Each eligible study participant will undergo a surgical procedure, which involves a full day visit, including a surgery. Prior to the procedure, study participants will be randomly assigned to one of two treatment groups: either the experimental gene therapy group or the placebo group. Neither you nor members of the study team will know which group you are in.
EXPERIMENTAL GENE THERAPY GROUP
Participants in this group will receive the experimental gene therapy that will be placed into a part of the brain that controls and coordinates movement via a surgical procedure performed under general anesthesia. Every participant will undergo general anesthesia so no one will know what treatment you receive.
Participants in this group will undergo a surgical procedure that externally mimics the experimental gene therapy procedure. Nothing will be placed in your brain. Every participant will undergo general anesthesia so no one will know what treatment you receive.
Why is there a placebo group?
A placebo group plays an important role in clinical studies. Placebo subjects allow researchers to compare the results of the placebo group to that of the experimental gene therapy group in order to better understand if any health-related changes occurred because of the experimental gene therapy or by chance.
All eligible study participants will have a 1-in-2 chance (50%) of being assigned to the experimental gene therapy group or to the placebo group.
The surgical procedure for both groups will include:
- An MRI scan during the procedure
- Your being asleep for the entire procedure
- A full day visit, which includes the surgery
- Approximately one to three days in the hospital, including the day you have surgery
- Other requirements as necessary