Testing VGN-R08b for Type III Gaucher Disease in Children
Part of Brain & nervous system, Genetic & congenital, Hormones & metabolism clinical trials.
This study tests whether a new drug called VGN-R08b can safely help children with Type III Gaucher disease—a rare genetic disorder affecting the brain and body. The drug is being tested in children aged 2–17 who have already tried other treatments but still have neurological symptoms, particularly eye movement problems.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You are between 2 and 18 years old
- You have been diagnosed with Type III Gaucher disease (confirmed by enzyme and genetic testing)
- You have eye movement problems (like difficulty moving eyes side-to-side) but can still walk and function day-to-day
- You are currently taking medication for Gaucher disease (such as substrate reduction therapy, ambroxol, or imiglucerase) and it has been stable for at least 2 months, OR you are willing to stop treatment
- Your blood counts and organ sizes meet certain targets: hemoglobin ≥11 g/dL (girls) or ≥12 g/dL (boys), platelets ≥100, and spleen/liver not severely enlarged
- You have not had a serious bone problem (like bone death or fracture with pain) in the past 3 months
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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