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

Recruitment & Eligibility

Status: ot Recruiting

Sex: All

Target Recruitment: 276

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 70 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.
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 218
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 58

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 from screening, whichever is longer, prior to randomization.
-The patient has, according to World Health Organization (WHO) Grading, a cortical cataract > onequarter 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, 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 safety 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.

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method

Secondary Outcome Measures

Name	Time	Method

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