risk for premature atherosclerotic cardiovascular disease (ASCVD) due to cancer treatment-related exposures, including radiation therapy and platinum-based chemotherapy. Current ASCVD risk assessment tools may underestimate cardiovascular risk in younger survivors. This observational study performs detailed cardiovascular phenotyping using imaging, blood-based biomarkers, and vascular function testing among adult survivors enrolled in the St. Jude Lifetime Cohort (SJLIFE), with comparison to community controls, to better characterize subclinical ASCVD risk and inform survivor-specific prevention strategies.\n\nPrimary Objective:\n\nPerform deeper phenotyping of SJLIFE participants at treatment-related risk of atherosclerotic cardiovascular disease \\[ASCVD\\] to facilitate early detection of pathophysiological targets appropriate for remediation.\n\nSecondary Objectives:\n\nDetermine the distribution of lipoprotein (a) levels and prevalence of elevated levels among survivors with any treatment related exposure-based risk for ASCVD overall and then compared to community controls.\n\nEvaluate prevalence of clinical and imaging markers of ASCVD risk among survivors exposed only to platinum chemotherapy and compare that to community controls.",[563],"Atherosclerotic Cardiovascular Disease",[400,565],"ASCVD","2026-07-14",{"date":521,"type":36},{"date":484,"type":19},{"date":570,"type":19},"2031-06",{"name":41,"class":42},{"id":573,"slug":574,"hasResults":12,"nctId":575,"briefTitle":576,"officialTitle":576,"acronym":4,"eligibilityCriteria":577,"healthyVolunteers":12,"sex":16,"minAge":132,"maxAge":4,"enrollmentInfo":578,"targetDuration":4,"studyType":20,"phases":580,"briefSummary":581,"conditions":582,"keywords":584,"overallStatus":32,"whyStopped":4,"lastUpdateSubmitDate":566,"lastUpdatePostDateStruct":588,"startDateStruct":589,"completionDateStruct":591,"leadSponsor":593,"locationsCount":43},"100573563","assessment-of-remote-approaches-for-identification-of-autonomic-dysfunction-among-survivors-of-leukemia-and-lymphoma-100573563","NCT06747910","Assessment of Remote Approaches for Identification of Autonomic Dysfunction Among Survivors of Leukemia and Lymphoma","Inclusion Criteria:\n\n* Participants enrolled in St. Jude Lifetime Cohort (SJLIFE) \\>18 years of age.\n* Primary diagnosis of acute lymphoblastic leukemia (ALL), Hodgkin's Lymphoma (HL), or Non-Hodgkin's Lymphoma (Non-HL).\n* Not currently taking beta-blocker medication.\n\nExclusion Criteria:\n\n* Individuals who cannot speak, read, and\u002For understand English.\n* Individuals who are unable to follow directions\u002Finstructions in order to complete the Ewing battery.\n* Individuals with acute heart failure (new or worsening signs and symptoms of heart failure, including a combination of the following: dyspnea, orthopnea, lower limb swelling, elevated jugular venous pressure, and pulmonary congestion).\n* Women who are currently pregnant.",{"count":579,"type":19},188,[22],"This study seeks to determine if diagnosing cardiac autonomic dysfunction (AD) can be done remotely with the same accuracy as in-person testing. If so, the identification of AD could happen sooner, facilitating remote studies of the condition and potentially reducing the risk of illness. Childhood cancer survivors, particularly survivors of acute lymphoblastic leukemia (ALL) and Hodgkins's lymphoma (HL), appear to be at increased risk for AD.\n\nPrimary Objectives:\n\n* To determine the sensitivity and specificity of heart rate variability (HRV), measured remotely with biosensor technology (Actigraph LEAP), compared to in-person assessment using the Ewing battery as the reference standard to identify cardiac autonomic dysfunction (AD) among survivors of leukemia and lymphoma.\n* To determine the sensitivity and specificity of the Composite Autonomic Symptom Scale 31 (COMPASS31) compared to the Ewing battery to identify AD among leukemia and lymphoma survivors.",[583],"Childhood Cancer",[585,114,586,587],"Survivor","Hodgkins's lymphoma","Autonomic",{"date":521,"type":36},{"date":590,"type":36},"2025-02-03",{"date":592,"type":19},"2027-06",{"name":41,"class":42},{"id":595,"slug":596,"hasResults":12,"nctId":597,"briefTitle":598,"officialTitle":599,"acronym":4,"eligibilityCriteria":600,"healthyVolunteers":12,"sex":16,"minAge":157,"maxAge":132,"enrollmentInfo":601,"targetDuration":4,"studyType":20,"phases":603,"briefSummary":605,"conditions":606,"keywords":607,"overallStatus":32,"whyStopped":4,"lastUpdateSubmitDate":609,"lastUpdatePostDateStruct":610,"startDateStruct":611,"completionDateStruct":613,"leadSponsor":615,"locationsCount":204},"100538349","phase-4-identification-of-necessary-information-for-treatment-induction-in-newly-diagnosed-acute-lymphoblastic-leukemialymphoma-100538349","NCT06289673","Identification