Phase 1/2 Clinical Trial of PR001 in Infants With Type 2 Gaucher Disease (PROVIDE)
Active, not recruiting · Phase 1, Phase 2 · NCT04411654
Conditions: Gaucher Disease, Type 2
At a glance
- Research payments reported, 2025
- $274,759
- U.S. investigators named
- 3
- Sites paid
- 4
- Planned or actual enrollment
- 7
About the trial
- Sponsor
- Prevail Therapeutics (with Eli Lilly and Company)
- Tested
- LY3884961 (genetic), Methylprednisolone (drug), Sirolimus (drug), Prednisone (drug)
- Dates
- Started 2021-06-29; completion 2027-02
- Results posted
- Not yet
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.
From ClinicalTrials.gov, last updated 2026-10-22. Full record, eligibility and contacts (opens in a new tab)
Industry funding
Eli Lilly and Company reported $274,759 in research payments for this study in 2025 (4 payments).
Research payments usually go to hospitals, universities and research sites to run a study, not to the investigators personally. A payment is not evidence of wrongdoing.
U.S. investigators named on this study
- Paul Harmatz, MD Pediatric Gastroenterology · Oakland, CA
- Deepa Soundara Rajan Pediatrics · Detroit, MI
- Chester B. Whitley, MD Pediatrics · Minneapolis, MN
Clinicians the company named as principal investigators in its Open Payments reports.
Sites paid to run it
- University of California Davis Medical Center, CA $194,318
- University of Pittsburgh $45,925
- Univ of Pittsburgh Cancer Institute Cancer Srvcs $23,707
- Regents of the University of Minnesota $10,809
Sources: CMS Open Payments research payments (program year 2025) and ClinicalTrials.gov, U.S. National Library of Medicine.