A Clinical Trial of PR001 (LY3884961) in Patients With Peripheral Manifestations of Gaucher Disease (PROCEED)

NCT05487599 | Recruiting Phase 1 FDA Drug

• 1 other identifier: J3Z-MC-OJAE
• Study Type: interventional
• Target: 15
• Locations: 6 countries
• Sites: 8

Brief 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

Gaucher Disease; Gaucher Disease, Type 1

Outcome Measures

Primary Outcomes (1)

• Incidence and severity of Treatment-emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)

  Treatment-emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) with AE's graded as mild, moderate, or severe.

  5 years

Secondary Outcomes (6)

• Spleen volume

  5 years

• Platelet count

  5 years

• GCase levels

  5 years

• GlcSph levels

  5 years

• Discontinuation of enzyme replacement therapy (ERT)/substrate reduction therapy (SRT)

  5 years

Study Arms (1)

LY3884961

EXPERIMENTAL

LY3884961 is an advanced therapy investigational medicinal product administered as a single intravenous infusion.

Genetic: LY3884961

Interventions

LY3884961 GENETIC

• 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.

LY3884961

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Age greater or equal to 18 years at the time of informed consent.
• Bi-allelic pathogenic GBA1 variants must be centrally confirmed.
• On ERT or SRT for at least 2 years and on a stable, maximum tolerated dose, for at least 3 months prior to screening.
• Capable of giving signed informed consent, including compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol.
• 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.
• 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.

You may not qualify if:

• Clinically significant neurological signs and symptoms and/or behavioral disturbances.
• Active and progressive bone disease expected to require surgical treatment in the next 6 months.
• Splenomegaly \> 10 MN as evaluated by centrally read abdominal magnetic resonance imaging (MRI)
• Evidence of clinically significant liver disease, fragile liver, or history of exposure to hepatotoxins.
• Thrombocytopenia with platelet count \< 40 × 10\^3 per μL.
• Severe hyperlipidemia (triglycerides \> 1,000 mg/dL).
• Current diagnosis of unstable or clinically significant cardiovascular conditions based on Investigator assessment.
• History of certain cancers within 5 years of Screening.
• 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.
• 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.
• 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.
• Any type of prior gene or cell therapy.
• Use of systemic immunosuppressant or steroid therapy other than protocol-specified immunosuppression.
• Participation in another therapeutic investigational drug or device study within 3 months or 5 half-lives of the study agent, whichever is longer.
• Have an anti-AAV9 antibody titer of \>1:40 as determined by central laboratory.

Sponsors & Collaborators

• Prevail Therapeutics: lead
• Eli Lilly and Company: collaborator

Study Sites (8)

Ann and Robert H Lurie Children's Hospital of Chicago

Chicago, Illinois, 60611, United States

RECRUITING

Duke University Health System

Durham, North Carolina, 27710-3017, United States

RECRUITING

Lysosomal & Rare Disorders Research and Treatment Center

Fairfax, Virginia, 22030-6066, United States

RECRUITING

Westmead Hospital-Cnr Hawkesbury and Darcy Rds

Westmead, New South Wales, 2145, Australia

COMPLETED

Hospital de Clinicas de Porto Alegre (HCPA)

Porto Alegre, Rio Grande do Sul, 90035-903, Brazil

RECRUITING

SphinCS Clinical Science for LSD

Höchheim, 65239, Germany

RECRUITING

Hospital Quironsalud Zaragoza, Paseo Mariano Renovales Sn

Zaragoza, 50006, Spain

RECRUITING

Royal Free Hospital NHS Trust

London, United Kingdom

RECRUITING

MeSH Terms

Conditions

Gaucher Disease

Condition Hierarchy (Ancestors)

Sphingolipidoses; Lysosomal Storage Diseases, Nervous System; Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Lipidoses; Lipid Metabolism, Inborn Errors; Lysosomal Storage Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases; Lipid Metabolism Disorders

Study Officials

• Aaron Tward, MD, PhD

  Prevail Therapeutics, a wholly owned subsidiary of Eli Lilly and Company

  STUDY DIRECTOR

Central Study Contacts

Prevail Therapeutics

CONTACT

(917) 336-9310; Prevail.Patients@lilly.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 1, 2022
• First Posted: August 4, 2022
• Study Start: December 20, 2022
• Primary Completion (Estimated): November 30, 2031
• Study Completion (Estimated): November 30, 2031
• Last Updated: April 15, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will not share

Locations

DE (1); BR (1); ES (1); US (3); GB (1); AU (1)