Gene therapy for chronic granulomatous disease
Part of Blood & lymphatic, Genetic & congenital, Immune system & allergy clinical trials.
This trial tests a new gene therapy for chronic granulomatous disease (CGD), a condition that makes people prone to severe infections. The therapy uses a modified virus to fix the faulty gene in your blood cells, which may help your immune system fight infections better.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must have chronic granulomatous disease (CGD) confirmed by genetic testing and lab evidence of low NADPH-oxidase activity.
- You should be in fairly good overall health, able to carry out most daily activities (Karnofsky score of 70% or higher).
- You must have had at least one serious infection or inflammation that needed hospital treatment.
- Adults must give written consent; children aged 7 and over must agree to take part (assent).
- You cannot join if you are pregnant or breastfeeding, or if you have conditions that make blood collection (leukapheresis) unsafe.
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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