Randomized, Double-blind, Safety and Efficacy of Recombinant Human Erythropoietin in Amyotrophic Lateral Sclerosis
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 发起方
- 入组人数
- 64
- 试验地点
- 1
- 主要终点
- changes of ALSFRS-R score
研究概览
简要总结
Erythropoietin is neuroprotective in animal models of neurodegenerative diseases including amyotrophic lateral sclerosis (ALS). The aim of this study was to determine the safety and feasibility of repetitive high-dose recombinant human erythropoietin (rhEPO) therapy in ALS patients.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Other
- 盲法
- Single (Participant)
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
- •Age between 25 to 80
- •upper motor neuron signs and lower motor neuron signs were identified in neurological examination.
- •Meet the revised El Escorial Criteria for clinically possible, probable-laboratory -supported, probable, definite ALS.
- •Disease duration < 3 years (Within 3 years from symptom onset)
- •ALSFRS-R score between 21 to 46
- •Patient who can visit an outpatient under the aid of his or her own walking or caregivers.
- •The person who have agreed in writing to participate in this clinical trial by themselves and the legal representative
- •FVC over 50% at screening
排除标准
- •Person who were not compatible with ALS
- •Patient with PLS or PMA
- •A group of patients who are concerned about the adverse effects of the drug administration (e.g. malignant hypertension,...)
- •ALSFRS-R score below 20 at screening
- •Ventilator user or Tracheostomy state patients at screening
- •Gastrostomy state at screening
- •FVC below 50% at screening or patient who cannot perform FVC test.
- •EKG abnormality, history of coronary stent , CABG at screening
- •Person who was given another clinical trial drug three months prior to screening.
- •History of seizure/ epilepsy
- •Abnormal renal function (serem creatinine > 2.0mg/dl)
- •Abnormal liver function(AST/ALT/bilirubin over 2 times the upper normal limit
- •Bleeding tendency at screening
- •Infectious disease at screening
- •Drug sensitivity
- •Person who injected erythropoietin 6 months prior to screening
- •Malignant tumor
- •Other neurological disease (stroke, parkinson's disease, dementia...)
- •Psychological disease
- •Hb more than 16g/dL
研究组 & 干预措施
Control group
Inject Normal saline 100ml * 12 times (1month apart)
干预措施: recombinant human erythropoietin(rhEPO) (Drug)
Low dose group
Inject 500IU/kg of EPO mixed with normal saline 100ml * 12 times (1month apart)
干预措施: recombinant human erythropoietin(rhEPO) (Drug)
High dose group
Inject 750IU/kg of EPO mixed with normal saline 100ml * 12 times (1month apart)
干预措施: recombinant human erythropoietin(rhEPO) (Drug)
结局指标
主要结局
changes of ALSFRS-R score
时间窗: Last visit [15th visit (15 months)]
changes between score of ALSFRS-R at initial and study end point
次要结局
未报告次要终点
研究者
Seung Hyun Kim
MD, PhD
Hanyang University Seoul Hospital