A Global Study to Assess the Drug Dynamics, Efficacy, and Safety of Venglustat (GZ/SAR402671) in Parkinson's Disease Patients Carrying a Glucocerebrosidase (GBA) Gene Mutation
Public ClinicalTrials.gov record NCT02906020. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
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
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
Primary Objectives: * Part 1: To determine the safety and tolerability of 4, 8, and 15 milligrams of GZ/SAR402671 (venglustat) administered orally for 4 weeks, as compared to placebo in participants with early-stage Parkinson's disease (PD) carrying a glucocerebrosidase gene (GBA) mutation or other pre-specified variants. * Part 2: To determine the efficacy of GZ/SAR402671 administered orally daily, as compared to placebo in participants with early-stage PD carrying a GBA mutation or other pre-specified variants. Secondary Objectives: Part 1: * To assess the pharmacokinetic (PK) profile of oral dosing of GZ/SAR402671 in plasma when administered in early-stage PD participants carrying a GBA mutation. * To assess the exposure of GZ/SAR402671 in cerebrospinal fluid (CSF) when administered in early-stage PD participants carrying a GBA mutation. Part 2: * To demonstrate overall safety and tolerability of GZ/SAR402671 administered orally for 52 weeks in early-stage PD participants 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 PD participants carrying a GBA mutation over a 52-week period.
Study identification
- NCT ID
- NCT02906020
- Recruitment status
- Terminated
- Study type
- Interventional
- Phase
- Phase 2
- Enrollment
- 273 participants
Conditions and interventions
Conditions
Eligibility (public fields only)
- Age range
- 18 Years to 80 Years
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Dec 15, 2016 (Actual)
- Primary completion
- Dec 18, 2020 (Actual)
- Completion
- May 27, 2021 (Actual)
- Last update posted
- May 24, 2022
2016 – 2021
United States locations
- U.S. sites
- 19
- U.S. states
- 11
- U.S. cities
- 13
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Investigational Site Number :8400017 | Scottsdale | Arizona | 85258 | — |
| Investigational Site Number :8400011 | Scottsdale | Arizona | 85259 | — |
| Investigational Site Number :8400004 | La Jolla | California | 92093 | — |
| Investigational Site Number :8400019 | Palo Alto | California | 94304 | — |
| Investigational Site Number :8400013 | Sunnyvale | California | 94085 | — |
| Investigational Site Number :8400015 | New Haven | Connecticut | 06510 | — |
| Investigational Site Number :8400008 | Boca Raton | Florida | 33486 | — |
| Investigational Site Number :8400016 | Chicago | Illinois | 60611 | — |
| Investigational Site Number :8400005 | Chicago | Illinois | 60612-3854 | — |
| Investigational Site Number :8400014 | Boston | Massachusetts | 02114 | — |
| Investigational Site Number :8400018 | New York | New York | 10003 | — |
| Investigational Site Number :8400010 | New York | New York | 10016 | — |
| Investigational Site Number :8400001 | New York | New York | 10032 | — |
| Investigational Site Number :8400021 | Portland | Oregon | 97210 | — |
| Investigational Site Number :8400020 | Portland | Oregon | 97225 | — |
| Investigational Site Number :8400009 | Portland | Oregon | 97239 | — |
| Investigational Site Number :8400002 | Philadelphia | Pennsylvania | 19107 | — |
| Investigational Site Number :8400006 | Fairfax | Virginia | 22030 | — |
| Investigational Site Number :8400012 | Kirkland | Washington | 98034 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
Non-U.S. locations
This page focuses on the U.S. directory. The official record also lists 33 non-U.S. sites.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT02906020, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted May 24, 2022 · Synced Oct 30, 2026
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Open the official record
The complete public registry record, listed eligibility criteria, and available contact information for NCT02906020 live on ClinicalTrials.gov.