[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"severe-aplastic-anemia-saa\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:severe-aplastic-anemia-saa":30},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,4,0,[8,59,89,114],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":15,"eligibilityCriteria":16,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":4,"enrollmentInfo":19,"targetDuration":4,"studyType":22,"phases":23,"briefSummary":26,"conditions":27,"keywords":34,"overallStatus":46,"whyStopped":4,"lastUpdateSubmitDate":47,"lastUpdatePostDateStruct":48,"startDateStruct":51,"completionDateStruct":53,"leadSponsor":55,"locationsCount":58},"100647654","phase-1-a-phase-12-study-to-evaluate-the-safety-and-efficacy-of-dibotatug-dr-01-in-adults-with-bone-marrow-failure-syndromes-100647654",false,"NCT07712562","A Phase 1\u002F2 Study to Evaluate the Safety and Efficacy of Dibotatug (DR-01) in Adults With Bone Marrow Failure Syndromes","A Phase 1\u002F2, Open-Label Study to Evaluate the Safety and Efficacy of Dibotatug in Adults With Bone Marrow Failure Syndromes","BMF","Inclusion Criteria:\n\n* Age ≥ 18 years old\n* Women of childbearing potential and males must agree to use 2 methods of effective contraception, with at least 1 method being highly effective.\n\nInclusion Criteria for Severe Aplastic Anemia (SAA) (Cohorts A and B):\n\n* Participants with SAA must have a current or prior diagnosis of SAA or very SAA.\n\nInclusion Criteria for Refractory Severe Aplastic Anemia (Cohort A) Participants with refractory SAA must have:\n\n* Received one ≥ 3-month course of ATG and\u002For CSA-based IST.\n* Refractory SAA, defined as failure to achieve CR or PR ≥ 3 months after starting ATG and\u002For CSA-based IST.\n\nInclusion Criteria for Relapsed Severe Aplastic Anemia (Cohort B):\n\n* Relapsed SAA, defined as relapse following a CR or PR that was achieved ≥ 3 months after starting ATG- and\u002For cyclosporine A (CSA)-based IST.\n\nInclusion Criteria for Relapsed or Refractory Transfusion-Dependent Non-Severe Aplastic Anemia (Cohort C):\n\n* Current or prior diagnosis of NSAA\n* No current or prior diagnosis of SAA.\n* Received at least 1 prior course of IST such as ATG- or CSA, with or without a TPO-R agonist (lasting ≥ 3 months).\n* Meets criteria for transfusion dependence (either RBC or platelet):\n* RBC transfusion dependence: transfusion of ≥ 2 units of RBCs in the past 56 days\n* Platelet transfusion dependence: transfusion of ≥ 1 unit of apheresis platelets in the past 28 days\n\nParticipants entering the Extension Treatment Period must meet the following criteria:\n\n* Signed informed consent form (ICF) for the Extension Treatment Period.\n* CR or PR by Week 24 during the Main Treatment Period\n\nExclusion Criteria:\n\n* Diagnosis of Fanconi anemia, dyskeratosis congenita, or other congenital BMF syndrome.\n* Prior HCT.\n* Planning to receive HCT as treatment for AA.\n* Evidence of a clonal disorder with poor risk cytogenetics per Revised International Prognostic Scoring System (IPSS-R) for MDS.\n* Diagnosis of PNH or a clonal hematologic bone marrow disorder such as LGLL. Existence of PNH clones, LGLL cells, or clonal hematopoiesis of indeterminate potential (CHIP) clones without a clinical diagnosis is not exclusionary.\n* Use of a T-cell depleting agent (e.g., ATG, alemtuzumab, thymoglobulin) within 3 months prior to Day 1.\n* Use of a B-cell depleting agent (e.g., rituximab, ocrelizumab, ofatumumab, ublituximab) within 28 days prior to Day 1.\n* Use of any of the following within 14 days of Day 1, unless used as an established therapy at screening and there is evidence of either progressive cytopenia or lack of count improvement over the 3 months before screening:\n* Calcineurin inhibitor (e.g., cyclosporine), TPO-R agonist (e.g., eltrombopag), Androgen (e.g., danazol), Oral Janus kinase (JAK) inhibitor\n* Regardless of their use as an established therapy at screening, use of the above medications is prohibited for all participants from 3 months after Day 1.\n* Current infection not adequately responding to appropriate therapy or requiring hospitalization.\n* Current uncontrolled or invasive infection with cytomegalovirus (CMV), Epstein Barr virus (EBV), varicella zoster virus (VZV), herpes simplex virus (HSV), or human T-lymphotropic virus-1 (HTLV-1), defined by polymerase chain reaction (PCR) at screening. Note that low level CMV, EBV, or VZV viremia is not exclusionary.