search
Back to results

Precise Transplantation of Human Amniotic Epithelial Stem Cells Into Lateral Ventricle for Parkinson's Disease

Primary Purpose

Idiopathic Parkinson's Disease

Status
Recruiting
Phase
Phase 1
Locations
China
Study Type
Interventional
Intervention
hAESCs
Sponsored by
Shanghai East Hospital
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Idiopathic Parkinson's Disease

Eligibility Criteria

40 Years - 70 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria: 40-70 years old, with more than 5 years of idiopathic PD history UPDRS-III off-time scores ≤49 MMSE scores ≥24 HAMD-17 scores < 25 H-Y on-time scores ≤4 reactive to levodopa or dopa agonists PD medication dose is stable for more than 2 months no general anesthesia contraindications, no stereotactic surgery contraindications or other conditions that interfere with clinical evaluation no abnormalities affecting cell transplantation by cranial MRI no participation of other clinical trials 3 months before signing the informed consent Exclusion Criteria: secondary PD or Parkinson's syndrome subcutaneous apomorphine treatment scoring ≥ 2 on UPDRS-I item 2, or ≥ 2 on UPDRS-II item 13; or ≥ 3 on UPDRS-II item14 history of intracranial surgery or device implantation, including deep brain stimulation, within 2 years prior to signing informed consent history of seizures or prophylactic application of antiepileptic drugs other serious central nervous system disorders history of stem cell therapy subject who had undergone a major surgery within 3 months and will undergo a major surgery within the next 6 months prior to signing informed consent autoimmune disease or current use of Immunosuppressants subjects with comorbid cardiac disease, for example, but not limited to, ischemic heart disease, congestive heart failure, significant arrhythmias or cardiac conduction block poorly controlled hypertension, diabetes mellitus, or comorbid endocrine system disorders, pulmonary disorders, gastrointestinal system disorders, serious infections, malignancies, etc. positive for hepatitis B surface antigen (HBsAg), hepatitis C virus (HCV) antibodies, human immunodeficiency virus (HIV) antibodies, or Treponema pallidum antibody abnormalities in liver or kidney function tests, including alanine aminotransferase (ALT), aspartate aminotransferase (AST) is less than 2.5 times the upper limit of normal, blood urea nitrogen (BUN) or creatinine(Cr) are less than 1.5 times the upper limit of normal, or serum albumin < 30.0 g/L abnormalities in hematologic test: coagulation disorders or ongoing anticoagulation therapy; moderate to severe anemia; platelet count < 80 × 10^9/L inability to undergo MRI and positron emission tomography (PET) examinations subject with severe allergies women of childbearing potential (WOCBP) or subject is a man with a WOCBP partner, who are unwilling to take contraception during the trial pregnant or lactating females other conditions deemed by the investigator to be inappropriate for enrollment.

Sites / Locations

  • Shanghai East HospitalRecruiting

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

hAESCs treatment

Arm Description

hAESCs will be administration through Ommaya reservoir implanted into the lateral ventricle. The tolerability, safety, and efficacy will be examined of 4 monthly doses of hAESCs for 3 months followed by 2 doses every 3 months in dose escalation through 3 cohorts. Dose A (5×10^7 cells/dose) Dose B (1.0×10^8 cells/dose) Dose C (1.5×10^8 cells/dose).

Outcomes

Primary Outcome Measures

Dose limiting toxicity (DLT)
The occurrence of DLT.
Number of participants with adverse event (AE), serious adverse event(SAE) and adverse events of special interest (AESI)
According to CTCAE V5.0, AE, SAE and AESI evaluated by laboratory examination, measurement of vital signs, physical examination, and subjects' symptoms to detect new abnormalities and/or deterioration of previous conditions. AESI is defined as intracranial infection, hemorrhage, rejection, edema, as well as acute allergic reactions and ectopic mass formation associated with the therapy.

