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Phase 1/2 Clinical Trial of PR001 in Infants With Type 2 Gaucher Disease (PROVIDE)

StatusActive, Not Recruiting
PhasePhase 1/2
Started2021-06-29
View on ClinicalTrials.gov ↗
J3Z-MC-OJAB is an open-label, Phase 1/2, multicenter study to evaluate the safety and efficacy of single-dose LY3884961 (formerly PR001) in infants diagnosed with Type 2 Gaucher disease (GD2). For each patient, the study will be approximately 5 years in duration. During the first 12 months after dosing, patients will be evaluated for the effects of LY3884961 on safety, tolerability, immunogenicity, biomarkers, and efficacy. Patients will be followed up for an additional 4 years to monitor safety and changes on selected biomarkers and clinical outcomes.

Amendment history 2 changes detected by DataLookout

2026-08-20
Primary completion moved earlier: 2028-05 → 2027-02
2026-08-20
Completion moved earlier: 2028-05 → 2027-02
Trial Details
NCT Number NCT04411654
Lead Sponsor Prevail Therapeutics
Collaborators: Eli Lilly and Company
Conditions Gaucher Disease, Type 2
Enrollment 7 participants
Start Date 2021-06-29
Primary Completion 2027-02 (estimated)
Study Completion 2027-02 (estimated)
Updated on ClinicalTrials.gov 2026-08-19