\n* Human immunodeficiency virus (HIV) infection.\n* Current or prior infection with hepatitis B virus (HBV)\n* Current hepatitis C virus (HCV) infection\n* Latent tuberculosis (TB) infection as indicated by IFN-γ release assay without documentation of appropriate treatment (appropriate therapy as defined by the World Health Organization \\[WHO\\] and\u002For the United States Centers for Disease Control and Prevention).\n* Estimated glomerular filtration rate \\\u003C 30 mL\u002Fmin\u002F1.73 m2 at screening (using the Chronic Kidney Disease Epidemiology Collaboration formula; Levey 2009).\n* Total bilirubin \\> 1.5 × upper limit of normal (ULN) (\\> 3 × ULN if known Gilbert's disease).\n* Aspartate aminotransferase or alanine aminotransferase \\> 2.5 × ULN (except in participants with known iron overload).","ALL","18 Years",{"count":20,"type":21},60,"ESTIMATED","INTERVENTIONAL",[24,25],"PHASE1","PHASE2","This is a multicenter, open-label, Phase 1\u002F2 basket study to evaluate the safety and efficacy of Dibotatug (DR-01) in adults with Bone Marrow Failure syndromes.",[28,29,30,31,32,33],"Bone Marrow Failure Syndromes","Aplastic Anemias","Severe Aplastic Anemia (SAA)","Severe Aplastic Anemia, Relapse","Severe Aplastic Anemia, Refractory","Non-severe Aplastic Anemia",[35,36,37,38,39,40,41,42,43,44,45],"DR-01","Dibotatug","aplastic anemia","transfusion dependent","hematologic diseases","hematology","Blood disorder","pancytopenia","stem cell disorder","red blood cell disease","white blood cell disease","NOT_YET_RECRUITING","2026-07-23",{"date":49,"type":50},"2026-07-24","ACTUAL",{"date":52,"type":21},"2026-08",{"date":54,"type":21},"2030-10",{"name":56,"class":57},"Dren Bio","INDUSTRY",10,{"id":60,"slug":61,"hasResults":11,"nctId":62,"briefTitle":63,"officialTitle":63,"acronym":4,"eligibilityCriteria":64,"healthyVolunteers":11,"sex":17,"minAge":65,"maxAge":66,"enrollmentInfo":67,"targetDuration":4,"studyType":22,"phases":69,"briefSummary":70,"conditions":71,"keywords":74,"overallStatus":77,"whyStopped":4,"lastUpdateSubmitDate":78,"lastUpdatePostDateStruct":79,"startDateStruct":81,"completionDateStruct":83,"leadSponsor":85,"locationsCount":88},"100325717","phase-2-haplo-identical-transplantation-for-severe-aplastic-anemia-hypo-plastic-mds-and-pnh-using-peripheral-blood-stem-cells-and-post-transplant-cyclophosphamide-for-gvhd-prophylaxis-100325717","NCT03520647","Haplo-identical Transplantation for Severe Aplastic Anemia, Hypo-plastic MDS and PNH Using Peripheral Blood Stem Cells and Post-transplant Cyclophosphamide for GVHD Prophylaxis","* INCLUSION CRITERIA - RECIPIENT:\n* Diagnosed with severe aplastic anemia with bone marrow cellularity \\\u003C30% (excluding lymphocytes) associated with RBC or platelet transfusion dependence and\u002For neutropenia (absolute neutrophil count less than or equal to 1000 cells\u002F microL or for patients receiving granulocyte transfusions, absolute neutrophil count less than or equal to 1000 cells\u002FmicroL before beginning granulocyte transfusions).\n\nOR\n\n--History of severe aplastic anemia transformed to MDS. that meet the following criteria: a) International Prognostic Scoring System (IPSS) risk category of INT-1 or greater, b) \\\u003C5% myeloblasts and \\\u003C30% of cellularity in the bone marrow on screening morphologic analysis.\n\nOR\n\n* PNH that is either refractory to treatment with eculizumab\u002Fravulizumab or occurs in patients who don t have access to treatment with eculizumab associated with either a) life- threatening thrombosis and\u002For b) cytopenia associated with transfusion dependence and\u002For c) recurrent and debilitating hemolytic crisis.\n\n  * Subjects with severe aplastic anemia, hypoplastic MDS or PNH with associated bone marrow failure syndromes who have intolerance of or failure to respond to immunosuppressive therapy. This also includes patients who have failed immunosuppressive therapy with ATG and cyclosporine or therapy with cyclosporine combined with eltrombopag in those who are intolerant of or do not have access to treatment with ATG.\n  * Availability of at least one HLA- haploidentical related donor (i.e. \\>= 5\u002F10 HLA match: HLA-A, B, C, DR, and DQ loci) to serve as a stem cell donor for the allogeneic transplant.