SNPMiner Trials (Home Page)
Report for Mutation E326K
Developed by Shray Alag, 2020.
SNP Clinical Trial Gene
There is one clinical trial.
Clinical Trials
1 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.
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/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 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.
NCT02906020 Parkinson's Disease Drug: GZ/SAR402671 Drug: Placebo MeSH:Parkinson Disease
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. --- E326K ---
Primary Outcomes
Measure: Change from baseline in Movement Disorder Society Unified Parkinson's Disease Rating Scale Part II and III score Time: From baseline to Week 8, and at Week 52Secondary Outcomes
Measure: Change from baseline in Parkinson's Disease Cognitive Rating Scale Time: From baseline to Week 52Measure: Change from baseline in Movement Disorder Society Unified Parkinson's Disease Rating Scale Part I, II, and III score Time: From baseline to Week 52
Measure: Change from baseline in Hoehn and Yahr score Time: From baseline to Week 52