Β -THALASSEMIA
Clinical trials for Β -THALASSEMIA explained in plain language.
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CRISPR gene therapy aims to free patients from lifelong transfusions
Disease control Recruiting nowThis study tests a single dose of a CRISPR-based gene therapy (CTX001) in 26 adults with transfusion-dependent beta-thalassemia or severe sickle cell disease. The treatment uses the patient's own blood stem cells, which are edited to produce more fetal hemoglobin, potentially red…
Matched conditions: Β -THALASSEMIA
Phase: PHASE3 • Sponsor: Vertex Pharmaceuticals Incorporated • Aim: Disease control
Last updated May 26, 2026 03:44 UTC
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New pill could reduce blood transfusions for thalassemia patients
Disease control Recruiting nowThis study tests an oral drug called AND017 in people with β-thalassemia, a blood disorder that often requires regular blood transfusions. The trial includes 64 adults who either need transfusions or do not. Participants receive AND017 or a placebo along with standard care. The g…
Matched conditions: Β -THALASSEMIA
Phase: PHASE2 • Sponsor: Kind Pharmaceuticals LLC • Aim: Disease control
Last updated May 26, 2026 03:38 UTC
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New Immune-Suppression strategy may make stem cell transplants safer for blood disorders
Disease control Recruiting nowThis study tests whether giving immune-suppressing drugs before a stem cell transplant can reduce serious side effects like graft failure and graft-versus-host disease. It includes adults and children aged 2 to 50 with sickle cell disease or beta-thalassemia who have a half-match…
Matched conditions: Β -THALASSEMIA
Phase: PHASE2 • Sponsor: Memorial Sloan Kettering Cancer Center • Aim: Disease control
Last updated May 20, 2026 11:57 UTC
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New pill shows promise for blood disorders in Long-Term trial
Disease control Recruiting nowThis study looks at the long-term safety and effectiveness of etavopivat, an experimental pill for sickle cell disease and thalassemia. It includes 480 adults, teens, and children who have already benefited from etavopivat in a previous study. Participants will take the drug for …
Matched conditions: Β -THALASSEMIA
Phase: PHASE3 • Sponsor: Novo Nordisk A/S • Aim: Disease control
Last updated May 18, 2026 12:07 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. About 200 people in …
Matched conditions: Β -THALASSEMIA
Sponsor: Bristol-Myers Squibb • Aim: Disease control
Last updated May 08, 2026 12:01 UTC
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Heart rhythm study aims to uncover clues in thalassemia patients
Knowledge-focused Recruiting nowThis study is for people with beta-thalassemia who need regular blood transfusions. Researchers want to compare those who have atrial fibrillation (a type of irregular heartbeat) with those who don't, to find differences in health tests and lab results. The goal is to learn more …
Matched conditions: Β -THALASSEMIA
Sponsor: University Hospital of Ferrara • Aim: Knowledge-focused
Last updated May 26, 2026 03:37 UTC
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Massive study tracks Real-World safety of blood disorder therapies
Knowledge-focused Recruiting nowThis study follows 3,000 people with non-cancer blood disorders (like hemophilia, sickle cell disease, and clotting issues) to see how well treatments work over time and what side effects may occur. Researchers collect safety data on approved therapies to understand long-term ris…
Matched conditions: Β -THALASSEMIA
Sponsor: American Thrombosis and Hemostasis Network • Aim: Knowledge-focused
Last updated May 26, 2026 03:35 UTC
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How long do red blood cells live? new study aims to find out
Knowledge-focused Recruiting nowThis study measures how long red blood cells survive in people with sickle cell disease and other inherited blood disorders. Researchers will label participants' own red blood cells with biotin (a vitamin) and track them over time. The goal is to compare cell survival between peo…
Matched conditions: Β -THALASSEMIA
Phase: EARLY_PHASE1 • Sponsor: National Heart, Lung, and Blood Institute (NHLBI) • Aim: Knowledge-focused
Last updated May 26, 2026 03:34 UTC