Β -THALASSEMIA
Clinical trials for Β -THALASSEMIA explained in plain language.
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New pill shows promise for thalassemia patients in early trial
Disease control OngoingThis study tested a drug called AG-348 in 20 adults with a type of thalassemia that doesn't require regular blood transfusions. The goal was to see if the drug could safely raise hemoglobin levels, which carry oxygen in the blood. Participants took the pill twice daily for up to …
Matched conditions: Β -THALASSEMIA
Phase: PHASE2 • Sponsor: Agios Pharmaceuticals, Inc. • Aim: Disease control
Last updated May 26, 2026 05:31 UTC
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CRISPR gene therapy: a lifelong watch begins for blood disease patients
Disease control ENROLLING_BY_INVITATIONThis study follows 160 children and adults with beta-thalassemia or sickle cell disease who already received an experimental CRISPR gene therapy called CTX001. Researchers will track participants for years to check for long-term side effects, new cancers, and how well the treatme…
Matched conditions: Β -THALASSEMIA
Phase: PHASE3 • Sponsor: Vertex Pharmaceuticals Incorporated • Aim: Disease control
Last updated May 26, 2026 04:37 UTC
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New Half-Matched transplant offers hope for sickle cell patients
Disease control OngoingThis study tests a new type of stem cell transplant for people with severe sickle cell disease who don't have a fully matched donor. It uses a half-matched relative as a donor and a milder chemotherapy to prepare the body, along with removing certain immune cells from the donated…
Matched conditions: Β -THALASSEMIA
Phase: PHASE1 • Sponsor: City of Hope Medical Center • Aim: Disease control
Last updated May 26, 2026 04:35 UTC
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CRISPR gene therapy may free kids with thalassemia from lifelong transfusions
Disease control OngoingThis study tests a single dose of CTX001, a gene therapy made from the patient's own blood stem cells edited with CRISPR, in 16 children with transfusion-dependent beta-thalassemia. The goal is to see if it can help them produce enough healthy red blood cells to stop needing regu…
Matched conditions: Β -THALASSEMIA
Phase: PHASE3 • Sponsor: Vertex Pharmaceuticals Incorporated • Aim: Disease control
Last updated May 22, 2026 14:01 UTC
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Blood disorder drug safety tracked in long-term follow-up study
Disease control OngoingThis study checks the long-term safety of the drug ruxolitinib (given alone or with panobinostat) in people with certain blood or immune conditions like myelofibrosis, polycythemia vera, graft-versus-host disease, acute myeloid leukemia, or thalassemia. Participants had already b…
Matched conditions: Β -THALASSEMIA
Phase: PHASE4 • Sponsor: Novartis Pharmaceuticals • Aim: Disease control
Last updated May 19, 2026 12:01 UTC