Clinical trial NCT04414813: 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
Overall status: COMPLETED
Phase: EARLY_PHASE1
Conditions: Parkinson's Disease
Keywords: Parkinson's Disease
stereotactic transplantation
Lead sponsor: Shanghai East Hospital
Sponsor class: OTHER
Collaborators: name: Shanghai iCELL Biotechnology Co., Ltd, Shanghai, China
First posted: 2020-06-04
Enrollment: 3
Sex: ALL
Minimum age (years): 30
Maximum age (years): 70
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Intervention model description: The study plans to recruit 3 patients who meet the criteria. 50 millions HAESCs will be transplanted to participants with PD.
Primary purpose: TREATMENT

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.

Eligibility criteria: Inclusion Criteria:

1. 30-70 years old, with primary Parkinson's disease more than 5 years, male or female;
2. 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% ;
3. The dosage of medicine is stable for more than 3 months;
4. Levodopa treatment was effective ;
5. Stable condition, good control of complications, no general anesthesia contraindications, no contraindications to stereotactic surgery and other conditions that interfere with clinical evaluation;
6. No abnormalities affecting cell transplantation were found through head MRI;
7. Patients shall be properly cared during treatment, and caregivers can provide assistance to the researcher if necessary.

Exclusion Criteria:

1. Atypical Parkinson's disease, such as Parkinson's syndrome, secondary Parkinson's disease;
2. Only having tremor syndrome;
3. Serious movement disorders and cannot complete any routine exercise tasks;
4. Symptoms of severe neurological deficits caused by other diseases;
5. Severe mental symptoms or dementia;
6. Patients are unwilling to cooperate or incapable of completing self-evaluation, and cannot complete the evaluator with the help of a doctor;
7. History of striatum or extrapyramidal surgery, including deep brain stimulation (DBS);
8. Ongoing treatment of apomorphine injecting ;
9. Coagulation disorders or ongoing anticoagulation therapy;
10. Women of childbearing age who do not take effective contraception;
11. Pregnant or lactation;
12. Patients who have participated in other clinical studies of drugs or medical devices within 3 months;
13. Botox toxin, phenol, subarachnoid injection of baclofen or interventional therapy for the treatment of dystonia or spasticity within 6 months;
14. History of seizures or taking prophylactic anti-epileptic drugs;
15. General anesthesia or stereotactic surgery contraindications, such as sleep apnea, chronic obstructive pulmonary disease;
16. Other circumstances judged by the investigator that may cause negative effect to the subject;
17. Alcohol or drug abuse;
18. Used diazepam within 3 months;
19. 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;
20. Chest CT shows active disease or tumor;
21. Currently suffering from or ever had a tumor other than cutaneous basal cell tumor and cervical carcinoma in situ;
22. 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;
23. 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.
24. Other situations not suitable for enrollment judged by investigator

Primary outcomes:
measure: Number of Participants with adverse event and serious adverse event
timeFrame: 12 months
Secondary outcomes:
measure: Changes in Unified Parkinson's Disease Rating Scale (UPDRS) in ON and OFF state
timeFrame: day 0, 1 month, 2 month, 3 month, 6 month, 9 month and 12 month.
measure: Changes in the Hoehn and Yahr scale
timeFrame: day 0, 1 month, 2 month, 3 month, 6 month, 9 month and 12 month.
measure: Changes in Parkinson's Disease Questionnaire (PDQ-39)
timeFrame: day 0, 1 month, 2 month, 3 month, 6 month, 9 month and 12 month.
measure: Changes in the Schwab and England score
timeFrame: day 0, 1 month, 2 month, 3 month, 6 month, 9 month and 12 month.
measure: Levodopa equivalent daily dose
timeFrame: day 0, 1 month, 2 month, 3 month, 4 month, 5 month, 6 month, 9 month and 12 month.
measure: Cranial doparmin transporter measured by positron emission tomography(PET)-magnetic resonance Imaging(MRI)
timeFrame: 4 month, 6 month
measure: Glucose metabolism measured by 18F-Fluoro-2-deoxy-glucose (FDG) PET scan
timeFrame: 4 month, 6 month
measure: Differences in biochemical indicators of cerebrospinal fluid
timeFrame: day 0, 1 month, 2 month, 3 month, 4 month,5 month,6 month, 9 month and 12 month.
Other outcomes:
Locations:
city: Shanghai
country: China
facility: WU Jingwen
state: Shanghai
Study officials:
affiliation: Shanghai East Hospital, China
name: WU Jingwen, Dr
role: PRINCIPAL_INVESTIGATOR
References: