HEMOGLOBINOPATHIES
Clinical trials for HEMOGLOBINOPATHIES explained in plain language.
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New bone marrow transplant trial offers hope for children with severe blood disorders
Disease control Recruiting nowThis study tests a bone marrow transplant from a partially matched family donor for children under 21 with severe non-cancer diseases like sickle cell disease, bone marrow failure, or immune disorders. The goal is to see if the transplant can safely replace the diseased bone marr…
Matched conditions: HEMOGLOBINOPATHIES
Phase: PHASE1, PHASE2 • Sponsor: Washington University School of Medicine • Aim: Disease control
Last updated May 26, 2026 04:43 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 genetic blood disorders who do not have a matched family donor. The main goal is to see how many patients survive one year after the transplan…
Matched conditions: HEMOGLOBINOPATHIES
Phase: PHASE2 • Sponsor: Memorial Sloan Kettering Cancer Center • Aim: Disease control
Last updated May 26, 2026 04:41 UTC
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New cell addback aims to slash infection risk in child stem cell recipients
Disease control Recruiting nowThis study tests whether adding a specific type of immune cell (memory T cells) after a stem cell transplant can help prevent serious viral and fungal infections. About 100 children and young adults with blood cancers, immune disorders, or bone marrow failure will receive this ex…
Matched conditions: HEMOGLOBINOPATHIES
Phase: PHASE1, PHASE2 • Sponsor: Children's Hospital of Philadelphia • Aim: Disease control
Last updated May 26, 2026 04:41 UTC
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CRISPR gene therapy aims to free patients from lifelong transfusions
Disease control Recruiting nowThis study tests a single dose of a CRISPR-based gene therapy (CTX001) in 26 adults with transfusion-dependent beta-thalassemia or severe sickle cell disease. The treatment uses the patient's own blood stem cells, which are edited to produce more fetal hemoglobin, potentially red…
Matched conditions: HEMOGLOBINOPATHIES
Phase: PHASE3 • Sponsor: Vertex Pharmaceuticals Incorporated • Aim: Disease control
Last updated May 26, 2026 04:39 UTC
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New stem cell trial offers hope for sickle cell and thalassemia patients
Disease control Recruiting nowThis study tests a stem cell transplant from a healthy donor for people with serious blood disorders like sickle cell disease or thalassemia. The goal is to replace the faulty blood cells with healthy ones, reducing the need for lifelong transfusions. Participants will receive a …
Matched conditions: HEMOGLOBINOPATHIES
Phase: PHASE2 • Sponsor: Masonic Cancer Center, University of Minnesota • Aim: Disease control
Last updated May 22, 2026 14:05 UTC
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Milder transplant approach aims to tame deadly immune diseases
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 pre-transplant treatment can help donor cells take hold with fewer severe side effects. About 220 participants will be followed…
Matched conditions: HEMOGLOBINOPATHIES
Phase: PHASE1, PHASE2 • Sponsor: Washington University School of Medicine • Aim: Disease control
Last updated May 22, 2026 14:04 UTC
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Leftover bone marrow could unlock cures for blood diseases
Knowledge-focused Recruiting nowThis study collects bone marrow that would otherwise be thrown away during surgery. Researchers will use these samples to develop better gene therapies and treatments for blood disorders like sickle cell disease and beta-thalassemia. Up to 50 people with and without these conditi…
Matched conditions: HEMOGLOBINOPATHIES
Sponsor: St. Jude Children's Research Hospital • Aim: Knowledge-focused
Last updated May 26, 2026 04:31 UTC