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NCT04411654 · ClinicalTrials.gov registry record · Phase 1
Phase 1/2 Clinical Trial of PR001 in Infants With Type 2 Gaucher Disease (PROVIDE)
A Phase 1 study of Gaucher Disease, Type 2, sponsored by Prevail Therapeutics.
- Active
- Registry status
- Phase 1
- Development phase
- 7
- Enrollment target
- 5
- Study locations
NCT04411654: Active Phase 1 study of Gaucher Disease, Type 2, sponsored by Prevail Therapeutics.
NCT04411654 is a Phase 1 study of Gaucher Disease, Type 2 that is active but no longer recruiting, run by Prevail Therapeutics. The registered enrollment target is 7 participants, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (88% lower). The trial reports 5 study locations across 5 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT04411654, a Phase 1 study of Gaucher Disease, Type 2, is active but no longer recruiting, sponsored by Prevail Therapeutics.
- ACTIVE NOT RECRUITING
- Registry status
- Phase 1
- Development phase
- 7 participants
- Enrollment target
- 5
- Study locations
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 |
What the registry record for NCT04411654 still lists
NCT04411654 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 7 participants, a relatively small participant target, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (88% lower).
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.
NCT04411654 lists 5 locations in 5 states (California, Minnesota, Pennsylvania).
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.
What interventions are being tested in trial NCT04411654?
The interventions under investigation include: Sirolimus (DRUG), Prednisone (DRUG), Methylprednisolone (DRUG), LY3884961 (GENETIC).
Who is sponsoring clinical trial NCT04411654?
This trial is sponsored by Prevail Therapeutics, which has 5 total clinical trials registered on ClinicalTrials.gov.
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.
Learn More About Clinical Trials
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Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.
Where NCT04411654's enrollment target sits among peer trials
7 1972nd of 2000 higher than 27 of 2,000 other Phase 1 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 1 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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