Secondary Outcome Measures

Change from baseline in the UPDRS-III (Unified Parkinson's Rating Scale part III/motor part) scores
UPDRS is used for evaluating the impairment and disability associated with PD. It consists of 4 sections: I Mentation, behavior, and mood; II Activities of daily living (ADLs); III Motor; and IV Complications.The UPDRS score ranges from 0 to 199, with higher score indicating greater disability
Change from baseline in the UPDRS off-time total scores
UPDRS is used for evaluating the impairment and disability associated with PD. It consists of 4 sections: I Mentation, behavior, and mood; II Activities of daily living (ADLs); III Motor; and IV Complications.The UPDRS score ranges from 0 to 199, with higher score indicating greater disability
Change from baseline in the UPDRS on-time total scores
UPDRS is used for evaluating the impairment and disability associated with PD. It consists of 4 sections: I Mentation, behavior, and mood; II Activities of daily living (ADLs); III Motor; and IV Complications.The UPDRS score ranges from 0 to 199, with higher score indicating greater disability
Change from baseline in sum of the UPDRS-II and UPDRS-III scores
UPDRS is used for evaluating the impairment and disability associated with PD. It consists of 4 sections: I Mentation, behavior, and mood; II Activities of daily living (ADLs); III Motor; and IV Complications.The UPDRS score ranges from 0 to 199, with higher score indicating greater disability
Changes from baseline in the Parkinson's Disease Questionnaire (PDQ-39) scores
The PDQ-39 is a self-completed questionnaire assessing how often people with Parkinson's experience difficulties across 8 dimensions of daily living with 39 questions . Questions are answered using a 5-point ordinal scoring system: 0 = never, 1 = occasionally, 2 = sometimes, 3 = often, 4 = always. Each scores range from 0 = never have difficulty to 100 = always have difficulty.The higher the score, the lower the quality of life of PD patients.
Changes in the Hamilton Depression-17 (HAMD-17) Scale scores
The HAMD-17 Scale is the most widely used scale in clinical evaluation of depression in PD patients. This project takes 8 points as the critical value of depression, and the total score over 35 points is considered as severe depression, more than 20 points is likely to be diagnosed with depression, less than 8 points is not depressed.
Change from baseline in the Parkinson's Disease Sleep Scale (PDSS) scores
The PDSS is a visual analogue scale addressing 15 commonly reported symptoms associated with sleep disturbance overall quality of night's sleep (item 1), sleep onset and maintenance insomnia (items 2 and 3), nocturnal restlessness (items 4 and 5), nocturnal psychosis (items 6 and 7), nocturia (items 8 and 9), nocturnal motor symptoms (items 10-13), sleep refreshment (item 14) and daytime dozing (item 15). Scores range from 0 (poorly or often) to 10 (very good or never), with a total score of <90 indicating sleep disturbance, with higher scores indicating better sleep quality.
Changes from baseline in the Hoehn and Yahr scale (H-Y)
The Hoehn and Yahr scale is used to provide a general estimate of clinical function of PD patients, combining functional deficits (disability) and objective signs (impairment). The Hoehn and Yahr score ranges from 0 to 5, with higher score indicating higher dysfunction of PD patients.
Changes from baseline in the Mini-Mental State Examination (MMSE)
The total score of tne MMSE ranges from 0 to 30, with a higher score indicating better function for Cognitive assessment.

Full Information

First Posted
January 10, 2023
Last Updated
February 16, 2023
Sponsor
Shanghai East Hospital
Collaborators
Shanghai iCELL Biotechnology Co., Ltd, Shanghai, China
search

1. Study Identification

Unique Protocol Identification Number
NCT05691114
Brief Title
Precise Transplantation of Human Amniotic Epithelial Stem Cells Into Lateral Ventricle for Parkinson's Disease
Official Title
Precise Transplantation of Human Amniotic Epithelial Stem Cells Into Lateral Ventricle for Parkinson's Disease
Study Type
Interventional

2. Study Status

Record Verification Date
February 2023
Overall Recruitment Status
Recruiting
Study Start Date
February 2023 (Anticipated)
Primary Completion Date
February 2025 (Anticipated)
Study Completion Date
February 2026 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Shanghai East Hospital
Collaborators
Shanghai iCELL Biotechnology Co., Ltd, Shanghai, China

4. Oversight

Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No

5. Study Description

Brief Summary
This is a single-center, single-arm, dose escalation study, to explore the safety, tolerability and efficacy of human amniotic epithelial stem cells (hAESCs) for idiopathic Parkinson's disease (PD).
Detailed Description
hAESCs will be administration through the Ommaya reservoir implanted into the lateral ventricle of subjects with idiopathic PD. This dose escalation will be followed by an exploratory expansion phase in 3 cohorts. Dose A (5×10^7 cells/dose) Dose B (1.0×10^8 cells/dose) Dose C (1.5×10^8 cells/dose).

