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A Clinical Trial of PR001 (LY3884961) in Patients With Peripheral Manifestations of Gaucher Disease (PROCEED)
NCT05487599 · View on ClinicalTrials.gov ↗
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.
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 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT05487599
The ClinicalTrials.gov registry entry for NCT05487599 describes a study currently listed as 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 15 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 2 conditions, with Gaucher Disease appearing as the primary indexed condition, and to 1 intervention — of which LY3884961 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: NCT05487599 reports 8 study locations spanning 6 distinct geographic areas — top geographies include Other, Illinois, North Carolina. 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 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. These conditions were identified from the trial registry and reflect the primary focus areas of the research.
What interventions are being tested in trial NCT05487599?
The interventions under investigation include: LY3884961 (GENETIC). Each intervention is being evaluated for safety and efficacy as part of this clinical study.
Who is sponsoring clinical trial NCT05487599?
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 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.
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