Gene therapy study for type 3 Gaucher disease
Part of Brain & nervous system, Genetic & congenital, Hormones & metabolism clinical trials.
This study tests a new gene therapy, VGN-R08b, for people with type 3 Gaucher disease. It aims to see if the treatment is safe and can help with neurological symptoms like eye movement problems.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You are between 2 and 17 years old.
- You have a confirmed diagnosis of type 3 Gaucher disease with GBA1 gene mutations.
- You have eye movement problems (like trouble moving eyes sideways) but are not bedridden.
- You are currently on stable treatment for Gaucher disease (like substrate reduction therapy or ambroxol) and are willing to either continue or stop it before the study.
- You are receiving and willing to continue stable treatment for peripheral symptoms (like enzyme replacement therapy) and have stable blood counts and spleen/liver sizes.
- If you are of reproductive age, you agree to use effective contraception for at least 1 year after treatment starts.
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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