HEMOGLOBINOPATHIES
Clinical trials for HEMOGLOBINOPATHIES explained in plain language.
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CRISPR gene therapy offers hope for kids with sickle cell disease
Disease control OngoingThis study tests a single dose of a gene-edited stem cell treatment (CTX001) in 13 children with severe sickle cell disease who cannot take or did not benefit from hydroxyurea. The goal is to see if the treatment can prevent severe pain crises and hospital stays for at least a ye…
Matched conditions: HEMOGLOBINOPATHIES
Phase: PHASE3 • Sponsor: Vertex Pharmaceuticals Incorporated • Aim: Disease control
Last updated May 17, 2026 04:26 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: HEMOGLOBINOPATHIES
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: HEMOGLOBINOPATHIES
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: HEMOGLOBINOPATHIES
Phase: PHASE3 • Sponsor: Vertex Pharmaceuticals Incorporated • Aim: Disease control
Last updated May 13, 2026 16:00 UTC
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New transplant technique aims to cut deadly side effects in half-matched donor patients
Disease control OngoingThis study tests a new way to do stem cell transplants using cells from a half-matched family donor. By removing certain immune cells before transplant, researchers hope to lower the risk of graft-versus-host disease, a serious complication. The goal is to help patients with bloo…
Matched conditions: HEMOGLOBINOPATHIES
Phase: NA • Sponsor: Baylor College of Medicine • Aim: Disease control
Last updated May 11, 2026 20:49 UTC