Gene therapy for children with GA-1 not helped by standard care
Part of Genetic & congenital, Hormones & metabolism clinical trials.
This trial tests a new gene therapy (VGM-R02b) for children with Glutaric Acidemia Type 1 (GA-1) whose symptoms are not well controlled by standard treatment. The goal is to see if the therapy is safe and can help improve neurological symptoms.
Summary written for real people, not researchers, by Clin2.
Who can take part
- Your child must be 6 years old or younger.
- They must have a confirmed diagnosis of Glutaric Acidemia Type 1 (GA-1) with a specific gene change (GCDH mutation).
- They must have at least one brain-related symptom, like a large head, muscle stiffness, developmental delays, seizures, or abnormal brain waves.
- They must be on standard treatment (like a special diet) but still have symptoms that are not well controlled.
- Blood tests must show high levels of glutaric acid (GA) or 3-hydroxyglutaric acid (3-OHGA).
- Children who have had gene therapy before, have severe liver or kidney disease, or have active infections cannot join.
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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