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

Phase 1

• Conditions: Parkinson's Disease
• Interventions: Biological: NPC transplantation; Drug: Levodopa

• Registration Number: NCT03119636
• Lead Sponsor: Chinese Academy of Sciences

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.

Study Timeline

• Status Verified: 2016-09
• Study First Submitted: 2017-04-06
• Study First Submitted QC: 2017-04-13
• Last Update Submitted: 2017-04-13
• Study First Posted: 2017-04-18
• Last Update Posted: 2017-04-18
• Study Start: 2017-05
• Primary Completion: 2018-11
• Study Completion: 2020-12

Recruitment & Eligibility

• Status: UNKNOWN
• Sex: All
• Target Recruitment: 50

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

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

Primary Outcome Measures

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

Secondary Outcome Measures

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

Trial Locations

Locations (1)

The first affiliated hospital of Zhengzhou university; 🇨🇳 Zhengzhou, Henan, China