Back to resultsSafety and Efficacy of Autologous iNSC-DAP in the Treatment of Parkinson's Disease
Primary Purpose
Parkinson's Disease
Status
Recruiting
Phase
Phase 1
Locations
China
Study Type
Interventional
Intervention
Autologous induced neural stem cell-derived DA precursor cells
Sponsored by
About this trial
This is an interventional treatment trial for Parkinson's Disease focused on measuring Autologous; iNSC; DA precursor cells; Parkinson's Disease; Cell therapy; Stereotaxic injection
Eligibility Criteria
Inclusion Criteria: Ages between 30 and 85 years, males or females; Diagnosed to be Parkinson's disease patients according to MDS Parkinson's disease diagnostic criteria; Disease history over 3 years; Hoehn and Yahr Stage less than or equal to 4 during the medication "on" time; Responsive to levodopa treatment (Maximum rate of improvement in MDS-UPDRS, part 3, is over 30%). Exclusion Criteria: Atypical Parkinsonian syndrome or secondary Parkinsonian syndrome; Accompanied with other central nervous system diseases; With other severe systemic diseases or dysfunction; With severe psychiatric disorders; Subjects are using hormone or cytotoxic drugs and cannot stop taking the drug during the trial; With cognitive disorders (MMSE<24); With severe dyskinesia (MDS-UPDRS part 4, score in 4.1/4.2 ≥ 2); Subjects have undergone previous brain surgery; Subjects are long-term user of anticoagulant; Subjects have intracranial lesions which may affect the surgery or follow-up studies as assessed by imaging; Subjects are unable to undergo MRI or AV133 PET examination; Pregnancy or in preparation for pregnancy; Not suitable to participate in this clinical trial as assessed by the study investigators/physicians.
Sites / Locations
- Xuanwu Hospital Capital Medical UniversityRecruiting
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Autologous induced neural stem cell-derived DA precursor cells
Arm Description
The patients will receive transplantation of autologous induced neural stem cell-derived DA precursor cells.
Outcomes
Primary Outcome Measures
Assessment 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.
Incidence and severity of transplant-related adverse events.
Secondary Outcome Measures
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.
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.
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.
Assessment of changes during the medication "off" time in motor function by using Purdue Pegboard test and gait analyzer, in comparison with baseline values.
Assessment of changes in the daily total medication "off" time in comparison with baseline values.
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.
Assessment of changes in daily levodopa-equivalent dose in comparison with baseline values.
Assessment of changes in Parkinson's Disease Questionnaire (PDQ-39) in comparison with baseline values.
Assessment of changes in AV133 PET imaging in comparison with baseline values.
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.
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT05901818
Brief Title
Safety and Efficacy of Autologous iNSC-DAP in the Treatment of Parkinson's Disease
Official Title
Safety and Efficacy of Autologous Induced Neural Stem Cell-derived Dopaminergic Precursor Cells in the Treatment of Parkinson's Disease
Study Type
Interventional
2. Study Status
Record Verification Date
May 2023
Overall Recruitment Status
Recruiting
Study Start Date
June 13, 2023 (Actual)
Primary Completion Date
December 31, 2025 (Anticipated)
Study Completion Date
December 31, 2026 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Xuanwu Hospital, Beijing
4. Oversight
Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
5. Study Description
Brief Summary
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.
Detailed Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Parkinson's Disease
Keywords
Autologous; iNSC; DA precursor cells; Parkinson's Disease; Cell therapy; Stereotaxic injection
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1
Interventional Study Model
Single Group Assignment
Model Description
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
10 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
Autologous induced neural stem cell-derived DA precursor cells
Arm Type
Experimental
Arm Description
The patients will receive transplantation of autologous induced neural stem cell-derived DA precursor cells.
Intervention Type
Drug
Intervention Name(s)
Autologous induced neural stem cell-derived DA precursor cells
Intervention Description
The autologous induced neural stem cell-derived DA precursor cells will be stereotactically implanted into the striatum of PD patients.
Primary Outcome Measure Information:
Title
Assessment 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.
Description
Minimum score: 0; Maximum score: 132; Higher scores mean a worse outcome.
