Gene therapy for X-linked severe combined immunodeficiency
Treatments studied
Part of Genetic & congenital, Immune system & allergy clinical trials.
This study tests a gene therapy to treat X-linked severe combined immunodeficiency (X-SCID) without a stem cell transplant from a matched sibling. It aims to improve immune function and reduce infections using your own stem cells modified to carry the correct gene.
Summary written for real people, not researchers, by Clin2.
Who can take part
- Age 3 years or older and weight at least 10 kg
- Diagnosis of X-linked severe combined immunodeficiency (X-SCID) with specific gene mutations
- If previously received a stem cell transplant, it must be at least 18 months ago
- Expected survival of at least 120 days
- No available HLA-matched sibling donor
- No history of cancer before age 18 in you or a first-degree relative, and no known genetic cancer predisposition
- Willing to share personal information with the Campath distribution program if needed
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
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