NCT02988297撤回2 期
Nebulized RNS60 for the Treatment of Amyotrophic Lateral Sclerosis
适应症
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 撤回
- 入组人数
- 140
- 主要终点
- ALS functional rating scale-revised (ALSFRS-R) score
研究概览
简要总结
The purpose of this study is to determine whether nebulized RNS60 is effective in the treatment of amyotrophic lateral sclerosis (ALS).
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
- Triple (Participant, 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
- •Probable, probable-laboratory supported, or definite ALS, either sporadic or familial according to modified El Escorial criteria
- •Disease duration < 3 years
- •Age 18 to 80
- •Able to provide informed consent and to comply with study procedures
- •Subjects must not have taken riluzole for at least 30 days, or be on a stable dose of riluzole for at least 30 days, prior to screening (riluzole-naïve participants are permitted in the study)
- •Women must not be lactating or able to become pregnant (e.g. post menopausal, surgically sterile, or using adequate birth control) for the duration of the study, and for 3 months after study completion
- •Men should practice contraception for the duration of the study and for 3 months after completion
排除标准
- •Diagnosis of a motor neuron disease other than ALS (e.g., primary lateral sclerosis, progressive muscular atrophy, progressive bulbar palsy)
- •Clinically significant unstable medical condition (other than ALS) that would pose a risk to the participant if they were to participate in the study or that would impact survival or functional disability in the next 12 months
- •Abnormal liver function defined as aspartate aminotransferase (AST) and/or alanine aminotransferase (ALT) > 3 times the upper limit of the normal
- •Renal insufficiency (Glomerular Filtration Rate < 60)
- •Active pulmonary disease
- •Prior poor compliance with an inhalation device
- •The presence of unstable psychiatric disease, cognitive impairment, or dementia that would impair ability of the participant to provide informed consent, according to judgment by the principal investigator.
- •History of human immunodeficiency virus (HIV) infection, clinically significant chronic hepatitis, or other active infection.
- •Active participation in another ALS clinical trial within 30 days of the Screening Visit
- •Other experimental treatments or anti-inflammatory/immunomodulatory medications used in the preceding 3 months
研究组 & 干预措施
Placebo
Placebo Comparator
Nebulized Placebo will be administered by daily inhalation for 24 weeks.
干预措施: Placebo (Drug)
RNS60
Experimental
Nebulized RNS60 will be administered by daily inhalation for 24 weeks.
干预措施: RNS60 (Drug)
结局指标
主要结局
ALS functional rating scale-revised (ALSFRS-R) score
时间窗: 24 weeks
The mean change of the ALSFRS-R total score
次要结局
- Slow vital capacity (SVC)(24 weeks)
- ALS assessment questionnaire (ALSAQ-40) score(24 weeks)
- Proportion of regulatory T cells (Treg)(24 weeks)
- Deaths or tracheostomies(28 weeks)
- Adverse events (AEs)(28 weeks)
研究者
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