Safety and Efficacy Study of Human ESC-derived Neural Precursor Cells in the Treatment of Parkinson's Disease

Study Description

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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.

Condition or disease	Intervention/treatment	Phase
Parkinson's Disease	Biological: NPC transplantation; Drug: Levodopa	Phase 1; Phase 2

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.

Study Design

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Study Type :	Interventional (Clinical Trial)
Estimated Enrollment :	50 participants
Allocation:	Non-Randomized
Intervention Model:	Single Group Assignment
Masking:	None (Open Label)
Primary Purpose:	Treatment
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
Estimated Study Start Date :	May 2017
Estimated Primary Completion Date :	November 2018
Estimated Study Completion Date :	December 2020

Arms and Interventions

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Arm	Intervention/treatment
Experimental: NPC transplantation; The patients will receive Levodopa combined with a regular neural precursor cell (NPC) transplantation	Biological: NPC transplantation; The cells are stereotactically implanted in the striatum.; Drug: Levodopa; Levodopa is used depending on the patient's condition
Experimental: HLA-matched NPC transplantation; The patients will receive Levodopa combined with a HLA-matched neural precursor cell (NPC) transplantation	Biological: NPC transplantation; The cells are stereotactically implanted in the striatum.; Drug: Levodopa; Levodopa is used depending on the patient's condition
Experimental: HLA-non-matched NPC transplantation; The patients will receive Levodopa combined with a HLA-non-matched neural precursor cell (NPC) transplantation	Biological: NPC transplantation; The cells are stereotactically implanted in the striatum.; Drug: Levodopa; Levodopa is used depending on the patient's condition

Outcome Measures

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Primary Outcome Measures :

1. Incidence of treatment-emergent adverse events (TEAEs), severe TEAEs as assessed by head MRI and blood examination [ Time Frame: 6 months ]
   Number of subjects with adverse events such as the evidence of graft failure or rejection

Secondary Outcome Measures :

1. Change in Unified Parkinson's Disease Rating Scale (UPDRS) score from baseline [ Time Frame: Baseline and 12 months ]
   The total Unified Parkinson's Disease Rating Scale (UPDRS) score is derived from Part I (Mentation, Behavior and Mood), Part II (Activities of Daily Living) and Part III (Motor Examination). Part I assesses 4 functions; Part II assesses 13 activities of daily living; Part III assesses 14 motor symptoms. Each item is rated on a scale from 0 (normal) to 4 (severe). The minimum total score possible is 0 and the maximum total score possible is 176. Each subject was independently rated by two observers at each study visit and a mean score was calculated for analysis.
2. Change in DATscan from baseline [ Time Frame: Baseline and 12 months ]
   DATscan is an imaging technology that uses small amounts of a radioactive drug to help determine how much dopamine is available in a person's brain.Its principle is based on using of radiopharmaceutical, which bind to dopamine transporters (DAT).
3. Change in Hoehn and Yahr Stage from baseline [ Time Frame: Baseline and 12 months ]
   The Hoehn and Yahr scale is a commonly used system for describing how the symptoms of Parkinson's disease progress mainly by observing balance and walk.

Eligibility Criteria

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Ages Eligible for Study:	50 Years to 80 Years (Adult, Older Adult)
Sexes Eligible for Study:	All
Accepts Healthy Volunteers:	Yes

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

Contacts and Locations

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Contacts

Contact: Wang Liu, Doctor	+86-01064807858	wangliu@ioz.ac.cn
Contact: Hao Jie, Doctor	+86-01062558737	haojie@ioz.ac.cn

Locations

China, Henan
The first affiliated hospital of Zhengzhou university	Recruiting
Zhengzhou, Henan, China, 450052

Sponsors and Collaborators

Chinese Academy of Sciences

The First Affiliated Hospital of Zhengzhou University

Investigators

Principal Investigator:	Zhou Qi, Doctor	Institute of zoology, Chinese Academy of Sciences

More Information

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Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

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.

Responsible Party:	Qi Zhou, Deputy director of Institute of zoology, Chinese academy of sciences, and vice president of medical school， University of Chinese academy of sciences, Chinese Academy of Sciences
ClinicalTrials.gov Identifier:	NCT03119636 History of Changes
Other Study ID Numbers:	ChineseASZQ-003
First Posted:	April 18, 2017
Last Update Posted:	April 18, 2017
Last Verified:	September 2016
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD:	Undecided

Studies a U.S. FDA-regulated Drug Product:		No
Studies a U.S. FDA-regulated Device Product:		No

Additional relevant MeSH terms:

Parkinson Disease; Parkinsonian Disorders; Basal Ganglia Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Movement Disorders; Neurodegenerative Diseases	Levodopa; Antiparkinson Agents; Anti-Dyskinesia Agents; Dopamine Agents; Neurotransmitter Agents; Molecular Mechanisms of Pharmacological Action; Physiological Effects of Drugs