Gaucher DiseasePhase 1/2NCT05487599
A Clinical Trial of PR001 (LY3884961) in Patients With Peripheral Manifestations of Gaucher Disease (PROCEED)
A Phase 1/2 study testing LY3884961 in people with Gaucher Disease and Gaucher Disease, Type 1, run by Prevail Therapeutics at 5 sites in 4 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Gaucher Disease, Gaucher Disease, Type 1
- What is being tested?
- LY3884961
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 8 participants
- Where
- 5 sitesin 4 countries
- Study dates
- 20 Dec 2022to 30 Aug 2031
- Phase
- Phase 1/2
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
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- 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.
- Clinically significant abnormalities in laboratory test results at Screening.
- Have any contraindications for MRI, including claustrophobia or the presence of contraindicated metal (ferromagnetic)implants/cardiac pacemaker.
Trial locations and contacts
5 sites in 4 countries.
5 sites in 4 countries
United States2 sites
Duke University Health System
Durham, North Carolina, 27710-3017
Lysosomal & Rare Disorders Research and Treatment Center
Fairfax, Virginia, 22030-6066
Australia1 site
Westmead Hospital-Cnr Hawkesbury and Darcy Rds
Westmead, New South Wales, 2145
Spain1 site
Hospital Quironsalud Zaragoza, Paseo Mariano Renovales Sn
Zaragoza, 50006
About this study
Official title: 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)
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
- Being tested
- LY3884961
- Medical subject headings
- Gaucher Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
LY3884961Genetic
- 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.
| Study group | Role | What they receive |
|---|---|---|
| LY3884961 | Experimental | LY3884961 is an advanced therapy investigational medicinal product administered as a single intravenous infusion. Receives: Genetic: LY3884961 |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 5 years
Secondary outcomes (6)
Spleen volume
Change and percent change from baseline
Measured: 5 years
Platelet count
Change from baseline
Measured: 5 years
GCase levels
Change from baseline
Measured: 5 years
GlcSph levels
Change from baseline
Measured: 5 years
Discontinuation of enzyme replacement therapy (ERT)/substrate reduction therapy (SRT)
Time from dosing to discontinuation of ERT/SRT
Measured: 5 years
Re-initiation of ERT/SRT (if necessary)
Time to development of criteria requiring re-initiation of ERT/SRT (if necessary)
Measured: 5 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Aug 2022
- Study start20 Dec 2022
- Primary completion30 Aug 2031 (estimated)
- Study completion30 Aug 2031 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Gaucher DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 8 participants (actual)
Sponsor and investigators
- Lead sponsor
- Prevail Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Eli Lilly and Company
- Responsible party
- Sponsor
- Investigators
- Aaron Tward, MD, PhD, Study director, Prevail Therapeutics, a wholly owned subsidiary of Eli Lilly and Company
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05487599
- Sponsor's protocol ID
- J3Z-MC-OJAE
- Status last verified
- Aug 2026
- First submitted
- 1 Jun 2022
- Last updated
- 17 Aug 2026
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No