Gene therapy trial for Artemis SCID using modified blood cells
Part of Genetic & congenital, Hormones & metabolism, Immune system & allergy clinical trials.
This trial tests whether gene therapy can safely and effectively treat children with a specific type of severe combined immunodeficiency (SCID) caused by an Artemis gene problem. It uses the patient’s own stem cells, modifies them to add a missing gene, and then returns them after chemotherapy to help the new cells work.
Summary written for real people, not researchers, by Clin2.
Who can take part
- Age up to 47 months (under 4 years old)
- You have SCID from confirmed Artemis (DCLRE1C) gene mutations (even if you still have some leftover function)
- No matching bone marrow donor available, or an unrelated donor can’t be found within 6 weeks of diagnosis
- Your treating team says gene therapy can start without dangerous delay because of a serious, life-threatening infection risk
- You (and a parent/guardian) can agree to long follow-up visits for the first 2 years and beyond
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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