TRANSFUSION-DEPENDENT BETA-THALASSEMIA
Clinical trials for TRANSFUSION-DEPENDENT BETA-THALASSEMIA explained in plain language.
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New pill may cut transfusions for thalassemia patients
Disease control OngoingThis phase 3 study tests whether mitapivat, an oral medication, can safely reduce the number of blood transfusions needed by adults with transfusion-dependent alpha- or beta-thalassemia. Participants receive either mitapivat or a placebo for 48 weeks. The main goal is to see if m…
Matched conditions: TRANSFUSION-DEPENDENT BETA-THALASSEMIA
Phase: PHASE3 • Sponsor: Agios Pharmaceuticals, Inc. • Aim: Disease control
Last updated Jun 27, 2026 14:03 UTC
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Gene therapy could free Beta-Thalassemia patients from lifelong transfusions
Disease control OngoingThis early-stage study tests a gene therapy for people with severe beta-thalassemia who need regular blood transfusions. The treatment modifies the patient's own blood stem cells to produce healthy hemoglobin, aiming to reduce or eliminate the need for transfusions. Six participa…
Matched conditions: TRANSFUSION-DEPENDENT BETA-THALASSEMIA
Phase: PHASE1, PHASE2 • Sponsor: Lantu Biopharma • Aim: Disease control
Last updated Jun 27, 2026 14:00 UTC
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New blood storage method could mean fewer transfusions for thalassemia patients
Disease control ENROLLING_BY_INVITATIONThis study looks at whether storing red blood cells in a low-oxygen environment (called hypoxic storage) can improve transfusion care for adults with transfusion-dependent beta-thalassemia. Participants already receiving these specially stored blood cells as part of their routine…
Matched conditions: TRANSFUSION-DEPENDENT BETA-THALASSEMIA
Sponsor: University Research Institute for the Study of Genetic & Malignant Disorders in Childhood • Aim: Disease control
Last updated Jun 27, 2026 08:04 UTC
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Gene therapy patients monitored for years to ensure lasting safety
Disease control OngoingThis study follows 8 people who received a one-time gene therapy (BIVV003 for sickle cell disease or ST-400 for beta-thalassemia) to see if it remains safe and effective over many years. Researchers will track serious side effects, survival, and blood markers like hemoglobin leve…
Matched conditions: TRANSFUSION-DEPENDENT BETA-THALASSEMIA
Sponsor: Sangamo Therapeutics • Aim: Disease control
Last updated Jun 27, 2026 07:57 UTC