Phase 1/2a Clinical Trial of PR001 in Patients With Parkinson's Disease With at Least One GBA1 Mutation (PROPEL)
Status:
Recruiting
Trial end date:
2027-06-01
Target enrollment:
Participant gender:
Summary
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.