Beta-thalassemia
📧 Sign up to get email alerts when something new happens for Beta-thalassemia.
Sign up freeTop drugs studied for this disease
-
CLINICAL_TRIALS trial_result · 2026-03-25Clinical Trial Update: A Long-term Follow-up Study in Participants Who Received CTX001 [Phase 3]
A long-term follow-up update on participants who received CTX001 in a Phase 3 study, reporting safety and durability data for beta-thalassem…
-
CLINICAL_TRIALS trial_result · 2026-02-23Clinical Trial Update: A Real-World Study to Evaluate Luspatercept in Adults With Transfusion-Dependent Beta-Thalassemia in the Middle East
A real-world clinical trial is ongoing to evaluate luspatercept in adults with transfusion-dependent beta-thalassemia in the Middle East; th…
-
CLINICAL_TRIALS trial_result · 2026-02-12Clinical Trial Update: A Clinical Study Evaluating the Safety and Efficacy of CS-101 in Treating Subjects With β-thalassemia [EARLY_Phase 1]
An early-phase trial evaluating CS-101 in Beta-thalassemia has completed. The study assessed the safety and preliminary efficacy of CS-101 i…
-
CLINICAL_TRIALS trial_result · 2025-12-17Clinical Trial Update: A Safety and Efficacy Study Evaluating CTX001 in Participants With Transfusion-Dependent β-Thalassemia [Phase 2, Phase 3]
A Phase 2/3 safety and efficacy study of CTX001 in transfusion-dependent beta-thalassemia has completed.
-
CLINICAL_TRIALS trial_result · 2025-11-12Clinical Trial Update: Long - Term Follow Up of Sickle Cell Disease and Beta-thalassemia Subjects Previously Exposed to BIVV003 or ST-400.
Sangamo Therapeutics provides an update on long-term follow-up for sickle cell disease and beta-thalassemia subjects previously treated with…
- Want full summaries, importance scores, and drug timelines? Upgrade to Plus or Pro.
-
CLINICAL_TRIALS trial_result · 2025-08-05Clinical Trial Update: Evaluation the Safety and Efficacy of KL003 Cell Injection in the Treatment of Transfusion-dependent β-thalassemia. [NA]
This update describes ongoing clinical evaluation of KL003 Cell Injection for safety and efficacy in patients with transfusion-dependent bet…
-
CLINICAL_TRIALS trial_result · 2025-08-01Clinical Trial Update: Impact of Virtual Reality on Pain and Anxiety During Intravenous Cannulation Procedures Among Thalassemia Patients in UAE. [NA]
A completed clinical trial evaluated whether virtual reality reduces pain and anxiety during intravenous cannulation in beta-thalassemia pat…
-
CLINICAL_TRIALS trial_result · 2025-05-30Clinical Trial Update: Long Term Beta Thalassemia Treatment: Findings From The Extension Period [Phase 2]
Long-term extension-period findings from a Phase 2 trial evaluating treatment for beta-thalassemia are reported.
-
CLINICAL_TRIALS trial_result · 2025-05-25Clinical Trial Update: A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-TDT) [Phase 3]
Phase 3 trial evaluating the efficacy and safety of mitapivat in transfusion-dependent alpha- and beta-thalassemia; results have been posted…
-
CLINICAL_TRIALS trial_result · 2025-04-02Clinical Trial Update: EDIT-301 for Autologous Hematopoietic Stem Cell Transplant (HSCT) in Participants With Transfusion-Dependent Beta Thalassemia (TDT) [Phase 1, Phase 2]
Editas Medicine reports an update for EDIT-301, an autologous hematopoietic stem cell gene-editing therapy, currently in Phase 1/Phase 2 for…
-
CLINICAL_TRIALS trial_result · 2025-03-30Clinical Trial Update: A Safety and Efficacy Study Evaluating ET-01 in Subjects with Transfusion Dependent Β-Thalassaemia [Phase 1]
A Phase 1 safety and efficacy study evaluating ET-01 for transfusion-dependent beta-thalassemia was terminated.
-
CLINICAL_TRIALS trial_result · 2025-03-30Clinical Trial Update: A Study to Evaluate the Safety and Efficacy of ET-01 Transplantation in Subjects with Transfusion Dependent Β-Thalassaemia. [NA]
A study evaluating ET-01 transplantation for transfusion-dependent beta-thalassemia has been terminated.
-
CLINICAL_TRIALS trial_result · 2025-02-11Clinical Trial Update: A Study of Participants with Β-Thalassemia Treated with Betibeglogene Autotemcel
A clinical trial update indicating recruitment is ongoing for participants with Beta-Thalassemia treated with Betibeglogene Autotemcel.
