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
试验速览
- 阶段
- 2 期
- 发起方
- 入组人数
- 12
- 试验地点
- 1
- 主要终点
- The change of Unified Parkinson's Disease Reting Scale(UPDRS)score from baseline
研究概览
简要总结
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,
详细描述
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 35 Years 至 70 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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.
排除标准
- •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
研究组 & 干预措施
h-NSC arm
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.
干预措施: human neural stem cell (Biological)
结局指标
主要结局
The change of Unified Parkinson's Disease Reting Scale(UPDRS)score from baseline
时间窗: 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％
次要结局
- motor function index(baseline and 16, 28 weeks)
- Non-motor function score:smell(baseline and 16, 28 weeks)
- Immunological index(baseline and 16, 28 weeks)
- Non-motor function score:cognitive function(baseline and 16, 28 weeks)
- Non-motor function score:fatigue(baseline and 16, 28 weeks)
- Non-motor function score:emotion(baseline and 16, 28 weeks)
- Non-motor function score:non-motor symptoms(baseline and 16, 28 weeks)
- Non-motor function score:autonomic symptoms(baseline and 16, 28 weeks)
- Non-motor function score:the quality of life.(baseline and 16, 28 weeks)
- Imaging index(baseline and 16, 28 weeks)
- Blood routine examination(baseline and 16, 28 weeks)
- Biochemical routine examination(baseline and 16, 28 weeks)
- Safety index(1,2,3,4,weeks and 16, 28 weeks)
研究者
Chun-Feng Liu
Professor
Second Affiliated Hospital of Soochow University