Gene therapy for X-linked SCID in young children
Part of Genetic & congenital, Immune system & allergy clinical trials.
This trial tests a gene therapy using a lentivirus to give missing immune system instructions in children with X-linked SCID (SCID-X1). It also uses a low dose of targeted chemotherapy (busulfan) to help the new genes work, and it requires long-term follow-up for safety.
Summary written for real people, not researchers, by Clin2.
Who can take part
- Your child must have X-linked SCID (SCID-X1) caused by a confirmed IL2RG gene change and very weak T-cell function
- There must be no matching close family donor for a transplant (an HLA-identical related donor)
- Your child must be 8 weeks to 5 years old when busulfan is given
- You (and your child’s guardian) must sign consent, and you must be willing to return for long-term follow-up for 15 years
- If your child previously had a related-donor transplant, they must show no donor T cells are still taking hold
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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