Gene therapy for immune disease in people with IPEX syndrome
Part of Genetic & congenital, Immune system & allergy clinical trials.
This study tests a gene therapy that delivers a correct copy of the FOXP3 gene to your immune cells (T-cells), turning them into cells that can calm the overactive immune response seen in IPEX syndrome. The goal is to reduce symptoms and the need for lifelong immunosuppressive drugs.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must be male and between the ages of 1 and 45 (the first three patients will be between 10 and 45).
- You must have IPEX syndrome caused by a specific change (mutation) in the FOXP3 gene.
- You must have already tried at least one treatment (like immunosuppressive drugs) and still have symptoms, or your disease is controlled but you still need treatment.
- You cannot have a suitable stem cell transplant from a matched sibling or unrelated donor, or you are not willing to have one.
- If you've had a stem cell transplant before, it must have failed (meaning the donor cells didn't take).
- You must be willing to come back for follow-up visits for up to 2 years, plus a longer follow-up of 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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