HEMOGLOBINOPATHIES
Clinical trials for HEMOGLOBINOPATHIES explained in plain language.
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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: HEMOGLOBINOPATHIES
Phase: PHASE1 • Sponsor: City of Hope Medical Center • Aim: Disease control
Last updated May 26, 2026 05:35 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: HEMOGLOBINOPATHIES
Phase: PHASE3 • Sponsor: Vertex Pharmaceuticals Incorporated • Aim: Disease control
Last updated May 26, 2026 04:37 UTC
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New transplant technique aims to cut deadly immune reaction in half-matched donors
Disease control OngoingThis study tests a stem cell transplant method that filters out certain immune cells (TCRαβ+ and CD19+) from a half-matched family donor's blood. The goal is to lower the risk of graft-versus-host disease, a serious complication, and allow patients to avoid long-term immune-suppr…
Matched conditions: HEMOGLOBINOPATHIES
Phase: NA • Sponsor: Baylor College of Medicine • Aim: Disease control
Last updated May 22, 2026 14:02 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: HEMOGLOBINOPATHIES
Phase: PHASE3 • Sponsor: Vertex Pharmaceuticals Incorporated • Aim: Disease control
Last updated May 22, 2026 14:01 UTC
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CRISPR hope for kids with sickle cell: One-Time treatment may stop pain crises
Disease control OngoingThis study tests a one-time gene-editing treatment (CTX001) in children with severe sickle cell disease who cannot take hydroxyurea. The treatment uses the child's own blood stem cells, modified with CRISPR-Cas9, to produce healthy red blood cells. The goal is to prevent severe p…
Matched conditions: HEMOGLOBINOPATHIES
Phase: PHASE3 • Sponsor: Vertex Pharmaceuticals Incorporated • Aim: Disease control
Last updated May 22, 2026 14:00 UTC