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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)

This study is currently recruiting participants.
See Contacts and Locations
Verified August 2017 by Sanofi ( Genzyme, a Sanofi Company )
Sponsor:
Information provided by (Responsible Party):
Sanofi ( Genzyme, a Sanofi Company )
ClinicalTrials.gov Identifier:
NCT02906020
First received: September 14, 2016
Last updated: August 2, 2017
Last verified: August 2017
  Purpose

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.

Condition Intervention Phase
Parkinson's Disease Drug: GZ/SAR402671 Drug: Placebo Phase 2

Study Type: Interventional
Study Design: 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.

Resource links provided by NLM:


Further study details as provided by Sanofi ( Genzyme, a Sanofi Company ):

Primary Outcome Measures:
  • Change from baseline in Movement Disorder Society Unified Parkinson's Disease Rating Scale Part II and III score [ Time Frame: From baseline to Week 8, and at Week 52 ]

Secondary Outcome Measures:
  • Change from baseline in Parkinson's Disease Cognitive Rating Scale [ Time Frame: From baseline to Week 52 ]
  • Change from baseline in Movement Disorder Society Unified Parkinson's Disease Rating Scale Part I, II, and III score [ Time Frame: From baseline to Week 52 ]
  • Change from baseline in Hoehn and Yahr score [ Time Frame: From baseline to Week 52 ]

Estimated Enrollment: 243
Study Start Date: December 15, 2016
Estimated Study Completion Date: April 7, 2022
Estimated Primary Completion Date: March 2021 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: GZ/SAR402671
Part 1: Increasing dose of GZ/SAR402671 will be administered once per day. Part 2: A dose of GZ/SAR402671 (determined in Part 1) will be administered once per day.
Drug: GZ/SAR402671
Pharmaceutical form:capsule Route of administration: oral
Placebo Comparator: Placebo
A matching placebo for Parts 1 and 2 will be administered once per day.
Drug: Placebo
Pharmaceutical form:capsule Route of administration: oral

Detailed Description:
The total study duration per subject in Part 2 will be approximately 168 weeks that will consist of 6.5 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 64 weeks in Japan.
  Eligibility

Ages Eligible for Study:   18 Years to 80 Years   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion criteria :

  • Male and female adults with a diagnosis of PD and who are heterozygous carriers of a GBA mutation associated with PD.
  • Patients carrying known sequence variants associated with GBA-PD (such as E326K) must have rapid eye movement (REM) sleep behavior disorder (RBD) confirmed by historically documented polysomnography or by questionnaire.
  • Age ≥18 years to 80 years inclusive at the time of informed consent signing.
  • Has symptoms of PD ≥2 years.
  • Hoehn and Yahr (H and Y) stage of 2 or lower at baseline.
  • Stable medication regimen of PD drugs for at least 30 days (at least 60 days for rasagiline) prior to randomization.
  • The patient is willing to abstain from grapefruit containing products for 72 hours prior to administration of the first dose of GZ/SAR402671 and for duration of the entire treatment period (Periods 2 and 3).
  • Signed written consent.

Exclusion criteria:

