NCT04127578

Phase 1/2a Clinical Trial of PR001 (LY3884961) 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 LY3884961 in Patients With Parkinson's Disease With at Least One GBA1 Mutation (PROPEL)

• Status: Active Not Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 32 (estimated)
• Sponsor: Prevail Therapeutics · Industry
• Sex: All
• Age: 35 Years – 80 Years
• Healthy volunteers: Not accepted

Summary

Study J3Z-MC-OJAA is a Phase 1/2a, multicenter, open-label, ascending dose, first in-human study that will evaluate the safety of intracisternal LY3884961 administration in patients with moderate to severe Parkinson's disease with at least 1 pathogenic GBA1 mutation. Two dose level cohorts of LY3884961 are planned (Dose Level 1 and Dose Level 2). The duration of the study is 5 years. During the first year, patients will be evaluated for the effect of LY3884961 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.

Conditions

• Parkinson Disease

Interventions

Type	Name	Description
BIOLOGICAL	LY3884961	Participants will receive a single dose of LY3884961, administered intra-cisterna magna
DRUG	Methylprednisolone	6 IV pulses administered as concomitant medication over 3 months
DRUG	Sirolimus	Back-up treatment if corticosteroid is not well tolerated. Loading dose, followed by maintenance dose, followed by dose tapering; administered as concomitant medication

Timeline

• Start date: 2020-01-03
• Primary completion: 2030-12-31
• Completion: 2030-12-31
• First posted: 2019-10-15
• Last updated: 2026-03-13

Locations

15 sites across 2 countries: United States, Israel

Regulatory

• FDA-regulated drug study