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A Study To Evaluate the Safety and Efficacy of Human Neural Stem Cells for Parkinson's Disease Patient (hNSCPD)

Primary Purpose

Parkinson Disease

Status
Unknown status
Phase
Phase 2
Locations
China
Study Type
Interventional
Intervention
human neural stem cell
Sponsored by
Second Affiliated Hospital of Soochow University
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Parkinson Disease focused on measuring Parkinson Disease, Neural stem cell, Nasal delivery

Eligibility Criteria

35 Years - 70 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  • Patient diagnosed with idiopathic PD, at least with two key symptoms (Static tremor, bradykinesia, rigidity ) , without any other evidence of Secondary Parkinson's syndrome.
  • Disease course ≥7 years，modified Hoehn-Yahr is 3-5 stage
  • Patient age ≥35 years
  • Patients receiving a stable dose of levodopa for at least 1 months with the expectation that the treatment will remain unchanged throughout the course of the study
  • The doses of levodopa ≥300mg •Signed informed consent form (ICF) by patient self or his law-in relationship before enrollment.

Exclusion Criteria:

  • Hepatic dysfunction(transaminase ≥1.5 normal range), Renal dysfunction(Cr>2.0mg/dl or 177μmol/L)，Cardiac dysfunction or other severe systematic diseases etc.
  • Suffering malignancy or during anti-cancer treatment period.
  • Pregancy, lactation or possible pregancy and plan to pregancy patient
  • Attended other intervention clinical trial within 3 months aftre getting ICF, or during other ongoing intervention clinical trial
  • Investigator think inappropriate patient for this protocol

Sites / Locations

  • Department of Neurology, Second Affiliated Hospital of Soochow University

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

h-NSC arm

Arm Description

human neural stem cell: 100ul/vessel，2 vessel/one bag，≥2×10 6cells/vessel，produced by Shanghai Angecon Biotechology Cooperate. One enrolled PD patient was given 2 vessels h-NSC througth nasal cavity weekly for 4 weeks。Total cell number will be over ≥4×10 6cells for one time.

Outcomes

Primary Outcome Measures

The change of Unified Parkinson's Disease Reting Scale(UPDRS)score from baseline
Improvement rate of UPDRS motor score defined as below: Reduction rate =（baseline score 16, 28 weeks score after therapy）/ baseline score×100%. Based on reduction rate, the efficacy can be defined as complete remission, partial remission, effective and invalid The reduction rate will be 100%, >50%, >25-50%, ≤ 25% for complete remission, partial remission, effective and invalid. The improvement rate =[(complete+partial+effective patient number)/total patient number]×100％

Secondary Outcome Measures

motor function index
Hoehn-Yahr modified score
Non-motor function score:cognitive function
Minimum Mental State Examination and Montreal Cognitive Assessment to assess cognitive function
Non-motor function score:smell
Argentina Hyposmia Rating Scales is used to detect the smell
Non-motor function score:fatigue
Fatigue Severity Scale to assess the extent of fatigue
Non-motor function score:emotion
Hamilton Depression Scale to evaluate the degree of depression, Hamilton Anxiety Scale to evaluate the degree of anxiety
Non-motor function score:non-motor symptoms
non-motor symptoms in PD patients is evaluated used Non-motor Symptoms Questionnaire
Non-motor function score:autonomic symptoms
The scale for outcomes in PD for autonomic symptoms to assess autonomic dysfunction
Non-motor function score:the quality of life.
The 39-item Parkinson's disease questionnaire to assess the quality of life.
Immunological index
CD3(%)，CD4(%)，CD8(%)，Treg cells(%)
Imaging index
Magnetic Resonance Imaging or positron emission tomography
Blood routine examination
Blood routine examination includes the total number of red blood cells, hemoglobin, total number of white blood cells, white blood cells count, platelet count.
Biochemical routine examination
Biochemical routine examination includes liver function, renal function, blood glucose, blood lipid
Safety index
Adverse Event and Serious Adverse Event

