A Clinical Trial to Evaluate the Safety and Efficacy of Fycompa in Subjects With Amyotrophic Lateral Sclerosis (ALS)
试验速览
- 阶段
- 不适用
- 状态
- 终止
- 入组人数
- 12
- 试验地点
- 1
- 主要终点
- Incidence of treatment-emergent adverse events
研究概览
简要总结
This is a pilot trial to test perampanel (Fycompa; Eisai, Inc.) in ALS patients. The investigators will focus on safety and preliminary signs of efficacy. Perampanel is approved by the FDA for treatment of seizures in patients with epilepsy. In this study, perampanel will be used off-label for adults with ALS at an oral medication dose on the low end of the recommended dose range for epilepsy. This study will consist of two treatments arms: perampanel and matching placebo randomized at a 1:1 ratio. Subjects will receive medication for 9 months.
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
- •diagnosis of ALS
- •first clinical weakness within past 3 years
- •slow vital capacity >= 60% of predicted within 1 month of treatment
- •may be on stable dose of riluzole for at least 30 days, or otherwise agree to not initiate riluzole for duration of the trial
- •may be on stable dose of edaravone for at least 30 days, otherwise agree to not initiate edaravone for duration of the trial
- •can travel to Stony Brook to receive medical care
- •must have a monitor who can be contacted at regular intervals to report on subject's clinical/psychiatric status
排除标准
- •use of tracheostomy or mechanical ventilation within last 3 months
- •hepatic insufficiency or abnormal liver function
- •renal insufficiency
- •clinically significant psychiatric disorder
- •active malignancy
- •history of HIV, clinically significant chronic hepatitis, or other active infection
- •history of stomach or intestinal surgery or condition that could interfere with absorption, distribution, metabolism or secretion of study drug
- •history of alcohol or substance abuse within 3 months prior to entry (subjects will be instructed to refrain from alcohol during the study)
- •use of strong cytochrome P4503A inhibitors or inducers, anticonvulsants or other drugs known to interact strongly with perampanel.
- •pregnancy or lactation
- •clinically significant medical condition (other than ALS) that would pose a risk to the subject if they were to participate
- •know hypersensitivity to perampanel
- •currently participating, or has participated in a study with an investigation or marketed compound within 3 months of entry
研究组 & 干预措施
placebo
placebo 1 tablet QD for 2 weeks placebo 2 tablets QD for 2 weeks placebo 3 tablets QD for 2 weeks placebo 4 tablets QD for 2 weeks
干预措施: Placebo Oral Tablet (Drug)
perampanel
perampanel 2mg QD for 2 weeks perampanel 4mg QD for 2 weeks perampanel 6mg QD for 2 weeks perampanel 8mg QD for 30 weeks
干预措施: Perampanel (Drug)
结局指标
主要结局
Incidence of treatment-emergent adverse events
时间窗: 9 months
次要结局
- Efficacy as measured by change in ALSFRS-R score (ALS functional rating scale-revised);(9 months)
研究者
Nurcan Gursoy
Clinical Assistant Professor
Stony Brook University