of Necessary Information for Treatment Induction in Newly Diagnosed Acute Lymphoblastic Leukemia\u002FLymphoma","INITIALL: Identification of Necessary Information for Treatment Induction in Newly Diagnosed Acute Lymphoblastic Leukemia\u002FLymphoma","Inclusion Criteria:\n\n* Age 1-18.99 years\n* Diagnosis of acute leukemia \u002F lymphoma as below:\n\n  * Acute lymphoblastic leukemia (ALL) with at least 25% bone marrow blasts or definitive evidence of ALL in peripheral blood (in those without an available bone marrow sample).\n  * Lymphoblastic lymphoma (LLy) with immunophenotypic evidence of a lymphoblastic population and \\\u003C25% bone marrow blasts and less than 1,000 circulating blasts\u002F microL.\n  * Mixed phenotype acute leukemia (MPAL) with or without 25% bone marrow involvement (i.e. patients with either leukemia or lymphoma are eligible).\n\nExclusion Criteria:\n\n* Pregnant or breastfeeding\n* Receipt of prior cancer directed therapy with the exclusion of up to 1 dose of intrathecal chemotherapy, 1 dose of vincristine, or emergency radiotherapy due to organ compromising malignant mass. There is no exclusion for prior steroid therapy.\n* Known to be currently ineligible for available SJALL therapeutic studies (e.g. receipt of prohibited therapy, no appropriate SJALL therapeutic study available, enrolled on competing trial, etc.).\n\nNote: The intention of this exclusion criterion is to enroll all newly diagnosed ALL\u002F LLy\u002F MPAL patients. If participant is screened as a potential participant for subsequent SJALL and later found to be ineligible due to information obtained during INITIALL, this will not make the participant ineligible for INITIALL.\n\n* Inability or unwillingness of research participant or legal guardian\u002Frepresentative to give written informed consent.\n* Major pre-existing abnormalities such as ataxia telangiectasia, Fanconi anemia, Charcot Marie Tooth, etc.",{"count":602,"type":19},850,[604],"PHASE4","The goal of this study is to provide sufficient therapy during the time a patients' B-cell Acute Lymphoblastic Leukemia (ALL) or Lymphoblastic Lymphoma (LLy) risk category is being determined. The term \"risk\" refers to the chance of the ALL or LLy coming back after treatment.\n\nPrimary Objectives\n\n* To provide sufficient therapy to enable testing of newly diagnosed acute lymphoblastic leukemia\u002Flymphoma and mixed phenotype acute leukemia\u002Flymphoma tumor samples to determine eligibility and appropriate risk stratification for SJALL therapeutic studies.\n* To develop a central database of genomic and clinical findings.\n\nSecondary Objectives\n\n* To assess event free and overall survival data of patients enrolled on this study.",[539,540,165],[167,608,539,540,170,168,169],"Risk Category","2026-07-13",{"date":566,"type":36},{"date":612,"type":36},"2024-12-26",{"date":614,"type":19},"2039-05",{"name":41,"class":42},{"id":617,"slug":618,"hasResults":12,"nctId":619,"briefTitle":620,"officialTitle":620,"acronym":4,"eligibilityCriteria":621,"healthyVolunteers":12,"sex":16,"minAge":4,"maxAge":4,"enrollmentInfo":622,"targetDuration":4,"studyType":107,"phases":4,"briefSummary":624,"conditions":625,"keywords":628,"overallStatus":32,"whyStopped":4,"lastUpdateSubmitDate":635,"lastUpdatePostDateStruct":636,"startDateStruct":637,"completionDateStruct":639,"leadSponsor":641,"locationsCount":43},"100617139","stakeholders-of-rare-diseases-informing-values-in-neuroethics-100617139","NCT07314736","Stakeholders of Rare Diseases Informing Values In Neuroethics","Inclusion Criteria:\n\nGroup 1 (Parental Caregiver and Patient Participants)\n\n* Parental\u002Fprimary caregiver with a child who has a genetic diagnosis of an ultrarare disorder with pediatric onset, or a clinical diagnosis with a suspected genetic etiology.\n* Child is under 21 years of age at the time of enrollment.\n* Child has an expected survival of at least one year following study enrollment.\n* Patients (age ≤ 25 years) with a genetic diagnosis of an ultrarare disorder with pediatric onset, or clinical diagnosis with suspected genetic etiology.\n* Willingness to provide verbal informed consent (or assent, as appropriate) to participate\n\nGroup 2 (Other Family)\n\n* Family member of a Group 1 participant who plays an active role in the child's life or care.