Gene therapy trial for IPEX syndrome
Part of Genetic & congenital, Immune system & allergy clinical trials.
This trial tests a gene therapy that adds a working FOXP3 gene to your own T cells to help them regulate your immune system. It's for people with IPEX syndrome whose disease is not controlled by standard treatments or who cannot have a stem cell transplant.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must be male and between 1 and 45 years old (the first three people treated will be 10 to 45).
- You have IPEX syndrome caused by a change in the FOXP3 gene.
- Your IPEX symptoms keep coming back even though you're on immune-suppressing medicines.
- A stem cell transplant is not possible or a suitable donor isn't available.
- You agree to use effective birth control for at least 12 months after the infusion.
- You are willing to come back for check-ups for 2 years and then for long-term follow-up for 15 years.
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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