A Phase 1/2a Study to Evaluate the Safety of two Dose Levels of PR001A in Patients With Parkinson s Disease who Have at Least one GBA1 Mutation

Brief description of study

The objective of the study is to evaluate the safety and effectiveness of an experimental drug called PR001A as a possible treatment for Parkinson’s disease in subjects with the presence of a specific gene mutation called GBA1. An experimental drug is one that is being tested and is not approved by the United States Food and Drug Administration (FDA). PR001A has not been tested previously in humans. It has been tested in animal experiments (mice and non-human primates) where no related adverse events (side effects) were observed at the dose that will be used in this clinical trial. There are currently no treatments that target the progression of the disease or that are specifically developed for patients who carry GBA1 mutations. The purpose of this study is to find out what effects, good or bad, PR001A has on Parkinson’s disease. PR001A is designed to deliver a normal GBA1 gene copy to increase the activity of an enzyme which is low in Parkinson’s disease patients with a GBA1 mutation.

Clinical Study Identifier: s18-01416
ClinicalTrials.gov Identifier: NCT04127578
Principal Investigator: Steven J. Frucht.
Other Investigator: Christine M. Stahl.

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