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NCT05487599 · ClinicalTrials.gov registry record · Phase 1
A Clinical Trial of PR001 (LY3884961) in Patients With Peripheral Manifestations of Gaucher Disease (PROCEED)
A Phase 1 study of Gaucher Disease and Gaucher Disease, Type 1, sponsored by Prevail Therapeutics.
- Recruiting
- Registry status
- Phase 1
- Development phase
- 15
- Enrollment target
- 8
- Study locations
NCT05487599: Recruiting Phase 1 study of Gaucher Disease and Gaucher Disease, Type 1, sponsored by Prevail Therapeutics.
NCT05487599 is a Phase 1 study of Gaucher Disease and Gaucher Disease, Type 1 that is actively recruiting participants, run by Prevail Therapeutics. The registered enrollment target is 15 participants, below the 1,057-participant average among 13 other Gaucher Disease trials with a reported enrollment target (99% lower). The trial reports 8 study locations across 6 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT05487599, a Phase 1 study of Gaucher Disease and Gaucher Disease, Type 1, is actively recruiting participants, sponsored by Prevail Therapeutics.
- RECRUITING
- Registry status
- Phase 1
- Development phase
- 15 participants
- Enrollment target
- 8
- Study locations
Study Summary
Study J3Z-MC-OJAE is a Phase 1/2, multicenter, open-label, dose-finding study of LY3884961 evaluating the safety and tolerability in adults with peripheral manifestations of GD. Up to 3 dose levels of LY3884961 will be assessed in 3 dose-finding cohorts of 3 patients. Following this, up to 6 patients may be enrolled in an expansion cohort. For each enrolled patient, the study will be approximately 5 years in duration, including up to a 60-day screening period. During the first 18 months after dosing, subjects will be evaluated for the effects of LY3884961 on safety, tolerability, immunogenicity, biomarkers, and efficacy. Patients will be followed for an additional 42 months to monitor safety, immunogenicity, and selected biomarker and efficacy parameters.
Primary Outcome
Treatment-emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) with AE's graded as mild, moderate, or severe.
Conditions Studied
Interventions
- GENETIC LY3884961
Study Locations (8)
Other
- SphinCS Clinical Science for LSD - Höchheim
- Hospital Quironsalud Zaragoza, Paseo Mariano Renovales Sn - Zaragoza
- Royal Free Hospital NHS Trust - London
Illinois
- Ann and Robert H Lurie Children's Hospital of Chicago - Chicago
North Carolina
- Duke University Health System - Durham
Virginia
- Lysosomal & Rare Disorders Research and Treatment Center - Fairfax
New South Wales
- Westmead Hospital-Cnr Hawkesbury and Darcy Rds - Westmead
Rio Grande do Sul
- Hospital de Clinicas de Porto Alegre (HCPA) - Porto Alegre
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 15 participants |
| Start Date | 2022-12-20 |
| Est. Completion | 2031-11-30 |
| Phase | Phase 1 |
What NCT05487599 shows while recruiting
NCT05487599 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 15 participants, a relatively small participant target, below the 1,057-participant average among 13 other Gaucher Disease trials with a reported enrollment target (99% lower).
The record links to 2 conditions, with Gaucher Disease appearing as the primary indexed condition, and to 1 intervention - of which LY3884961 is the first listed.
NCT05487599 lists 8 locations in 6 states (Other, Illinois, North Carolina).
Frequently Asked Questions
What is clinical trial NCT05487599 about?
NCT05487599 is a clinical study titled "A Clinical Trial of PR001 (LY3884961) in Patients With Peripheral Manifestations of Gaucher Disease (PROCEED)". Study J3Z-MC-OJAE is a Phase 1/2, multicenter, open-label, dose-finding study of LY3884961 evaluating the safety and tolerability in adults with peripheral manifestations of GD. Up to 3 dose levels of LY3884961 will be assessed in 3 dose-finding cohorts of 3 patients. Following this, up to 6 patien...
What is the current status of trial NCT05487599?
This trial is currently recruiting. It is a Phase 1 study. The enrollment target is 15 participants. The study started on 2022-12-20. Estimated completion is 2031-11-30.
What conditions does trial NCT05487599 study?
This clinical trial studies the following conditions: Gaucher Disease, Gaucher Disease, Type 1.
What interventions are being tested in trial NCT05487599?
The interventions under investigation include: LY3884961 (GENETIC).
Who is sponsoring clinical trial NCT05487599?
This trial is sponsored by Prevail Therapeutics, which has 5 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT05487599 being conducted?
This trial has 8 study locations across Illinois, North Carolina, Virginia, New South Wales, Rio Grande do Sul. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
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Where NCT05487599's enrollment target sits among peer trials
15 13th of 13 higher than 1 of 13 other Gaucher Disease trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Gaucher Disease 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.
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