Gene therapy for chronic granulomatous disease (CGD)
Part of Blood & lymphatic, Genetic & congenital, Immune system & allergy clinical trials.
This trial tests a lentiviral gene therapy to improve immune function in people with CGD, a condition where the body can’t fight certain infections well. It may help by addressing the underlying genetic problem, but it also requires a procedure to collect immune cells and special “conditioning” medicines beforehand.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You have CGD and are at least 0 years old (any age).
- Your CGD diagnosis is confirmed by genetic (DNA) testing and lab tests showing very low or absent NADPH-oxidase activity.
- Your overall ability to function is good enough (Karnofsky score 70 or higher).
- You have had at least one severe infection and/or inflammation problem that has kept coming back, not improved, and/or required hospital care despite treatments.
- You can provide consent (or assent if you are age 7+), and a parent/guardian can consent for children.
- You can safely undergo leukapheresis (cell collection) and the medicines needed to prepare your body (no major anemia, unstable heart/blood pressure, or serious bleeding/clotting problems).
- If you are female, you are not pregnant and not breastfeeding.
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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