Clinical trial NCT05435755: Precise Transplantation of hAESCs Into the Ventricle for Parkinson's Disease.
Official title: The Precise Transplantation of Human Amniotic Epithelial Stem Cells Into the Ventricle Through Surgical Robot in the Treatment of Parkinson's Disease.
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: EARLY_PHASE1
Conditions: Parkinson's Disease
Keywords: Parkinson's Disease; stem cell
Lead sponsor: Shanghai East Hospital
Sponsor class: OTHER
Collaborators: name: Shanghai iCELL Biotechnology Co., Ltd, Shanghai, China
First posted: 2022-06-28
Enrollment: 12
Sex: ALL
Minimum age (years): 30
Maximum age (years): 70
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Intervention model description: The study plans to recruit 12 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 of multiple treatments of hAESCs treatments, while exploring the effectiveness of hAESCs treatments for PD.

Detailed description: The study plans to use randomized controlled trial and recruit 12 participants with Idiopathic Parkinson's disease. The trial will be divided into two groups, hAESCs treatment group and control group, each group 6 subjects. Through surgical robot technology, hAESCs will be precisely multiple transplanted into the lateral ventricles, that is two times consolidation hAESCs treatments or placebo were added to the four times basic hAESCs treatments. Therapeutic safety and effectiveness of multiple treatments of hAESCs on PD will be evaluated to further develop the optimal stem cell treatment strategy.

Eligibility criteria: Inclusion Criteria:

1. 30-70 years old, with more than 5 years primary Parkinson's disease history, male or female;
2. The "off-age" UPDRS exercise score is between 38 and 70 points, two times before the operation, the "down period" UPDRS exercise score was stable, that is, (high value - low value) / high value ≤ 10%;
3. The Parkinson treatment is stable for more than 3 months;
4. Levodopa treatment was effective;
5. 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 the cranial MRI;
7. Patients shall be properly cared during treatment, and caregivers can provide transplantation situations and 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. "off period" or "on period" has 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. Injecting apomorphine treatment;
9. Abnormal blood coagulation 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 prophylactic anti-epileptic drugs;
15. Alcohol or drug abuse;
16. 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;
17. Malignant tumor or active infection within 5 years;
18. Serum virology showed positive results for hepatitis C virus (HCV) , treponema pallidum (TP) or HIV; Active hepatitis B (hepatitis B surface antigen (HBsAg) and/or hepatitis B core antibody (HBcAb) positive, and peripheral blood HBV DNA titer ≥1×10\^3 IU/ mL);
19. Abnormal liver and kidney 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.
20. Other conditions considered ineligible for inclusion by the investigator.

Primary outcomes:
measure: Number of participants with adverse event (AE), serious adverse event(SAE) and adverse events of special interest(AESI)
timeFrame: 12 Months
Secondary outcomes:
measure: Changes in Unified Parkinson's Disease Rating Scale (UPDRS) in OFF state between the experimental group and the control group at 12 months after the first hAESCs treatment
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 the Schwab and England score
timeFrame: day 0, 1 month, 2 month, 3 month, 6 month, 9 month and 12 month.
measure: Changes in the 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 Hamilton Depression (HAMD) Scale
timeFrame: day 0, 1 month, 2 month, 3 month, 6 month, 9 month and 12 month.
measure: Differences in biochemical indicators of cerebrospinal fluid
timeFrame: day 0, 1 month, 2 month, 3 month, 6 month, 9 month and 12 month.
measure: Cranial dopamin transporter measured by positron emission tomography (PET)- magnetic resonance imaging (MRI)
timeFrame: 4 month, 6 month, 12 month
measure: Glucose metabolism measured by 18F-Fluoro-2-deoxy-glucose (FDG) PET scan
timeFrame: 4 month, 6 month, 12 month
Other outcomes:
Locations:
city: Shanghai
country: China
facility: Jingwen Wu
state: Shanghai
Study officials:
affiliation: Shanghai East Hospital, China
name: Jingwen Wu, Dr
role: PRINCIPAL_INVESTIGATOR
References: