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
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
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 applicableNot Yet RecruitingNCT07614217What this trial is testingOral Health Status in Children With β-Thalassemia MajorWho this might be right forB Thalassemia Major Cairo University 45
Testing effectiveness (Phase 2)Ended earlyNCT01571635What this trial is testingStudy to Determine the Safety and Tolerability of Sotatercept (ACE-011) in Adults With Beta( β)- Thalassemia.Who this might be right forBeta Thalassemia MajorBeta Thalassemia Intermedia Celgene 46
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 applicableUnknownNCT05133388What this trial is testingThe Prevelence of HBB c.93-21 G-A in β Thalassemia PatientsWho this might be right forBeta-Thalassemia Assiut University 150
Not applicableUnknownNCT01935661What this trial is testingA Case Control Study to Evaluate the Cognitive and Brain Function of β-thalassemia Patients.Who this might be right forThalassemiaBrain Function (Measured by ERP Level) HaEmek Medical Center, Israel 120
Very early researchEnrolling By InvitationNCT06328764What this trial is testingCS-101 in Patients With β-thalassemiaWho this might be right forBeta-Thalassemia CorrectSequence Therapeutics Co., Ltd 10
Testing effectiveness (Phase 2)Study completedNCT07292259What this trial is testingCombination of Thalidomide and Hydroxyuria in Transfusion Dependent ThalasemmiaWho this might be right forTransfusion Dependent Beta Thalassemia Pakistan Blood and Marrow Transplant (PBMT) Group 100
Not applicableLooking for participantsNCT06734520What this trial is testingClinical Study of Super Transplantation in the Treatment of Severe β-thalassemiaWho this might be right forThalassemia Majors (Beta-Thalassemia Major)Haplo-identical Donors Guangzhou Women and Children's Medical Center 3
Very early researchLooking for participantsNCT05864170What this trial is testingThe Safety and Efficacy Evaluation of HGI-001 Injection in Patients With Transfusion-Dependent β-ThalassemiaWho this might be right forβ-thalassemia Shenzhen Hemogen 3
Not applicableStudy completedNCT01319552What this trial is testingFresh Versus Old Red Blood Cells for TransfusionWho this might be right forIron, Abnormal Blood LevelOther Abnormal Blood Chemistry Columbia University 14
Not applicableLooking for participantsNCT05773729What this trial is testingSafety and Efficacy of Gene Modified Autologous Hematopoietic Stem Cells to Treat Transfusion-dependent Beta-thalassemiaWho this might be right forβ-thalassemia Shanghai BDgene Co., Ltd. 10
Large-scale testing (Phase 3)Active Not RecruitingNCT05356195What this trial is testingEvaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Transfusion-Dependent β-Thalassemia (TDT)Who this might be right forBeta-ThalassemiaThalassemiaGenetic Diseases, Inborn+2 more Vertex Pharmaceuticals Incorporated 16
Early research (Phase 1)Active Not RecruitingNCT01639690What this trial is testingSS-Thalassemia Major With Autologous CD34+ Hematopoietic Progenitor Cells Transduced With TNS9.3.55 a Lentiviral Vector Encoding the Normal Human ß-Globin GeneWho this might be right forConfirmed Diagnosis of ß-thalassemia Major San Rocco Therapeutics 10
Large-scale testing (Phase 3)Study completedNCT06146478What this trial is testingDeciphering Effects of Thalidomide on Red Blood Cells in Transfusion Dependents Beta Thalassemia PatientsWho this might be right forTransfusion-dependent Beta-Thalassemia Blood Care Clinic 200
Not applicableLooking for participantsNCT06479616What this trial is testingA Long-term Follow-up Study in Participants Who Received CS-101Who this might be right forBeta-Thalassemia Children's Hospital of Fudan University 5
Early research (Phase 1)Study completedNCT06291961What this trial is testingA Safety and Efficacy Study Evaluating CS-101 in Subjects With β-Thalassemia MajorWho this might be right forBeta-Thalassemia Major CorrectSequence Therapeutics Co., Ltd 9