Gene therapy for severe combined immunodeficiency (SCID-X1)
Part of Genetic & congenital, Immune system & allergy clinical trials.
This trial tests a gene therapy that aims to fix the genetic cause of SCID-X1, a condition that leaves babies without a working immune system. By introducing a corrected gene into your child's own cells, the treatment may help their body fight off infections.
Summary written for real people, not researchers, by Clin2.
Who can take part
- A genetic test confirms SCID-X1 due to a specific gene change
- No matched sibling or other family member is available for a stem cell transplant
- Your child has had severe infections such as pneumonia, severe diarrhea, or certain viral infections
- No previous stem cell transplant and no current cancer
- Your child is HIV-negative and not taking chemotherapy or immunosuppressive drugs
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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