HEMOGLOBINOPATHIES
Clinical trials for HEMOGLOBINOPATHIES explained in plain language.
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Cord blood transplants offer hope for kids without a donor match
Disease control Recruiting nowThis study tests a cord blood transplant in 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 goal is to see if this approach lowers the risk of death from treatment one year afte…
Matched conditions: HEMOGLOBINOPATHIES
Phase: PHASE2 • Sponsor: Memorial Sloan Kettering Cancer Center • Aim: Disease control
Last updated Jun 19, 2026 08:29 UTC
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New immune cell boost may shield kids from Post-Transplant infections
Disease control Recruiting nowThis study tests whether adding memory immune cells (CD45RO) to a stem cell transplant can help children fight off dangerous viral and fungal infections after the procedure. The transplant uses donor stem cells that have been stripped of certain cells to prevent graft-versus-host…
Matched conditions: HEMOGLOBINOPATHIES
Phase: PHASE1, PHASE2 • Sponsor: Children's Hospital of Philadelphia • Aim: Disease control
Last updated Jun 18, 2026 11:50 UTC
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New bone marrow transplant trial offers hope for kids with severe blood disorders
Disease control Recruiting nowThis study tests a bone marrow transplant from a family donor for children under 21 with severe non-cancer blood disorders like sickle cell disease, bone marrow failure, or immune problems. The goal is to see if the transplant can replace the diseased cells with healthy donor cel…
Matched conditions: HEMOGLOBINOPATHIES
Phase: PHASE1, PHASE2 • Sponsor: Washington University School of Medicine • Aim: Disease control
Last updated Jun 18, 2026 11:46 UTC
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New stem cell transplant 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 serious blood disorders like sickle cell disease and thalassemia. About 62 participants will receive the transplant to see if it can reduce complications like graft-versus-host disease. The goal is to help …
Matched conditions: HEMOGLOBINOPATHIES
Phase: PHASE2 • Sponsor: Masonic Cancer Center, University of Minnesota • Aim: Disease control
Last updated Jun 15, 2026 19:08 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 person's own CRISPR-edited stem cells to treat two inherited blood disorders: transfusion-dependent beta-thalassemia and severe sickle cell disease. The goal is to help the body produce healthy red blood cells and reduce or eliminate the need f…
Matched conditions: HEMOGLOBINOPATHIES
Phase: PHASE3 • Sponsor: Vertex Pharmaceuticals Incorporated • Aim: Disease control
Last updated Jun 15, 2026 19:07 UTC
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Gentler transplant offers hope for blood disorders without cancer
Disease control Recruiting nowThis study is testing a milder type of stem cell transplant for people with non-cancerous blood, immune, or metabolic disorders. The goal is to safely get donor cells to grow in the patient's body while reducing harsh side effects. About 220 participants will receive a reduced-in…
Matched conditions: HEMOGLOBINOPATHIES
Phase: PHASE1, PHASE2 • Sponsor: Washington University School of Medicine • Aim: Disease control
Last updated Jun 15, 2026 18:57 UTC
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Leftover bone marrow could unlock better gene therapies for sickle cell disease
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. Researchers will use these samples in the lab to learn how to better manipulate stem cells for gene therapy and to study cell health.…
Matched conditions: HEMOGLOBINOPATHIES
Sponsor: St. Jude Children's Research Hospital • Aim: Knowledge-focused
Last updated Jun 19, 2026 08:42 UTC
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New biobank aims to unlock better treatments for blood diseases
Knowledge-focused Recruiting nowThis study collects blood samples and health information from 375 people with non-cancerous blood disorders like aplastic anemia, sickle cell disease, or thalassemia who are receiving a stem cell transplant or gene therapy. The goal is to create a biobank that helps researchers u…
Matched conditions: HEMOGLOBINOPATHIES
Sponsor: Medical College of Wisconsin • Aim: Knowledge-focused
Last updated Jun 18, 2026 11:49 UTC