  • Parkinsonism due to drug(s) or toxin(s).
  • Patients carrying mutations in genes other than GBA that have been associated with an increased risk for PD, specifically LRKK2 (G2019S).
  • Patients with Gaucher disease (GD), defined by the presence of 2 mutated GBA alleles.
  • Montreal Cognitive Assessment score <20 at Screening Visit.
  • Patients with prior surgical history of deep brain stimulation (DBS).
  • Patients with baseline brain MRI without contrast showing a structural abnormality that is a possible cause of their PD signs or symptoms.
  • Hepatic insufficiency with liver function tests (LFT) >2 times upper limit of normal at Screening Visit.
  • The patient has prior known positive result on any of the following tests: hepatitis B surface antigen (HBsAg), anti-hepatitis C virus (anti-HCV) antibodies, anti-human immunodeficiency virus 1 and 2 antibodies (anti-HIV 1 and anti-HIV 2 Ab). Patients with a positive hepatitis B surface antibody (HBsAb) test with a history of prior hepatitis B immunization are eligible if other criteria are met (ie, negative tests for : HBsAg, hepatitis B core antibody [HBcAb],and hepatitis C [HCVAb].
  • Renal insufficiency as defined by creatine >1.5 times normal at Screening Visit.
  • The patient has received strong or moderate inducers or inhibitors of CYP3A4 within 30 days or 5 half-lives prior to randomization, whichever is longer.
  • The patient has, according to World Health Organization (WHO) Grading, a cortical cataract > one-quarter the lens circumference (grade cortical catact-2 [COR-2]) or a posterior subcapsular cataract >2 mm (grade posterior subscapsular cataract [PSC-2]). Patient with nuclear cataracts will not be excluded.
  • The patient is currently receiving potentially cataractogenic medications, including chronic regimen (more frequently than every 2 weeks) of any dose or route of corticosteroids or any medication that can cause cataract or worsen the vision of patients with cataract (eg, glaucoma medications) according to the Prescribing Information.
  • If female, pregnancy (defined as positive beta-human chorionic gonadotrophin [Beta-HCG] blood test) or lactating or breast-feeding.
  • Any medical disorders that, in the opinion of the Investigator, could interfere with study-related procedures. This includes condition(s) that preclude the safe performance of routine lumbar punctures, such as prohibitive spinal diseases, bleeding diasthesis, or clinically significant coagulopathy or thrombocytopenia.
  • Current participation in another investigational interventional study.
  • Any medications specifically used for treating memory dysfunction, such as, but not limited to cholinesterase inhibitors or memantine, are prohibited.

The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.

  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT02906020

Contacts
Contact: For site information, send an email with site number to Contact-Us@sanofi.com

Locations
United States, Connecticut
Investigational Site Number 8400015 Recruiting
New Haven, Connecticut, United States, 06510
United States, Florida
Investigational Site Number 8400008 Recruiting
Boca Raton, Florida, United States, 33486
United States, Illinois
Investigational Site Number 8400016 Recruiting
Chicago, Illinois, United States, 60611
United States, Massachusetts
Investigational Site Number 8400014 Recruiting
Boston, Massachusetts, United States, 02114
United States, New York
Investigational Site Number 8400001 Recruiting
New York, New York, United States, 10032
France
Investigational Site Number 2500001 Recruiting
Paris, France, 75013
Germany
Investigational Site Number 2760002 Recruiting
Kiel, Germany, 24105
Investigational Site Number 2760001 Recruiting
Tübingen, Germany, 72076
Italy
Investigational Site Number 3800001 Recruiting
Catanzaro, Italy, 88100
Portugal
Investigational Site Number 6200003 Recruiting
Torres Vedras, Portugal, 2560-280
Spain
Investigational Site Number 7240002 Recruiting
Barcelona, Spain, 08025
Investigational Site Number 7240001 Recruiting
Sevilla, Spain, 41013
Sweden
Investigational Site Number 7520001 Recruiting
Stockholm, Sweden, 14186
Sponsors and Collaborators
Genzyme, a Sanofi Company
Investigators
Study Director: Clinical Sciences & Operations Sanofi
  More Information

Responsible Party: Genzyme, a Sanofi Company
ClinicalTrials.gov Identifier: NCT02906020     History of Changes
Other Study ID Numbers: ACT14820
2016-000657-12 ( EudraCT Number )
U1111-1180-6918 ( Other Identifier: UTN )
Study First Received: September 14, 2016
Last Updated: August 2, 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
Plan Description: Individual participant data (IPD) and supporting clinical documents are available for request at clinicalstudydatarequest.com. While making information available Sanofi continues to protect the privacy of the participants in clinical trials and to remove commercially confidential information (CCI). Details on Data Sharing criteria and process for requesting access can be found at this web address: clinicalstudydatarequest.com

Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:
Parkinson Disease
Parkinsonian Disorders
Basal Ganglia Diseases
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Movement Disorders
Neurodegenerative Diseases

ClinicalTrials.gov processed this record on August 18, 2017