Gene therapy for Parkinson's disease with GBA1 mutations
Part of Brain & nervous system clinical trials.
This trial tests a new gene therapy for Parkinson's disease in people who have a specific genetic change (GBA1 mutation) that may be causing their symptoms. The treatment aims to fix the root cause by delivering a corrected gene to the brain, which might help improve motor symptoms and reduce 'off' time.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You are between 30 and 70 years old.
- You have Parkinson's disease with a confirmed GBA1 gene mutation and low glucocerebrosidase enzyme activity.
- Your Parkinson's symptoms are moderate to severe (Hoehn-Yahr stage 3-4) and you have significant 'off' time despite optimized medication.
- You can walk without a walker or wheelchair.
- You have a study partner who can help with visits and provide information about your health.
- You must be willing to use effective contraception and not donate sperm or eggs for at least a year after dosing.
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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