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Clinical Trials/2022-500281-10-02
2022-500281-10-02
Recruiting
Phase 1/2

An Open-label, Dose-Finding, Phase 1/2 Study to Evaluate the Safety and Tolerability of a Single Intravenous Dose of LY3884961 in Subjects with Peripheral Manifestations of Gaucher Disease

Prevail Therapeutics Inc.3 sites in 2 countries6 target enrollmentStarted: September 5, 2023Last updated:
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Overview

Phase
Phase 1/2
Status
Recruiting
Enrollment
6
Locations
3
Primary Endpoint
Incidence and severity of TEAEs and SAEs, including clinically significant changes in vital signs, clinical laboratory parameter assessments, 12-lead ECGs, results of abdominal and bone MRIs, physical and neurological examinations, and waist circumference and weight over time.
OverviewStudy DesignEligibility CriteriaOutcomesInvestigatorsSitesRecords & IdentifiersSimilar Trials

Overview

Brief Summary

To evaluate the safety and tolerability of LY3884961

Study Design

Allocation
Not Applicable
Primary Purpose
Expansion Period
Masking
None

Eligibility Criteria

Ages
18 years to 65+ years (65+ Years, 18-64 Years)
Accepts Healthy Volunteers
Yes

Inclusion Criteria

  • •Age ≥ 18years 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 donations for at least the first year of the study; and must agree to abstain from tissue and organ donation for the duration of the study, including long-term follow-up.

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
  • •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 × 103 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.

Outcomes

Primary Outcomes

Incidence and severity of TEAEs and SAEs, including clinically significant changes in vital signs, clinical laboratory parameter assessments, 12-lead ECGs, results of abdominal and bone MRIs, physical and neurological examinations, and waist circumference and weight over time.

Incidence and severity of TEAEs and SAEs, including clinically significant changes in vital signs, clinical laboratory parameter assessments, 12-lead ECGs, results of abdominal and bone MRIs, physical and neurological examinations, and waist circumference and weight over time.

Secondary Outcomes

  • Change and percent change from baseline in spleen volume (MN)
  • Change from baseline in platelet count
  • Change from baseline in GCase enzyme activity and protein levels and GluSph levels
  • Time from LY3884961 to ERT/SRT discontinuation.
  • Time from discontinuation of ERT/SRT to re-initiation of ERT/SRT.

Investigators

Sponsor Class
Pharmaceutical company
Responsible Party
Principal Investigator
Principal Investigator

Regulatory Department

Scientific

Prevail Therapeutics Inc.

Study Sites (3)

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