Gene therapy by vein for SCID-X1 immune deficiency
Part of Genetic & congenital, Immune system & allergy clinical trials.
This trial tests an experimental gene therapy given through a vein to help children with SCID-X1 make needed immune cells. It may help reduce severe infections when standard options like a matched donor are not available.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must have SCID-X1 confirmed by DNA testing for a specific gene mutation (IL2RG).
- Your T-cell immune system must be very weak based on blood or lab test results.
- There must be no fully matched related donor (an HLA-identical family donor).
- You must be free of HIV infection and able to provide informed consent (or have a guardian consent).
- You must not have signs of blood cancer risk on bone marrow/ chromosome testing.
- You must not have had a prior stem cell transplant, and your expected life expectancy must be at least 3 months.
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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