THALASSEMIA
Clinical trials for THALASSEMIA explained in plain language.
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CRISPR gene therapy aims to free kids from lifelong blood transfusions
Disease control OngoingThis phase 3 trial tests a single dose of CTX001, a CRISPR gene therapy made from the child's own blood stem cells, for children with transfusion-dependent beta-thalassemia. The goal is to help them become transfusion-free for at least 12 months. Sixteen children will receive the…
Matched conditions: THALASSEMIA
Phase: PHASE3 • Sponsor: Vertex Pharmaceuticals Incorporated • Aim: Disease control
Last updated Jul 02, 2026 02:00 UTC
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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 a day for up to …
Matched conditions: THALASSEMIA
Phase: PHASE2 • Sponsor: Agios Pharmaceuticals, Inc. • Aim: Disease control
Last updated Jun 27, 2026 12:03 UTC
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CRISPR gene therapy: a Long-Term safety check for blood disease patients
Disease control ENROLLING_BY_INVITATIONThis study follows 160 children and adults with beta-thalassemia or sickle cell disease who received a one-time treatment of their own CRISPR-edited stem cells (CTX001). The goal is to monitor long-term safety, including any new cancers or blood disorders, and to see how well the…
Matched conditions: THALASSEMIA
Phase: PHASE3 • Sponsor: Vertex Pharmaceuticals Incorporated • Aim: Disease control
Last updated Jun 27, 2026 12:00 UTC
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New blood treatment could block zika in transfusions
Disease control OngoingThis study tests a special treatment for red blood cells that aims to kill the Zika virus, making transfusions safer. About 692 people who need blood transfusions will receive either treated or standard blood. The goal is to see if the treated blood works just as well and is safe…
Matched conditions: THALASSEMIA
Phase: PHASE3 • Sponsor: Cerus Corporation • Aim: Disease control
Last updated Jun 27, 2026 11:03 UTC
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New transplant approach offers hope for sickle cell patients without a perfect donor match
Disease control OngoingThis early-phase study tests a new type of stem cell transplant for people with severe sickle cell disease who don't have a fully matched sibling donor. It uses a half-matched family donor and a milder chemotherapy regimen to prepare the body, along with removing certain immune c…
Matched conditions: THALASSEMIA
Phase: PHASE1 • Sponsor: City of Hope Medical Center • Aim: Disease control
Last updated Jun 27, 2026 09:10 UTC