Gene therapy for p47 chronic granulomatous disease
Part of Blood & lymphatic, Genetic & congenital, Immune system & allergy clinical trials.
This trial tests a new gene therapy for people with a specific type of chronic granulomatous disease (p47 CGD). The treatment uses your own blood stem cells, which are modified in a lab to help your body fight infections better. It is for people who do not have a matched stem cell donor and have had serious infections.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must have a confirmed diagnosis of p47 chronic granulomatous disease (CGD).
- You must be at least 3 years old and weigh at least 33 pounds (15 kg).
- You must have had a serious CGD-related infection or inflammation that needed hospital care, or have an ongoing infection that is not getting better with treatment.
- You cannot have a matched stem cell donor (brother, sister, or unrelated).
- You must be willing to use effective birth control if you are of childbearing age, and avoid pregnancy for at least 3 months after the treatment.
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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