Familial severe combined immunodeficiency
MONDO:003152024 clinical trials for this condition and its sub-types.
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Broader categories
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Gene therapy offers hope for babies with 'Bubble Boy' disease
Cure Recruiting nowThis study tests a new gene therapy for children with a severe immune disorder called Artemis-SCID, where the body cannot fight infections. Doctors take the child's own blood stem cells, fix the faulty gene in a lab, and put the corrected cells back. The goal is to rebuild a work…
Phase 1/2 • Sponsor: Assistance Publique - Hôpitaux de Paris • Aim: Cure
Last updated Jun 27, 2026 12:00 UTC
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New gene therapy offers hope for older kids and adults with 'bubble boy' disease
Disease control Recruiting nowThis study tests a gene therapy for people aged 2 to 50 with X-linked severe combined immunodeficiency (SCID-X1), a condition that leaves them without a working immune system. Participants receive their own blood stem cells that are genetically corrected in a lab, then infused ba…
Phase 1/2 • Sponsor: National Institute of Allergy and Infectious Diseases (NIAID) • Aim: Disease control
Last updated Sep 09, 2026 16:00 UTC
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Stem cell transplant offers new hope for rare immune diseases
Disease control Recruiting nowThis study tests a stem cell transplant for people with common variable immunodeficiency (CVID) and other immune disorders. The goal is to replace the faulty immune system with a healthy donor's stem cells. Participants are aged 5 to 40 and will receive a reduced-intensity transp…
Phase 2 • Sponsor: Paul Szabolcs • Aim: Disease control
Last updated Jun 27, 2026 12:33 UTC
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Gene therapy offers new hope for babies with rare 'Bubble Boy' disease
Disease control Recruiting nowThis study tests a gene therapy for Artemis-deficient severe combined immunodeficiency (ART-SCID), a life-threatening immune disorder. It involves taking the patient's own blood stem cells, adding a working copy of the faulty gene, and infusing them back after mild chemotherapy. …
Phase 1/2 • Sponsor: University of California, San Francisco • Aim: Disease control
Last updated Jun 27, 2026 09:04 UTC
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Gene therapy offers hope for children with SCID-X1
Disease control Recruiting nowThis trial tests a gene therapy for children with X-linked severe combined immunodeficiency (SCID-X1), also known as 'bubble boy' disease. Participants receive a low dose of busulfan followed by an infusion of their own blood stem cells that have been genetically corrected with a…
Phase 1/2 • Sponsor: David Williams • Aim: Disease control
Last updated Jun 27, 2026 08:03 UTC
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Scientists launch major study to track rare immune diseases
Knowledge-focused Recruiting nowThis study is observing up to 3,500 people with immune system problems, including rare genetic conditions like chronic granulomatous disease and severe combined immunodeficiency. Researchers will track how these diseases change over time through blood tests, imaging, and checkups…
Sponsor: National Institute of Allergy and Infectious Diseases (NIAID) • Aim: Knowledge-focused
Last updated Sep 03, 2026 00:00 UTC