A Global Study to Assess the Drug Dynamics, Efficacy, and Safety of GZ/SAR402671 in Parkinson's Disease Patients Carrying a Glucocerebrosidase (GBA) Gene Mutation (MOVES-PD)

Brief Summary:
Primary Objectives:
- Part 1: To determine the safety and tolerability of GZ/SAR402671 administered orally, as compared to placebo in patients with early-stage Parkinson's disease (PD) carrying a GBA mutation or other pre-specified variants.
- Part 2: To determine the efficacy of GZ/SAR402671 administered orally daily, as compared to placebo in patients with early-stage Parkinson's disease carrying a GBA mutation or other pre-specified variants.

Secondary Objectives:
- Part 1:
To assess the pharmacokinetic (PK) profile of oral dosing of GZ/SAR4027671 in plasma when administered in early-stage Parkinson's disease patients carrying a GBA mutation.
To assess the exposure of GZ/SAR402671 in cerebrospinal fluid (CSF) when administered in early-stage Parkinson's disease patients carrying a GBA mutation.

- Part 2:
To demonstrate overall safety and tolerability of GZ/SAR4027671 administered orally in early-stage Parkinson's disease patients carrying a GBA mutation as compared to placebo.
To assess the pharmacodynamic response to daily oral dosing of GZ/SAR402671 in plasma and CSF as measured by glucosylceramide (GL-1) when administered in early-stage Parkinson's disease patients carrying a GBA mutation.

Detailed Description:
The total study duration per subject in Part 2 will be approximately 170 weeks that will consist of 8.5 weeks of screening period, 52 weeks of treatment period, 104 weeks of follow-up period, and 6 weeks of post-treatment observation period. Part 1 maximal duration will be up to 48 weeks outside Japan, and up to 66 weeks in Japan.


Study Type: Interventional  (Clinical Trial)
Estimated Enrollment: 243 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple (Participant, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: Multicenter, Randomized, Double-blind, Placebo Controlled Study to Assess the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of GZ/SAR402671 in Patients With Early-stage Parkinson's Disease Carrying a GBA Mutation or Other Pre-specified Variant.
Actual Study Start Date: December 15, 2016
Estimated Primary Completion Date: October 2021
Estimated Study Completion Date: January 2023

Arms:
- Experimental: GZ/SAR402671
- Placebo Comparator: Placebo