An Open-label, Phase 1 Trial for Safety and Efficacy Study of 2nd Cycle Treatment After 6 Months of 1st Cycle HLA-haplo Matched Allogenic Bone Marrow Derived Stem Cell("HYNR-CS-Allo Inj") Treatment in Amyotrophic Lateral Sclerosis(ALS)
试验速览
- 阶段
- 1 期
- 发起方
- 入组人数
- 6
- 试验地点
- 2
- 主要终点
- The Incidence of any treatment related serious adverse events(SAE)
研究概览
简要总结
The purpose of this study is to evaluate the safety and efficacy of HLA-haplo matched Allogenic Bone Marrow Derived stem cells("HYNRCS-Allo-ALS-02 inj"), through intrathecal delivery for the repeated treatment after 6 months of first treatment in patients with amyotrophic lateral sclerosis(ALS).
This study is an open label, single-dose study to assess the safety and efficacy of HLA-haplo matched Allogenic Bone Marrow Derived stem cells("HYNRCS-Allo-ALS-02 inj")
详细描述
Amyotrophic lateral sclerosis is a progressive neurodegenerative disease characterized by motor neuron loss. Despite of many trials for disease-modifying, no treatment has so far changed natural course of disease.
The investigators had performed the pre-clinical and clinical studies using autologous bone marrow-derived stem cells in ALS. In the investigators' results of clinical trial, intrathecal injection of autologous bone marrow-derived stem cells is safe and could slow down disease progression and might be used as a disease modifying strategy in patients with ALS.
In the new field, like cell therapy, it is an important issue whether a bone marrow derived mesenchymal stem cells can be used as an allograft. Many investigators had showed that the immunoprivileged and immunosuppressive properties of mesenchymal stem cells result from the absence of major histocompatibility class II antigens and the secretion of T helper type 2 cytokines.
One potential advantage of allogenic bone marrow derived cells could be avoiding the need for procedural delay before treatment. And it is also hypothesized that the function of autologous bone marrow derived cells could be impaired in patients with co-morbidities or advanced age.
This study is to evaluate safety and efficacy of repeated treatment after 6 months of first HYNRCS-Allo-ALS-02 inj(HLA-haplo matched Allogenic bone marrow-derived stem cells) treatment in patients with ALS.
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
- 25 Years 至 80 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients between 25 and 80 years old
- •Patients diagnosed as 'Possible with lab-supported' or 'Possible' or 'Probable' or 'Definite' ALS according to the World Federation of Neurology El Escorial criteria
- •Patients whose duration of disease is within 5 years from the first diagnosis
- •Patients with ALSFRS-R score within 21 to 46 at screening
- •Patients who can visit to a hospital by walk personally or by protector's help
- •Patients who provide the written consent by oneself or his/her legal representative
- •Patients who has HLA-haplo matched Bone marrow donor
排除标准
- •Patients who doesn't appropriate to the diagnostic criteria of ALS
- •Patients who doesn't have HLA-haplo-matched bone marrow donor
- •Patients suspected of adverse effect after stem cell injection(patients suspected of malignant tumor, risk group of psychogenic shock, patients with serious hypertension)
- •Patients with ALSFRS-R score below 21 at screening
- •Patients performed Tracheostomy at screening
- •Patients with suspected 20% or less of Forced vital capacity(FVC) at screening
- •Patients who doesn't agree with written consent form by oneself of his/her legal representative
- •Patients who have taken any other drug for clinical trial within the past 3 months at screening entry
- •Patients with epilepsy
- •Patients with severe medical disease
- •Pregnant woman, lactating woman, female patients who has a pregnancy planning or who doesn't agree with adoption of contraception methods proper medically, male patients who doesn't agree with adoption of contraception methods proper to his partner during participating this study
- •Patients with hemorrhagic tendency at screening
- •Patients with a known history of hypersensitivity/allergy to penicillin and streptomycin
- •Patients with severe psychotic diseases (such as alzheimer, schizophrenia excepts slight cognitive dysfunction and secondary emotional disorder)
研究组 & 干预措施
HYNRCS-Allo inj.
2 cycles of HYNRCS-Allo inj. with 6 months interval through intrathecal injection.
*1 cycle of HYNRCS-Allo inj. is 2 times administration with 28 days interval by intrathecal.
干预措施: HYNRCS-Allo inj (Biological)
结局指标
主要结局
The Incidence of any treatment related serious adverse events(SAE)
时间窗: 12 months
次要结局
- ALS-Functional rating scales(ALS-FRS)(12 months)
- Incidence & Degree of Adverse Events(AE)(12 months)
- PRA test to identify generation status of HLA antibody(12 months)
研究者
Seung Hyun Kim
Principal Investigator
Hanyang University Seoul Hospital