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Idiopathic Parkinson's Disease

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 1
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
18 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
hAESCs treatment
Arm Type
Experimental
Arm Description
hAESCs will be administration through Ommaya reservoir implanted into the lateral ventricle. The tolerability, safety, and efficacy will be examined of 4 monthly doses of hAESCs for 3 months followed by 2 doses every 3 months in dose escalation through 3 cohorts. Dose A (5×10^7 cells/dose) Dose B (1.0×10^8 cells/dose) Dose C (1.5×10^8 cells/dose).
Intervention Type
Biological
Intervention Name(s)
hAESCs
Intervention Description
human Amniotic Epithelial Stem Cells
Primary Outcome Measure Information:
Title
Dose limiting toxicity (DLT)
Description
The occurrence of DLT.
Time Frame
12 months
Title
Number of participants with adverse event (AE), serious adverse event(SAE) and adverse events of special interest (AESI)
Description
According to CTCAE V5.0, AE, SAE and AESI evaluated by laboratory examination, measurement of vital signs, physical examination, and subjects' symptoms to detect new abnormalities and/or deterioration of previous conditions. AESI is defined as intracranial infection, hemorrhage, rejection, edema, as well as acute allergic reactions and ectopic mass formation associated with the therapy.
Time Frame
12 Months
Secondary Outcome Measure Information:
Title
Change from baseline in the UPDRS-III (Unified Parkinson's Rating Scale part III/motor part) scores
Description
UPDRS is used for evaluating the impairment and disability associated with PD. It consists of 4 sections: I Mentation, behavior, and mood; II Activities of daily living (ADLs); III Motor; and IV Complications.The UPDRS score ranges from 0 to 199, with higher score indicating greater disability
Time Frame
12 months
Title
Change from baseline in the UPDRS off-time total scores
Description
UPDRS is used for evaluating the impairment and disability associated with PD. It consists of 4 sections: I Mentation, behavior, and mood; II Activities of daily living (ADLs); III Motor; and IV Complications.The UPDRS score ranges from 0 to 199, with higher score indicating greater disability
Time Frame
12 months
Title
Change from baseline in the UPDRS on-time total scores
Description
UPDRS is used for evaluating the impairment and disability associated with PD. It consists of 4 sections: I Mentation, behavior, and mood; II Activities of daily living (ADLs); III Motor; and IV Complications.The UPDRS score ranges from 0 to 199, with higher score indicating greater disability
Time Frame
12 months
Title
Change from baseline in sum of the UPDRS-II and UPDRS-III scores
Description
UPDRS is used for evaluating the impairment and disability associated with PD. It consists of 4 sections: I Mentation, behavior, and mood; II Activities of daily living (ADLs); III Motor; and IV Complications.The UPDRS score ranges from 0 to 199, with higher score indicating greater disability
Time Frame
12 months
Title
Changes from baseline in the Parkinson's Disease Questionnaire (PDQ-39) scores
Description
The PDQ-39 is a self-completed questionnaire assessing how often people with Parkinson's experience difficulties across 8 dimensions of daily living with 39 questions . Questions are answered using a 5-point ordinal scoring system: 0 = never, 1 = occasionally, 2 = sometimes, 3 = often, 4 = always. Each scores range from 0 = never have difficulty to 100 = always have difficulty.The higher the score, the lower the quality of life of PD patients.
Time Frame
12 months
Title
Changes in the Hamilton Depression-17 (HAMD-17) Scale scores
Description
The HAMD-17 Scale is the most widely used scale in clinical evaluation of depression in PD patients. This project takes 8 points as the critical value of depression, and the total score over 35 points is considered as severe depression, more than 20 points is likely to be diagnosed with depression, less than 8 points is not depressed.
Time Frame
12 months
Title
Change from baseline in the Parkinson's Disease Sleep Scale (PDSS) scores
Description
The PDSS is a visual analogue scale addressing 15 commonly reported symptoms associated with sleep disturbance overall quality of night's sleep (item 1), sleep onset and maintenance insomnia (items 2 and 3), nocturnal restlessness (items 4 and 5), nocturnal psychosis (items 6 and 7), nocturia (items 8 and 9), nocturnal motor symptoms (items 10-13), sleep refreshment (item 14) and daytime dozing (item 15). Scores range from 0 (poorly or often) to 10 (very good or never), with a total score of <90 indicating sleep disturbance, with higher scores indicating better sleep quality.
Time Frame
12 months
Title
Changes from baseline in the Hoehn and Yahr scale (H-Y)
Description
The Hoehn and Yahr scale is used to provide a general estimate of clinical function of PD patients, combining functional deficits (disability) and objective signs (impairment). The Hoehn and Yahr score ranges from 0 to 5, with higher score indicating higher dysfunction of PD patients.
Time Frame
12 months
Title
Changes from baseline in the Mini-Mental State Examination (MMSE)
Description
The total score of tne MMSE ranges from 0 to 30, with a higher score indicating better function for Cognitive assessment.
Time Frame
12 months