\n  * Availability of a backup stem cell source in the event of graft rejection:\n* at least one additional haploidentical related alternative donor (i.e. HLA- haploidentical related donor (i.e. \\>= 5\u002F10 HLA match: HLA-A, B, C, DR, and DQ loci) or \\>= 9\u002F10 HLA matched unrelated donor who is available to serve as a stem cell donor for a salvage allogeneic transplant in the event that the haplo-transplant has been rejected.\n* umbilical cord blood unit\u002Fs that can be used for a salvage cord blood transplant in the event that the haplo-transplant has been rejected\n\n  * The patient does not have any HLA antibodies detectable against any of the mismatched HLA alleles expressed by the haplo-donor.\n  * Ages 4-60 years inclusive.\n  * Ability to comprehend the investigational nature of the study and provide informed consent. The procedure will be explained to subjects aged 4-17 years with formal consent being obtained from parents or legal guardian.\n\nEXCLUSION CRITERIA - RECIPIENT (ANY OF THE FOLLOWING):\n\n* Availability of an HLA identical (12\u002F12) matched related or unrelated donor who is available within optimal timeline and suitable considering graft source and established donor selection factors (e.g. age, sex, viral exposure, ABO compatibility, pregnancy status, etc) per PI discretion.\n* The patient is deemed to be a candidate for a 12\u002F12 HLA matched unrelated stem cell transplant (availability of a donor and resources required for such a transplant).\n* ECOG performance status of 2 or more.\n* Major anticipated illness or organ failure incompatible with survival from transplant.\n* Current pregnancy, or unwillingness to take oral contraceptives or use a barrier method of birth control or practice abstinence to refrain from pregnancy, if of childbearing potential for one year.\n* HIV positive.\n* Diagnosis of Fanconi s anemia (by chromosome breakage study).\n* Diffusion capacity of carbon monoxide (DLCO) \\\u003C40% using DLCO corrected for Hgb or lung volumes (patients under the age of 10 may be excluded from this criterion if they have difficulty performing the test correctly and thus are unable to have their DLCO assessed).\n* Left ventricular ejection fraction \\\u003C40% (evaluated by ECHO)\n* Transaminases \\> 5x upper limit of normal.\n* Direct bilirubin \\>3 mg\u002Fdl.\n* Creatinine clearance \\\u003C 50 cc\u002Fmin\u002FBSAm\\^2 by 24-hour urine collection adjusted by body surface area.\n* Serum creatinine \\> 2.5 mg\u002Fdl\n* Presence of an active infection not adequately responding to appropriate therapy.\n* History of a malignant disease liable to relapse or progress within 5 years.\n\nINCLUSION CRITERIA - DONOR:\n\nDonor deemed suitable and eligible, and willing to donate, per clinical evaluations who are additionally willing to donate blood for research. Donors will be evaluated in accordance with existing Standard NIH Policies and Procedures for determination of eligibility and suitability for clinical donation. Note that participation in this study is offered to all donors, but is not required for a donor to make a stem cell donation, so it is possible that not all donors will enroll onto this study.\n\nEXCLUSION CRITERIA - RELATED DONOR:\n\nNone","4 Years","75 Years",{"count":68,"type":21},56,[25],"Background:\n\nSevere aplastic anemia (SAA), and myelodysplastic syndrome (MDS), and paroxysmal nocturnal hemoglobinuria\n\n(PNH) cause serious blood problems. Stem cell transplants using bone marrow or blood plus chemotherapy can help. Researchers want to see if using peripheral blood stem cells (PBSCs) rather than bone marrow cells works too. PBSCs are easier to collect and have more cells that help transplants.\n\nObjectives:\n\nTo see how safely and effectively SAA, MDS and PNH are treated using peripheral blood hematopoietic stem cells from a family member plus chemotherapy.\n\nEligibility:\n\nRecipients ages 4-60 with SAA, MDS or PNH and their relative donors ages 4-75\n\nDesign:\n\nRecipients will have:\n\n* Blood, urine, heart, and lung tests\n* Scans\n* Bone marrow sample\n\nRecipients will need a caregiver for several months. They may make fertility plans and a power of attorney.\n\nDonors will have blood and tissue tests, then injections to boost stem cells for 5-7 days.