BETA-THALASSEMIA
Clinical trials for BETA-THALASSEMIA explained in plain language.
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Gene editing could cure sickle cell and thalassemia – new trial launches
⭐️ CURE ⭐️ Recruiting nowThis study tests a gene therapy that edits a patient's own blood stem cells to produce more fetal hemoglobin, a healthy type that can replace the defective hemoglobin causing sickle cell disease or beta-thalassemia. Up to 10 people aged 13-40 with severe forms of these blood diso…
Matched conditions: BETA-THALASSEMIA
Phase: PHASE1 • Sponsor: Daniel Bauer • Aim: ⭐️ CURE ⭐️
Last updated May 26, 2026 06:19 UTC
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Gene therapy for Beta-Thalassemia: will the fix last?
Disease control Recruiting nowThis study checks the long-term safety and effectiveness of a gene therapy called CS-101 in 5 people with beta-thalassemia who already received the treatment. Researchers will watch for serious side effects, new cancers, and whether patients can stop needing blood transfusions fo…
Matched conditions: BETA-THALASSEMIA
Phase: NA • Sponsor: Children's Hospital of Fudan University • Aim: Disease control
Last updated May 26, 2026 06:27 UTC
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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: BETA-THALASSEMIA
Phase: PHASE3 • Sponsor: Vertex Pharmaceuticals Incorporated • Aim: Disease control
Last updated May 26, 2026 06:27 UTC
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New stem cell transplant aims to boost donor cells in blood disorders
Disease control Recruiting nowThis study tests a partial stem cell transplant for people with severe sickle cell disease or beta-thalassemia who need regular blood transfusions. The goal is to get more donor cells to stay in the body long-term, which could reduce disease complications. About 90 participants w…
Matched conditions: BETA-THALASSEMIA
Phase: PHASE1, PHASE2 • Sponsor: National Heart, Lung, and Blood Institute (NHLBI) • Aim: Disease control
Last updated May 26, 2026 06:25 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 people with a severe blood disorder called beta-thalassemia, who currently need regular blood transfusions. The therapy involves taking the patient's own blood stem cells, adding a healthy gene to help produce normal red blood cells, and re…
Matched conditions: BETA-THALASSEMIA
Phase: PHASE1, PHASE2 • Sponsor: Children's Hospital of Philadelphia • Aim: Disease control
Last updated May 26, 2026 06:25 UTC
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Gene therapy may free Beta-Thalassemia patients from lifelong transfusions
Disease control Recruiting nowThis early-stage study tests a one-time gene therapy for people with a severe form of beta-thalassemia that requires regular blood transfusions. The treatment uses the patient's own blood stem cells, which are modified in a lab to produce healthy hemoglobin. The goal is to see if…
Matched conditions: BETA-THALASSEMIA
Phase: PHASE1, PHASE2 • Sponsor: Lantu Biopharma • Aim: Disease control
Last updated May 26, 2026 06:23 UTC
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Blood disorder drug luspatercept under Long-Term safety review
Disease control Recruiting nowThis study looks at the long-term safety of luspatercept in people with certain blood disorders (MDS, beta-thalassemia, or myelofibrosis) who were already in a luspatercept trial. Participants either continue treatment or enter long-term follow-up to monitor side effects, disease…
Matched conditions: BETA-THALASSEMIA
Phase: PHASE3 • Sponsor: Celgene • Aim: Disease control
Last updated May 26, 2026 04:37 UTC
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New hope for kids with blood disorder: drug may cut transfusions
Disease control Recruiting nowThis study tests a drug called luspatercept in children aged 6 to 18 with beta-thalassemia, a blood disorder that often requires regular blood transfusions. The goal is to find a safe dose that can reduce the need for transfusions or raise hemoglobin levels. Participants will be …
Matched conditions: BETA-THALASSEMIA
Phase: PHASE2 • Sponsor: Celgene • Aim: Disease control
Last updated May 22, 2026 13:51 UTC