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

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ClinicalTrials.gov Identifier: NCT05487599
Recruitment Status : Recruiting
First Posted : August 4, 2022
Last Update Posted : May 13, 2024
Sponsor:
Collaborator:
Eli Lilly and Company
Information provided by (Responsible Party):
Prevail Therapeutics

Study Description
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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 into an expansion cohort.

For each enrolled patient, the study will be approximately 5 years in duration, including up to a 45-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 up for an additional 42 months to monitor safety, immunogenicity, and selected biomarker and efficacy parameters.


Condition or disease Intervention/treatment Phase
Gaucher Disease Gaucher Disease, Type 1 Genetic: LY3884961 Phase 1 Phase 2

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 15 participants
Allocation: N/A
Intervention Model: Sequential Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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)
Actual Study Start Date : December 20, 2022
Estimated Primary Completion Date : October 2, 2030
Estimated Study Completion Date : October 2, 2030

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Gaucher Disease

Arms and Interventions
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Arm Intervention/treatment
Experimental: LY3884961
LY3884961 is an advanced therapy investigational medicinal product administered as a single intravenous infusion.
 Genetic: 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.


Outcome Measures
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Primary Outcome Measures :
  1. Incidence and severity of Treatment-emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) [ Time Frame: 5 years ]
    Treatment-emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) with AE's graded as mild, moderate, or severe.


Secondary Outcome Measures :
  1. Spleen volume [ Time Frame: 5 years ]
    Change and percent change from baseline

  2. Platelet count [ Time Frame: 5 years ]
    Change from baseline

  3. GCase levels [ Time Frame: 5 years ]
    Change from baseline

  4. GluSph levels [ Time Frame: 5 years ]
    Change from baseline

  5. Discontinuation of enzyme replacement therapy (ERT)/substrate reduction therapy (SRT) [ Time Frame: 5 years ]
    Time from dosing to discontinuation of ERT/SRT

  6. Re-initiation of ERT/SRT (if necessary) [ Time Frame: 5 years ]
    Time to re-initiation of ERT/SRT (if necessary) Time to re-initiation of ERT/SRT (if necessary)


Eligibility Criteria
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Ages Eligible for Study:   18 Years to 65 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Age 18-65 years inclusive at the time of informed consent.
  2. Bi-allelic GBA1 mutations 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 donations for at least the first year of the study.

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 MRI
  5. Evidence of clinically significant liver disease, fragile liver, or history of exposure to hepatotoxins.
  6. Thrombocytopenia with platelet count < 40 × 103 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 Day 1) 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 magnetic resonance imaging (MRI), including claustrophobia or the presence of contraindicated metal (ferromagnetic)implants/cardiac pacemaker.
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05487599


Contacts
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Contact: Prevail Therapeutics (917) 336-9310 Prevail.Patients@lilly.com

Locations
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United States, North Carolina
Duke University Health System Recruiting
Durham, North Carolina, United States, 27710-3017
Contact: Gretchen Clinical Research Coordinator    919-660-0757    Gretchen.nichting@duke.edu   
United States, Virginia
Lysosomal Rare Disorders Research and Treatment Center Recruiting
Fairfax, Virginia, United States, 22030-6066
Contact: Lauren Noll    571-732-4655    lnoll@ldrtc.org   
Germany
SphinCS Clinical Science for LSD Recruiting
Hochheim, Germany, 65239
Contact: Eugen Mengel    496146904820    eugen.mengel@sphincs.de   
Spain
Hospital Universitario Ramon y Cajal, Calle Colmenar Viejo Km 9100 Recruiting
Madrid, Spain, 28034
Contact: Jesus Villarrubia    +34913368967    jesus.villarrubia@salud.madrid.org   
Hospital Quironsalud Zaragoza, Paseo Mariano Renovales Sn Recruiting
Zaragoza, Spain, 50006
Contact: Teresa Navarro    +690762382    teresanair@feeteg.org   
Sponsors and Collaborators
Prevail Therapeutics
Eli Lilly and Company
Investigators
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Study Director: Sarah Neuhaus, DO Prevail Therapeutics, a wholly owned subsidiary of Eli Lilly and Company
More Information
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Responsible Party: Prevail Therapeutics
ClinicalTrials.gov Identifier: NCT05487599    
Other Study ID Numbers: J3Z-MC-OJAE
First Posted: August 4, 2022    Key Record Dates
Last Update Posted: May 13, 2024
Last Verified: May 2024
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Gaucher Disease
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
Lipidoses
Lipid Metabolism, Inborn Errors
Lysosomal Storage Diseases
Metabolic Diseases
Lipid Metabolism Disorders