|Clinical Trial Title
|Open to Enrollment
|Doctors & Locations
|Early Stage Parkinson's Disease
This is a two-part, multicenter, multiple-country, randomized, double-blinded, placebo-controlled study in early-stage Parkinson’s Disease (PD) patients carrying a GBA mutation.
The purpose of this study is to evaluate the possible risks and the effectiveness of GZ/SAR402671 (study medication) once daily compared to placebo in the treatment of PD patients carrying a specific type of gene mutation, called the GBA (glucocerebrosidase) mutation.
GZ/SAR402671 has never been tested in patients with Parkinson’s disease, including Parkinson’s patients who have mutations in genes (such as the glucocerebrosidase gene, GBA). Research by other people indicates that GBA is a genetic risk factor for PD. Based on this research, it is believed that PD patients with a GBA mutation (GBA-PD) have an associated reduction in a brain protein that leads to an increase in the levels of a lipid component in brain cells It is thought that increases in this lipid component in the brain may disrupt normal brain functioning and lead to motor problems (for example, shaking hands, stiff muscles, short steps, poor balance, and falls) and nonmotor problems (for example, sleep, memory, long term attention, and ability to plan hard tasks).
The study is divided into 2 consecutive parts: Part 1 determined the selection of the dose of GZ/SAR402671 for Part 2. Part 1 is complete. Only Part 2 is being done at Legacy.
Part 2 will include 4 main periods:
Subjects will be taking a capsule once daily.
Ages Eligible for Study: 18 years to 80 years
Sexes Eligible for Study: All
Inclusion criteria :
Age ≥18 years to 80 years inclusive at the time of informed consent signing.
|Richard Rosenbaum, MD