10. Eligibility

Sex
All
Minimum Age & Unit of Time
40 Years
Maximum Age & Unit of Time
70 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: 40-70 years old, with more than 5 years of idiopathic PD history UPDRS-III off-time scores ≤49 MMSE scores ≥24 HAMD-17 scores < 25 H-Y on-time scores ≤4 reactive to levodopa or dopa agonists PD medication dose is stable for more than 2 months no general anesthesia contraindications, no stereotactic surgery contraindications or other conditions that interfere with clinical evaluation no abnormalities affecting cell transplantation by cranial MRI no participation of other clinical trials 3 months before signing the informed consent Exclusion Criteria: secondary PD or Parkinson's syndrome subcutaneous apomorphine treatment scoring ≥ 2 on UPDRS-I item 2, or ≥ 2 on UPDRS-II item 13; or ≥ 3 on UPDRS-II item14 history of intracranial surgery or device implantation, including deep brain stimulation, within 2 years prior to signing informed consent history of seizures or prophylactic application of antiepileptic drugs other serious central nervous system disorders history of stem cell therapy subject who had undergone a major surgery within 3 months and will undergo a major surgery within the next 6 months prior to signing informed consent autoimmune disease or current use of Immunosuppressants subjects with comorbid cardiac disease, for example, but not limited to, ischemic heart disease, congestive heart failure, significant arrhythmias or cardiac conduction block poorly controlled hypertension, diabetes mellitus, or comorbid endocrine system disorders, pulmonary disorders, gastrointestinal system disorders, serious infections, malignancies, etc. positive for hepatitis B surface antigen (HBsAg), hepatitis C virus (HCV) antibodies, human immunodeficiency virus (HIV) antibodies, or Treponema pallidum antibody abnormalities in liver or kidney function tests, including alanine aminotransferase (ALT), aspartate aminotransferase (AST) is less than 2.5 times the upper limit of normal, blood urea nitrogen (BUN) or creatinine(Cr) are less than 1.5 times the upper limit of normal, or serum albumin < 30.0 g/L abnormalities in hematologic test: coagulation disorders or ongoing anticoagulation therapy; moderate to severe anemia; platelet count < 80 × 10^9/L inability to undergo MRI and positron emission tomography (PET) examinations subject with severe allergies women of childbearing potential (WOCBP) or subject is a man with a WOCBP partner, who are unwilling to take contraception during the trial pregnant or lactating females other conditions deemed by the investigator to be inappropriate for enrollment.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Jingwen Wu, M.D.
Phone
021-38804518
Email
wujingwendongfang@163.com
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Jingwen Wu, M.D.
Organizational Affiliation
Shanghai East Hospital
Official's Role
Principal Investigator
Facility Information:
Facility Name
Shanghai East Hospital
City
Shanghai
State/Province
Shanghai
ZIP/Postal Code
200000
Country
China
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Jingwen Wu, M.D.
Phone
18916111526
Email
wujingwendongfang@163.com

12. IPD Sharing Statement

Plan to Share IPD
No

Learn more about this trial

Precise Transplantation of Human Amniotic Epithelial Stem Cells Into Lateral Ventricle for Parkinson's Disease

We'll reach out to this number within 24 hrs