Gene therapy for children with severe immune deficiency (XSCID)
Treatments studied
Part of Genetic & congenital, Immune system & allergy clinical trials.
This trial tests a one-time gene therapy approach to help children with X-linked SCID (a serious inherited immune disorder) make better immune cells. It’s for people who don’t have a fully matched sibling donor and need long-term follow-up after treatment.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You have a confirmed XSCID-causing genetic change in the common gamma chain gene (from a DNA test).
- You have no fully tissue-typed matched sibling donor available.
- You are age 2 to 50, and you weigh at least 10 kg (about 22 lb).
- If you had a transplant before, it must be at least 18 months ago.
- You must be able to store blood and tissue samples for the study, and your doctor must feel your home/social situation will let you complete the study and long-term follow-up.
- You must have signs of severe immune weakness (for example very low immunity proteins like IgG, or needing regular IVIG) plus either lab signs (like very low CD4/T-cells or low immune cell numbers) or clinical signs (like frequent significant infections or chronic lung/GI problems).
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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