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

NCT04802733 | Completed Phase 1 DMC FDA Drug FDA Device

• 1 other identifier: MSK-DA01-101
• Study Type: interventional
• Target: 12
• Locations: 2 countries
• Sites: 3

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.

Conditions

Advanced Parkinson's Disease

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (4)

• Evidence of Cell Survival

  Baseline to 1 Year Post-Transplant and Baseline to 2 Years Post-Transplant

• Changes in Motor Function

  Baseline to 1 Year Post-Transplant and 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

• Continued Safety and Tolerability

  Baseline to 1 Year Post-Transplant and Baseline to 2 Years Post-Transplant

Study Arms (1)

MSK-DA01

EXPERIMENTAL

Biological: MSK-DA01; Device: MSK-DA01 Cell Delivery Device

Interventions

MSK-DA01 BIOLOGICAL

MSK-DA01 is an experimental product derived from human embryonic stem cells. The stem cells were converted into brain cells that produce dopamine.

MSK-DA01

MSK-DA01 Cell Delivery Device 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.

MSK-DA01

Eligibility Criteria

• Age: 50 Years - 78 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• BlueRock Therapeutics: lead
• Memorial Sloan Kettering Cancer Center: collaborator

Study Sites (3)

University of California Irvine

Orange, California, 92868, United States

Location

Weill Cornell Medical College

New York, New York, 10065, United States

Location

Toronto Western Hospital

Toronto, Ontario, M5T 2S8, Canada

Location

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 12, 2021
• First Posted: March 17, 2021
• Study Start: May 3, 2021
• Primary Completion: May 18, 2023
• Study Completion: June 10, 2024
• Last Updated: November 1, 2024

Record last verified: 2024-10

Data Sharing

• IPD Sharing: Will not share

Locations

CA (1); US (2)