Venglustat for type 3 Gaucher disease
Part of Brain & nervous system, Genetic & congenital, Hormones & metabolism clinical trials.
This trial provides access to venglustat, an experimental oral medication for people with type 3 Gaucher disease that affects the brain and nervous system. It is designed for those who are already on a stable dose of enzyme replacement therapy (ERT) and are looking for additional treatment options.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You are at least 12 years old and weigh at least 15 kg (33 pounds).
- You have a confirmed diagnosis of type 3 Gaucher disease with at least one symptom affecting your brain or nerves.
- You have been on a steady dose of enzyme replacement therapy (ERT) for the last 6 months or more.
- Your blood cell counts, spleen, and liver size have been stable and not getting worse.
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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