HEMOGLOBINOPATHIES
Clinical trials for HEMOGLOBINOPATHIES explained in plain language.
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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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Cord blood hope for kids with blood cancers who have no donor
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 late…
Matched conditions: HEMOGLOBINOPATHIES
Phase: PHASE2 • Sponsor: Memorial Sloan Kettering Cancer Center • Aim: Disease control
Last updated Jun 15, 2026 19:07 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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New bone marrow transplant offers hope for kids with sickle cell and other disorders
Disease control Recruiting nowThis study is testing a bone marrow transplant from a family donor (like a parent or sibling) for children under 21 with severe non-cancerous blood disorders, such as sickle cell disease or bone marrow failure. The goal is to see if a milder pre-transplant treatment can help the …
Matched conditions: HEMOGLOBINOPATHIES
Phase: PHASE1, PHASE2 • Sponsor: Washington University School of Medicine • Aim: Disease control
Last updated Jun 15, 2026 19:04 UTC
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New cell addback may shield kids from deadly infections after transplant
Disease control Recruiting nowThis study tests whether adding a special type of immune cell (memory T cells) after a stem cell transplant can help children fight off viral and fungal infections. About 100 children and young adults with blood cancers, immune disorders, or bone marrow failure will receive this …
Matched conditions: HEMOGLOBINOPATHIES
Phase: PHASE1, PHASE2 • Sponsor: Children's Hospital of Philadelphia • Aim: Disease control
Last updated Jun 15, 2026 19:04 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 cures for sickle cell disease
Knowledge-focused Recruiting nowThis study collects bone marrow samples that would otherwise be thrown away during surgery. Researchers will use them to develop better gene therapies and treatments for sickle cell disease and thalassemia. Up to 50 children and adults with or without these blood disorders will p…
Matched conditions: HEMOGLOBINOPATHIES
Sponsor: St. Jude Children's Research Hospital • Aim: Knowledge-focused
Last updated Jun 01, 2026 17:55 UTC