The goal of this clinical trial is to assess the safety and tolerability of the surgical transplantation of dopaminergic progenitor cells into the brains of participants with Parkinson's disease. The transplanted dopaminergic cells will be derived from the participant's own skin cells.
This Phase I, open-label clinical trial aims to assess the feasibility and safety of autologous midbrain dopaminergic progenitor cell (mDAP) transplantation for the treatment of Parkinson's disease. mDAPs will be produced for each participant from a fibroblast sample and then transplanted bilaterally into the putamen under general anesthesia. The study will assess the safety and tolerability of the cell transplant procedure through clinical assessments and neuroimaging (CT, MRI and 18F-DOPA PET) over a 2-year follow-up period.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
8
Dopaminergic progenitor cells derived from autologous induced pluripotent stem cells will be injected into the brain in two cohorts of Parkinson's patients, one receiving low dose and the other high dose (4 and 8 million cells, respectively)
Massachusetts General Hospital
Boston, Massachusetts, United States
RECRUITINGSafety and Tolerability
Incidence and severity of adverse events and serious adverse events
Time frame: 2 years from time of implantation
18-F DOPA PET uptake
Change in putaminal 18F DOPA PET uptake from baseline to 12 and 24-month follow-up.
Time frame: 2 years from time of implantation
Change in motor function
Change in the on-state and practically defined off-state motor function as assessed by the MDS-UPDRS part III from baseline to 12 and 24-month follow-up.
Time frame: 2 years from time of implantation
Change in "off" hours
Change in the number of waking hours spent in the "off" state as measured using a self-reported symptom diary, comparing baseline to 12 and 24-month follow-up.
Time frame: 2 years from time of implantation
Change in dyskinesia
Change in the frequency and severity of dyskinesia as assessed by the MDS-UDysRS from baseline to 12 and 24-month follow-up.
Time frame: 2 years from time of implantation
Change in PD medication usage
Change in the Parkinson's disease medication usage as defined by the levodopa equivalent daily dose (LEDD) from baseline to 12 and 24-month follow-up.
Time frame: 2 years from time of implantation
Change in Parkinson's disease related quality of life
Change in the Parkinson's disease-related quality of life as assessed by the change in PDQ-39 from baseline to 12 and 24-month follow-up.
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Time frame: 2 years from time of implantation
Global impression of change
Patient and Clinician Global Impression of Change (PGI-I and CGI-I) assessed at 12 and 24-months after implantation
Time frame: 2 years from time of implantation