Venglustat in Combination With Cerezyme in Adult Patients With Gaucher Disease Type 3
Status:
Active, not recruiting
Trial end date:
2024-01-11
Target enrollment:
Participant gender:
Summary
Primary Objectives:
Part 1: Biomarker evaluation/screening phase
- Evaluate cerebrospinal fluid (CSF) biomarkers in adult Gaucher disease Type 3 (GD3)
patients that distinguish GD3 from adult Gaucher disease Type 1 (GD1) patients
- Screen adult GD3 patients who qualify for treatment with venglustat in Parts 2 and 3
Parts 2 and 3: Treatment phases
- Evaluate short-term (Part 2) and long-term (Part 3) safety and tolerability of
venglustat in combination with Cerezyme in adult GD3 patients
- Evaluate the change in CSF central nervous system (CNS) biomarkers (glucosylceramide
[GL-1] and lyso-glucosylceramide [lyso-GL-1]) from adult GD3 patients receiving
venglustat in combination with Cerezyme (Part 2 only)
Secondary Objectives:
- Evaluate the pharmacokinetics (PK) of venglustat in adult GD3 patients
- Explore the efficacy of venglustat in combination with Cerezyme in infiltrative lung
disease (ILD) in adult GD3 patients (Part 2 only)
- Explore the efficacy of venglustat in combination with Cerezyme in systemic disease in
adult GD3 patients
- Explore the efficacy of venglustat in combination with Cerezyme on neurological function
and on exploratory CSF biomarkers other than lyso-GL-1 and GL-1 in adult GD3 patients