THALASSEMIA
Clinical trials for THALASSEMIA explained in plain language.
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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 64 adults with β-thalassemia, a blood disorder that often requires lifelong transfusions. Participants receive either AND017 or a placebo alongside standard care. The goal is to see if the drug safely raises hemoglobin levels and red…
Matched conditions: THALASSEMIA
Phase: PHASE2 • Sponsor: Kind Pharmaceuticals LLC • Aim: Disease control
Last updated May 16, 2026 23:37 UTC
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CRISPR gene editing offers hope for blood disorder patients
Disease control Recruiting nowThis study tests a single dose of a CRISPR-modified stem cell therapy (CTX001) in 26 people with transfusion-dependent β-thalassemia or severe sickle cell disease. The treatment uses the patient's own blood stem cells, edited to produce fetal hemoglobin, aiming to reduce or elimi…
Matched conditions: THALASSEMIA
Phase: PHASE3 • Sponsor: Vertex Pharmaceuticals Incorporated • Aim: Disease control
Last updated May 16, 2026 23:36 UTC
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New stem cell approach aims to reduce transplant risks for blood disorders
Disease control Recruiting nowThis study tests a new way to prepare people with sickle cell disease or beta-thalassemia for a stem cell transplant from a half-matched donor. Before the transplant, participants receive drugs to lower their immune system, which may help prevent serious side effects like graft f…
Matched conditions: THALASSEMIA
Phase: PHASE2 • Sponsor: Memorial Sloan Kettering Cancer Center • Aim: Disease control
Last updated May 16, 2026 23:32 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: THALASSEMIA
Phase: PHASE3 • Sponsor: Novo Nordisk A/S • Aim: Disease control
Last updated May 16, 2026 23:31 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: THALASSEMIA
Sponsor: Bristol-Myers Squibb • Aim: Disease control
Last updated May 08, 2026 12:01 UTC
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Heart rhythm secrets in thalassemia patients under the microscope
Knowledge-focused Recruiting nowThis study looks at 350 people with beta-thalassemia to find out what makes some develop atrial fibrillation (an irregular heartbeat) while others do not. Researchers will compare medical tests and lab results between the two groups. The goal is to better understand the condition…
Matched conditions: THALASSEMIA
Sponsor: University Hospital of Ferrara • Aim: Knowledge-focused
Last updated May 16, 2026 23:35 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 tho…
Matched conditions: THALASSEMIA
Phase: EARLY_PHASE1 • Sponsor: National Heart, Lung, and Blood Institute (NHLBI) • Aim: Knowledge-focused
Last updated May 14, 2026 12:04 UTC
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Massive new study tracks Real-World safety of blood disorder treatments
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 and what side effects occur over time. Researchers will collect safety data, including allergic reactions, infections, and o…
Matched conditions: THALASSEMIA
Sponsor: American Thrombosis and Hemostasis Network • Aim: Knowledge-focused
Last updated May 06, 2026 16:01 UTC