Advanced Parkinson's Disease
Conditions
Brief summary
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.
Detailed description
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.
Interventions
BIOLOGICALMSK-DA01
MSK-DA01 is an experimental product derived from human embryonic stem cells. The stem cells were converted into brain cells that produce dopamine.
DEVICEMSK-DA01 Cell Delivery Device
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.
Sponsors
Memorial Sloan Kettering Cancer Center
BlueRock Therapeutics
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
50 Years to 78 Years
Healthy volunteers
No
Inclusion criteria
* Age 50-78 years old (Canada) * Age 60-78 years old (United States) * Diagnosis of Parkinson's Disease made between 3 to 20 years ago * Taking levodopa, but with complications of therapy such as wearing off and/or dyskinesia * Able to participate in all study visits and evaluations, including brain MRI and PET scan * Existence of a study partner who may act as potential surrogate over long term for ongoing consent
Exclusion criteria
* Diagnosis of primary mitochondrial disorder, epilepsy, stroke, multiple sclerosis or other neurodegenerative diseases such as Alzheimer's disease * Prior Deep Brain Stimulation , lesion therapy, or gene therapy for PD * Prior surgical or radiation therapy to the brain or spinal cord * Any medical condition resulting in high risk of immunosuppressive drugs, including any active infectious disease * Inability to temporarily stop anti-platelet agents or other anti-coagulant medications without serious risk * Previous or currently active malignant disease within the past 5 years, except basal cell carcinoma or in situ uterine cervical carcinoma that have been treated * Severe obesity (\>350 lbs) or any condition that prevents use of PET/MRI * Pregnancy or breastfeeding * Contraindication to surgery or general anesthesia * In the opinion of the investigator, any other condition regarded as making subject unsuitable for trial
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Safety and Tolerability | Baseline to 1 Year Post-Transplant | The incidence of Serious Adverse Events (SAEs) at 1 year post-transplant. or abnormal tissue overgrowth related to presence of transplanted cells; |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Evidence of Cell Survival | Baseline to 1 Year Post-Transplant and Baseline to 2 Years Post-Transplant | Change in 18F-DOPA uptake using positron emission tomography (PET) from baseline to 1 and 2 years |
| Changes in Motor Function | Baseline to 1 Year Post-Transplant and Baseline to 2 Years Post-Transplant | Changes in MDS-Unified Parkinson's Disease Rating Scale (UPDRS) motor sub-score in the off state from baseline to 2 years post-transplant. |
| Changes in Waking Hours in Off State | Baseline to 1 Year Post-Transplant and Baseline to 2 Years Post-Transplant | Changes in number of waking hours in the off state from baseline to 2 years post-transplant. |
| Continued Safety and Tolerability | Baseline to 1 Year Post-Transplant and Baseline to 2 Years Post-Transplant | Incidence of SAEs at 2 years post-transplant and incidence and type of AEs at 1 and 2 years post-transplant. |
Countries
Canada, United States
Outcome results
None listed