Gene therapy for x-linked chronic granulomatous disease
Treatments studied
Part of Blood & lymphatic, Genetic & congenital, Immune system & allergy clinical trials.
This trial tests a new gene therapy for X-linked chronic granulomatous disease (CGD), a condition where the immune system cannot fight certain infections. The treatment uses your own blood stem cells, which are modified in the lab to help your body produce working immune cells.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must have X-linked chronic granulomatous disease, with the specific gene change confirmed by a blood test.
- You have had at least one serious infection or inflammation that required hospital care in the past.
- There is no perfectly matched bone marrow donor available (a 10/10 match).
- You must be at least 3 years old and weigh at least 15 kg (about 33 pounds).
- Your kidney, liver, and heart function must be within safe limits, and you must not have HIV or active hepatitis B or C.
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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