Back to resultsStereotactic Transplantation of hAESCs for Parkinson's Disease
Primary Purpose
Parkinson's Disease
Status
Completed
Phase
Early Phase 1
Locations
China
Study Type
Interventional
Intervention
Human Amniotic Epithelial Stem Cells
Sponsored by
About this trial
This is an interventional treatment trial for Parkinson's Disease focused on measuring Parkinson's Disease, stereotactic transplantation
Eligibility Criteria
Inclusion Criteria:
- 30-70 years old, with primary Parkinson's disease more than 5 years, male or female;
- The "off-stage"Hohen and Yahr grade of Parkinson's disease is from 2 to 4; and the"off-stage"UPDRS score is 38-70, difference between two scores before treatment is less than 10% ;
- The dosage of medicine is stable for more than 3 months;
- Levodopa treatment was effective ;
- Stable condition, good control of complications, no general anesthesia contraindications, no contraindications to stereotactic surgery and other conditions that interfere with clinical evaluation;
- No abnormalities affecting cell transplantation were found through head MRI;
- Patients shall be properly cared during treatment, and caregivers can provide assistance to the researcher if necessary.
Exclusion Criteria:
- Atypical Parkinson's disease, such as Parkinson's syndrome, secondary Parkinson's disease;
- Only having tremor syndrome;
- Serious movement disorders and cannot complete any routine exercise tasks;
- Symptoms of severe neurological deficits caused by other diseases;
- Severe mental symptoms or dementia;
- Patients are unwilling to cooperate or incapable of completing self-evaluation, and cannot complete the evaluator with the help of a doctor;
- History of striatum or extrapyramidal surgery, including deep brain stimulation (DBS);
- Ongoing treatment of apomorphine injecting ;
- Coagulation disorders or ongoing anticoagulation therapy;
- Women of childbearing age who do not take effective contraception;
- Pregnant or lactation;
- Patients who have participated in other clinical studies of drugs or medical devices within 3 months;
- Botox toxin, phenol, subarachnoid injection of baclofen or interventional therapy for the treatment of dystonia or spasticity within 6 months;
- History of seizures or taking prophylactic anti-epileptic drugs;
- General anesthesia or stereotactic surgery contraindications, such as sleep apnea, chronic obstructive pulmonary disease;
- Other circumstances judged by the investigator that may cause negative effect to the subject;
- Alcohol or drug abuse;
- Used diazepam within 3 months;
- Severe cognitive impairment, depression, or behavioral disorder, defined as the Mini-Mental State Assessment Scale (MMSE) of less than 26 points, and the Hamilton Depression Rating Scale (HAMD) of greater than 35 points;
- Chest CT shows active disease or tumor;
- Currently suffering from or ever had a tumor other than cutaneous basal cell tumor and cervical carcinoma in situ;
- The detection of HIV, hepatitis B virus(HBV), hepatitis C virus (HCV), syphilis and other infection indicators before surgery can not exclude HIV and syphilis infection;
- Abnormal liver and kidney normal function tests in the laboratory, liver and kidney function is defined as Alanine aminotransferase（ALT）, Aspartate aminotransferase（AST） is less than 2.5 times the upper limit of normal, blood urea nitrogen (BUN) and Creatinine（Cr） are less than the upper limit of normal.
- Other situations not suitable for enrollment judged by investigator
Sites / Locations
- WU Jingwen
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
hAESCs treatment
Arm Description
50 millions hAESCs are transplanted to participants with PD.
Outcomes
Primary Outcome Measures
Number of Participants with adverse event and serious adverse event
An adverse event (AE) is defined as any untoward medical occurrence in a clinical trial participant. The event does not necessarily have a causal relationship with study treatment. A serious adverse event is defined as an adverse event that meets at least one of the following serious criteria: • fatal • life threatening • requires in-patient hospitalization or prolongation of existing hospitalization • results in persistent or significant disability/incapacity • congenital anomaly/birth defect • other significant medical hazard. Whether an adverse event was treatment-related (TRAE) or not was determined by investigator.
Secondary Outcome Measures
Changes in Unified Parkinson's Disease Rating Scale (UPDRS) in ON and OFF state
UPDRS is used for evaluating the impairment and disability associated with Parkinson's Disease (PD). It consists of four sections: (1) Mentation, behavior, and mood; (2) Activities of daily living (ADLs); (3) Motor; and (4) Complications.
The UPDRS score ranges from 0 to 199, with higher score indicating greater disability.
Changes in the Hoehn and Yahr scale
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 in Parkinson's Disease Questionnaire (PDQ-39)
PDQ-39 provides the evidence of the quality of life of a PD patient. The higher the score, the lower the quality of life of PD patients.
Changes in the Schwab and England score
The Schwab and England scale develop a scale that describes the capacity of daily living shown by a PD patient. It measures the following three areas: dependence, abilities, and awareness. The Schwab and England scores range from 0% to 100%, with higher scores indicating greater healthy status.
