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

Primary Purpose

Parkinson's Disease

Status
Unknown status
Phase
Phase 1
Locations
China
Study Type
Interventional
Intervention
NPC transplantation
Levodopa
Sponsored by
Chinese Academy of Sciences
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Parkinson's Disease

Eligibility Criteria

50 Years - 80 Years (Adult, Older Adult)All SexesAccepts Healthy Volunteers

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

Sites / Locations

  • The first affiliated hospital of Zhengzhou universityRecruiting

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm Type

Experimental

Experimental

Experimental

Arm Label

NPC transplantation

HLA-matched NPC transplantation

HLA-non-matched NPC transplantation

Arm Description

The patients will receive Levodopa combined with a regular neural precursor cell (NPC) transplantation

The patients will receive Levodopa combined with a HLA-matched neural precursor cell (NPC) transplantation

The patients will receive Levodopa combined with a HLA-non-matched neural precursor cell (NPC) transplantation

Outcomes

Primary Outcome Measures

Incidence of treatment-emergent adverse events (TEAEs), severe TEAEs as assessed by head MRI and blood examination
Number of subjects with adverse events such as the evidence of graft failure or rejection

Secondary Outcome Measures

Change in Unified Parkinson's Disease Rating Scale (UPDRS) score from baseline
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.
Change in DATscan from baseline
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).
Change in Hoehn and Yahr Stage from baseline
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.

Full Information

First Posted
April 6, 2017
Last Updated
April 13, 2017
Sponsor
Chinese Academy of Sciences
Collaborators
The First Affiliated Hospital of Zhengzhou University
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1. Study Identification

Unique Protocol Identification Number
NCT03119636
Brief Title
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

2. Study Status

Record Verification Date
September 2016
Overall Recruitment Status
Unknown status
Study Start Date
May 2017 (Anticipated)
Primary Completion Date
November 2018 (Anticipated)
Study Completion Date
December 2020 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Chinese Academy of Sciences
Collaborators
The First Affiliated Hospital of Zhengzhou University

4. Oversight

Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Parkinson's Disease

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 1, Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
50 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
NPC transplantation
Arm Type
Experimental
Arm Description
The patients will receive Levodopa combined with a regular neural precursor cell (NPC) transplantation
Arm Title
HLA-matched NPC transplantation
Arm Type
Experimental
Arm Description
The patients will receive Levodopa combined with a HLA-matched neural precursor cell (NPC) transplantation
Arm Title
HLA-non-matched NPC transplantation
Arm Type
Experimental
Arm Description
The patients will receive Levodopa combined with a HLA-non-matched neural precursor cell (NPC) transplantation
Intervention Type
Biological
Intervention Name(s)
NPC transplantation
Intervention Description
The cells are stereotactically implanted in the striatum.
Intervention Type
Drug
Intervention Name(s)
Levodopa
Intervention Description
Levodopa is used depending on the patient's condition
Primary Outcome Measure Information:
Title
Incidence of treatment-emergent adverse events (TEAEs), severe TEAEs as assessed by head MRI and blood examination
Description
Number of subjects with adverse events such as the evidence of graft failure or rejection
Time Frame
6 months
Secondary Outcome Measure Information:
Title
Change in Unified Parkinson's Disease Rating Scale (UPDRS) score from baseline
Description
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.
Time Frame
Baseline and 12 months
Title
Change in DATscan from baseline
Description
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).
Time Frame
Baseline and 12 months
Title
Change in Hoehn and Yahr Stage from baseline
Description
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.
Time Frame
Baseline and 12 months

10. Eligibility

Sex
All
Minimum Age & Unit of Time
50 Years
Maximum Age & Unit of Time
80 Years
Accepts Healthy Volunteers
Accepts Healthy Volunteers
Eligibility Criteria
Inclusion Criteria: Primary Parkinson's disease patients，a history over 5 years，females or males; Cannot effectively control the PD or tolerate the side effects of drugs; Hoehn and Yahr Stage 3 or 4 in the off state at screening Age between 50 and 80 years; Dopamine is effective or once; Sign the informed consent Exclusion Criteria: Atypical Parkinsonian syndrome or only having tremor syndrome; Having been done pallidotomy, DBS, striatum or extrapyramidal surgery; Subjects are using apomorphine or anticoagulant; Subjects used immunosuppressant or antipsychotic drugs in last 3 months; Subjects used botulinum toxin, phenol, or other drugs for the treatment of dystonia or muscle cramps in last 6 months; During the period of active epilepsy preventing epilepsy with antiepileptic; Coagulant function abnormality or other obviously abnormal laboratory test results; Having skin basal cell carcinoma or cervical cancer and other pre-cancerous lesions; Subject has a history of chronic alcohol or drug abuse ; Pregnancy or lactation; Subjects participated in other clinical trials in recent 3 months; Subject is considered as dementia, a serious mental disorder (depression or mania), and personality or behavioral disorders through cognitive and behavioral test; Cannot cooperate on the research; Severe brain atrophy, or existing brain injury such as cerebral infarction, cerebral vascular malformation or trauma; Severe systemic diseases; Severe dyskinesia or frequent "OFF" or "ON" states Severe infectious diseases (eg HIV, HCV, HBV, syphilis positive) Not suitable to participate in this clinical trial assessed by other physicians
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Wang Liu, Doctor
Phone
+86-01064807858
Email
wangliu@ioz.ac.cn
First Name & Middle Initial & Last Name or Official Title & Degree
Hao Jie, Doctor
Phone
+86-01062558737
Email
haojie@ioz.ac.cn
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Zhou Qi, Doctor
Organizational Affiliation
Institute of zoology, Chinese Academy of Sciences
Official's Role
Principal Investigator
Facility Information:
Facility Name
The first affiliated hospital of Zhengzhou university
City
Zhengzhou
State/Province
Henan
ZIP/Postal Code
450052
Country
China
Individual Site Status
Recruiting

12. IPD Sharing Statement

Plan to Share IPD
Undecided
Citations:
PubMed Identifier
29910127
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.
Results Reference
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Safety and Efficacy Study of Human ESC-derived Neural Precursor Cells in the Treatment of Parkinson's Disease

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