Actively Recruiting
A Clinical Trial of PR001 (LY3884961) in Patients With Peripheral Manifestations of Gaucher Disease (PROCEED)
Led by Prevail Therapeutics · Updated on 2026-04-15
15
Participants Needed
8
Research Sites
466 weeks
Total Duration
On this page
Sponsors
P
Prevail Therapeutics
Lead Sponsor
E
Eli Lilly and Company
Collaborating Sponsor
AI-Summary
What this Trial Is About
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
Official Title
A Clinical Trial of PR001 (LY3884961) in Patients With Peripheral Manifestations of Gaucher Disease (PROCEED)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Age 18 years or older at consent
- Confirmed bi-allelic pathogenic GBA1 variants
- On enzyme replacement or substrate reduction therapy for at least 2 years, stable dose for 3 months before screening
- Able to provide signed informed consent and comply with study requirements
- Both females and males eligible; those of childbearing potential must use effective contraception throughout the study
- Agree to abstain from blood, tissue, and organ donation during the study and follow-up
You will not qualify if you...
- Significant neurological symptoms or behavioral disturbances
- Active progressive bone disease likely needing surgery within 6 months
- History or planned total splenectomy during first 18 months (partial splenectomy allowed)
- Splenomegaly larger than 10 MN by MRI
- Significant liver disease, fragile liver, or hepatotoxin exposure history
- Platelet count below 40 x 10^3 per microliter
- Severe hyperlipidemia (triglycerides over 1,000 mg/dL)
- Unstable or significant cardiovascular conditions
- Cancer history within 5 years before screening
- Other diseases or treatments posing unacceptable risk or interfering with study
- Pregnant, breastfeeding, or intending pregnancy during study
- Use of GD-related chaperone therapy within 4 weeks before screening or expected need during first 18 months
- Previous gene or cell therapy
- Use of systemic immunosuppressants or steroids except protocol-specified
- Participation in another investigational drug or device study within 3 months or 5 half-lives
- Anti-AAV9 antibody titer greater than 1:40
- Significant abnormal lab test results at screening
- Contraindications for MRI, including claustrophobia or ferromagnetic implants/pacemaker
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 8 locations
1
Ann and Robert H Lurie Children's Hospital of Chicago
Chicago, Illinois, United States, 60611
Actively Recruiting
2
Duke University Health System
Durham, North Carolina, United States, 27710-3017
Actively Recruiting
3
Lysosomal & Rare Disorders Research and Treatment Center
Fairfax, Virginia, United States, 22030-6066
Actively Recruiting
4
Westmead Hospital-Cnr Hawkesbury and Darcy Rds
Westmead, New South Wales, Australia, 2145
Completed
5
Hospital de Clinicas de Porto Alegre (HCPA)
Porto Alegre, Rio Grande do Sul, Brazil, 90035-903
Actively Recruiting
6
SphinCS Clinical Science for LSD
Höchheim, Germany, 65239
Actively Recruiting
7
Hospital Quironsalud Zaragoza, Paseo Mariano Renovales Sn
Zaragoza, Spain, 50006
Actively Recruiting
8
Royal Free Hospital NHS Trust
London, United Kingdom
Actively Recruiting
Research Team
P
Prevail Therapeutics
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
1
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