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/