Sanofi's specialist care global business unit Sanofi Genzyme has initiated a Phase II trial of an investigational oral therapy GZ/SAR402671 to treat patients with Parkinson’s disease, who carry a single copy mutation of the glucocerebrosidase (GBA) gene.
The trial is designed to assess the dynamics, efficacy and safety of the drug, which is being developed to decrease the production of glycosphingolipids.
Expected to enrol more than 200 patients, the randomised, double blind Phase II trial will be conducted in two phases.
The first phase will be a dose escalation study, while the other will evaluate the drug's efficacy and safety.
Sanofi R&D early development for Parkinson’s disease and movement disorders global project head Tanya Fischer said: “We are excited to be able to bring the results of our many years of research in GBA gene mutations to a new therapeutic area with the potential to benefit patients with Parkinson’s disease.
"We look forward to evaluating whether this molecule, which has been shown to cross into the brain in preclinical studies, may positively impact the devastating neurologic effects of this disease.”
The trial's primary endpoint is to determine the change in score from baseline with Movement Disorder Society Unified Parkinson's Disease Rating Scale Part II and III, a scale used to measure Parkinson’s disease progression.
The end point also includes self-evaluation of daily life activities and motor experience, as well as a clinician-scored motor evaluation.