Date: 2017-02-14
Type of information: Initiation of the trial
phase: 2
Announcement: initiation of the trial
Company: Genzyme (USA - MA), a Sanofi company (France)
Product: GZ/SAR402671
Action
mechanism: glucosylceramide synthase inhibitor. GZ/SAR402671 is a glucosylceramide synthase inhibitor that blocks the formation of glucosylceramide (GL-1), a key intermediate in the synthesis of GL-3.
Disease: Parkinson's disease
Therapeutic area: Neurodegenerative diseases
Country: USA
Trial
details: MOVES-PD is a 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/SAR4027671 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/SAR4027671 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)
Latest
news: * On February 14, 2017, Sanofi Genzyme announced the start of a Phase 2 trial of an investigational oral therapy, GZ/SAR402671, for patients with Parkinson’s disease who carry a a mutation of the glucocerebrosidase (GBA) gene that allows lipids called glycosphingolipids to build up in cells. This gene mutation is the most common genetic risk factor for the disease. People with GBA mutations in both copies of the gene, as opposed to a single mutation in GBA Parkinson’s disease, have Gaucher disease. GZ/SAR402671 reduces the production of glycosphingolipids. The MOVES-PD trial will assess the drug’s dynamics, efficacy and safety. This is the first industry-sponsored Phase 2 clinical trial in a genetically defined population of Parkinson’s disease. It will be run in two phases: a dose escalation study followed by a study of efficacy and safety. The randomized, double blind study will enroll more than 200 patients at trial sites around the world. The primary endpoint of the study is the change in score from baseline in a scale commonly used to measure Parkinson’s disease progression known as the Movement Disorder Society Unified Parkinson's Disease Rating Scale Part II and III. This includes self-evaluation of daily life activities and motor experience, and a clinician-scored motor evaluation.
