Clinical trial NCT03128450: A Study To Evaluate the Safety and Efficacy of Human Neural Stem Cells for Parkinson's Disease Patient
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
Overall status: UNKNOWN
Phase: PHASE2
PHASE3
Conditions: Parkinson Disease
Keywords: Parkinson Disease
Neural stem cell
Nasal delivery
Lead sponsor: Second Affiliated Hospital of Soochow University
Sponsor class: OTHER
First posted: 2017-04-25
Enrollment: 12
Sex: ALL
Minimum age (years): 35
Maximum age (years): 70
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.

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

Primary outcomes:
measure: The change of Unified Parkinson's Disease Reting Scale(UPDRS)score from baseline
timeFrame: Baseline and 16, 28 weeks
Secondary outcomes:
measure: motor function index
timeFrame: baseline and 16, 28 weeks
measure: Non-motor function score:cognitive function
timeFrame: baseline and 16, 28 weeks
measure: Non-motor function score:smell
timeFrame: baseline and 16, 28 weeks
measure: Non-motor function score:fatigue
timeFrame: baseline and 16, 28 weeks
measure: Non-motor function score:emotion
timeFrame: baseline and 16, 28 weeks
measure: Non-motor function score:non-motor symptoms
timeFrame: baseline and 16, 28 weeks
measure: Non-motor function score:autonomic symptoms
timeFrame: baseline and 16, 28 weeks
measure: Non-motor function score:the quality of life.
timeFrame: baseline and 16, 28 weeks
measure: Immunological index
timeFrame: baseline and 16, 28 weeks
measure: Imaging index
timeFrame: baseline and 16, 28 weeks
measure: Blood routine examination
timeFrame: baseline and 16, 28 weeks
measure: Biochemical routine examination
timeFrame: baseline and 16, 28 weeks
measure: Safety index
timeFrame: 1,2,3,4,weeks and 16, 28 weeks
Other outcomes:
measure: PD therapy drugs
timeFrame: baseline and 16, 28 weeks
Locations:
city: Suzhou
country: China
facility: Department of Neurology, Second Affiliated Hospital of Soochow University
state: Jiangsu
Study officials:
affiliation: Department of Neurology, Second Affiliated Hospital of Soochow University, Suzhou
name: Jie Li
role: PRINCIPAL_INVESTIGATOR
References:
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.
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.