Gene therapy for X-linked SCID using a modified virus
Part of Genetic & congenital, Immune system & allergy clinical trials.
This early-stage study tests a one-time lentiviral gene therapy to help children with X-linked severe combined immunodeficiency (SCID-X1) make infection-fighting T cells. It may reduce the need for a matching donor transplant, but it requires long-term follow-up for safety over many years.
Summary written for real people, not researchers, by Clin2.
Who can take part
- Your child must have SCID-X1 confirmed by a specific IL2RG gene change and very low T-cell function
- There must be no fully HLA-matched related donor (a closely matched family tissue type match)
- Your child must be younger than 5 years old
- Your child must be at least 8 weeks old when they receive busulfan (a chemotherapy medicine before gene therapy)
- You must agree to long-term follow-up for 15 years after the infusion
- The study team must feel your child can safely receive the collection and infusion process (including anesthesia and ability to return for visits)
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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