Clinical trial NCT03119636: Safety and Efficacy Study of Human ESC-derived Neural Precursor Cells in the Treatment of Parkinson's Disease
Official title: A Phase I/II, Open-Label Study to Assess the Safety and Efficacy of Striatum Transplantation of Human Embryonic Stem Cells-derived Neural Precursor Cells in Patients With Parkinson's Disease
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE1
PHASE2
Conditions: Parkinson's Disease
Lead sponsor: Chinese Academy of Sciences
Sponsor class: OTHER_GOV
Collaborators: name: The First Affiliated Hospital of Zhengzhou University
First posted: 2017-04-18
Enrollment: 50
Sex: ALL
Minimum age (years): 50
Maximum age (years): 80
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: yes
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: This study will evaluate the safety and efficacy of intracerebral transplantation of human embryonic stem cells-derived neural precursor cells in patients with Parkinson's Disease.

Detailed description: This study is a Phase I/II, open-label, non randomized clinical trial. The study will enroll 50 patients for cell injection, administering a single dose of neural precursor cells by stereotaxic intra-striatal injection.

Eligibility criteria: Inclusion Criteria:

1. Primary Parkinson's disease patients，a history over 5 years，females or males;
2. Cannot effectively control the PD or tolerate the side effects of drugs;
3. Hoehn and Yahr Stage 3 or 4 in the off state at screening
4. Age between 50 and 80 years;
5. Dopamine is effective or once;
6. Sign the informed consent

Exclusion Criteria:

1. Atypical Parkinsonian syndrome or only having tremor syndrome;
2. Having been done pallidotomy, DBS, striatum or extrapyramidal surgery;
3. Subjects are using apomorphine or anticoagulant;
4. Subjects used immunosuppressant or antipsychotic drugs in last 3 months;
5. Subjects used botulinum toxin, phenol, or other drugs for the treatment of dystonia or muscle cramps in last 6 months;
6. During the period of active epilepsy preventing epilepsy with antiepileptic;
7. Coagulant function abnormality or other obviously abnormal laboratory test results;
8. Having skin basal cell carcinoma or cervical cancer and other pre-cancerous lesions;
9. Subject has a history of chronic alcohol or drug abuse ;
10. Pregnancy or lactation;
11. Subjects participated in other clinical trials in recent 3 months;
12. Subject is considered as dementia, a serious mental disorder (depression or mania), and personality or behavioral disorders through cognitive and behavioral test;
13. Cannot cooperate on the research;
14. Severe brain atrophy, or existing brain injury such as cerebral infarction, cerebral vascular malformation or trauma;
15. Severe systemic diseases;
16. Severe dyskinesia or frequent "OFF" or "ON" states
17. Severe infectious diseases (eg HIV, HCV, HBV, syphilis positive)
18. Not suitable to participate in this clinical trial assessed by other physicians

Primary outcomes:
measure: Incidence of treatment-emergent adverse events (TEAEs), severe TEAEs as assessed by head MRI and blood examination
timeFrame: 6 months
Secondary outcomes:
measure: Change in Unified Parkinson's Disease Rating Scale (UPDRS) score from baseline
timeFrame: Baseline and 12 months
measure: Change in DATscan from baseline
timeFrame: Baseline and 12 months
measure: Change in Hoehn and Yahr Stage from baseline
timeFrame: Baseline and 12 months
Other outcomes:
Locations:
city: Zhengzhou
country: China
facility: The first affiliated hospital of Zhengzhou university
state: Henan
Study officials:
affiliation: Institute of Zoology, Chinese Academy of Sciences
name: Zhou Qi, Doctor
role: PRINCIPAL_INVESTIGATOR
References:
citation: Wang YK, Zhu WW, Wu MH, Wu YH, Liu ZX, Liang LM, Sheng C, Hao J, Wang L, Li W, Zhou Q, Hu BY. Human Clinical-Grade Parthenogenetic ESC-Derived Dopaminergic Neurons Recover Locomotive Defects of Nonhuman Primate Models of Parkinson's Disease. Stem Cell Reports. 2018 Jul 10;11(1):171-182. doi: 10.1016/j.stemcr.2018.05.010. Epub 2018 Jun 14.