-
CLINICAL_TRIALS trial_result · 2024-11-29Clinical Trial Update: Nonmyeloablative Peripheral Blood Mobilized Hematopoietic Precursor Cell Transplantation for Sickle Cell Disease and Beta-thalassemia in People With Higher Risk of Transplant Failure [Phase 2]
Phase 2 trial update examining a nonmyeloablative peripheral blood stem cell transplantation approach for sickle cell disease and beta-thala…
-
CLINICAL_TRIALS trial_result · 2024-11-07Clinical Trial Update: A Pilot Study on the Safety and Efficacy of YOLT-204 for Transfusion-Dependent Beta-Thalassemia [EARLY_Phase 1]
A pilot Phase 1 trial evaluating the safety and preliminary efficacy of YOLT-204 in transfusion-dependent beta-thalassemia; the study status…
-
CLINICAL_TRIALS trial_result · 2024-07-19Clinical Trial Update: Vitamin E Supplementation for Children With Transfusion Dependent Beta Thalassemia on Different Iron Chelation Regimen [NA]
This completed clinical trial evaluates Vitamin E supplementation in children with transfusion-dependent beta-thalassemia on different iron …
-
CLINICAL_TRIALS trial_result · 2024-07-18Clinical Trial Update: Study to Evaluate the Safety and Efficacy of Luspatercept (ACE-536) in Participants With Beta-thalassemia (A536-04/MK-6143-002) [Phase 2]
A Phase 2 study evaluating the safety and efficacy of luspatercept for beta-thalassemia has completed and results have been posted.
-
CLINICAL_TRIALS trial_result · 2024-07-12Clinical Trial Update: Study to Evaluate Additional Risk Minimisation Measures (aRMMs) for REBLOZYL Among Healthcare Professionals (HCPs)
A completed clinical trial evaluated additional risk minimisation measures for REBLOZYL among healthcare professionals.
-
CLINICAL_TRIALS trial_result · 2024-06-14Clinical Trial Update: Safety and Efficacy of Gene Modified Autologous Hematopoietic Stem Cells to Treat Transfusion-dependent Beta-thalassemia [NA]
This entry notes a clinical trial evaluating safety and efficacy of gene-modified autologous hematopoietic stem cells in transfusion-depende…
-
CLINICAL_TRIALS trial_result · 2024-06-13Clinical Trial Update: Stem Cell Transplant in Sickle Cell Disease and Thalassemia [Phase 2]
Phase 2 clinical trial update on hematopoietic stem cell transplantation for sickle cell disease and beta-thalassemia; the study is currentl…
-
CLINICAL_TRIALS trial_result · 2024-06-04Clinical Trial Update: Evaluation of Low-cost Techniques for Detecting Sickle Cell Disease and β-thalassemia in Nepal and Canada [NA]
A completed clinical trial evaluated low-cost testing methods for detecting sickle cell disease and beta-thalassemia in Nepal and Canada, wi…
-
CLINICAL_TRIALS trial_result · 2024-03-12Clinical Trial Update: Comparison Of Efficacy And Safety Of Thalidomide Vs Hydroxyurea In Thalassemia Patients: A Single-Centre Pilot Study. [Phase 2]
A Phase 2 pilot trial comparing the efficacy and safety of thalidomide versus hydroxyurea in patients with beta-thalassemia was completed at…
-
CLINICAL_TRIALS trial_result · 2024-01-23Clinical Trial Update: Testing SIROLIMUS in Beta-thalassemia Transfusion Dependent Patients [Phase 2]
Phase 2 trial update for sirolimus in transfusion-dependent beta-thalassemia patients has completed.
-
CLINICAL_TRIALS trial_result · 2023-12-29Clinical Trial Update: A Study Evaluating the Efficacy and Safety of the LentiGlobin® BB305 Drug Product in Participants With Transfusion-Dependent β-Thalassemia [Phase 3]
Phase 3 trial evaluating LentiGlobin BB305 for transfusion-dependent beta-thalassemia has completed with results posted in 2023.
-
CLINICAL_TRIALS trial_result · 2023-12-14Clinical Trial Update: Safety, Tolerability, Pharmacokinetics (PK), Pharmacodynamics (PD) and Preliminary Efficacy of VIT-2763 in β-thalassaemia [Phase 2]
Phase 2 trial updates the safety, tolerability, PK/PD, and preliminary efficacy of VIT-2763 in beta-thalassemia with non-transfusion-depende…