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Trial record 3 of 3 for:    genzyme parkinson's

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 (MOVES-PD)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02906020
Recruitment Status : Active, not recruiting
First Posted : September 19, 2016
Last Update Posted : November 20, 2020
Sponsor:
Information provided by (Responsible Party):
Sanofi ( Genzyme, a Sanofi Company )

Brief Summary:

Primary Objectives:

  • Part 1: To determine the safety and tolerability of GZ/SAR402671 administered orally for 4 weeks, 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/SAR402671 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/SAR402671 administered orally for 52 weeks 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 over a 52-week period.

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

Detailed Description:

Part 1: the total duration will be up to approximately 50 weeks (or 68 weeks in Japan). Part 2: the total duration will be up to approximately 222 weeks that will consist of 8.5 weeks of screening period, 52 weeks of treatment period, 156 weeks of long term follow-up period and 6 weeks of post-treatment period.

At the end of a 52-week main placebo-controlled treatment period, all patients will be evaluated for possibility to transition to receive active treatment for 156 weeks plus 6 week post-treatment observation.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 270 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 : December 2020
Estimated Study Completion Date : February 2024

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
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
Other Name: venglustat

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




Primary Outcome Measures :
  1. Part 1: Number of patients with AE [ Time Frame: During treatment emergent period (from first IMP to 6 weeks after last IMP) ]
  2. Part 2: Change in Movement Disorder Society Unified Parkinson's Disease Rating Scale Part II and III score, performed during the OFF state [ Time Frame: From baseline to Week 52 ]

Secondary Outcome Measures :
  1. Part 2: Change in Parkinson's Disease Cognitive Rating Scale (total score) [ Time Frame: From baseline to Week 52 ]
  2. Part 2: Change in Movement Disorder Society Unified Parkinson's Disease Rating Scale Part I, II, and III score, performed during the OFF state [ Time Frame: From baseline to Week 52 ]
  3. Part 2: Change in Hoehn and Yahr score [ Time Frame: From baseline to Week 52 ]


Information from the National Library of Medicine

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, Learn About Clinical Studies.


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Ages Eligible for Study:   18 Years to 80 Years   (Adult, Older Adult)
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 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 (Part 1 and Part 2, Periods 2 and 3).
  • Signed written consent.

Exclusion criteria:

  • Parkinsonism due to drug(s) or toxin(s).
  • Patients carrying the LRRK2 G2019S mutation.
  • Patients with Gaucher disease (GD) as defined by clinical signs and symptoms (ie, hepatosplenomegaly, cytopenia, skeletal disease) and/or marked deficiency of GCase activity compatible with GD.
  • Montreal Cognitive Assessment score <20.
  • 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 a documented diagnosis, as per local regulations, of any of the following infections: hepatitis B, hepatitis C, human immunodeficiency virus 1 or 2.
  • 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 and/or clinically relevant findings 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, within 30 days or 5 half-lives of these medications prior to randomization, whichever is longer.

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


Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02906020


Locations
Show Show 53 study locations
Sponsors and Collaborators
Genzyme, a Sanofi Company
Investigators
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Study Director: Clinical Sciences & Operations Sanofi
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Responsible Party: Genzyme, a Sanofi Company
ClinicalTrials.gov Identifier: NCT02906020    
Other Study ID Numbers: ACT14820
2016-000657-12 ( EudraCT Number )
U1111-1180-6918 ( Other Identifier: UTN )
First Posted: September 19, 2016    Key Record Dates
Last Update Posted: November 20, 2020
Last Verified: November 2020
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

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Parkinson Disease
Parkinsonian Disorders
Basal Ganglia Diseases
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Movement Disorders
Neurodegenerative Diseases