Hemoglobinopathies
Clinical trials for Hemoglobinopathies explained in plain language.
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CRISPR gene therapy: a Long-Term safety check for blood disease patients
Disease control By invitation onlyThis 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: HEMOGLOBINOPATHIES
Phase 3 • Sponsor: Vertex Pharmaceuticals Incorporated • Aim: Disease control
Last updated Aug 13, 2026 00:00 UTC
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CRISPR stem cell therapy shows promise for kids with sickle cell
Disease control OngoingThis phase 3 trial tests a single dose of CTX001, a CRISPR-edited stem cell therapy, in 13 children with severe sickle cell disease who have not responded to standard treatments. The goal is to see if it can prevent severe pain crises for at least a year. Participants receive the…
Matched conditions: HEMOGLOBINOPATHIES
Phase 3 • Sponsor: Vertex Pharmaceuticals Incorporated • Aim: Disease control
Last updated Jul 03, 2026 00:00 UTC
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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: HEMOGLOBINOPATHIES
Phase 3 • Sponsor: Vertex Pharmaceuticals Incorporated • Aim: Disease control
Last updated Jul 03, 2026 00:00 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: HEMOGLOBINOPATHIES
Phase 1 • Sponsor: City of Hope Medical Center • Aim: Disease control
Last updated Jun 27, 2026 09:10 UTC
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New transplant method aims to cut dangerous immune reaction in half-matched donors
Disease control OngoingThis study tests a stem cell transplant method that removes certain immune cells from the donor's blood to lower the chance of graft-versus-host disease (GvHD), a serious complication. It involves 3 patients with blood cancers or non-cancerous blood disorders who lack a perfectly…
Matched conditions: HEMOGLOBINOPATHIES
Sponsor: Baylor College of Medicine • Aim: Disease control
Last updated Jun 27, 2026 07:56 UTC