To evaluate the safety and efficacy of transplantation of human induced pluripotent stem cell-derived dopaminergic progenitors, CT1-DAP001, into the corpus striatum in patients with Parkinson's disease
The subject has a diagnosis of PD (clinically established or clinically probable) in accordance with the MDS Clinical Diagnostic Criteria for Parkinson's Disease (2015).
The subject has an inadequate response to drug treatments.
The subject is ≥ 40 years and ≤ 75 years of age at the time of informed consent.
The subject has had PD for at least 5 years.
The subject has an abnormal brain MRI suggestive of brain pathology other than Parkinson's disease.
Atypical parkinsonism (Parkinsonism-Plus syndrome, secondary parkinsonism, hereditary parkinsonism).
The subject has clinical indication or diagnosis of abnormal immune function.
The subject has been diagnosed with a major neurocognitive disorder such as dementia, or is high risk for this.
Single group
Human induced pluripotent stem cell-derived dopaminergic progenitors (CT1-DAP001)
Combination productInvestigational PET agents will be synthesized at UCSD and administered to subjects according to the local institutional guidelines. IND applications for these agents are linked with the IND application for the study product, CT1-DAP001. The IND applications for the PET radiopharmaceuticals contains the manufacturing method, specifications, quality testing, clinical usage, and safety and efficacy information for individual PET agents. Investigational Cell Injector Suniviion needle: The investigational instrument will be used to administer dopaminergic progenitors into the putamen. After aspirating cells, the instrument will be attached to a Leksell stereotactic frame to administer the cells into the brain.
Incidence And Severity Of Adverse Events
For adverse events reported, the severity will be assessed, and the incidence in the treatment period will be determined.
ACCEPTABILITY
Assessed by presence or absence of graft expansion (\> 3 cm3) in the brain at 24 months after transplantation
Improvement or worsening of involuntary movements
Assessed by dyskinesia score, measured as the change in dyskinesia score from Baseline to each post-transplantation time point.
ACCURACY
Assessed by FDOPA (MRI) of tissue expansion The expansion on MRI will be assessed at each time point of measurement * day before transplantation, * immediately after transplantation, * day after transplantation, * 4 weeks, 12 weeks, 6 months, 18 months, (if clinically indicated) * 12 months, 24 months after transplantation
MDS-UPDRS Part III totalscore (at OFF time)
This endpoint is defined as the change in UPDRS Part III score at OFF time from Baseline to each post-transplantation time point. The efficacy will be assessed based on MDS-UPDRS Part III total score at OFF time at 24 months after cell transplantation. 1. Excellent response defined as a decrease of ≥ 5 2. Good response defined as a decrease of 0 to 4
MDS-UPDRS Part III totalscore (at ON time)
This endpoint is defined as the change in UPDRS Part III score at ON time from Baseline to each post-transplantation time point.
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University of California, San Diego
Lead sponsor
Sumitomo Pharma America, Inc.
Collaborator
Kyoto University
Collaborator
Sumitomo Pharma Co., Ltd.
Collaborator
