NCT04411654

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

An Open-label, Phase 1/2 Study to Evaluate the Safety and Efficacy of Single-dose LY3884961 in Infants With Type 2 Gaucher Disease

• Status: Active Not Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 7 (actual)
• Sponsor: Prevail Therapeutics · Industry
• Sex: All
• Age: 0 Months – 24 Months
• Healthy volunteers: Not accepted

Summary

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.

Conditions

• Gaucher Disease, Type 2

Interventions

Type	Name	Description
GENETIC	LY3884961	Participants will receive a single dose of LY3884961 administered intracisternally.
DRUG	Methylprednisolone	Single IV pulse administered as concomitant medication.
DRUG	Sirolimus	Loading dose, followed by maintenance doses, followed by dose tapering; administered as concomitant medication.
DRUG	Prednisone	Administered orally as concomitant medication, followed by dose tapering.

Timeline

• Start date: 2021-06-29
• Primary completion: 2028-05-01
• Completion: 2028-05-01
• First posted: 2020-06-02
• Last updated: 2026-04-01

Locations

5 sites across 2 countries: United States, United Kingdom

Regulatory

• FDA-regulated drug study