THALASSEMIA MAJOR
Clinical trials for THALASSEMIA MAJOR explained in plain language.
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Gentler transplant shows promise for kids with blood diseases
Disease control CompletedThis study tests a milder chemotherapy and immune-suppressing regimen before a stem cell transplant for children and young adults with non-malignant blood disorders like sickle cell disease or immune deficiencies. The goal is to safely achieve donor cell engraftment with fewer si…
Matched conditions: THALASSEMIA MAJOR
Phase: PHASE2 • Sponsor: Children's Hospital of Philadelphia • Aim: Disease control
Last updated Jul 23, 2026 00:00 UTC
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Gene-Editing breakthrough offers hope for blood disorder patients
Disease control CompletedThis early-stage study tested a new gene-edited stem cell transplant in 2 children with severe β-thalassemia, a serious blood disorder requiring lifelong transfusions. The treatment aims to help the body produce healthy red blood cells and reduce or stop the need for transfusions…
Matched conditions: THALASSEMIA MAJOR
Phase: EARLY_PHASE1 • Sponsor: Children's Hospital of Fudan University • Aim: Disease control
Last updated Jun 27, 2026 12:32 UTC
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New stem cell transplant approach shows promise for blood disorders
Disease control CompletedThis pilot study tested a stem cell transplant from closely matched unrelated donors for 8 patients with severe sickle cell disease or thalassemia major. The donated stem cells were specially processed to remove certain immune cells to reduce the risk of rejection and graft-versu…
Matched conditions: THALASSEMIA MAJOR
Phase: NA • Sponsor: Timothy Olson • Aim: Disease control
Last updated Jun 27, 2026 12:24 UTC