This is a phase I, interventional, single arm, open-label, clinical study to evaluate the safety and efficacy of the striatal transplantation of autologous induced neural stem cell-derived DA precursor cells in Parkinson's Disease patients.
Parkinson's Disease (PD) is the second most common neurodegenerative disease, caused by progressive depletion of midbrain dopaminergic neurons in the substantia nigra pars compacta. This clinical study will include the preparation of dopaminergic neural precursor cells derived from neural stem cells through reprogramming of patient's peripheral blood mononuclear cells (PBMCs), and transplantation of the obtained cells into the brains of PD patients by stereotaxic injection. Safety, tolerability, evidence of cell survival (using PET scan), and the efficacy on PD symptoms will be assessed at different time points up to 12 months post-transplantation.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
6
The autologous induced neural stem cell-derived DA precursor cells will be stereotactically implanted into the striatum of PD patients.
Xuanwu Hospital Capital Medical University
Beijing, Beijing Municipality, China
RECRUITINGAssessment of changes during the medication "off" time in Movement Disorder Society-Sponsored Revision of the Unified Parkinson's Disease Rating Scale (MDS-UPDRS), part III, in comparison with baseline values.
Minimum score: 0; Maximum score: 132; Higher scores mean a worse outcome.
Time frame: Within 12 months post-transplantation
Incidence and severity of transplant-related adverse events.
Time frame: Within 12 months post-transplantation
Assessment of changes in Movement Disorder Society-Sponsored Revision of the Unified Parkinson's Disease Rating Scale (MDS-UPDRS), part I, in comparison with baseline values.
Minimum score: 0; Maximum score: 52; Higher scores mean a worse outcome.
Time frame: Within 12 months post-transplantation
Assessment of changes in Movement Disorder Society-Sponsored Revision of the Unified Parkinson's Disease Rating Scale (MDS-UPDRS), part II, in comparison with baseline values.
Minimum score: 0; Maximum score: 52; Higher scores mean a worse outcome.
Time frame: Within 12 months post-transplantation
Assessment of changes in Movement Disorder Society-Sponsored Revision of the Unified Parkinson's Disease Rating Scale (MDS-UPDRS), part IV, in comparison with baseline values.
Minimum score: 0; Maximum score: 24; Higher scores mean a worse outcome.
Time frame: Within 12 months post-transplantation
Assessment of changes during the medication "off" time in motor function by using Purdue Pegboard test and gait analyzer, in comparison with baseline values.
Time frame: Within 12 months post-transplantation
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Assessment of changes from baseline in total daily OFF time (or ratio of OFF time to daytime waking hours) at 12 months post-surgery.
Time frame: Within 12 months post-transplantation
Assessment of changes in Unified Dyskinesia Rating Scale in comparison with baseline values.
Minimum score: 0; Maximum score: 104; Higher scores mean a worse outcome.
Time frame: Within 12 months post-transplantation
Assessment of changes in daily levodopa-equivalent dose in comparison with baseline values.
Time frame: Within 12 months post-transplantation
Assessment of changes in Parkinson's Disease Questionnaire (PDQ-39) in comparison with baseline values.
Time frame: Within 12 months post-transplantation
Assessment of changes in AV133 PET imaging in comparison with baseline values.
Time frame: Within 12 months post-transplantation
Assessment of changes in International Parkinson and Movement Disorder Society - Non-Motor Rating Scale (NMSS), in comparison with baseline values.
Minimum score: 0; Maximum score: 960; Higher scores mean a worse outcome.
Time frame: Within 12 months post-transplantation