Gene therapy study for X-linked chronic granulomatous disease
Part of Blood & lymphatic, Genetic & congenital, Immune system & allergy clinical trials.
This study tests a new gene therapy called EN-374 for people with X-linked chronic granulomatous disease (X-CGD). It aims to fix the genetic cause of the disease to prevent severe infections and inflammation.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must be male and 18 years or older (or 3 months or older in later phases).
- You must have X-CGD with less than 5% of white blood cells showing normal function (DHR test) and a specific mutation in the CYBB gene.
- You must have had at least one severe infection that needed medical treatment or a long-term inflammatory condition.
- You do not have a suitable sibling or other donor who matches perfectly for a stem cell transplant.
- You must have adequate organ function and be willing to use contraception if sexually active.
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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