Gene therapy for p47 form of chronic granulomatous disease
Part of Blood & lymphatic, 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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This early-stage study tests a one-time lentiviral gene therapy to help children with X-linked severe combined immunodeficiency (SCID-X1) make infection-fighting T cells. It may reduce the need for a matching donor transplant, but it requires long-term follow-up for safety over many years.
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.
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.
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