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

Phase 1

• Conditions: Parkinson's disease (PD) carrying a GBA mutation; MedDRA version: 20.0Level: PTClassification code 10061536Term: Parkinson's diseaseSystem Organ Class: 10029205 - Nervous system disorders; Therapeutic area: Diseases [C] - Nervous System Diseases [C10]

• Registration Number: EUCTR2016-000657-12-AT
• Lead Sponsor: Genzyme Corporation

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2016-10-17
• Last Update Posted: 14 June 2021

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 290

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.
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 229
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 61

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 > 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 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 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.
-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.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified