This clinical trial is designed to test whether surgically injecting nerve cells that make dopamine into the brain of Parkinson's disease patients is safe, and to monitor for potential side effects.
Subjects will undergo surgical transplantation of the dopamine-producing cells under general anesthesia into a part of the brain called the putamen. Subjects then take medicines to partially suppress their immune system (aimed to prevent the body from rejecting the cells) for 1 year. Safety, tolerability, evidence of cell survival (using MRI and PET scans of the brain), and effect on Parkinson's disease symptoms are assessed for 2 years post-transplant.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
12
MSK-DA01 is an experimental product derived from human embryonic stem cells. The stem cells were converted into brain cells that produce dopamine.
A device that is used for injection of fluids into the brain will be used. Some minor modifications have been made to the device to allow delivery of MSK-DA01 cells.
University of California Irvine
Orange, California, United States
Weill Cornell Medical College
New York, New York, United States
Toronto Western Hospital
Toronto, Ontario, Canada
Safety and Tolerability
The incidence of Serious Adverse Events (SAEs) at 1 year post-transplant. or abnormal tissue overgrowth related to presence of transplanted cells;
Time frame: Baseline to 1 Year Post-Transplant
Evidence of Cell Survival
Change in 18F-DOPA uptake using positron emission tomography (PET) from baseline to 1 and 2 years
Time frame: Baseline to 1 Year Post-Transplant and Baseline to 2 Years Post-Transplant
Changes in Motor Function
Changes in MDS-Unified Parkinson's Disease Rating Scale (UPDRS) motor sub-score in the "off" state from baseline to 2 years post-transplant.
Time frame: Baseline to 1 Year Post-Transplant and Baseline to 2 Years Post-Transplant
Changes in Waking Hours in "Off" State
Changes in number of waking hours in the "off" state from baseline to 2 years post-transplant.
Time frame: Baseline to 1 Year Post-Transplant and Baseline to 2 Years Post-Transplant
Continued Safety and Tolerability
Incidence of SAEs at 2 years post-transplant and incidence and type of AEs at 1 and 2 years post-transplant.
Time frame: Baseline to 1 Year Post-Transplant and Baseline to 2 Years Post-Transplant
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