Phase 1 Safety and Tolerability Study of MSK-DA01 Cell Therapy for Advanced Parkinson's Disease

Phase 1 Study To Assess the Safety and Tolerability of Human Embryonic Stem Cell-Derived Midbrain Dopamine Neuron Cell Therapy (MSK-DA01) For Advanced Parkinson's Disease

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.

Study Overview

• Status: Completed
• Conditions: Advanced Parkinson's Disease
• Intervention / Treatment: Biological: MSK-DA01; Device: MSK-DA01 Cell Delivery Device

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.

• Study Type: Interventional
• Enrollment (Actual): 12
• Phase: Phase 1

Contacts and Locations

Study Locations

• Canada
  • Ontario
    • Toronto, Ontario, Canada, M5T 2S8
      • Toronto Western Hospital
• United States
  • California
    • Orange, California, United States, 92868
      • University of California Irvine
  • New York
    • New York, New York, United States, 10065
      • Weill Cornell Medical College

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 50 years to 78 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: MSK-DA01	Biological: MSK-DA01; MSK-DA01 is an experimental product derived from human embryonic stem cells. The stem cells were converted into brain cells that produce dopamine.; Device: MSK-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.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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;	Baseline to 1 Year Post-Transplant

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Evidence of Cell Survival	Change in 18F-DOPA uptake using positron emission tomography (PET) from baseline to 1 and 2 years	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.	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.	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.	Baseline to 1 Year Post-Transplant and Baseline to 2 Years Post-Transplant

Collaborators and Investigators

• Sponsor: BlueRock Therapeutics
• Collaborators: Memorial Sloan Kettering Cancer Center

Study record dates

Study Major Dates

• Study Start (Actual): May 3, 2021
• Primary Completion (Actual): May 18, 2023
• Study Completion (Actual): June 10, 2024

Study Registration Dates

• First Submitted: March 12, 2021
• First Submitted That Met QC Criteria: March 15, 2021
• First Posted (Actual): March 17, 2021

Study Record Updates

• Last Update Posted (Estimated): November 1, 2024
• Last Update Submitted That Met QC Criteria: October 30, 2024
• Last Verified: October 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Synucleinopathies; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Neurodegenerative Diseases; Movement Disorders; Parkinsonian Disorders; Basal Ganglia Diseases; Parkinson Disease
• Other Study ID Numbers: MSK-DA01-101

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: Yes