Venglustat in Combination With Cerezyme in Adult Patients With Gaucher Disease Type 3 With Venglustat Monotherapy Extension — LEAP  

Gaucher Disease Type 1 Clinical Trial

Official title: A 4-part, Open-label, Multicenter, Multinational Study of the Safety, Tolerability, Pharmacokinetics, Pharmacodynamic, and Exploratory Efficacy of Venglustat in Combination With Cerezyme in Adult Patients With Gaucher Disease Type 3 With Venglustat Monotherapy Extension

Status Active, not recruiting

Clinical Trial Summary

Part 1: Biomarker evaluation/screening phase

Primary Objectives:

• 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, Part 3, and Part 4

Parts 2 and 3: Combination treatment phases

Primary objectives:

• 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)

Part 4: Extended treatment phase with monotherapy

Primary objectives:

• Evaluate safety and tolerability of venglustat monotherapy in adult GD3 patients who have remained systemically stable on venglustat in combination with Cerezyme

Parts 2 and 3: Combination treatment phases

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 in adult GD3 patients

• Explore plasma biomarkers (lyso-GL-1 and GL-1) in adult GD3 patients

• Explore CSF biomarkers other than lyso-GL-1 and GL-1 in adult GD3 patients (Part 2 only)

Part 4: Extended treatment phase with monotherapy

Secondary objectives:

• Explore the efficacy of venglustat in systemic disease in adult GD3 patients

• Explore the efficacy of venglustat on neurological function in adult GD3 patients

• Explore plasma biomarkers (lyso-GL-1 and GL-1) in adult GD3 patients

Clinical Trial Description

The total duration for GD1 participants is 45 days (Part 1), while for GD3 participants the total duration is up to approximately 8.7 years ;

Related Conditions & MeSH terms

• Disease
• Gaucher Disease
• Gaucher Disease Type 1
• Gaucher Disease Type 3

• NCT number: NCT02843035
• Study type: Interventional
• Source: Sanofi
• Status: Active, not recruiting
• Phase: Phase 2
• Start date: January 4, 2017
• Completion date: September 30, 2025