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

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.