Plasmapheresis in Amyotrophic Lateral Sclerosis With Autoantibody Against NRIP 2 (PALADIN2)
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 入组人数
- 20
- 试验地点
- 1
- 主要终点
- Change in disease progression rates measuring using ALSFRS-R (Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised)
研究概览
简要总结
10-20% of patients with ALS have anti-NRIP autoantibody and the titer of anti-NRIP autoantibody is correlated with motor functional decline and mortality in ALS. The PALADIN2 clinical trial is a single arm study, which intends to enroll 20 ALS patients having anti-NRIP autoantibody in plasma. Patients will receive 3 courses of plasmapheresis per 3 months in order to maintain low concentration of anti-NRIP autoantibody in plasma. The study will follow up these patients for another 6 months after plasmapheresis. This project will potentially confirm the efficacy and safety of plasmapheresis for ALS patients having anti-NRIP autoantibody.
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
- 20 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •ALS patients above 20-year-old who have anti-NRIP autoantibody in plasma
- •Agree to receive plasmapheresis treatment
- •Agree to participate in the study and receive serial examinations
排除标准
- •Under permanent ventilator support
- •Cannot receive plasmapheresis treatment or serial examinations
- •Under pregnancy
- •Blood fibrinogen level below 50 mg/dl
- •Belong to special subtype of ALS, such as primary lateral sclerosis, progressive muscular atrophy, flail arm syndrome, flail leg syndrome.
结局指标
主要结局
Change in disease progression rates measuring using ALSFRS-R (Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised)
时间窗: 12 months
Difference between \[(ALSFRS-R at month -3 - ALSFRS-R at month 0)/3\] and \[(ALSFRS-R at month 0 - ALSFRS-R at month 9)/9\]; Months -3 to 0 vs. Months 0 to 9; the higher above ratio means higher disease progression rate
次要结局
- Change in disease progression rates measuring using EQ-5D (EuroQol Group Life Quality Score)(15 months)
- Change in disease progression rates measuring using ALSFRS-R (Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised)(15 months)
- Change in disease progression rates measuring using ALSFRS-R (Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised)(6 months)
- Change in disease severity measuring using ALSFRS-R (Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised)(12 months)
- Change in disease progression rates measuring using MRC (Medical Research Council score) mega-score(15 months)
- Change in disease progression rates measuring using Grips force (lb)(15 months)
- Change in disease progression rates measuring using 6MWT (6 minute walking test); distance in meters(15 months)
- Change in disease progression rates measuring using respiratory vital capacity (standardized vital capacity, %)(15 months)
- Change in disease progression rates measuring using CMAP (compound muscle action potential, mV)(15 months)
- Change in disease progression rates measuring using MUNE (motor unit number estimation; number)(15 months)
- Change in disease severity measuring using plasma NF-L (Neurofilament-light chain; pg/mL)(12 months)
- Any side effect under plasmapheresis(12 months)