What is the Purpose of this Study?
The purpose of this study is to evaluate the safety and tolerability of an experimental drug called LY3884961 in people with Gaucher disease (GD). GD causes the collection of fats in some organs and bones, which affects the function of organs, destroy blood cells, and can weaken bones. LY3884961 is a type of gene therapy (treatment that involves adding or changing genetic material in a person’s cells to correct abnormal genes or to make a beneficial protein).
In addition to examining the safety of LY3884961 and identifying an acceptable dose of the drug in GD patients, researchers aim to measure its effects on certain GD clinical and laboratory test results. Procedures include medical history review, physical examination, electrocardiogram, lung function tests, imaging procedures, questionnaires, blood tests, and urinalysis.
Eligibility
- 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.
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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.
- 11. 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.
- 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 MRI, including claustrophobia or the presence of contraindicated metal (ferromagnetic)implants/cardiac pacemaker.
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Where can I participate?
Beverly
More about this Clinical Trial
What is the full name of this clinical trial?
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