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 to treat severe immune system problems in 57 people. The goal is to help the body make healthy blood cells and fight infections. Patients must have a specific immune disorder and meet health criteria to join.
Matched conditions: WISKOTT-ALDRICH SYNDROME
Phase: NA • Sponsor: Masonic Cancer Center, University of Minnesota • Aim: Disease control
Last updated May 26, 2026 12:11 UTC
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Gene therapy offers hope for rare immune disorder
Disease control OngoingThis study tests a gene therapy for people with Wiskott-Aldrich syndrome, a rare genetic disease that weakens the immune system and causes bleeding. The treatment uses the patient's own blood stem cells, which are modified in a lab to fix the faulty gene and then returned to the …
Matched conditions: WISKOTT-ALDRICH SYNDROME
Phase: PHASE3 • Sponsor: Fondazione Telethon • Aim: Disease control
Last updated May 26, 2026 12:04 UTC
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Milder stem cell transplant offers hope for kids with rare immune diseases
Disease control OngoingThis study tests a less intense stem cell transplant for children and young adults up to age 28 with various immune system disorders. The goal is to see if a milder pre-transplant treatment can help donor cells take hold with fewer serious side effects. The study tracks engraftme…
Matched conditions: WISKOTT-ALDRICH SYNDROME
Phase: PHASE2 • Sponsor: Washington University School of Medicine • Aim: Disease control
Last updated May 26, 2026 12:01 UTC
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Double transplant offers hope for kids with rare immune diseases
Disease control ENROLLING_BY_INVITATIONThis study tests a new approach for people aged 5 to 45 with primary immune disorders and severe lung damage. Participants receive a double lung transplant followed by a stem cell transplant from the same donor. The goal is to see if this combination is safe and can help the body…
Matched conditions: WISKOTT-ALDRICH SYNDROME
Phase: PHASE1, PHASE2 • Sponsor: Paul Szabolcs • Aim: Disease control
Last updated May 07, 2026 18:41 UTC