Full Information

First Posted
February 9, 2017
Last Updated
September 13, 2017
Sponsor
Second Affiliated Hospital of Soochow University
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1. Study Identification

Unique Protocol Identification Number
NCT03128450
Brief Title
A Study To Evaluate the Safety and Efficacy of Human Neural Stem Cells for Parkinson's Disease Patient
Acronym
hNSCPD
Official Title
A Single Arm, Open-Label，Pilot Study to Evaluate the Safety and Efficacy of Human Neural Stem Cells Injection (ANGE-S003) Through Nasal Way Delivery to Patients With Parkinson's Disease
Study Type
Interventional

2. Study Status

Record Verification Date
May 2017
Overall Recruitment Status
Unknown status
Study Start Date
April 15, 2017 (Actual)
Primary Completion Date
June 1, 2018 (Anticipated)
Study Completion Date
November 1, 2018 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Second Affiliated Hospital of Soochow 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 Pilot study will evaluate the safety and Efficacy of an investigational cell transplantation therapy, h-NSC, in patients with Parkinson's disease, through nasal drug delivery, a new delivery way. All patients will receive the therapy, which consists of human neural stem cells,
Detailed Description
h-NSC is a cellular therapeutic consisting of human fetal neural stem cells (h-NSC). h-NSC injection will be delivered through nasal way for patients with Parkinson's disease (PD). The study will enroll 12 moderate to severe PD patients to be treated with the cell injection therapy at the same dose. The total therapy course will be four weeks，one dose for one week. The follow up will be two times within 24 weeks after finishing the treatment. The main objective of the study is to evaluate the efficacy and safety of the cell transplantation by this new delivery way.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Parkinson Disease
Keywords
Parkinson Disease, Neural stem cell, Nasal delivery

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2, Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
12 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
h-NSC arm
Arm Type
Experimental
Arm Description
human neural stem cell: 100ul/vessel，2 vessel/one bag，≥2×10 6cells/vessel，produced by Shanghai Angecon Biotechology Cooperate. One enrolled PD patient was given 2 vessels h-NSC througth nasal cavity weekly for 4 weeks。Total cell number will be over ≥4×10 6cells for one time.
Intervention Type
Biological
Intervention Name(s)
human neural stem cell
Intervention Description
human neural stem cell: 100ul/vessel，2 vessel/one bag，≥2×10 6cells/vessel
Primary Outcome Measure Information:
Title
The change of Unified Parkinson's Disease Reting Scale(UPDRS)score from baseline
Description
Improvement rate of UPDRS motor score defined as below: Reduction rate =（baseline score 16, 28 weeks score after therapy）/ baseline score×100%. Based on reduction rate, the efficacy can be defined as complete remission, partial remission, effective and invalid The reduction rate will be 100%, >50%, >25-50%, ≤ 25% for complete remission, partial remission, effective and invalid. The improvement rate =[(complete+partial+effective patient number)/total patient number]×100％
Time Frame
Baseline and 16, 28 weeks
Secondary Outcome Measure Information:
Title
motor function index
Description
Hoehn-Yahr modified score
Time Frame
baseline and 16, 28 weeks
Title
Non-motor function score:cognitive function
Description
Minimum Mental State Examination and Montreal Cognitive Assessment to assess cognitive function
Time Frame
baseline and 16, 28 weeks
Title
Non-motor function score:smell
Description
Argentina Hyposmia Rating Scales is used to detect the smell
Time Frame
baseline and 16, 28 weeks
Title
Non-motor function score:fatigue
Description
Fatigue Severity Scale to assess the extent of fatigue
Time Frame
baseline and 16, 28 weeks
Title
Non-motor function score:emotion
Description
Hamilton Depression Scale to evaluate the degree of depression, Hamilton Anxiety Scale to evaluate the degree of anxiety
Time Frame
baseline and 16, 28 weeks
Title
Non-motor function score:non-motor symptoms
Description
non-motor symptoms in PD patients is evaluated used Non-motor Symptoms Questionnaire
Time Frame
baseline and 16, 28 weeks
Title
Non-motor function score:autonomic symptoms
Description
The scale for outcomes in PD for autonomic symptoms to assess autonomic dysfunction
Time Frame
baseline and 16, 28 weeks
Title
Non-motor function score:the quality of life.
Description
The 39-item Parkinson's disease questionnaire to assess the quality of life.
Time Frame
baseline and 16, 28 weeks
Title
Immunological index
Description
CD3(%)，CD4(%)，CD8(%)，Treg cells(%)
Time Frame
baseline and 16, 28 weeks
Title
Imaging index
Description
Magnetic Resonance Imaging or positron emission tomography
Time Frame
baseline and 16, 28 weeks
Title
Blood routine examination
Description
Blood routine examination includes the total number of red blood cells, hemoglobin, total number of white blood cells, white blood cells count, platelet count.
Time Frame
baseline and 16, 28 weeks
Title
Biochemical routine examination
Description
Biochemical routine examination includes liver function, renal function, blood glucose, blood lipid
Time Frame
baseline and 16, 28 weeks
Title
Safety index
Description
Adverse Event and Serious Adverse Event
Time Frame
1,2,3,4,weeks and 16, 28 weeks
Other Pre-specified Outcome Measures:
Title
PD therapy drugs
Description
Reduction rate of PD therapy drugs
Time Frame
baseline and 16, 28 weeks

