Gene therapy for X-linked severe combined immunodeficiency
Treatments studied
Part of Genetic & congenital, Immune system & allergy clinical trials.
This trial tests a gene therapy that uses base-edited stem cells to treat X-SCID, a genetic immune disorder. It may help improve immune function and reduce infections in patients who still have low immune cells or severe infections despite previous treatments.
Summary written for real people, not researchers, by Clin2.
Who can take part
- Aged 3 years or older and weigh at least 10 kilograms.
- Diagnosed with X-SCID and have a specific mutation in the IL2RG gene (Q144X, R289X, Q235X, or R226H).
- If you had a prior stem cell transplant, it must be at least 18 months ago.
- You must be able to undergo a procedure to collect stem cells from your blood (apheresis).
- You must have low immune function, such as low CD4+ cell counts or low antibodies, and have had recent infections or other complications due to your immune deficiency.
- You must agree to use effective contraception for at least 2 years after treatment if you are of reproductive age.
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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