Beta-Thalassemia Clinical Trials

105 clinical trials for Beta-Thalassemia across international registries, including 19 currently recruiting participants. Compare status, phase, eligibility criteria and locations.

• A Long-term Follow-up Study in Participants Who Received CTX001 — RECRUITING · PHASE3
• Partial Stem Cell Transplant for Sickle Cell Disease From Matched Donors — RECRUITING · PHASE1
• Study of Safety & PK of Luspatercept (ACE-536) in Pediatric Participants With Beta (β)-Thalassemia — RECRUITING · PHASE2
• A Study to Evaluate Long-term Safety in Participants Who Have Participated in Other Luspatercept (ACE-536) Clinical Trials — RECRUITING · PHASE3
• CS-101 in Patients With β-thalassemia — RECRUITING · PHASE1
• ALS20-101 Lentiviral Gene Therapy for Beta Thalassemia — RECRUITING · PHASE1
• Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease — RECRUITING · PHASE3
• A Follow-up Study to Evaluate the Long-term Safety and Efficacy in Participants Who Received CS-101 Injection — RECRUITING
• A Long-term Follow-up Study in Participants Who Received CS-101 — RECRUITING
• Hematopoietic Stem Cell BCL11A Enhancer Gene Editing for Severe β-Hemoglobinopathies — RECRUITING · PHASE1
• A Long-term Follow-up Study in Participants Who Received CS-101 — RECRUITING · NA
• AlloSCT for Malignant and Non-malignant Hematologic Diseases Utilizing Alpha/Beta T Cell and CD19+ B Cell Depletion — RECRUITING · PHASE2
• A Study of Participants with Β-Thalassemia Treated with Betibeglogene Autotemcel — RECRUITING
• A Multiple Ascending Dose Study of 9MW3011 in Patients With Non-transfusion-dependent β-thalassemia — RECRUITING · PHASE1
• FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases — RECRUITING
• A Post-Marketing Surveillance Study to Assess Safety of Luspatercept in Korean Patients With Myelodysplastic Syndrome or β-thalassemia — RECRUITING
• Adherence of Beta Thalssemia Patients to Oral Chelation Therapy — RECRUITING · NA
• A LONG-TERM FOLLOW-UP STUDY OF SUBJECTS WITH BETA-THALASSEMIA OR SICKLE CELL DISEASE TREATED WITH AUTOLOGOUS CRISPR-CAS9 MODIFIED HEMATOPOIETIC STEM CELLS (CTX001) — RECRUITING
• A PHASE 3B, OPEN-LABEL, SINGLE-ARM, ROLLOVER STUDY TO EVALUATE LONG-TERM SAFETY IN SUBJECTS WHO HAVE PARTICIPATED IN OTHER LUSPATERCEPT(ACE-536) CLINICAL TRIALS — RECRUITING · PHASE3
• Safety and Efficacy Evaluation of BRL-101 in Subjects With Transfusion-Dependent β-Thalassemia — ACTIVE_NOT_RECRUITING · PHASE1
• A Study to Evaluate Luspatercept Utilization in Patients With Lower Risk Myelodysplastic Syndromes or Beta-thalassemia — TERMINATED
• Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Transfusion-Dependent β-Thalassemia (TDT) — ACTIVE_NOT_RECRUITING · PHASE3
• Early Check: Expanded Screening in Newborns — ACTIVE_NOT_RECRUITING
• An Open-label Study of a Gene Therapy Product (Vebeglogene Autotemcel) in Transfusion Dependent Beta-Thalassemia — ACTIVE_NOT_RECRUITING · PHASE1
• Base-edited Autologous Hematopoietic Stem Cell Transplantation in Treating Patients With β-thalassemia Major — COMPLETED · PHASE1
• Evaluation of Micro RNA-155 Expression in Relation to Alloantibody Formation in Transfusion-Dependent Patients — NOT_YET_RECRUITING
• Addition of JSP191 (C-kit Antibody) to Nonmyeloablative Hematopoietic Cell Transplantation for Sickle Cell Disease and Beta-Thalassemia — ACTIVE_NOT_RECRUITING · PHASE1
• A Clinical Study Evaluating the Safety and Efficacy of CS-101 in Treating Subjects With β-thalassemia — COMPLETED · PHASE1
• A Safety and Efficacy Study Evaluating CTX001 in Participants With Transfusion-Dependent β-Thalassemia — COMPLETED · PHASE2
• Evaluating the Effect of N-Acetyl Cysteine and Alpha Lipoic Acid in Patients With Beta Thalassemia — NOT_YET_RECRUITING · PHASE3
• A Study to Evaluate Luspatercept (ACE-536) in Chinese Participants Who Require Regular Red Blood Cell Transfusions Due to Beta (β)-Thalassemia. — ACTIVE_NOT_RECRUITING · PHASE2
• Long Term Beta Thalassemia Treatment: Findings From The Extension Period — ACTIVE_NOT_RECRUITING · PHASE2
• ERN-EuroBloodNet Registry on Patients With Rare Red Blood Cell Defects and COVID-19 — COMPLETED
• Plasticizer Exposure and Its Consequences on Health — NOT_YET_RECRUITING
• Vitamin E Supplementation for Children With Transfusion Dependent Beta Thalassemia on Different Iron Chelation Regimen — COMPLETED · NA
• Study to Evaluate Additional Risk Minimisation Measures (aRMMs) for REBLOZYL Among Healthcare Professionals (HCPs) — COMPLETED
• Stem Cell Transplant in Sickle Cell Disease and Thalassemia — OTHER · PHASE2
• Evaluation of Low-cost Techniques for Detecting Sickle Cell Disease and β-thalassemia in Nepal and Canada — COMPLETED · NA
• Tissue Repository Providing Annotated Biospecimens for Approved Investigator-directed Biomedical Research Initiatives — OTHER
• Thrombophilia In Beta Thalassemia — OTHER
• At-Home Research Study for Patients With Autoimmune, Inflammatory, Genetic, Hematological, Infectious, Neurological, CNS, Oncological, Respiratory, Metabolic Conditions — COMPLETED
• Comparison Of Efficacy And Safety Of Thalidomide Vs Hydroxyurea In Thalassemia Patients: A Single-Centre Pilot Study. — COMPLETED · PHASE2
• A Study Evaluating the Efficacy and Safety of the LentiGlobin® BB305 Drug Product in Participants With Transfusion-Dependent β-Thalassemia — COMPLETED · PHASE3
• Testing SIROLIMUS in Beta-thalassemia Transfusion Dependent Patients — COMPLETED · PHASE2
• Safety, Tolerability, Pharmacokinetics (PK), Pharmacodynamics (PD) and Preliminary Efficacy of VIT-2763 in β-thalassaemia — COMPLETED · PHASE2
• Assessment of Cognitive Function, Fatigue and Health Related Quality of Life in Children With Beta Thalassemia — COMPLETED
• A Study Evaluating the Efficacy and Safety of the LentiGlobin® BB305 Drug Product in Participants With Transfusion-Dependent β-Thalassemia, Who do Not Have a β0/β0 Genotype — COMPLETED · PHASE3
• An Efficacy and Safety Study of Luspatercept (ACE-536) Versus Placebo in Adults Who Require Regular Red Blood Cell Transfusions Due to Beta (β) Thalassemia — COMPLETED · PHASE3
• Phase I/II Pilot Study of Mixed Chimerism to Treat Hemoglobinopathies — WITHDRAWN · PHASE1
• The Prevelence of HBB c.93-21 G-A in β Thalassemia Patients — OTHER

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