Levodopa equivalent daily dose
Differences in daily dosage of levodopamine
Cranial doparmin transporter measured by positron emission tomography(PET)-magnetic resonance Imaging(MRI)
Changes of cranial expression of dopamine transporter investigated by PET-MR
Glucose metabolism measured by 18F-Fluoro-2-deoxy-glucose (FDG) PET scan
Changes in cranial glucose metabolism investigated by PET-MR
Differences in biochemical indicators of cerebrospinal fluid
Cerebrospinal fluid indicator tests include biochemical indicators and the following parameters: Dopamine (DA), high vanillic acid (HVA), 5-hydroxytryptamine (5-HT), 5-hydroxyindoleacetic acid (5-HIAA), norepinephrine (MHPG), α synuclein (α-synuclein), amyloid deposition (Aβ42), Tau protein (Tau) Protein, Neurofilament light (NF-L), and yKL-40 (YKL-40).
Full Information
NCT ID
NCT04414813
First Posted
May 21, 2019
Last Updated
April 11, 2023
Sponsor
Shanghai East Hospital
Collaborators
Shanghai iCELL Biotechnology Co., Ltd, Shanghai, China
1. Study Identification
Unique Protocol Identification Number
NCT04414813
Brief Title
Stereotactic Transplantation of hAESCs for Parkinson's Disease
Official Title
Clinical Study of Stereotactic Transplantation of Human Amniotic Epithelial Stem Cells (hAESCs) in the Treatment of Parkinson's Disease (PD)
Study Type
Interventional
2. Study Status
Record Verification Date
April 2023
Overall Recruitment Status
Completed
Study Start Date
October 8, 2020 (Actual)
Primary Completion Date
February 8, 2023 (Actual)
Study Completion Date
February 8, 2023 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
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
Yes
5. Study Description
Brief Summary
The purpose of this study is to evaluate the safety and efficacy of stereotactic transplantation of hAESCs for Parkinson's disease.
Detailed Description
The study plans to recruit 3 participants with Parkinson's disease . Using stereotactic technology, hAESCs will be accurately transplanted into the lateral ventricle. Therapeutic effectiveness and safety of hAESCs on PD will be evaluated. hAESCs are derived from placental amnion donated after cesarean section in healthy women. hAESCs is developed from the epiblast as early as 8 days after fertilization, recent reports indicate that hAESCs have some characteristics of neural stem cell. These cells are able to differentiate into dopaminergic neurons and secrete dopamine and various neurotrophic factors. These HAESCs could be seen as one of the best potential stem cell source for treating Parkinson's disease.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Parkinson's Disease
Keywords
Parkinson's Disease, stereotactic transplantation
7. Study Design
Primary Purpose
Treatment
Study Phase
Early Phase 1
Interventional Study Model
Single Group Assignment
Model Description
The study plans to recruit 3 patients who meet the criteria. 50 millions HAESCs will be transplanted to participants with PD.
Masking
None (Open Label)
Allocation
N/A
Enrollment
3 (Actual)
8. Arms, Groups, and Interventions
Arm Title
hAESCs treatment
Arm Type
Experimental
Arm Description
50 millions hAESCs are transplanted to participants with PD.
Intervention Type
Biological
Intervention Name(s)
Human Amniotic Epithelial Stem Cells
Intervention Description
Stereotactic transplantation of hAESCs for participants with PD.
Primary Outcome Measure Information:
Title
Number of Participants with adverse event and serious adverse event
Description
An adverse event (AE) is defined as any untoward medical occurrence in a clinical trial participant. The event does not necessarily have a causal relationship with study treatment. A serious adverse event is defined as an adverse event that meets at least one of the following serious criteria: • fatal • life threatening • requires in-patient hospitalization or prolongation of existing hospitalization • results in persistent or significant disability/incapacity • congenital anomaly/birth defect • other significant medical hazard. Whether an adverse event was treatment-related (TRAE) or not was determined by investigator.
Time Frame
12 months
Secondary Outcome Measure Information:
Title
Changes in Unified Parkinson's Disease Rating Scale (UPDRS) in ON and OFF state
Description
UPDRS is used for evaluating the impairment and disability associated with Parkinson's Disease (PD). It consists of four sections: (1) Mentation, behavior, and mood; (2) Activities of daily living (ADLs); (3) Motor; and (4) Complications.
The UPDRS score ranges from 0 to 199, with higher score indicating greater disability.
Time Frame
day 0, 1 month, 2 month, 3 month, 6 month, 9 month and 12 month.
Title
Changes in the Hoehn and Yahr scale
Description
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
day 0, 1 month, 2 month, 3 month, 6 month, 9 month and 12 month.
Title
Changes in Parkinson's Disease Questionnaire (PDQ-39)
Description
PDQ-39 provides the evidence of the quality of life of a PD patient. The higher the score, the lower the quality of life of PD patients.
Time Frame
day 0, 1 month, 2 month, 3 month, 6 month, 9 month and 12 month.
