Gene therapy for p47 form of chronic granulomatous disease
Part of Immune system & allergy clinical trials.
This trial tests an individualized gene therapy made from your own stem cells to help the immune system work better in p47-related chronic granulomatous disease (CGD). It’s designed for children who have serious infections or inflammation despite usual treatments and who don’t have a fully matched bone marrow donor.
Summary written for real people, not researchers, by Clin2.
Who can take part
- Must be older than 23 months (at least 2 years old).
- Must be diagnosed with p47 CGD confirmed by specific lab genetic testing and enzyme activity being very low.
- Must have had at least one severe infection and/or inflammation problem that led to hospital care despite conventional treatment.
- Must not have a fully matched (10/10) donor available, after checking donor registries.
- Must be free of certain infections (like HIV, hepatitis B, and hepatitis C) and other listed infections around the time of treatment.
- Must meet blood, heart, lung, kidney, liver, and overall health limits to safely tolerate the stem-cell procedure and conditioning medicines.
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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