Venglustat for type 3 Gaucher disease
Part of Brain & nervous system, Genetic & congenital, Hormones & metabolism clinical trials.
This special access program provides an experimental oral medicine called venglustat (taken as a tablet) to patients with Type 3 Gaucher disease, which affects the brain. It is designed for patients who are already on stable enzyme replacement therapy and aims to see if the drug can help manage neurological symptoms.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must be 12 years old or older.
- You must weigh at least 33 pounds (15 kg).
- You must have a diagnosis of Type 3 Gaucher disease with at least one sign that the disease affects your brain or nervous system.
- You must be on a stable dose of enzyme replacement therapy (ERT) for at least 6 months and be stable in your blood counts and organ sizes.
- If you have seizures, they need to be well controlled without certain medications that could interact with the study drug.
- For women who can get pregnant, you must have a negative pregnancy test and use birth control as required by local rules.
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 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.
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This trial tests a treatment called velaglucerase beta, which replaces an enzyme that people with Gaucher disease lack. It may help reduce enlarged spleen and liver, improve anemia, and increase platelet counts in children under 12.
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This study tests a new gene therapy called LY-M001 for adults with Type 1 Gaucher disease. It aims to help the body produce the missing enzyme and reduce symptoms like low blood counts or enlarged organs.
This trial tests a gene therapy called FLT201 for adults with Gaucher disease type 1. It aims to provide a long-term treatment option for people who have been stable on enzyme replacement therapy (ERT) or substrate reduction therapy (SRT) for at least two years.
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