Gene therapy for inherited immune disease
Part of Blood & lymphatic, Genetic & congenital, Immune system & allergy clinical trials.
This trial tests a new gene therapy (SGX-001) for chronic granulomatous disease caused by a specific genetic change. The treatment tries to fix the gene in your immune cells so they can fight infections better, and the trial will follow participants for up to 15 years to make sure it's safe and effective.
Summary written for real people, not researchers, by Clin2.
Who can take part
- Have a confirmed diagnosis of chronic granulomatous disease caused by a mutation in the NCF1 gene.
- Be at least 1.5 years old and weigh at least 10 kilograms (about 22 pounds).
- Have had one or more serious or repeated infections or inflammatory problems.
- Have no fully matched brother or sister available for a bone marrow transplant.
- Be free of active infections and other major health problems that could make the treatment 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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