\n\nDonors will have blood collected from a tube in an arm or leg vein. A machine will separate stem cells and maybe white blood cells. The rest of the blood will be returned into the other arm or leg.\n\nIn the hospital for about 1 month, recipients will have:\n\n* Central line inserted in the neck or chest\n* Medicines for side effects\n* Chemotherapy over 8 days and radiation 1 time\n* Stem cell transplant over 4 hours\n\nUp to 6 months after transplant, recipients will stay near NIH for weekly physical exams and blood tests.\n\nAt day 180, recipients will go home. They will have tests at their doctor s office and NIH several times over 5 years.",[30,72,73],"Hypo-Plastic Myelodysplastic Syndrome (MDS)","Paroxysmal Nocturnal Hemoglobinuria (PNH)",[75,76],"Haploidentical CD34+cells","Non-Myeloablative","RECRUITING","2026-07-01",{"date":80,"type":50},"2026-07-02",{"date":82,"type":50},"2019-02-19",{"date":84,"type":21},"2028-06-01",{"name":86,"class":87},"National Heart, Lung, and Blood Institute (NHLBI)","NIH",1,{"id":90,"slug":91,"hasResults":11,"nctId":92,"briefTitle":93,"officialTitle":94,"acronym":95,"eligibilityCriteria":96,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":97,"enrollmentInfo":98,"targetDuration":4,"studyType":22,"phases":100,"briefSummary":102,"conditions":103,"keywords":4,"overallStatus":46,"whyStopped":4,"lastUpdateSubmitDate":104,"lastUpdatePostDateStruct":105,"startDateStruct":107,"completionDateStruct":109,"leadSponsor":111,"locationsCount":4},"100638952","peripheral-blood-versus-bone-marrow-plus-peripheral-blood-grafts-for-haploidentical-transplantation-in-severe-aplastic-anemia-100638952","NCT07629947","Peripheral Blood Versus Bone Marrow Plus Peripheral Blood Grafts for Haploidentical Transplantation in Severe Aplastic Anemia","A Randomized Controlled Trial Comparing Different Graft Sources for Haploidentical Transplantation in Aplastic Anemia","PB-Graft Haplo","Inclusion Criteria:\n\nPatients with newly diagnosed or relapsed severe aplastic anemia (SAA) or very severe aplastic anemia (vSAA) according to the Camitta criteria. SAA is defined as bone marrow cellularity \\\u003C25% and at least two of the following peripheral blood criteria: absolute neutrophil count \\\u003C0.5 × 10⁹\u002FL, platelet count \\\u003C20 × 10⁹\u002FL, or reticulocyte count \\\u003C20 × 10⁹\u002FL. vSAA is defined as SAA with an absolute neutrophil count \\\u003C0.2 × 10⁹\u002FL.\n\nAge 18 to 40 years. Availability of a haploidentical related donor. Eastern Cooperative Oncology Group (ECOG) performance status of 0 to 2.\n\nAdequate organ function, defined as:\n\nCardiac function: left ventricular ejection fraction ≥50% and no severe arrhythmia; Hepatic function: total bilirubin ≤2 times the upper limit of normal and alanine aminotransferase\u002Faspartate aminotransferase ≤3 times the upper limit of normal; Renal function: creatinine clearance ≥60 mL\u002Fmin. No active infection. For women of childbearing potential, a negative pregnancy test is required. All patients must agree to use effective contraception during the study period.\n\nWritten informed consent provided by the patient or legal representative.\n\nExclusion Criteria:\n\nPresence of hematologic malignancy or myelodysplastic syndrome-related chromosomal abnormalities, such as +8 or del(7q).\n\nPrior allogeneic hematopoietic stem cell transplantation or prior organ transplantation.\n\nAvailability of an HLA-matched sibling donor. Active uncontrolled infection, including unresolved invasive fungal disease, active tuberculosis, sepsis, or other uncontrolled infections.\n\nSevere comorbidities, defined as Hematopoietic Cell Transplantation-Comorbidity Index (HCT-CI) score ≥4, or uncontrolled autoimmune disease.\n\nDonor-related contraindications, including donor age \\\u003C12 years or \\>65 years, donor body mass index \\\u003C18 or \\>35 kg\u002Fm², contraindications to anesthesia, or contraindications to bone marrow harvest.\n\nPsychiatric illness that prevents cooperation with the study procedures, or any condition considered by the investigator to make the patient unsuitable for study participation.","40 Years",{"count":99,"type":21},180,[101],"NA","Severe aplastic anemia is a life-threatening bone marrow failure disorder. Haploidentical hematopoietic stem cell transplantation has become an important curative treatment option for patients who do not have an HLA-matched sibling donor.