Testing effectiveness (Phase 2)Ended earlyNCT03993613What this trial is testingApotransferrin in Patients With β-thalassemiaWho this might be right forβ-thalassemia Intermedia Prothya Biosolutions 10
Testing effectiveness (Phase 2)Active Not RecruitingNCT05577312What this trial is testingSafety and Efficacy Evaluation of BRL-101 in Subjects With Transfusion-Dependent β-ThalassemiaWho this might be right forBeta-Thalassemia Bioray Laboratories 39
Testing effectiveness (Phase 2)Looking for participantsNCT06280378What this trial is testingΒ-Thalassemia Treatment with KL003 Cell InjectionWho this might be right forTransfusion-dependent Beta-Thalassemia Kanglin Biotechnology (Hangzhou) Co., Ltd. 41
Testing effectiveness (Phase 2)Ended earlyNCT04411082What this trial is testingIMR-687 in Subjects With Beta ThalassemiaWho this might be right forβ Thalassemia Cardurion Pharmaceuticals, Inc. 122
Testing effectiveness (Phase 2)Looking for participantsNCT04143724What this trial is testingStudy of Safety & PK of Luspatercept (ACE-536) in Pediatric Participants With Beta (β)-ThalassemiaWho this might be right forBeta-Thalassemia Celgene 99
Not applicableUnknownNCT06041620What this trial is testingSafety and Efficacy Evaluation of Autologous CRISPR-Cas12b Edited Hematopoietic Stem CellsWho this might be right forThalassemia, BetaThalassemia Major Institute of Hematology & Blood Diseases Hospital, China 2
Testing effectiveness (Phase 2)Study completedNCT02435901What this trial is testingHSCT For Patients With High Risk Hemoglobinopathies Using Reduced IntensityWho this might be right forSickle Cell DiseaseBeta Thalassemia-Major Northwell Health 29
Not applicableLooking for participantsNCT06073860What this trial is testingA Post-Marketing Surveillance Study to Assess Safety of Luspatercept in Korean Patients With Myelodysplastic Syndrome or β-thalassemiaWho this might be right forMyelodysplastic SyndromeBeta Thalassemia Bristol-Myers Squibb 104
Large-scale testing (Phase 3)Active Not RecruitingNCT04770779What this trial is testingA Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-TDT)Who this might be right forTransfusion-dependent Alpha-ThalassemiaTransfusion-dependent Beta-Thalassemia Agios Pharmaceuticals, Inc. 258
Not applicableLooking for participantsNCT06298630What this trial is testingLong-term Follow-up Study of BRL-101 for TDTWho this might be right forThalassemia, Beta Bioray Laboratories 45
Large-scale testing (Phase 3)Study completedNCT03851055What this trial is testingZinc Supplementation in Patients With β-Thalassemia Major Complicated With Diabetes MellitusWho this might be right forBeta-thalassemia Major Complicated With Diabetes Ain Shams University 80
Testing effectiveness (Phase 2)Active Not RecruitingNCT06421636What this trial is testingTest the Safety, Tolerability, and Efficacy of an Antibody, REGN7999, Injected Under the Skin for the Treatment of Iron Overload in Adult Participants With Non-Transfusion Dependent β-thalassemia, Using MRI Scans to Measure Iron Levels in the BodyWho this might be right forNon-Transfusion Dependent Beta-Thalassemia (NTDT) Regeneron Pharmaceuticals 19
Not applicableActive Not RecruitingNCT05860595What this trial is testingEvaluation the Safety and Efficacy of KL003 Cell Injection in the Treatment of Transfusion-dependent β-thalassemia.Who this might be right forTransfusion-dependent Beta-Thalassemia Institute of Hematology & Blood Diseases Hospital, China 3
Testing effectiveness (Phase 2)Study completedNCT01749540What this trial is testingStudy to Evaluate the Safety and Efficacy of Luspatercept (ACE-536) in Participants With Beta-thalassemia (A536-04/MK-6143-002)Who this might be right forB-Thalassemia Acceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway, NJ USA 64
Not applicableEnded earlyNCT05752123What this trial is testingEvaluate the Safety and Efficacy of ET-01 Transplantation in Subjects with Transfusion Dependent Β-Thalassaemia.Who this might be right forTransfusion Dependent Beta-Thalassaemia EdiGene (GuangZhou) Inc. 2
Testing effectiveness (Phase 2)Study completedNCT04364269What this trial is testingSafety, Tolerability, Pharmacokinetics (PK), Pharmacodynamics (PD) and Preliminary Efficacy of VIT-2763 in β-thalassaemiaWho this might be right forBeta-ThalassemiaNon-transfusion-dependent Thalassemia Vifor (International) Inc. 35
Early research (Phase 1)Looking for participantsNCT06647979What this trial is testingHematopoietic Stem Cell BCL11A Enhancer Gene Editing for Severe β-HemoglobinopathiesWho this might be right forSickle Cell DiseaseSickle Cell Anemia (HbSS, or HbSβ-thalassemia0)Beta-Thalassemia+1 more Daniel Bauer 10
Not applicableStudy completedNCT02626689What this trial is testingTo Document the Burden of Illness on the Quality of Life and the Impact on Healthcare Utilization in (Beta) β-thalassemia Subjects Who Are Transfusion Dependent (TD) and Non-transfusion Dependent (NTD) Receiving Standard of CareWho this might be right forBeta-Thalassemia Celgene 100
Not applicableActive Not RecruitingNCT02633943What this trial is testingLong-term Follow-up of Subjects With Transfusion-Dependent β-Thalassemia (TDT) Treated With Ex Vivo Gene TherapyWho this might be right forTransfusion-dependent Beta-Thalassemia Genetix Biotherapeutics Inc. 66
Testing effectiveness (Phase 2)UnknownNCT03014882What this trial is testingSperm DNA Damage in β-thalassemia Major: Is There a Role for Antioxidants?Who this might be right forEndocrine DiseaseFertility Decreased MaleDNA Damage Ain Shams University 30