NCT04802733

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

• Status: Completed
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 12 (actual)
• Sponsor: BlueRock Therapeutics · Industry
• Sex: All
• Age: 50 Years – 78 Years
• Healthy volunteers: Not accepted

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

Interventions

Type	Name	Description
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.

Timeline

• Start date: 2021-05-03
• Primary completion: 2023-05-18
• Completion: 2024-06-10
• First posted: 2021-03-17
• Last updated: 2024-11-01

Locations

3 sites across 2 countries: United States, Canada

Regulatory

• FDA-regulated drug study
• FDA-regulated device study