\n* Includes siblings (≥ 13 years of age), grandparents, or other non-primary caregivers directly affected by the child's diagnosis.\n* Demonstrated familiarity with the child's medical and family experience.\n* Willingness to provide verbal informed consent (or assent, as appropriate) to participate.\n\nGroup 3 (Non-Family Stakeholders)\n\n* Individuals currently engaged, or recently active, in clinical care, research, advocacy or policy work related to pediatric-onset rare genetic disorders.\n* May include clinicians (e.g., neurologists, genetic counselors, nurses, child-life specialists, home-health staff), members of patient-advocacy organizations, institutional-review-board (IRB) members, payers, sponsors, funders, or representatives of hospital systems or regulatory agencies.\n* Willingness to provide verbal informed consent to participate in semi-structured interviews or focus groups\n\nExclusion Criteria:\n\n* Limited English proficiency\n* Unable to complete the survey materials or complete the interviews in English.\n* Inability or unwillingness of research participant to give verbal informed consent (in English)\n* Condition or chronic illness, which in the opinion of the PI\u002FCo-I, makes participation unsafe or untenable (i.e., cognitive impairment, concurrent acute morbidity).",{"count":623,"type":19},385,"The purpose of this research study is to learn more about the perspectives of key stakeholders-patients, families, healthcare providers, and researchers-on the ethical challenges of small-scale, personalized treatment trials for rare neurological diseases (RND).",[626,627],"Rare Disorder","Disorder, Neurologic",[629,630,631,632,633,634],"Rare Neurological Disorders (RND)","Stakeholders","Patient Participant","Caregiver","Other Family","Non-Family Stakeholder","2026-07-10",{"date":609,"type":36},{"date":638,"type":36},"2026-07-08",{"date":640,"type":19},"2031-01",{"name":41,"class":42},{"id":643,"slug":644,"hasResults":12,"nctId":645,"briefTitle":646,"officialTitle":646,"acronym":4,"eligibilityCriteria":647,"healthyVolunteers":51,"sex":16,"minAge":4,"maxAge":4,"enrollmentInfo":648,"targetDuration":4,"studyType":20,"phases":649,"briefSummary":650,"conditions":651,"keywords":652,"overallStatus":32,"whyStopped":4,"lastUpdateSubmitDate":635,"lastUpdatePostDateStruct":655,"startDateStruct":656,"completionDateStruct":658,"leadSponsor":659,"locationsCount":43},"100598409","seminar-in-unwavering-empowering-presence-optimized-for-rehabilitation-teams-100598409","NCT07071116","Seminar in Unwavering Empowering Presence Optimized for Rehabilitation Teams","Inclusion Criteria: Intervention Participants\n\n* Licensed rehabilitation professionals (e.g., physical therapists, occupational therapists, speech-language pathologists) employed at St. Jude Children's Research Hospital actively involved in the care of pediatric oncology patients.\n* Willingness to participate in the communication skills training (CST) intervention and associated study activities.\n\nInclusion Criteria: Intervention Facilitators\n\n* St. Jude Bereaved Parent Educators who have participated as an educator in at least one other institutional educational event\n* Willingness to facilitate the communication skills training (CST) intervention and complete associated study activities.\n\nExclusion Criteria: Intervention Participants\n\n* Non-rehabilitation professionals\n* Individuals unable to attend the CST intervention session.\n\nExclusion Criteria: Intervention Facilitators\n\n* Individuals unable to attend the CST intervention session.",{"count":7,"type":19},[22],"The goal of this study to test the feasibility, acceptability, and potential impact of a serious illness communication skills training (CST) tailored to rehabilitation professionals to improve their comfort and confidence in navigating difficult conversations with patients and families.\n\nPrimary Objectives:\n\nAim 1: To assess feasibility and acceptability of a multidisciplinary co-designed interactive CST program for rehabilitation professionals who care for children with serious illness and their families.\n\nAim 2: To characterize the potential impact of this CST intervention on pediatric rehabilitation professionals.\n\nSecondary Objective:\n\nAim 3: To examine the perspectives of bereaved parent educators on participation in the implementation of communication training for rehabilitation professionals.",[59],[653,654],"Communication Skills Training (CST)","Rehabilitation Professionals",{"date":609,"type":36},{"date":657,"type":36},"2025-07-09",{"date":362,"type":19},{"name":41,"class":42},""]