10. Eligibility

Sex
All
Minimum Age & Unit of Time
35 Years
Maximum Age & Unit of Time
70 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Patient diagnosed with idiopathic PD, at least with two key symptoms (Static tremor, bradykinesia, rigidity ) , without any other evidence of Secondary Parkinson's syndrome. Disease course ≥7 years，modified Hoehn-Yahr is 3-5 stage Patient age ≥35 years Patients receiving a stable dose of levodopa for at least 1 months with the expectation that the treatment will remain unchanged throughout the course of the study The doses of levodopa ≥300mg •Signed informed consent form (ICF) by patient self or his law-in relationship before enrollment. Exclusion Criteria: Hepatic dysfunction(transaminase ≥1.5 normal range), Renal dysfunction(Cr>2.0mg/dl or 177μmol/L)，Cardiac dysfunction or other severe systematic diseases etc. Suffering malignancy or during anti-cancer treatment period. Pregancy, lactation or possible pregancy and plan to pregancy patient Attended other intervention clinical trial within 3 months aftre getting ICF, or during other ongoing intervention clinical trial Investigator think inappropriate patient for this protocol
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Jie Li
Organizational Affiliation
Department of Neurology, Second Affiliated Hospital of Soochow University, Suzhou
Official's Role
Principal Investigator
Facility Information:
Facility Name
Department of Neurology, Second Affiliated Hospital of Soochow University
City
Suzhou
State/Province
Jiangsu
ZIP/Postal Code
215004
Country
China

12. IPD Sharing Statement

Plan to Share IPD
Undecided
IPD Sharing Plan Description
Will decisde if the IPD based on patient consent
Citations:
PubMed Identifier
24910427
Citation
Hallett PJ, Cooper O, Sadi D, Robertson H, Mendez I, Isacson O. Long-term health of dopaminergic neuron transplants in Parkinson's disease patients. Cell Rep. 2014 Jun 26;7(6):1755-61. doi: 10.1016/j.celrep.2014.05.027. Epub 2014 Jun 6.
Results Reference
result
PubMed Identifier
24217017
Citation
Kefalopoulou Z, Politis M, Piccini P, Mencacci N, Bhatia K, Jahanshahi M, Widner H, Rehncrona S, Brundin P, Bjorklund A, Lindvall O, Limousin P, Quinn N, Foltynie T. Long-term clinical outcome of fetal cell transplantation for Parkinson disease: two case reports. JAMA Neurol. 2014 Jan;71(1):83-7. doi: 10.1001/jamaneurol.2013.4749.
Results Reference
result

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A Study To Evaluate the Safety and Efficacy of Human Neural Stem Cells for Parkinson's Disease Patient

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