HEMOGLOBINOPATHIES
Clinical trials for HEMOGLOBINOPATHIES explained in plain language.
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CRISPR breakthrough trial aims to cure kids of lifelong blood disease
⭐️ CURE ⭐️ OngoingThis study is testing a one-time gene therapy called CTX001 in children with severe transfusion-dependent beta-thalassemia. Doctors use CRISPR gene-editing technology to modify a patient's own blood stem cells, then infuse them back, aiming to eliminate the need for lifelong bloo…
Matched conditions: HEMOGLOBINOPATHIES
Phase: PHASE3 • Sponsor: Vertex Pharmaceuticals Incorporated • Aim: ⭐️ CURE ⭐️
Last updated Mar 23, 2026 15:18 UTC
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CRISPR gene therapy patients monitored for years in groundbreaking study
Disease control ENROLLING_BY_INVITATIONThis study tracks the long-term safety and effectiveness of a one-time CRISPR gene therapy called CTX001 in people with sickle cell disease or beta-thalassemia. It follows 160 children and adults who have already received the therapy in earlier studies. The main goal is to monito…
Matched conditions: HEMOGLOBINOPATHIES
Phase: PHASE3 • Sponsor: Vertex Pharmaceuticals Incorporated • Aim: Disease control
Last updated Mar 27, 2026 12:41 UTC
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New transplant method aims to cure blood diseases with fewer side effects
Disease control OngoingThis study is testing a modified bone marrow transplant procedure for adults and children with serious blood cancers and disorders. The goal is to treat the underlying disease while reducing the risk of a dangerous complication called graft-versus-host disease. Researchers use a …
Matched conditions: HEMOGLOBINOPATHIES
Phase: NA • Sponsor: Baylor College of Medicine • Aim: Disease control
Last updated Mar 25, 2026 14:09 UTC
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New hope for sickle cell patients without perfect donor matches
Disease control OngoingThis early-stage study is testing a new type of stem cell transplant for adults with severe sickle cell disease. It uses a half-matched family donor and a milder chemotherapy regimen to prepare the body, aiming to reduce transplant risks. The main goals are to see if the procedur…
Matched conditions: HEMOGLOBINOPATHIES
Phase: PHASE1 • Sponsor: City of Hope Medical Center • Aim: Disease control
Last updated Mar 24, 2026 12:02 UTC
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CRISPR breakthrough: One-Shot gene therapy aims to free kids from sickle cell pain crises
Disease control OngoingThis study is testing a one-time treatment called CTX001 in children with severe sickle cell disease who don't respond well to the standard medication, hydroxyurea. The treatment uses the patient's own modified stem cells (a type of gene therapy using CRISPR technology) to try to…
Matched conditions: HEMOGLOBINOPATHIES
Phase: PHASE3 • Sponsor: Vertex Pharmaceuticals Incorporated • Aim: Disease control
Last updated Mar 20, 2026 14:48 UTC