Autologous iPSC-Derived Dopamine Neuron Transplantation for Parkinson's Disease

General Information

Summary This research study is evaluating an investigational cell product called autologous induced pluripotent stem cell (iPSC)-derived dopamine neurons. This research study is a single-center Phase 1 clinical trial, which will test the safety of injecting the investigational cell product into the brain of subjects with Parkinson's disease.
Description The goal of this research study is to test a new treatment for Parkinson's disease. Parkinson's disease is a progressive disease that causes people to lose specific brain cells called midbrain dopamine neurons. When these dopamine neurons are lost, it leads to a lack of dopamine in the brain. When there is not enough dopamine, people with Parkinson's disease experience problems with their movement. This trial will test whether new dopamine neurons made from blood cells from subjects with Parkinson's disease are safe when surgically injected into the area of the brain affected (called the putamen) of the same subjects (called autologous transplantation). The trial will assess the safety of the injected cells and will also measure the effects of the transplanted autologous dopamine neurons on Parkinson's disease symptoms.
Clinical trials phase Phase 1
Start date (estimated) 2024-06-01
End date (estimated) 2027-08-31
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 NCT06422208
ICTRP weblink
Other study identifiers
Name 2023P003609
Name U01NS109463
Description U.S. NIH Grant/Contract
Source weblink
Regulatory body approval
Name Food and Drug Administration (FDA)
United States
Public contact
Public email
First name Penelope
Last name Hallett
Phone +1 617-732-6564
City Boston
United States
Address freetext 115 Mill St, Belmont, MA 02478, Vereinigte Staaten
Sponsors Penelope J. Hallett, Ph.D.


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


Recruitment Status Not yet recruiting
Estimated number of participants 6