Actively Recruiting
An Open-label, Dose-Finding, Phase 1/2 Study to Evaluate the Safety and Tolerability of a Single Intravenous Dose of LY3884961 in Patients With Peripheral Manifestations of Gaucher Disease (PROCEED)
Led by Prevail Therapeutics · Updated on 2026-05-22
15
Participants Needed
9
Research Sites
N/A
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
Researchers are conducting a Phase 1/2, multicenter, open-label study to evaluate the safety and tolerability of LY3884961 in adults with peripheral manifestations of Gaucher Disease. The study focuses on finding the right dose of LY3884961, a gene therapy delivered as a single intravenous infusion. The trial aims to assess safety, immune response, biomarkers, and efficacy over time in patients who have specific genetic variants and have been on enzyme replacement or substrate reduction therapy. The study includes up to three dose-finding groups, each with three patients, followed by an expansion group of up to six patients. Patients receive one dose of LY3884961 and are monitored closely. The total participation time is about five years, with an initial 60-day screening period. During the first 18 months after dosing, detailed evaluations of safety, immune response, biomarkers, and efficacy take place, followed by an extended 42-month follow-up focusing on safety and selected measures. Participants will undergo regular assessments including monitoring for adverse events, blood tests for platelet counts and Gaucher disease markers, and imaging to evaluate spleen volume. Researchers will track treatment-emergent adverse events and serious adverse events for five years. The study also monitors whether patients can reduce or stop their enzyme replacement or substrate reduction therapies. Long-term follow-up helps ensure ongoing evaluation of safety and treatment effects throughout the study duration.
CONDITIONS
Brief 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 the time of informed consent
- Confirmed bi-allelic pathogenic GBA1 variants
- On enzyme replacement or substrate reduction therapy for at least 2 years, stable at maximum tolerated dose for at least 3 months prior to screening
- Able to provide signed informed consent and comply with study requirements
- Men and women eligible; those of childbearing potential must use highly 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...
- Clinically significant neurological signs, symptoms, or behavioral disturbances
- Active and progressive bone disease expected to require surgery within 6 months
- History of total splenectomy or planned total splenectomy in first 18 months (partial splenectomy allowed)
- Splenomegaly greater than 10 MN by central MRI
- Significant liver disease, fragile liver, or exposure to hepatotoxins
- Platelet count below 40 x 10^3 per µL
- Severe hyperlipidemia with triglycerides over 1,000 mg/dL
- Unstable or significant cardiovascular conditions
- History of certain cancers within 5 years of screening
- Other diseases or treatments posing unacceptable risk or interfering with study compliance
- Pregnant, breastfeeding, or intending pregnancy during trial
- Use of GD-related chaperone therapy within 4 weeks prior to screening or expected need during first 18 months
- Prior gene or cell therapy
- Use of systemic immunosuppressants or steroids except protocol-specified
- Participation in another investigational study within 3 months or 5 half-lives of that agent
- Anti-AAV9 antibody titer greater than 1:40
- Significant laboratory abnormalities at screening
- Contraindications for MRI including claustrophobia or ferromagnetic implants/cardiac pacemaker
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Single dose with safety monitoring up to 5 years
Participants receive a single intravenous infusion of LY3884961, an investigational gene therapy, and are monitored for safety and tolerability.
Multiple visits over 5 years for safety and efficacy assessments
Trial Site Locations
Total: 9 locations
1
Cedars-Sinai
Los Angeles, California, United States, 90048
Actively Recruiting
2
Ann and Robert H Lurie Children's Hospital of Chicago
Chicago, Illinois, United States, 60611
Actively Recruiting
3
Duke University Health System
Durham, North Carolina, United States, 27710-3017
Actively Recruiting
4
Lysosomal & Rare Disorders Research and Treatment Center
Fairfax, Virginia, United States, 22030-6066
Actively Recruiting
5
Westmead Hospital-Cnr Hawkesbury and Darcy Rds
Westmead, New South Wales, Australia, 2145
Completed
6
Hospital de Clinicas de Porto Alegre (HCPA)
Porto Alegre, Rio Grande do Sul, Brazil, 90035-903
Actively Recruiting
7
SphinCS Clinical Science for LSD
Höchheim, Germany, 65239
Actively Recruiting
8
Hospital Quironsalud Zaragoza, Paseo Mariano Renovales Sn
Zaragoza, Spain, 50006
Actively Recruiting
9
Royal Free Hospital NHS Trust
London, United Kingdom
Actively Recruiting
Research Team
P
Prevail Therapeutics
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
1
Similar Trials
Frequently Asked Questions
Have more questions? Get in touch with our team for quick support
Not the Right Trial for You?
Explore thousands of other clinical trials that might be a better match.
Sign up to get personalized trial recommendations delivered to your inbox.
Already have an account? Log in here