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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 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. The trial reports 5 study locations across 5 states.
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 |
Interested in This Trial?
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, categorized as Phase 1. 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.
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 reports 5 study locations spanning 5 distinct geographic areas - top geographies include 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.
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