Testing effectiveness (Phase 2)Ended earlyNCT04205435What this trial is testingΒ-globin Restored Autologous HSC in β-thalassemia Major PatientsWho this might be right forβ-thalassemia Major Bioray Laboratories 2
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 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)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 applicableLooking for participantsNCT06314529What this trial is testingLong-term Follow-up Study of BHC001 for TDTWho this might be right forThalassemia, Beta Bioray Laboratories 6
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
Testing effectiveness (Phase 2)Study completedNCT02151526What this trial is testingA Study Evaluating the Safety and Efficacy of LentiGlobin BB305 Drug Product in β-Thalassemia Major (Also Referred to as Transfusion-dependent β-Thalassemia [TDT]) and Sickle Cell DiseaseWho this might be right forBeta-Thalassemia MajorSickle Cell Disease Genetix Biotherapeutics Inc. 7
Not applicableUnknownNCT05494333What this trial is testingCorrelation Between Pulmonary Functions and Physical Fitness in Children With β-thalassemiaWho this might be right forβ-ThalassemiaPulmonary FunctionPhysical Fitness Cairo University 40
Post-approval studies (Phase 4)Active Not RecruitingNCT05891249What this trial is testingEvaluate the Safety and Effectiveness of Luspatercept for the Treatment of Transfusion-dependent (TD) Anemia Associated With Myelodysplastic Syndromes (MDS) & Beta-thalassemia (β-Thal) in IndiaWho this might be right forAnemia Bristol-Myers Squibb 85
Not applicableUnknownNCT01905787What this trial is testingSickle Cell Anemia - A Comparative Study Between Three Ethnical Communities, a Multicenter StudyWho this might be right forSickle Cell AnemiaSickle Cell β+ or β0 Thalassemia HaEmek Medical Center, Israel 300
Very early researchLooking for participantsNCT05745532What this trial is testingSafety and Efficacy Evaluation of β-globin Restored Autologous Hematopoietic Stem Cells in β-thalassemia Major PatientsWho this might be right forβ-thalassemia Shenzhen Hemogen 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
Testing effectiveness (Phase 2)Looking for participantsNCT07338344What this trial is testingEvaluating the Clinical Efficacy and Safety of Luspatercept Combined With Thalidomide in the Treatment of β-TDT PatientsWho this might be right forTransfusion-dependent β-thalassemia Patients Rongrong Liu 78
Not applicableLooking for participantsNCT06271512What this trial is testingParticipants with Β-Thalassemia Treated with Betibeglogene AutotemcelWho this might be right forBeta-Thalassemia Genetix Biotherapeutics Inc. 150
Not applicableStudy completedNCT01369719What this trial is testingEfficacy and Safety of Desferal Versus Osveral in Transfusional Iron OverloadWho this might be right forThalassemiaIron OverloadTransfusion Related Complications Hormozgan University of Medical Sciences 138
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
Large-scale testing (Phase 3)Study completedNCT03207009What this trial is testingA Study Evaluating the Efficacy and Safety of the LentiGlobin® BB305 Drug Product in Participants With Transfusion-Dependent β-ThalassemiaWho this might be right forBeta-Thalassemia Genetix Biotherapeutics Inc. 19
Not applicableUnknownNCT02126046What this trial is testingUnrelated Umbilical Cord Blood Following HLA-haploidentical Hematopoietic Stem Cell Transplantation in Patients With β-thalassemia MajorWho this might be right forThalassemia Major Nanfang Hospital, Southern Medical University 30
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
Early research (Phase 1)Study completedNCT02950857What this trial is testingEPEG in Beta Thalassemia PatientsWho this might be right forBeta-Thalassemia Prolong Pharmaceuticals 6