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Clinical Trial Report

Gene Therapy Trial Report

Summary

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


NCTID NCT04127578 (View at clinicaltrials.gov)
Description
Development Status Active
Indication Parkinson Disease
Disease Ontology Term DOID:14330
Compound Name PR001
Compound Alias LY3884961
Compound Description AAV9-CMV-CBA-GBA1
Sponsor Prevail Therapeutics
Funder Type Industry
Recruitment Status
Recruiting
Enrollment Count 20
Results Posted Not Available

Therapy Information


Target Gene/Variant GBA1
Therapy Type Gene transfer
Therapy Route In-vivo
Mechanism of Action Overexpression of protective allele/gene
Route of Administration Intracisterna magna
Drug Product Type Viral vector
Target Tissue/Cell
Delivery System Viral transduction
Vector Type AAV9
Editor Type none
Dose 1 Undisclosed dose 1
Dose 2 Undisclosed dose 2
Dose 3
Dose 4
Dose 5

Study Record Dates


Current Stage Phase1, Phase2
Submit Date 2019-10-14
Completion Date 2030-12-31
Last Update 2025-04-10

Participation Criteria


Eligible Age 35 Years - 80 Years
Standard Ages Adult, Older adult
Sexes Eligible for Study ALL

Locations


No.of Trial Sites 13
Locations Netherlands,United States,Israel

Regulatory Information


Has US IND True
FDA Designations Fast Track
Recent Updates

Resources/Links