Stereotactic Transplantation of hAESCs for Parkinson's Disease

Parkinson's Disease Clinical Trial

Official title:

Clinical Study of Stereotactic Transplantation of Human Amniotic Epithelial Stem Cells (hAESCs) in the Treatment of Parkinson's Disease (PD)

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT04414813
• Other study ID #: DFSC-2019(CR)-001
• Status: Completed
• Phase: Early Phase 1
• Start date: October 8, 2020
• Est. completion date: February 8, 2023

Study information

• Verified date: April 2023
• Source: Shanghai East Hospital
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

The purpose of this study is to evaluate the safety and efficacy of stereotactic transplantation of hAESCs for Parkinson's disease.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 3
• Est. completion date: February 8, 2023
• Est. primary completion date: February 8, 2023
• Accepts healthy volunteers: No
• Gender: All
• Age group: 30 Years to 70 Years

Eligibility

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

Related Conditions & MeSH terms

• Parkinson Disease
• Parkinson's Disease

Intervention

Biological:
• Human Amniotic Epithelial Stem Cells
Stereotactic transplantation of hAESCs for participants with PD.

Locations

Country	Name	City	State
China	WU Jingwen	Shanghai	Shanghai

Sponsors (2)

Lead Sponsor	Collaborator
Shanghai East Hospital	Shanghai iCELL Biotechnology Co., Ltd, Shanghai, China

Country where clinical trial is conducted

China, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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.	12 months
Secondary	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.	day 0, 1 month, 2 month, 3 month, 6 month, 9 month and 12 month.
Secondary	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.	day 0, 1 month, 2 month, 3 month, 6 month, 9 month and 12 month.
Secondary	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.	day 0, 1 month, 2 month, 3 month, 6 month, 9 month and 12 month.
Secondary	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.	day 0, 1 month, 2 month, 3 month, 6 month, 9 month and 12 month.
Secondary	Levodopa equivalent daily dose	Differences in daily dosage of levodopamine	day 0, 1 month, 2 month, 3 month, 4 month, 5 month, 6 month, 9 month and 12 month.
Secondary	Cranial doparmin transporter measured by positron emission tomography(PET)-magnetic resonance Imaging(MRI)	Changes of cranial expression of dopamine transporter investigated by PET-MR	4 month, 6 month
Secondary	Glucose metabolism measured by 18F-Fluoro-2-deoxy-glucose (FDG) PET scan	Changes in cranial glucose metabolism investigated by PET-MR	4 month, 6 month
Secondary	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), a synuclein (a-synuclein), amyloid deposition (Aß42), Tau protein (Tau) Protein, Neurofilament light (NF-L), and yKL-40 (YKL-40).	day 0, 1 month, 2 month, 3 month, 4 month,5 month,6 month, 9 month and 12 month.