A Phase I, Open Label, Single Arm, Dose Escalation and Dose Expansion Clinical Trial to Evaluate the Safety, Tolerability and Efficacy of Human Induced Pluripotent Stem Cell Derived Dopaminergic Progenitor Cells (NCR201) Injection in the Treatment of Subjects With Parkinson's Disease
Trial Snapshot
- Phase
- Phase 1
- Status
- Recruiting
- Enrollment
- 48
- Locations
- 1
- Primary Endpoint
- Incidence and severity of adverse events.
Study Overview
Brief Summary
The goal of this clinical trial is to evaluate the safety, tolerability and preliminary efficacy that NCR201 has on Parkinson's disease (PD) patients.
Detailed Description
Parkinson's disease (PD) is a common neurodegenerative disease in the middle-aged and elderly. It is the "third killer" of the middle-aged and elderly after tumors and cardiovascular and cerebrovascular diseases. Its main clinical manifestations are resting tremor, reduced voluntary movement, muscle rigidity, postural reflex impairment, and autonomic dysfunction, which seriously affect patients' work ability and quality of life. It is estimated that nearly 100,000 people in China become new Parkinson's patients every year. Experts from the World Health Organization predict that the number of Parkinson's patients in China will reach 5 million in 2030, which will be more than half of the world's total. As the disease progresses, the symptoms of Parkinson's patients will become increasingly severe. The high prevalence and high disability rate of Parkinson's disease bring heavy burdens to individuals, families, and society.
Study Design
- Study Type
- Interventional
- Allocation
- Non Randomized
- Intervention Model
- Sequential
- Primary Purpose
- Treatment
- Masking
- None
Eligibility Criteria
- Ages
- 40 Years to 75 Years (Adult, Older Adult)
- Sex
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- •Ages between 40 and 75 years;
- •Diagnosed to be Parkinson's disease according to Parkinson's disease diagnostic criteria;
- •Disease history over 5 years;
- •Stable dose of dopamine treatment;
- •Able to undergo PET/CT/MRI detection;
Exclusion Criteria
- •Patients who have previously undergone brain surgery;
- •Past use of stem cell therapy or participation in stem cell clinical research;
- •Cognitive impairment;
- •History of mental disorders;
- •Patients with other serious systemic diseases;
- •Past or current metastatic malignant tumors.
Arms & Interventions
Low Dose
MRI-guided bilateral stereotactic cell implantation
Intervention: Allogeneic dopaminergic neural precursor cell(NCR201) (Drug)
High Dose
MRI-guided bilateral stereotactic cell implantation
Intervention: Allogeneic dopaminergic neural precursor cell(NCR201) (Drug)
Outcomes
Primary Outcomes
Incidence and severity of adverse events.
Time Frame: Within 24 weeks post-transplantation
Safety and tolerability
Secondary Outcomes
- Assessment of changes in Movement Disorder Society-Sponsored Revision of the Unified Parkinson's Disease Rating Scale (MDS-UPDRS), part III, in comparison with baseline values.(Within 24 months post-transplantation)
- Assessment of changes in Hamilton Anxiety Scale (HAMA)-14 in comparison with baseline values.(Within 24 months post-transplantation)
- Assessment of changes in Parkinson's Disease Questionnaire-39 (PDQ-39) in comparison with baseline values.(Within 24 months post-transplantation)
- Assessment of changes in Hoehn & Yahr scale in comparison with baseline values.(Within 24 months post-transplantation)
- Assessment of changes in Movement Disorder Society-Sponsored Revision of the Unified Parkinson's Disease Rating Scale (MDS-UPDRS), part I,II,IV, in comparison with baseline values.(Within 24 months post-transplantation)
- Assessment of changes in Hamilton Depression Scale (HAMD)-17 in comparison with baseline values.(Within 24 months post-transplantation)
- Bilateral putamen standardized uptake value as demonstrated on positron emission tomography(PET) compared with baseline in the 'off' state.(Within 24 months post-transplantation)
- Patient L-dopa equivalent dose compared with baseline.(Within 24 months post-transplantation)
- Incidence and severity of adverse events.(Within 24 months post-transplantation)