WISKOTT-ALDRICH SYNDROME
Clinical trials for WISKOTT-ALDRICH SYNDROME explained in plain language.
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Stem cell transplant offers new hope for kids with rare immune diseases
Disease control OngoingThis study tests a donor stem cell transplant for people with severe immune system problems, like SCID and Wiskott-Aldrich syndrome. The goal is to help the body make healthy blood cells and fight infections. Participants receive donated stem cells to rebuild their immune system.…
Matched conditions: WISKOTT-ALDRICH SYNDROME
Phase: NA • Sponsor: Masonic Cancer Center, University of Minnesota • Aim: Disease control
Last updated Jun 27, 2026 14:00 UTC
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Gentler transplant shows promise for kids with blood diseases
Disease control OngoingThis 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: WISKOTT-ALDRICH SYNDROME
Phase: PHASE2 • Sponsor: Children's Hospital of Philadelphia • Aim: Disease control
Last updated Jun 27, 2026 12:28 UTC
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Milder stem cell transplant shows promise for kids with immune disorders
Disease control OngoingThis study tests a stem cell transplant using a milder chemotherapy-like regimen to treat children and young adults (up to age 28) with various immune system disorders. The goal is to help the donor stem cells settle in the body with fewer side effects. The study involves 20 part…
Matched conditions: WISKOTT-ALDRICH SYNDROME
Phase: PHASE2 • Sponsor: Washington University School of Medicine • Aim: Disease control
Last updated Jun 27, 2026 09:06 UTC
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Double transplant breakthrough offers hope for rare immune disorder patients
Disease control ENROLLING_BY_INVITATIONThis study tests a new approach for people aged 5 to 45 with primary immune deficiencies and end-stage lung disease. Participants receive a double lung transplant followed by a stem cell transplant from the same donor. The goal is to see if this combined procedure is safe and can…
Matched conditions: WISKOTT-ALDRICH SYNDROME
Phase: PHASE1, PHASE2 • Sponsor: Paul Szabolcs • Aim: Disease control
Last updated Jun 27, 2026 08:04 UTC
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New stem cell approach could fix immune systems in kids
Disease control OngoingThis Phase II trial tests a stem cell transplant from unrelated or partially matched family donors for children and young adults with severe immune deficiencies. The stem cells are specially processed to remove certain immune cells, aiming to reduce complications. The goal is to …
Matched conditions: WISKOTT-ALDRICH SYNDROME
Phase: PHASE2 • Sponsor: Children's Hospital of Philadelphia • Aim: Disease control
Last updated Jun 27, 2026 07:56 UTC
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Gene therapy aims to stop severe infections in rare immune disorder
Disease control OngoingThis trial tests a gene therapy called OTL-103 for people with Wiskott-Aldrich syndrome, a rare genetic disorder that weakens the immune system and causes bleeding. The therapy uses the patient's own blood stem cells, modified in a lab to carry a working copy of the faulty gene, …
Matched conditions: WISKOTT-ALDRICH SYNDROME
Phase: PHASE3 • Sponsor: Fondazione Telethon • Aim: Disease control
Last updated Jun 27, 2026 07:52 UTC