Phase 1/2a Clinical Trial of PR001 in Patients With Parkinson's Disease With at Least One GBA1 Mutation (PROPEL)

A Phase 1/2a Open-Label Ascending Dose Study to Evaluate the Safety and Effects of PR001A in Patients With Parkinson's Disease With at Least One GBA1 Mutation

Study PRV-PD101 is a Phase 1/2a, multicenter, open-label, ascending dose, first in-human study that will evaluate the safety of intracisternal PR001 administration in patients with moderate to severe Parkinson's disease with at least 1 pathogenic GBA1 mutation. Two escalating dose cohorts are planned (low dose and high dose). The duration of the study is 5 years. During the first year, patients will be evaluated for the effect of PR001 on safety, tolerability, immunogenicity, biomarkers, and clinical efficacy measures. Patients will continue to be followed for an additional 4 years to continue to monitor safety as well as selected biomarker and efficacy measures.

Phase 1/2a Clinical Trial of PR001 in Patients With Parkinson's Disease With at Least One GBA1 Mutation (PROPEL)

Recruitment

Summary

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