THALASSEMIA
Clinical trials for THALASSEMIA explained in plain language.
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CRISPR breakthrough trial aims to cure kids of lifelong blood disease
⭐️ CURE ⭐️ OngoingThis study is testing a one-time gene therapy called CTX001 in children with severe transfusion-dependent beta-thalassemia. Doctors use CRISPR gene-editing technology to modify a patient's own blood stem cells, then infuse them back, aiming to eliminate the need for lifelong bloo…
Matched conditions: THALASSEMIA
Phase: PHASE3 • Sponsor: Vertex Pharmaceuticals Incorporated • Aim: ⭐️ CURE ⭐️
Last updated Mar 23, 2026 15:18 UTC
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Experimental drug aims to boost blood health in thalassemia patients
Disease control OngoingThis study tested whether the drug AG-348 could safely increase hemoglobin levels in adults with a less severe form of thalassemia who don't regularly need blood transfusions. Twenty participants took the drug twice daily for up to 24 weeks, with the option to continue in a long-…
Matched conditions: THALASSEMIA
Phase: PHASE2 • Sponsor: Agios Pharmaceuticals, Inc. • Aim: Disease control
Last updated Apr 03, 2026 14:43 UTC
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CRISPR gene therapy patients monitored for years in groundbreaking study
Disease control ENROLLING_BY_INVITATIONThis study tracks the long-term safety and effectiveness of a one-time CRISPR gene therapy called CTX001 in people with sickle cell disease or beta-thalassemia. It follows 160 children and adults who have already received the therapy in earlier studies. The main goal is to monito…
Matched conditions: THALASSEMIA
Phase: PHASE3 • Sponsor: Vertex Pharmaceuticals Incorporated • Aim: Disease control
Last updated Mar 27, 2026 12:41 UTC
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Patients continue life-extending blood cancer drugs in safety study
Disease control OngoingThis study allows patients who benefited from previous clinical trials of ruxolitinib (alone or with panobinostat) to continue receiving their treatment while researchers monitor long-term safety. The study includes 279 patients with various blood conditions like myelofibrosis an…
Matched conditions: THALASSEMIA
Phase: PHASE4 • Sponsor: Novartis Pharmaceuticals • Aim: Disease control
Last updated Mar 27, 2026 12:39 UTC
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New hope for sickle cell patients without perfect donor matches
Disease control OngoingThis early-stage study is testing a new type of stem cell transplant for adults with severe sickle cell disease. It uses a half-matched family donor and a milder chemotherapy regimen to prepare the body, aiming to reduce transplant risks. The main goals are to see if the procedur…
Matched conditions: THALASSEMIA
Phase: PHASE1 • Sponsor: City of Hope Medical Center • Aim: Disease control
Last updated Mar 24, 2026 12:02 UTC