Time Frame
Within 12 months post-transplantation
Title
Incidence and severity of transplant-related adverse events.
Time Frame
Within 12 months post-transplantation
Secondary Outcome Measure Information:
Title
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.
Description
Minimum score: 0; Maximum score: 52; Higher scores mean a worse outcome.
Time Frame
Within 12 months post-transplantation
Title
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.
Description
Minimum score: 0; Maximum score: 52; Higher scores mean a worse outcome.
Time Frame
Within 12 months post-transplantation
Title
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.
Description
Minimum score: 0; Maximum score: 24; Higher scores mean a worse outcome.
Time Frame
Within 12 months post-transplantation
Title
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
Title
Assessment of changes in the daily total medication "off" time in comparison with baseline values.
Time Frame
Within 12 months post-transplantation
Title
Assessment of changes in Unified Dyskinesia Rating Scale in comparison with baseline values.
Description
Minimum score: 0; Maximum score: 104; Higher scores mean a worse outcome.
Time Frame
Within 12 months post-transplantation
Title
Assessment of changes in daily levodopa-equivalent dose in comparison with baseline values.
Time Frame
Within 12 months post-transplantation
Title
Assessment of changes in Parkinson's Disease Questionnaire (PDQ-39) in comparison with baseline values.
Time Frame
Within 12 months post-transplantation
Title
Assessment of changes in AV133 PET imaging in comparison with baseline values.
Time Frame
Within 12 months post-transplantation
Title
Assessment of changes in International Parkinson and Movement Disorder Society - Non-Motor Rating Scale (NMSS), in comparison with baseline values.
Description
Minimum score: 0; Maximum score: 960; Higher scores mean a worse outcome.
Time Frame
Within 12 months post-transplantation
10. Eligibility
Sex
All
Minimum Age & Unit of Time
30 Years
Maximum Age & Unit of Time
85 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Ages between 30 and 85 years, males or females; Diagnosed to be Parkinson's disease patients according to MDS Parkinson's disease diagnostic criteria; Disease history over 3 years; Hoehn and Yahr Stage less than or equal to 4 during the medication "on" time; Responsive to levodopa treatment (Maximum rate of improvement in MDS-UPDRS, part 3, is over 30%).
Exclusion Criteria:
Atypical Parkinsonian syndrome or secondary Parkinsonian syndrome; Accompanied with other central nervous system diseases; With other severe systemic diseases or dysfunction; With severe psychiatric disorders; Subjects are using hormone or cytotoxic drugs and cannot stop taking the drug during the trial; With cognitive disorders (MMSE<24); With severe dyskinesia (MDS-UPDRS part 4, score in 4.1/4.2 ≥ 2); Subjects have undergone previous brain surgery; Subjects are long-term user of anticoagulant; Subjects have intracranial lesions which may affect the surgery or follow-up studies as assessed by imaging; Subjects are unable to undergo MRI or AV133 PET examination; Pregnancy or in preparation for pregnancy; Not suitable to participate in this clinical trial as assessed by the study investigators/physicians.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Zhiguo Chen, Ph.D.
Phone
+86-10-83198889
Email
chenzhiguo@gmail.com
First Name & Middle Initial & Last Name or Official Title & Degree
Jinghong Ma, M.D.
Phone
+86-10-83198677
Email
jinghongma@163.com
Facility Information:
Facility Name
Xuanwu Hospital Capital Medical University
City
Beijing
State/Province
Beijing
ZIP/Postal Code
100053
Country
China
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Zhiguo Chen, Ph.D.
Phone
86-10-83198889
Email
chenzhiguo@gmail.com
First Name & Middle Initial & Last Name & Degree
Zhiguo Chen, Ph.D.
First Name & Middle Initial & Last Name & Degree
Biao Chen, M.D., Ph.D.
First Name & Middle Initial & Last Name & Degree
Guoguang Zhao, M.D., Ph.D.
First Name & Middle Initial & Last Name & Degree
Jie Lu, M.D., Ph.D.
12. IPD Sharing Statement
Learn more about this trial
Safety and Efficacy of Autologous iNSC-DAP in the Treatment of Parkinson's Disease
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