Title
Changes in the Schwab and England score
Description
The Schwab and England scale develop a scale that describes the capacity of daily living shown by a PD patient. It measures the following three areas: dependence, abilities, and awareness. The Schwab and England scores range from 0% to 100%, with higher scores indicating greater healthy status.
Time Frame
day 0, 1 month, 2 month, 3 month, 6 month, 9 month and 12 month.
Title
Levodopa equivalent daily dose
Description
Differences in daily dosage of levodopamine
Time Frame
day 0, 1 month, 2 month, 3 month, 4 month, 5 month, 6 month, 9 month and 12 month.
Title
Cranial doparmin transporter measured by positron emission tomography(PET)-magnetic resonance Imaging(MRI)
Description
Changes of cranial expression of dopamine transporter investigated by PET-MR
Time Frame
4 month, 6 month
Title
Glucose metabolism measured by 18F-Fluoro-2-deoxy-glucose (FDG) PET scan
Description
Changes in cranial glucose metabolism investigated by PET-MR
Time Frame
4 month, 6 month
Title
Differences in biochemical indicators of cerebrospinal fluid
Description
Cerebrospinal fluid indicator tests include biochemical indicators and the following parameters: Dopamine (DA), high vanillic acid (HVA), 5-hydroxytryptamine (5-HT), 5-hydroxyindoleacetic acid (5-HIAA), norepinephrine (MHPG), α synuclein (α-synuclein), amyloid deposition (Aβ42), Tau protein (Tau) Protein, Neurofilament light (NF-L), and yKL-40 (YKL-40).
Time Frame
day 0, 1 month, 2 month, 3 month, 4 month,5 month,6 month, 9 month and 12 month.
10. Eligibility
Sex
All
Minimum Age & Unit of Time
30 Years
Maximum Age & Unit of Time
70 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
30-70 years old, with primary Parkinson's disease more than 5 years, male or female;
The "off-stage"Hohen and Yahr grade of Parkinson's disease is from 2 to 4; and the"off-stage"UPDRS score is 38-70, difference between two scores before treatment is less than 10% ;
The dosage of medicine is stable for more than 3 months;
Levodopa treatment was effective ;
Stable condition, good control of complications, no general anesthesia contraindications, no contraindications to stereotactic surgery and other conditions that interfere with clinical evaluation;
No abnormalities affecting cell transplantation were found through head MRI;
Patients shall be properly cared during treatment, and caregivers can provide assistance to the researcher if necessary.
Exclusion Criteria:
Atypical Parkinson's disease, such as Parkinson's syndrome, secondary Parkinson's disease;
Only having tremor syndrome;
Serious movement disorders and cannot complete any routine exercise tasks;
Symptoms of severe neurological deficits caused by other diseases;
Severe mental symptoms or dementia;
Patients are unwilling to cooperate or incapable of completing self-evaluation, and cannot complete the evaluator with the help of a doctor;
History of striatum or extrapyramidal surgery, including deep brain stimulation (DBS);
Ongoing treatment of apomorphine injecting ;
Coagulation disorders or ongoing anticoagulation therapy;
Women of childbearing age who do not take effective contraception;
Pregnant or lactation;
Patients who have participated in other clinical studies of drugs or medical devices within 3 months;
Botox toxin, phenol, subarachnoid injection of baclofen or interventional therapy for the treatment of dystonia or spasticity within 6 months;
History of seizures or taking prophylactic anti-epileptic drugs;
General anesthesia or stereotactic surgery contraindications, such as sleep apnea, chronic obstructive pulmonary disease;
Other circumstances judged by the investigator that may cause negative effect to the subject;
Alcohol or drug abuse;
Used diazepam within 3 months;
Severe cognitive impairment, depression, or behavioral disorder, defined as the Mini-Mental State Assessment Scale (MMSE) of less than 26 points, and the Hamilton Depression Rating Scale (HAMD) of greater than 35 points;
Chest CT shows active disease or tumor;
Currently suffering from or ever had a tumor other than cutaneous basal cell tumor and cervical carcinoma in situ;
The detection of HIV, hepatitis B virus(HBV), hepatitis C virus (HCV), syphilis and other infection indicators before surgery can not exclude HIV and syphilis infection;
Abnormal liver and kidney normal function tests in the laboratory, liver and kidney function is defined as Alanine aminotransferase（ALT）, Aspartate aminotransferase（AST） is less than 2.5 times the upper limit of normal, blood urea nitrogen (BUN) and Creatinine（Cr） are less than the upper limit of normal.
Other situations not suitable for enrollment judged by investigator
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
WU Jingwen, Dr
Organizational Affiliation
Shanghai East Hospital, China
Official's Role
Principal Investigator
Facility Information:
Facility Name
WU Jingwen
City
Shanghai
State/Province
Shanghai
ZIP/Postal Code
200000
Country
China
12. IPD Sharing Statement
Plan to Share IPD
No
Learn more about this trial
Stereotactic Transplantation of hAESCs for Parkinson's Disease
We'll reach out to this number within 24 hrs