Wiskott-Aldrich syndrome
MONDO:0010518Wiskott-Aldrich syndrome (WAS) is a primary immunodeficiency disease characterized by microthrombocytopenia, eczema, infections and an increased risk for autoimmune manifestations and malignancies.
Also known as: WAS, Wiskott Aldrich syndrome, Wiskott-Aldrich syndrome, Wiskott-Aldrich syndrome 1, Wiskott-Aldrich syndrome, X-linked recessive, eczema-thrombocytopenia-immunodeficiency syndrome, immunodeficiency 2, Aldrich syndrome
47 clinical trials for this condition and its sub-types, 10 tagged with Wiskott-Aldrich syndrome itself.
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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.…
Sponsor: Masonic Cancer Center, University of Minnesota • Aim: Disease control
Last updated Jun 27, 2026 14:00 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…
Phase 2 • 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 By invitation onlyThis 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…
Phase 1/2 • 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 …
Phase 2 • 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, …
Phase 3 • Sponsor: Fondazione Telethon • Aim: Disease control
Last updated Jun 27, 2026 07:52 UTC
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Brazilian study to reveal how well medical cannabis really works in daily life
Knowledge-focused By invitation onlyThis observational study will follow 384 patients in Brazil who are using cannabis-based products (like CBD or THC oils) prescribed by their own doctors for chronic pain, anxiety, or depression. Researchers will not provide the medication but will track patients' quality of life,…
Sponsor: Santa Catarina Federal University • Aim: Knowledge-focused
Last updated Jun 27, 2026 12:02 UTC