Β-THALASSEMIA
Clinical trials for Β-THALASSEMIA explained in plain language.
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Half-Match transplant offers potential cure for devasting blood disorders
⭐️ CURE ⭐️ Recruiting nowThis study is testing a modified stem cell transplant procedure for children and adults with severe sickle cell disease or beta-thalassemia. The goal is to cure these genetic blood disorders by replacing a patient's faulty blood-making cells with healthy ones from a half-matched …
Matched conditions: Β-THALASSEMIA
Phase: PHASE2 • Sponsor: Memorial Sloan Kettering Cancer Center • Aim: ⭐️ CURE ⭐️
Last updated Mar 30, 2026 14:29 UTC
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One-Shot CRISPR cure trial for devastating blood diseases
⭐️ CURE ⭐️ Recruiting nowThis study is testing a single treatment using the patient's own stem cells that have been genetically modified with CRISPR technology. The goal is to see if this one-time therapy can eliminate the need for lifelong blood transfusions in beta-thalassemia patients and stop painful…
Matched conditions: Β-THALASSEMIA
Phase: PHASE3 • Sponsor: Vertex Pharmaceuticals Incorporated • Aim: ⭐️ CURE ⭐️
Last updated Mar 25, 2026 14:08 UTC
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Study tracks new Drug's power to free patients from frequent blood transfusions
Disease control Recruiting nowThis study is observing how a drug called luspatercept works for adults with severe beta-thalassemia in the Middle East. The goal is to see if the drug can help patients need fewer blood transfusions and improve their hemoglobin levels. Researchers will follow 200 patients who ar…
Matched conditions: Β-THALASSEMIA
Sponsor: Bristol-Myers Squibb • Aim: Disease control
Last updated Apr 01, 2026 14:42 UTC
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New pill could reduce blood transfusions for thalassemia patients
Disease control Recruiting nowThis study is testing whether an oral medication called AND017 can help people with β-thalassemia. Researchers want to see if it safely reduces the need for blood transfusions in those who require them regularly, and increases hemoglobin levels in those who don't. The trial invol…
Matched conditions: Β-THALASSEMIA
Phase: PHASE2 • Sponsor: Kind Pharmaceuticals LLC • Aim: Disease control
Last updated Mar 24, 2026 12:03 UTC
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Five-Year Follow-Up for promising blood disorder drug
Disease control Recruiting nowThis study aims to understand the long-term safety and effectiveness of etavopivat, an investigational drug for sickle cell disease and thalassemia. It will follow about 480 adults, adolescents, and children who have already taken etavopivat in a previous study and benefited from…
Matched conditions: Β-THALASSEMIA
Phase: PHASE3 • Sponsor: Novo Nordisk A/S • Aim: Disease control
Last updated Mar 10, 2026 12:53 UTC
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Heart study seeks clues to thalassemia stroke risk
Knowledge-focused Recruiting nowThis study aims to understand why some people with beta-thalassemia develop atrial fibrillation (an irregular heart rhythm) while others don't. Researchers will compare 350 thalassemia patients with and without this heart condition to identify differences in their health data. Th…
Matched conditions: Β-THALASSEMIA
Sponsor: University Hospital of Ferrara • Aim: Knowledge-focused
Last updated Mar 27, 2026 12:38 UTC
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Massive 15-Year study launches to track safety of new blood disorder drugs
Knowledge-focused Recruiting nowThis study aims to collect long-term safety and effectiveness data on treatments for various non-cancerous blood disorders, including hemophilia, von Willebrand disease, and platelet disorders. It will follow up to 3,000 participants for at least 15 years to see how newer, FDA-ap…
Matched conditions: Β-THALASSEMIA
Sponsor: American Thrombosis and Hemostasis Network • Aim: Knowledge-focused
Last updated Mar 27, 2026 12:38 UTC
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Scientists track blood cell 'Expiration Dates' to unlock clues for sickle cell cure
Knowledge-focused Recruiting nowThis study aims to understand how long red blood cells survive in people with sickle cell disease, thalassemia, and other inherited blood disorders. Researchers will label a small sample of a participant's own blood cells with a safe vitamin (biotin) and track them over several m…
Matched conditions: Β-THALASSEMIA
Phase: EARLY_PHASE1 • Sponsor: National Heart, Lung, and Blood Institute (NHLBI) • Aim: Knowledge-focused
Last updated Mar 23, 2026 15:19 UTC