A Clinical Trial of PR001 (LY3884961) in Patients With Peripheral Manifestations of Gaucher Disease (PROCEED)
NCT ID: NCT05487599
Last Updated: 2025-12-08
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.
RECRUITING
PHASE1/PHASE2
15 participants
INTERVENTIONAL
2022-12-20
2031-11-30
Brief Summary
Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.
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.
Related Clinical Trials
Explore similar clinical trials based on study characteristics and research focus.
Phase 1/2 Clinical Trial of PR001 in Infants With Type 2 Gaucher Disease (PROVIDE)
NCT04411654
Clinical Study of LY-M001 Injection in the Treatment of Adolescents With Type I Gaucher Disease
NCT06528080
A Gaucher Disease Gene Therapy Trial With FLT201
NCT07223944
A Gene Therapy Study in Patients With Gaucher Disease Type 1
NCT05324943
A Multicenter Extension Study of Taliglucerase Alfa in Adult Subjects With Gaucher Disease
NCT01422187
Detailed Description
Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.
Conditions
See the medical conditions and disease areas that this research is targeting or investigating.
Study Design
Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.
NA
SEQUENTIAL
TREATMENT
NONE
Study Groups
Review each arm or cohort in the study, along with the interventions and objectives associated with them.
LY3884961
LY3884961 is an advanced therapy investigational medicinal product administered as a single intravenous infusion.
LY3884961
• LY3884961 is a replication-incompetent recombinant adeno-associated virus (AAV) vector. The vector is composed of a ss DNA genome packaged in an AAV-derived protein capsid.
Interventions
Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.
LY3884961
• LY3884961 is a replication-incompetent recombinant adeno-associated virus (AAV) vector. The vector is composed of a ss DNA genome packaged in an AAV-derived protein capsid.
Eligibility Criteria
Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.
Inclusion Criteria
2. Bi-allelic pathogenic GBA1 variants must be centrally confirmed.
3. On ERT or SRT for at least 2 years and on a stable, maximum tolerated dose, for at least 3 months prior to screening.
4. Capable of giving signed informed consent, including compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol.
5. Females and males will be eligible for this study. Men and women of childbearing potential must use a highly effective method of contraception consistently and correctly for the duration of the study, including the long-term follow-up.
6. Patients must agree to abstain from blood, tissue and organ donation; and must agree to abstain from tissue and organ donation for the duration of the study, including long-term follow-up.
Exclusion Criteria
2. Active and progressive bone disease expected to require surgical treatment in the next 6 months.
3. History of total splenectomy or planned total splenectomy during the first 18 months of the study. (Partial splenectomy not exclusionary).
4. Splenomegaly \> 10 MN as evaluated by centrally read abdominal magnetic resonance imaging (MRI)
5. Evidence of clinically significant liver disease, fragile liver, or history of exposure to hepatotoxins.
6. Thrombocytopenia with platelet count \< 40 × 10\^3 per μL.
7. Severe hyperlipidemia (triglycerides \> 1,000 mg/dL).
8. Current diagnosis of unstable or clinically significant cardiovascular conditions based on Investigator assessment.
9. History of certain cancers within 5 years of Screening.
10. Concomitant disease, condition or treatment which, in the opinion of the Investigator, would pose an unacceptable risk to the patient or interfere with the patient's ability to comply with study procedures or interfere with the conduct of the study.
11. Women of childbearing potential, pregnant (i.e., positive serum pregnancy result at Screening and/or Check-in) or breastfeeding or intending to become pregnant during the course of the trial.
12. Use of any GD-related chaperone therapy within 4 weeks prior to Screening or expected need to initiate chaperone therapy during at least the first 18 months of the study.
13. Any type of prior gene or cell therapy.
14. Use of systemic immunosuppressant or steroid therapy other than protocol-specified immunosuppression.
15. Participation in another therapeutic investigational drug or device study within 3 months or 5 half-lives of the study agent, whichever is longer.
16. Have an anti-AAV9 antibody titer of \>1:40 as determined by central laboratory.
17. Clinically significant abnormalities in laboratory test results at Screening.
18. Have any contraindications for MRI, including claustrophobia or the presence of contraindicated metal (ferromagnetic)implants/cardiac pacemaker.
18 Years
ALL
No
Sponsors
Meet the organizations funding or collaborating on the study and learn about their roles.
Eli Lilly and Company
INDUSTRY
Prevail Therapeutics
INDUSTRY
Responsible Party
Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.
Principal Investigators
Learn about the lead researchers overseeing the trial and their institutional affiliations.
Sarah Neuhaus, DO
Role: STUDY_DIRECTOR
Prevail Therapeutics, a wholly owned subsidiary of Eli Lilly and Company
Locations
Explore where the study is taking place and check the recruitment status at each participating site.
Ann and Robert H Lurie Children's Hospital of Chicago
Chicago, Illinois, United States
Duke University Health System
Durham, North Carolina, United States
Lysosomal & Rare Disorders Research and Treatment Center
Fairfax, Virginia, United States
Westmead Hospital-Cnr Hawkesbury and Darcy Rds
Westmead, New South Wales, Australia
Hospital de Clinicas de Porto Alegre (HCPA)
Porto Alegre, Rio Grande do Sul, Brazil
SphinCS Clinical Science for LSD
Höchheim, , Germany
Hospital Quironsalud Zaragoza, Paseo Mariano Renovales Sn
Zaragoza, , Spain
Royal Free Hospital NHS Trust
London, , United Kingdom
Countries
Review the countries where the study has at least one active or historical site.
Central Contacts
Reach out to these primary contacts for questions about participation or study logistics.
Facility Contacts
Find local site contact details for specific facilities participating in the trial.
Other Identifiers
Review additional registry numbers or institutional identifiers associated with this trial.
J3Z-MC-OJAE
Identifier Type: -
Identifier Source: org_study_id
More Related Trials
Additional clinical trials that may be relevant based on similarity analysis.