Gene editing trial for chronic granulomatous disease
Part of Blood & lymphatic, Genetic & congenital, Immune system & allergy clinical trials.
This trial tests a new gene editing treatment called PM359 for people with a specific type of chronic granulomatous disease (CGD) caused by a delGT mutation in the NCF1 gene. The goal is to see if it's safe and helps fight infections and inflammation.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must have CGD caused by the delGT mutation in the NCF1 gene (p47phox deficiency).
- You have been treated at a specialized CGD center for at least 2 years.
- You are willing to take part in this study and a long-term follow-up for up to 15 years total.
- You have had at least one serious CGD infection, or have a current serious infection that is not responding to treatment, OR you have an active CGD-related autoimmune or inflammatory condition that needs treatment.
- If you are under 16, you cannot have a fully matched related stem cell donor available.
- You cannot have active blood infections or other conditions that would make stem cell transplant 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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