Gene Therapy for Chronic Granulomatous Disease
Part of Blood & lymphatic, Genetic & congenital, Immune system & allergy clinical trials.
This study tests a new gene therapy for people with a rare immune disorder called chronic granulomatous disease (CGD), which causes repeated infections. The treatment uses a modified virus to deliver a healthy gene to your blood stem cells, potentially helping your immune system fight off bacteria and fungi. The goal is to reduce the risk of severe infections and improve overall health.
Summary written for real people, not researchers, by Clin2.
Who can take part
- Must weigh at least 15 kg (about 33 pounds).
- Must have a confirmed diagnosis of a specific type of chronic granulomatous disease (CGD) – your doctor can confirm this with a blood test.
- Must be willing to sign an informed consent form (or have a parent/guardian sign if under 18).
- Must agree to use effective birth control (if applicable) for the duration of the study.
- Must not have an active, untreated infection at the time of enrollment. However, if you have a persistent infection despite treatment, you may still qualify.
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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