TRANSFUSION-DEPENDENT BETA-THALASSEMIA
Clinical trials for TRANSFUSION-DEPENDENT BETA-THALASSEMIA explained in plain language.
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New pill may cut blood transfusions for kids with thalassemia
Disease control Recruiting nowThis study tests a drug called mitapivat in children aged 1 to 17 with alpha- or beta-thalassemia who need regular blood transfusions. The goal is to see if mitapivat can reduce the number of transfusions needed compared to a placebo. The study involves 54 participants and lasts …
Matched conditions: TRANSFUSION-DEPENDENT BETA-THALASSEMIA
Phase: PHASE3 • Sponsor: Agios Pharmaceuticals, Inc. • Aim: Disease control
Last updated Aug 05, 2026 00:00 UTC
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Gene therapy aims to free Beta-Thalassemia patients from lifelong transfusions
Disease control Recruiting nowThis study tests a single-dose gene therapy for people with transfusion-dependent beta-thalassemia, a blood disorder that requires regular red blood cell transfusions. The therapy uses a patient's own stem cells, modified with a virus to produce healthy hemoglobin, and then infus…
Matched conditions: TRANSFUSION-DEPENDENT BETA-THALASSEMIA
Phase: PHASE2 • Sponsor: Fondazione Telethon • Aim: Disease control
Last updated Jul 29, 2026 00:00 UTC
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New stem cell transplant trial aims to treat sickle cell disease and other blood disorders
Disease control Recruiting nowThis phase 2 trial tests a stem cell transplant from a donor for people with high-risk sickle cell disease, thalassemia, and other red blood cell disorders. Participants receive drugs and low-dose radiation before the transplant to help the new cells take hold. The study will tra…
Matched conditions: TRANSFUSION-DEPENDENT BETA-THALASSEMIA
Phase: PHASE2 • Sponsor: Masonic Cancer Center, University of Minnesota • Aim: Disease control
Last updated Jun 27, 2026 13:02 UTC
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New hope for thalassemia patients: drug may cut blood transfusions
Disease control Recruiting nowThis study is testing a drug called luspatercept in adults with a severe blood disorder called beta-thalassemia who need regular blood transfusions. The goal is to see if the drug can reduce how many transfusions they need and improve their hemoglobin levels. Researchers will fol…
Matched conditions: TRANSFUSION-DEPENDENT BETA-THALASSEMIA
Sponsor: Bristol-Myers Squibb • Aim: Disease control
Last updated Jun 27, 2026 13:00 UTC
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Could a One-Time gene fix free thalassemia patients from lifelong transfusions?
Disease control Recruiting nowThis study tests a new gene therapy for adults with transfusion-dependent beta-thalassemia, a severe blood disorder. Doctors take the patient's own blood stem cells, add a healthy gene to make normal hemoglobin, and return the cells. The goal is to reduce or stop the need for reg…
Matched conditions: TRANSFUSION-DEPENDENT BETA-THALASSEMIA
Phase: PHASE1, PHASE2 • Sponsor: Children's Hospital of Philadelphia • Aim: Disease control
Last updated Jun 27, 2026 12:08 UTC