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

RECRUITINGPHASE1/PHASE2

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

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)

NCT05487599•Prevail Therapeutics

View on ClinicalTrials.gov

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.

Eligibility

Age

18 - No limit years

Sex

ALL

Healthy Volunteers

Inclusion Criteria

•1. Age greater or equal to 18 years at the time of informed consent.
•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

•1. Clinically significant neurological signs and symptoms and/or behavioral disturbances.
•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.
+8 more criteria

Locations (8)

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

Key Dates

Start Date

December 20, 2022

Primary Completion Date

November 30, 2031

Completion Date

November 30, 2031

Last Updated

December 8, 2025

Enrollment

ESTIMATED participants

Conditions

Gaucher DiseaseGaucher Disease, Type 1

Interventions

LY3884961

GENETIC

Contacts

Prevail Therapeutics

CONTACT

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

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Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: December 8, 2025Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

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

Inclusion Criteria

Exclusion Criteria

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