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

Launched by PREVAIL THERAPEUTICS · Aug 1, 2022

Keywords

ClinConnect Summary

The PROCEED trial is studying a new treatment called PR001 (also known as LY3884961) for adults with Gaucher Disease, specifically those experiencing peripheral symptoms. This study aims to find out if the treatment is safe and well-tolerated by patients. Up to three different doses of the medication will be tested, with a small group of patients receiving each dose. The trial will take about five years, during which researchers will carefully monitor patients for any side effects and measure how well the treatment is working.

To participate, individuals must be between 18 and 65 years old, have specific genetic mutations related to Gaucher Disease, and have been stable on existing treatments for at least two years. Participants will be required to sign consent forms and follow some guidelines, like not donating blood during the study. Throughout the trial, patients can expect regular check-ups and assessments to ensure their safety and the effectiveness of the treatment. This is an exciting opportunity for those affected by Gaucher Disease to potentially benefit from a new therapy while contributing to important research.

Gender

ALL

Eligibility criteria

• 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.
• 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 × 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 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.