HEMOGLOBINOPATHIES
Clinical trials for HEMOGLOBINOPATHIES explained in plain language.
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CRISPR gene therapy offers hope for blood disorder patients
Disease control Recruiting nowThis study tests a single treatment using CRISPR to modify a person's own blood stem cells, aiming to help them produce healthy red blood cells and reduce or eliminate the need for blood transfusions. It is for adults with transfusion-dependent beta-thalassemia or severe sickle c…
Matched conditions: HEMOGLOBINOPATHIES
Phase: PHASE3 • Sponsor: Vertex Pharmaceuticals Incorporated • Aim: Disease control
Last updated May 01, 2026 15:57 UTC
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Cord blood transplants offer hope for kids without a donor match
Disease control Recruiting nowThis study tests whether a cord blood transplant can help children and young adults (up to age 21) with life-threatening blood cancers or non-cancer blood disorders who do not have a matched family donor. The main goal is to see how many patients are alive without treatment-relat…
Matched conditions: HEMOGLOBINOPATHIES
Phase: PHASE2 • Sponsor: Memorial Sloan Kettering Cancer Center • Aim: Disease control
Last updated Apr 30, 2026 15:52 UTC
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Milder stem cell transplant offers hope for Non-Cancer blood disorders
Disease control Recruiting nowThis study tests a less intense stem cell transplant for people with non-cancerous blood, immune, or metabolic disorders. The goal is to see if a milder preparation helps donor cells take hold safely. About 220 participants will be enrolled to monitor engraftment and side effects…
Matched conditions: HEMOGLOBINOPATHIES
Phase: PHASE1, PHASE2 • Sponsor: Washington University School of Medicine • Aim: Disease control
Last updated Apr 30, 2026 15:50 UTC