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Phase 1/2 Clinical Trial of PR001 in Infants With Type 2 Gaucher Disease (PROVIDE)
NCT04411654 · View on ClinicalTrials.gov ↗
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 Studied
Interventions
- DRUG Sirolimus
- DRUG Prednisone
- DRUG Methylprednisolone
- GENETIC LY3884961
Study Locations (5)
California
- UCSF Benioff Children's Hospital, 5700 Martin Luther King Jr Way — Oakland
Minnesota
- University of Minnesota Masonic Children's Hospital, 2450 Riverside Avenue — Minneapolis
Pennsylvania
- Children's Hospital of Pittsburgh, 4401 Penn Avenue — Pittsburgh
Virginia
- Lysosomal & Rare Disorders Research and Treatment Center — Fairfax
Other
- Manchester Centre for Genomic Medicine, 6th Floor, St Mary's Hospital, Oxford Road — Manchester
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 7 participants |
| Start Date | 2021-06-29 |
| Est. Completion | 2028-05 |
| Phase | Phase 1 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT04411654
The ClinicalTrials.gov registry entry for NCT04411654 describes a study currently listed as active not recruiting. It is categorized as Phase 1, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 7 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Prevail Therapeutics, which has 5 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.
The record links to 1 condition, with Gaucher Disease, Type 2 appearing as the primary indexed condition, and to 4 interventions — of which Sirolimus is the first listed. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.
Geographic footprint matters for practical reasons: NCT04411654 reports 5 study locations spanning 5 distinct geographic areas — top geographies include California, Minnesota, Pennsylvania. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.
Frequently Asked Questions
What is clinical trial NCT04411654 about?
NCT04411654 is a clinical study titled "Phase 1/2 Clinical Trial of PR001 in Infants With Type 2 Gaucher Disease (PROVIDE)". 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 dos...
What is the current status of trial NCT04411654?
This trial is currently active not recruiting. It is a Phase 1 study. The enrollment target is 7 participants. The study started on 2021-06-29. Estimated completion is 2028-05.
What conditions does trial NCT04411654 study?
This clinical trial studies the following conditions: Gaucher Disease, Type 2. These conditions were identified from the trial registry and reflect the primary focus areas of the research.
What interventions are being tested in trial NCT04411654?
The interventions under investigation include: Sirolimus (DRUG), Prednisone (DRUG), Methylprednisolone (DRUG), LY3884961 (GENETIC). Each intervention is being evaluated for safety and efficacy as part of this clinical study.
Who is sponsoring clinical trial NCT04411654?
This trial is sponsored by Prevail Therapeutics, which has 5 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.
Where is trial NCT04411654 being conducted?
This trial has 5 study locations across California, Minnesota, Pennsylvania, Virginia. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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