SNPMiner Trials by Shray Alag


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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 52

Secondary Outcomes

Measure: Change from baseline in Parkinson's Disease Cognitive Rating Scale

Time: From baseline to Week 52

Measure: 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


HPO Nodes