Phase 1/2 Clinical Trial of PR001 in Infants With Type 2 Gaucher Disease (PROVIDE)
Public ClinicalTrials.gov record NCT04411654. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
An Open-label, Phase 1/2 Study to Evaluate the Safety and Efficacy of Single-dose LY3884961 in Infants With Type 2 Gaucher Disease
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
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.
Study identification
- NCT ID
- NCT04411654
- Recruitment status
- Active, not recruiting
- Study type
- Interventional
- Phase
- Phase 1, Phase 2
- Enrollment
- 7 participants
Conditions and interventions
Conditions
Interventions
- LY3884961 Genetic
- Methylprednisolone Drug
- Sirolimus Drug
- Prednisone Drug
Genetic · Drug
Eligibility (public fields only)
- Age range
- 0 Months to 24 Months
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Jun 28, 2021
- Primary completion
- Jan 31, 2027
- Completion
- Jan 31, 2027
- Last update posted
- Aug 18, 2026
2021 – 2027
United States locations
- U.S. sites
- 5
- U.S. states
- 5
- U.S. cities
- 5
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| UCSF Benioff Children's Hospital, 747 52nd St | Oakland | California | 94609 | — |
| University of Minnesota Masonic Children's Hospital, 2450 Riverside Avenue | Minneapolis | Minnesota | 55454 | — |
| NYU Medical Center, 305 Second Ave, Suite 16 | New York | New York | 10003 | — |
| Children's Hospital of Pittsburgh, 4401 Penn Avenue | Pittsburgh | Pennsylvania | 15224 | — |
| Lysosomal & Rare Disorders Research and Treatment Center | Fairfax | Virginia | 22030 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
Non-U.S. locations
This page focuses on the U.S. directory. The official record also lists 1 non-U.S. site.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT04411654, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Aug 18, 2026 · Synced Sep 5, 2026
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Open the official record
The complete protocol, eligibility criteria, and contact information for NCT04411654 live on ClinicalTrials.gov.