Parkinson Disease
Conditions
Keywords
Parkinson Disease, Neural stem cell, Nasal delivery
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.
Interventions
BIOLOGICALhuman neural stem cell
human neural stem cell: 100ul/vessel，2 vessel/one bag，≥2×10 6cells/vessel
Sponsors
Second Affiliated Hospital of Soochow University
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
35 Years to 70 Years
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| The change of Unified Parkinson's Disease Reting Scale(UPDRS)score from baseline | Baseline and 16, 28 weeks | 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
| Measure | Time frame | Description |
|---|---|---|
| motor function index | baseline and 16, 28 weeks | Hoehn-Yahr modified score |
| Non-motor function score:cognitive function | baseline and 16, 28 weeks | Minimum Mental State Examination and Montreal Cognitive Assessment to assess cognitive function |
| Non-motor function score:smell | baseline and 16, 28 weeks | Argentina Hyposmia Rating Scales is used to detect the smell |
| Non-motor function score:fatigue | baseline and 16, 28 weeks | Fatigue Severity Scale to assess the extent of fatigue |
| Non-motor function score:emotion | baseline and 16, 28 weeks | 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 | baseline and 16, 28 weeks | non-motor symptoms in PD patients is evaluated used Non-motor Symptoms Questionnaire |
| Non-motor function score:the quality of life. | baseline and 16, 28 weeks | The 39-item Parkinson's disease questionnaire to assess the quality of life. |
| Immunological index | baseline and 16, 28 weeks | CD3(%)，CD4(%)，CD8(%)，Treg cells(%) |
| Imaging index | baseline and 16, 28 weeks | Magnetic Resonance Imaging or positron emission tomography |
| Blood routine examination | baseline and 16, 28 weeks | 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 | baseline and 16, 28 weeks | Biochemical routine examination includes liver function, renal function, blood glucose, blood lipid |
| Safety index | 1,2,3,4,weeks and 16, 28 weeks | Adverse Event and Serious Adverse Event |
| Non-motor function score:autonomic symptoms | baseline and 16, 28 weeks | The scale for outcomes in PD for autonomic symptoms to assess autonomic dysfunction |
Other
| Measure | Time frame | Description |
|---|---|---|
| PD therapy drugs | baseline and 16, 28 weeks | Reduction rate of PD therapy drugs |
Countries
China
Outcome results
None listed