A Phase 2 Randomized, Double-Blind, Placebo-Controlled, Multicenter Study of NP001 in Subjects With Amyotrophic Lateral Sclerosis (ALS) and Evidence of Elevated Systemic Inflammation
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 138
- 试验地点
- 22
- 主要终点
- Change from baseline in score on ALS Functional Rating Scale-Revised (ALSFRS-R) questionnaire
研究概览
简要总结
This study evaluates NP001 in patients with amyotrophic lateral sclerosis (ALS) and evidence of systemic inflammation. Half of participants will receive NP001 and the other half will receive placebo.
详细描述
This is a randomized, double-blind, placebo-controlled study of NP001 in subjects with ALS and evidence of elevated systemic inflammation. Subjects will be allocated (1:1) to NP001 and placebo. Drug or placebo will be given intravenously.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 21 Years 至 80 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosis of clinically possible, clinically probable (with or without laboratory support), or clinically definite ALS (using the revised El Escorial Criteria)
- •Forced vital capacity greater than or equal to 65% of that predicted for age and height
- •Onset of ALS-related weakness less than 3 years prior to first dose of study drug
- •Plasma high sensitivity C-reactive protein (hs-CRP) concentration of greater than or equal to 0.113 mg/dL at pre-screening/screening
- •Stable dose (greater than 30 days) of riluzole if undergoing treatment with this agent
- •For females: Not be of childbearing potential or agree to use adequate birth control during the study
排除标准
- •Life expectancy of less than 6 months
- •Tracheotomy or be using ventilatory assistance, including Bi-level Positive Airway Pressure (BiPAP) or Continuous Positive Airway Pressure (CPAP)
- •Active pulmonary disease
- •Gastrostomy
- •Stem cell therapy
- •Immune modulator therapy or participation in studies of other agents within 12 weeks of pre-screening/screening
- •Unstable medical condition other than ALS
研究组 & 干预措施
NP001
NP001 2 mg/kg by intravenous administration for 5 consecutive days in Month 1 and for 3 consecutive days in Months 2 through 6
干预措施: NP001 (Drug)
Placebo
Normal saline by intravenous administration for 5 consecutive days in Month 1 and for 3 consecutive days in Months 2 through 6
干预措施: Placebo (Drug)
结局指标
主要结局
Change from baseline in score on ALS Functional Rating Scale-Revised (ALSFRS-R) questionnaire
时间窗: Baseline and 6 months
次要结局
- Change in pulmonary function as measured by slow vital capacity readings(Baseline and 6 months)
- Change in levels of blood inflammatory biomarkers(Baseline, 3 and 6 months)
- Time to tracheotomy(Up to 6 months)