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

NCT ID: NCT04802733

Last Updated: 2024-11-01

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE1
• Total Enrollment: 12 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2021-05-03
• Study Completion Date: 2024-06-10

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.

Conditions

Advanced Parkinson's Disease

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

MSK-DA01

Group Type: EXPERIMENTAL

MSK-DA01

Intervention Type: 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 Cell Delivery Device

Intervention Type: 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.

Interventions

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.

Intervention Type: BIOLOGICAL

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.

Intervention Type: DEVICE

Eligibility Criteria

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

• Minimum Eligible Age: 50 Years
• Maximum Eligible Age: 78 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Memorial Sloan Kettering Cancer Center

OTHER

Sponsor Role: collaborator

BlueRock Therapeutics

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

University of California Irvine

Orange, California, United States

Weill Cornell Medical College

New York, New York, United States

Toronto Western Hospital

Toronto, Ontario, Canada

Countries

United States; Canada

Other Identifiers

MSK-DA01-101

Identifier Type: -

Identifier Source: org_study_id