Who is eligible for clinical trial NCT05487599?

Participants must meet the protocol's inclusion criteria for Gaucher Disease. The stated age range is 18 Years. Confirm all requirements with the study coordinator.

Does clinical trial NCT05487599 provide travel or cost reimbursement?

Check with the site coordinator at the participating facility for travel and lodging stipend availability, and for which routine-care costs are covered.

🏛️ Verified NIH Record • Updated 2026-08-14

A Clinical Trial of PR001 (LY3884961) in Patients With Peripheral Manifestations of Gaucher Disease (PROCEED) (NCT05487599)

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)

🔵 Active, Not Recruiting Phase1 / Phase2 📋 Clinical Research Protocol Sponsor: Prevail Therapeutics

🩺 Plain-English Summary (8th-Grade Level)

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.

Who can join: To join this study, participants generally need to meet the listed inclusion criteria (6 items) and avoid the listed exclusions (19 items).

Location: Duke University Health System — Durham, North Carolina

Age range: 18 Years

🧪 Interventions in This Study

LY3884961 (GENETIC)

📋 "Do I Qualify?" — 1-Minute Self Screener

Instant Checklist

Check every box below. If you can check all of them, you may meet the study's basic screening requirements (final eligibility is confirmed by the site team).

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.

Usually NOT eligible if any of the following apply:

Exclusion Criteria:
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.
History of total splenectomy or planned total splenectomy during the first 18 months of the study. (Partial splenectomy not exclusionary).
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.

🔍 Extracted verbatim from the official NIH eligibility criteria. Always confirm with the study coordinator.

📍 All Participating Trial Locations (5 Sites)

Duke University Health System — Durham, North Carolina
Lysosomal & Rare Disorders Research and Treatment Center — Fairfax, Virginia
Westmead Hospital-Cnr Hawkesbury and Darcy Rds — Westmead, New South Wales
SphinCS Clinical Science for LSD — Höchheim,
Hospital Quironsalud Zaragoza, Paseo Mariano Renovales Sn — Zaragoza,

💰 Cost, Insurance & Patient Rights

100% Free Items: The investigational treatment and protocol-required procedures are supplied at zero cost by the sponsor.
Routine Patient Care: Under federal law (ACA § 2709) and many state statutes, routine care costs are covered during participation.
Voluntary Participation: You may withdraw at any time without affecting your standard medical care.

More studies in Gaucher Disease

International Collaborative Gaucher Group (ICGG) Gaucher Disease Registry & Pregnancy Sub-registry — Phoenix, Arizona
A Long-term Follow-up Study of Gaucher Disease — Durham, North Carolina
Gaucher Disease Outcome Survey (GOS) — Lexington, Massachusetts
Long Term Follow-Up for Safety of AVR-RD-02 — Iowa City, Iowa
Extracellular Vesicles as Potential Biomarkers and Therapeutic Target in Gaucher Disease — Minneapolis, Minnesota

Explore by condition: All Gaucher Disease clinical trials

💡 How to Participate

Check the checklist above to see if you may qualify.
Review the official NIH record below to find the coordinating site.
Ask about the visit schedule, what is covered at no cost, and any travel support.
Confirm with your own doctor before making a decision.

🏛️ View Official NIH Record ↗