A Phase 2a, Double-Blinded, Randomized, Placebo-Controlled, and Active-Treatment Extension Study to Assess the Safety, Tolerability, Efficacy, Pharmacokinetics, and Immunogenicity of ARGX-119 in Participants With Amyotrophic Lateral Sclerosis
试验速览
- 阶段
- 2 期
- 状态
- 终止
- 发起方
- argenx
- 入组人数
- 60
- 试验地点
- 18
- 主要终点
- Assessment of adverse events (AEs)
研究概览
简要总结
This study aims to evaluate the safety of ARGX-119 in adults with ALS. The study will also assess the impact of ARGX-119 on ALS disease outcomes, including muscle function. The study consists of 2 periods: a treatment period when participants will receive one of three ARGX-119 doses or placebo and an extension period when all participants will receive the same dose of ARGX-119. Participation in the study will last up to approximately 100 weeks.
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
- 18 Years 至 80 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •The participant is at least 18 and ≤80 years of age
- •The participant is diagnosed with familial or sporadic ALS according to Gold Coast criteria
- •The participant has a Treatment Research Initiative to Cure ALS (TRICALS) risk profile of ≥ -6.0 to < -2.0
- •Slow vital capacity (SVC) of ≥ 60% of the predicted value according to Global Lung Function Initiative 2012
排除标准
- •Use of noninvasive ventilation more than 10 hours a day or use of a tracheostomy for ventilatory support
- •Any history of or current exposure to any gene or cell therapies (off-label use or investigational) for ALS
- •Pregnant or lactating state or intention to become pregnant during the study
研究组 & 干预措施
ARGX-119 - Dose 1
Participants will receive first dosage level of ARGX-119 intravenously during the double blinded treatment period followed by ARGX-119 in active treatment extension period
干预措施: ARGX-119 (Biological)
ARGX-119 - Dose 2
Participants will receive second dosage level of ARGX-119 intravenously during the double blinded treatment period followed by ARGX-119 in active treatment extension period
干预措施: ARGX-119 (Biological)
ARGX-119 - Dose 3
Participants will receive third dosage level of ARGX-119 intravenously during the double blinded treatment period followed by ARGX-119 in active treatment extension period
干预措施: ARGX-119 (Biological)
Placebo
Participants will receive placebo intravenously during the double-blinded treatment period followed by ARGX-119 in active treatment extension period
干预措施: ARGX-119 (Biological)
Placebo
Participants will receive placebo intravenously during the double-blinded treatment period followed by ARGX-119 in active treatment extension period
干预措施: Placebo (Other)
结局指标
主要结局
Assessment of adverse events (AEs)
时间窗: Up to week 96
次要结局
- Incidence of anti-drug antibodies (ADA) against ARGX-119 in serum over time(Up to week 96)
- Rate of change from baseline in electrophysiological muscle scan (MScan)-derived motor unit number (MUN)(Up to week 24)
- Prevalence of anti-drug antibodies (ADA) against ARGX-119 in serum over time(Up to week 96)
- Maximum observed serum concentration (Cmax) of ARGX-119(Up to week 96)