HEMOGLOBINOPATHIES
Clinical trials for HEMOGLOBINOPATHIES explained in plain language.
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CRISPR gene editing offers hope for blood disorder patients
Disease control Recruiting nowThis study tests a single dose of a CRISPR-modified stem cell therapy (CTX001) in 26 people with transfusion-dependent β-thalassemia or severe sickle cell disease. The treatment uses the patient's own blood stem cells, edited to produce fetal hemoglobin, aiming to reduce or elimi…
Matched conditions: HEMOGLOBINOPATHIES
Phase: PHASE3 • Sponsor: Vertex Pharmaceuticals Incorporated • Aim: Disease control
Last updated May 17, 2026 04:14 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 donor to treat people with severe blood disorders like sickle cell disease and thalassemia. About 62 participants will receive the transplant and be monitored for side effects like graft-versus-host disease. The goal is to control th…
Matched conditions: HEMOGLOBINOPATHIES
Phase: PHASE2 • Sponsor: Masonic Cancer Center, University of Minnesota • Aim: Disease control
Last updated May 15, 2026 11:55 UTC
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Immune cell boost may shield kids from Post-Transplant infections
Disease control Recruiting nowThis study tests whether adding a special type of immune cell (memory T cells) after a donor stem cell transplant can help prevent serious viral and fungal infections. It involves up to 100 children and young adults with blood cancers or immune disorders. The approach aims to spe…
Matched conditions: HEMOGLOBINOPATHIES
Phase: PHASE1, PHASE2 • Sponsor: Children's Hospital of Philadelphia • Aim: Disease control
Last updated May 15, 2026 11:54 UTC
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Gentler stem cell transplant offers hope for blood disorders
Disease control Recruiting nowThis study is testing a milder stem cell transplant for people with non-cancerous diseases like metabolic disorders, sickle cell disease, and thalassemia. The goal is to safely get donor stem cells to grow in the patient's body while reducing harsh side effects. Up to 220 partici…
Matched conditions: HEMOGLOBINOPATHIES
Phase: PHASE1, PHASE2 • Sponsor: Washington University School of Medicine • Aim: Disease control
Last updated May 12, 2026 13:43 UTC
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New bone marrow transplant could help kids with sickle cell and immune 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 control the disease with fewer side e…
Matched conditions: HEMOGLOBINOPATHIES
Phase: PHASE1, PHASE2 • Sponsor: Washington University School of Medicine • Aim: Disease control
Last updated May 07, 2026 18:42 UTC
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Cord blood transplants offer hope for kids with blood cancers who lack 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-cancerous blood disorders who do not have a matched family donor. The main goal is to see how many patients are alive without treatment-re…
Matched conditions: HEMOGLOBINOPATHIES
Phase: PHASE2 • Sponsor: Memorial Sloan Kettering Cancer Center • Aim: Disease control
Last updated May 07, 2026 18:38 UTC
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Leftover bone marrow could unlock cures for sickle cell and thalassemia
Knowledge-focused Recruiting nowThis study collects bone marrow that is normally thrown away during surgery from people with and without blood disorders like sickle cell disease and beta-thalassemia. Researchers will use these samples to develop better gene therapies and drug treatments that could one day cure …
Matched conditions: HEMOGLOBINOPATHIES
Sponsor: St. Jude Children's Research Hospital • Aim: Knowledge-focused
Last updated May 14, 2026 12:03 UTC