\n\nTraditionally, haploidentical transplantation for severe aplastic anemia uses a graft composed of granulocyte colony-stimulating factor-primed bone marrow plus peripheral blood stem cells. However, bone marrow collection is invasive and may increase donor burden. Peripheral blood stem cell collection is simpler and less invasive, but it remains unclear whether using peripheral blood stem cells alone provides similar clinical outcomes without increasing the risk of graft-versus-host disease.\n\nThis multicenter, randomized, open-label, non-inferiority trial will compare granulocyte colony-stimulating factor-primed peripheral blood stem cells alone with granulocyte colony-stimulating factor-primed bone marrow plus peripheral blood stem cells as graft sources for haploidentical transplantation in patients with severe or very severe aplastic anemia. Participants will be randomly assigned in a 1:1 ratio to either graft source group. The primary outcome is the cumulative incidence of grade II-IV acute graft-versus-host disease within 100 days after transplantation. Secondary outcomes include engraftment, chronic graft-versus-host disease, infections, immune reconstitution, survival, and donor safety.",[30],"2026-06-01",{"date":106,"type":50},"2026-06-05",{"date":108,"type":21},"2026-06-15",{"date":110,"type":21},"2029-12-31",{"name":112,"class":113},"Peking University People's Hospital","OTHER",{"id":115,"slug":116,"hasResults":11,"nctId":117,"briefTitle":118,"officialTitle":119,"acronym":4,"eligibilityCriteria":120,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":4,"enrollmentInfo":121,"targetDuration":4,"studyType":22,"phases":123,"briefSummary":124,"conditions":125,"keywords":4,"overallStatus":46,"whyStopped":4,"lastUpdateSubmitDate":127,"lastUpdatePostDateStruct":128,"startDateStruct":130,"completionDateStruct":132,"leadSponsor":134,"locationsCount":4},"100635880","phase-2-csaepaghpagromiplostim-n01-in-newly-diagnosed-saatd-nsaa-100635880","NCT07558434","CsA+EPAG\u002FHPAG+Romiplostim N01 in Newly-diagnosed SAA\u002FTD-NSAA","Cyclosporine + Eltrombopag\u002FHetrombopag + Romiplostim N01 in the Treatment of Newly-diagnosed Transfusion-dependent Non-severe Aplastic Anemia\u002F Severe Aplastic Anemia","Inclusion Criteria:\n\n1. Age ≥ 18 years old;\n2. Diagnosed with aplastic anemia (AA) through routine blood tests, bone marrow puncture, bone marrow biopsy, and exclusion tests, and determined as transfusion-dependent non-severe aplastic anemia (TD-NSAA) or severe aplastic anemia (SAA) according to the Camitta criteria; Platelet \\\u003C 30×10\\^9\u002FL;\n3. Had no HLA-matched donors or was not suitable for first-line allogeneic hematopoietic stem cell transplantation (HSCT);\n4. Not suitable for ATG, due to reasons such as age, complications, and the patient's own wishes;\n5. With baseline liver and kidney functions \\\u003C2 ULN;\n6. ECOG score ≤ 2;\n7. Signed the informed consent;\n\nExclusion Criteria:\n\n1. Had other primary or secondary bone marrow failure (BMF) diseases, such as Fanconi anemia, congenital keratinization disorder, etc.;\n2. With evidence of clonal hematological bone marrow diseases (MDS, AML) in cytogenetics;\n3. PNH clone ≥ 50%;\n4. Received HSCT before enrollment;\n5. Previously used immunosuppressive treatments such as ATG, CsA, TPO receptor agonists (TPO-RAs);\n6. Allergic or intolerant to romiplostim N01, eltrombopag, hetrombopag, or CsA;\n7. Pregnant or lactating patients;\n8. Severe bleeding or infection that cannot be controlled by standard treatment;\n9. History of arterial or venous thrombosis;\n10. Complicated with malignant tumors;\n11. Participated in other clinical trials within 3 months;\n12. Patients considered not suitable to participate in this study by the investigator.",{"count":122,"type":21},43,[25],"This study aimed to explore the efficacy and safety of cyclosporine (CsA)\n\n+eltrombopag (EPAG)\u002Fhetrombopag (HPAG)+romiplostim N01 in the treatment of newly-diagnosed transfusion-dependent aplastic anemia (TD-NSAA) and severe aplastic anemia (SAA)",[30,126],"Transfusion-dependent Non-severe Aplastic Anemia","2026-04-23",{"date":129,"type":50},"2026-04-30",{"date":131,"type":21},"2026-04",{"date":133,"type":21},"2029-12",{"name":135,"class":113},"Peking Union Medical College Hospital"]