A Study To Evaluate the Safety and Efficacy of Human Neural Stem Cells for Parkinson's Disease Patient
- Conditions
- Parkinson Disease
- Registration Number
- NCT03128450
- Lead Sponsor
- Second Affiliated Hospital of Soochow University
- 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.
Recruitment & Eligibility
- Status
- UNKNOWN
- Sex
- All
- Target Recruitment
- 12
- 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.
- 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
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Primary Outcome Measures
Name Time Method 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 Outcome Measures
Name Time Method motor function index baseline and 16, 28 weeks Hoehn-Yahr modified score
Non-motor function score:smell baseline and 16, 28 weeks Argentina Hyposmia Rating Scales is used to detect the smell
Immunological index baseline and 16, 28 weeks CD3(%)，CD4(%)，CD8(%)，Treg cells(%)
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: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:autonomic symptoms baseline and 16, 28 weeks The scale for outcomes in PD for autonomic symptoms to assess autonomic dysfunction
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.
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
Trial Locations
- Locations (1)
Department of Neurology, Second Affiliated Hospital of Soochow University
🇨🇳Suzhou, Jiangsu, China
Department of Neurology, Second Affiliated Hospital of Soochow University🇨🇳Suzhou, Jiangsu, China