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

NCT04411654 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 7

Last updated 2026-04-01

No results posted yet for this study

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

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.

Sponsors & Collaborators

Principal Investigators

  • Hamzeh Migdadi, M.D. · Prevail Therapeutics

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
0 Months
Max Age
24 Months
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2021-06-29
Primary Completion
2028-05-31
Completion
2028-05-31
FDA Drug
Yes

Countries

  • United States
  • United Kingdom

Study Locations

More Related Trials

Entities

Drugs
Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04411654 on ClinicalTrials.gov