THALASSEMIA
Clinical trials for THALASSEMIA explained in plain language.
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New pill shows promise for thalassemia patients in early trial
Disease control OngoingThis study tested an experimental drug called AG-348 in 20 adults with a form of thalassemia that does not 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 d…
Matched conditions: THALASSEMIA
Phase: PHASE2 • Sponsor: Agios Pharmaceuticals, Inc. • Aim: Disease control
Last updated May 17, 2026 00:28 UTC
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Blood disorder patients continue promising drug combo in extended safety trial
Disease control OngoingThis study looks at the long-term safety of the drugs ruxolitinib (alone or with panobinostat) in people with certain blood diseases like myelofibrosis, polycythemia vera, graft-versus-host disease, acute myeloid leukemia, or thalassemia. Participants had already been taking thes…
Matched conditions: THALASSEMIA
Phase: PHASE4 • Sponsor: Novartis Pharmaceuticals • Aim: Disease control
Last updated May 15, 2026 11:57 UTC
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New Half-Matched transplant could ease sickle cell suffering
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 family donor and a milder chemotherapy to prepare the body, along with removing certain immune cells from the donor cells to…
Matched conditions: THALASSEMIA
Phase: PHASE1 • Sponsor: City of Hope Medical Center • Aim: Disease control
Last updated May 15, 2026 11:53 UTC
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CRISPR treatment frees kids from lifelong blood transfusions
Disease control OngoingThis study tests a one-time gene-editing treatment (CTX001) in 16 children with transfusion-dependent beta-thalassemia, a severe blood disorder. The therapy uses the child's own stem cells, modified with CRISPR-Cas9, to produce healthy red blood cells. The main goal is to see if …
Matched conditions: THALASSEMIA
Phase: PHASE3 • Sponsor: Vertex Pharmaceuticals Incorporated • Aim: Disease control
Last updated May 14, 2026 12:04 UTC
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CRISPR gene therapy: a Long-Term look at safety 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). Researchers will monitor participants for years to check for side effects, new cancers, and how well the thera…
Matched conditions: THALASSEMIA
Phase: PHASE3 • Sponsor: Vertex Pharmaceuticals Incorporated • Aim: Disease control
Last updated May 13, 2026 16:00 UTC