Phase 1 Safety and Tolerability Study of MSK-DA01 Cell Therapy for Advanced Parkinson's Disease
- Conditions
- Advanced Parkinson's Disease
- Interventions
- Device: MSK-DA01 Cell Delivery Device
- Registration Number
- NCT04802733
- Lead Sponsor
- BlueRock Therapeutics
- 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.
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 12
- 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
- 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
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description MSK-DA01 MSK-DA01 - MSK-DA01 MSK-DA01 Cell Delivery Device -
- Primary Outcome Measures
Name Time Method 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 Outcome Measures
Name Time Method 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.
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.
Trial Locations
- Locations (3)
Toronto Western Hospital
🇨🇦Toronto, Ontario, Canada
Weill Cornell Medical College
🇺🇸New York, New York, United States
University of California Irvine
🇺🇸Orange, California, United States