Phase 1 Study To Assess the Safety and Tolerability of Human Embryonic Stem Cell-Derived Midbrain Dopamine Neuron

General Information

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.
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.
Clinical trials phase Phase 1
Start date (estimated) 2021-05-03
End date (estimated) 2024-05-31
Clinical feature
Label Parkinson's disease
Description A synucleinopathy that has_material_basis_in degeneration of the central nervous system that often impairs motor skills, speech, and other functions.; Xref MGI. OMIM mapping confirmed by DO. [SN].

Administrative Information

NCT number NCT04802733
ICTRP weblink
Other study identifiers
Name MSK-DA01-101
Source weblink
Regulatory body approval
Name Food and Drug Administration (FDA)
United States
Public contact
Public email
Last name BlueRock Therapeutics
Phone +1 857-777-0512
United States
Sponsors BlueRock Therapeutics


Which differentiated cell type is used
Label dopaminergic neuron
Description A neuron that releases dopamine as a neurotransmitter.


Recruitment Status Active, not recruiting
Estimated number of participants 12