HEMOGLOBINOPATHIES
Clinical trials for HEMOGLOBINOPATHIES explained in plain language.
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One-Shot CRISPR cure trial for devastating blood diseases
⭐️ CURE ⭐️ Recruiting nowThis study is testing a single treatment using the patient's own stem cells that have been genetically modified with CRISPR technology. The goal is to see if this one-time therapy can eliminate the need for lifelong blood transfusions in beta-thalassemia patients and stop painful…
Matched conditions: HEMOGLOBINOPATHIES
Phase: PHASE3 • Sponsor: Vertex Pharmaceuticals Incorporated • Aim: ⭐️ CURE ⭐️
Last updated Mar 25, 2026 14:08 UTC
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Gentler transplant offers hope for kids with rare blood diseases
Disease control Recruiting nowThis study is testing a less intense stem cell transplant procedure for children and young adults with inherited disorders like sickle cell disease and metabolic conditions. The goal is to replace a patient's faulty blood-forming system with a healthy donor's cells while reducing…
Matched conditions: HEMOGLOBINOPATHIES
Phase: PHASE1, PHASE2 • Sponsor: Washington University School of Medicine • Aim: Disease control
Last updated Apr 01, 2026 21:26 UTC
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Lifesaving cord blood transplants tested for kids without matched donors
Disease control Recruiting nowThis study is testing the safety of using cord blood for transplants in children and young adults (up to age 21) with life-threatening blood cancers or genetic blood disorders who do not have a perfectly matched donor. The main goal is to measure how many patients survive the fir…
Matched conditions: HEMOGLOBINOPATHIES
Phase: PHASE2 • Sponsor: Memorial Sloan Kettering Cancer Center • Aim: Disease control
Last updated Apr 01, 2026 21:26 UTC
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New transplant trial aims to free patients from lifelong blood transfusions
Disease control Recruiting nowThis study is testing a donor stem cell transplant to treat people with severe inherited blood disorders like sickle cell disease and thalassemia, who currently depend on regular blood transfusions. The goal is to replace the patient's faulty blood-making system with a healthy do…
Matched conditions: HEMOGLOBINOPATHIES
Phase: PHASE2 • Sponsor: Masonic Cancer Center, University of Minnesota • Aim: Disease control
Last updated Mar 27, 2026 12:41 UTC
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New hope for kids: gentler transplant uses family donors to fight devastating blood diseases
Disease control Recruiting nowThis trial is testing a new, less intense bone marrow transplant procedure for children and young adults with severe, non-cancerous blood disorders like sickle cell disease. The goal is to see if using a partially matched donor from the patient's own family is safe and can succes…
Matched conditions: HEMOGLOBINOPATHIES
Phase: PHASE1, PHASE2 • Sponsor: Washington University School of Medicine • Aim: Disease control
Last updated Mar 16, 2026 15:24 UTC
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Scientists hunt for cures in discarded bones
Knowledge-focused Recruiting nowThis study aims to collect bone marrow samples that are normally thrown away during orthopedic surgery. Researchers will use these samples to learn how to better use gene therapy and gene editing techniques to treat serious inherited blood disorders like sickle cell disease and t…
Matched conditions: HEMOGLOBINOPATHIES
Sponsor: St. Jude Children's Research Hospital • Aim: Knowledge-focused
Last updated Mar 16, 2026 15:25 UTC