Millions of people live with Parkinson’s disease, a condition that causes damage to a part of the brain in ways that make daily life challenging for patients. While the exact causes are unknown, an estimated 5 – 10% of cases are linked to a genetic mutation (a GBA mutation) that puts them at risk of having an earlier appearance of PD symptoms and faster loss of mental and motor skills than those PD patients with no GBA mutation. However, a study of PD patients with this mutation and treatment has not been done yet. This research study is the first study to identify patients with this mutation (using a saliva sample) and evaluate how they respond to an investigational treatment that targets the pathway of the mutation. The drug being tested targets the pathway where this mutation is located and ‘normalizes’ the cell activity. The drug is called SAR402671, and it is an experimental medication (one that has not been approved yet) being developed by the Sponsor of the clinical trial. There will be 2 parts to the study. Part 1 will first evaluate how tolerable the drug is in a small number of patients. Part 2 will then evaluate how effective the drug is in improving symptoms of GBA-mutation positive PD patients. If selected to be in this study, the total study duration in Part 1 will be up to 9 months. Part 2 will be approximately 42 months that will include 6.5 weeks of screening period, 12 months of treatment period, 26 months of follow-up period, and 6 weeks of post-treatment observation period. This study will also help further researchers’ understanding of GBA mutations, which may be helpful to other patients. If interested in learning more visit: http://mediaroom.sanofi.com/persistence-and-accidental-inspiration-propel-parkinsons-research/
The total study duration per subject in Part 2 will be approximately 168 weeks that will consist of 6.5 weeks of screening period, 52 weeks of treatment period, 104 weeks of follow-up period, and 6 weeks of post-treatment observation period. Part 1 maximal duration will be up to 48 weeks outside Japan, and up to 64 weeks in Japan.