A Proof of Concept Study of the Effect of Amifampridine (Firdapse®) on Neuromuscular Transmission in Patients Treated With OnabotulinumtoxinA (Botox®, BTX-A)
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 入组人数
- 21
- 试验地点
- 1
- 主要终点
- Percentage of abnormal pairs
研究概览
简要总结
if amifampridine can improve neuromuscular transmission in muscles previously injected with OnabotulinumtoxinA (BTX-A)
详细描述
Amifampridine may also be effective in treating the sequelae of botulinum toxin injections and this study will determine if neuromuscular transmission as measured by single fiber electromyography is improved in patients treated with BTX-A following administration of amifampridine
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
- 18 Years 至 80 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Age: 18-80 years of either gender and of any racial background
- •Underwent BTX-A injection of facial muscles including frontalis with a total dose between 100-200 units between 80 and 150 days prior to study
- •Have decision-making capacity to provide informed consent to study drug dosing and Single Fiber Electromyography (SFEMG)
排除标准
- •History of cardiac arrhythmia
- •History of seizures or uncontrolled asthma
- •History of renal or hepatic disease
- •History of any generalized neuromuscular disease
- •History of Bell's Palsy or facial nerve trauma
- •History of treatment with or sensitivity to amifampridine, 3,4 diaminopyridine (DAP) or 4-aminopyridine (Ampyra®)
- •Currently experiencing sequelae of previous BTX-A treatment
- •Current use of pyridostigmine (known to alter neuromuscular transmission)
- •Use of any investigational drug or device within 30 days of enrollment
研究组 & 干预措施
Amifampridine will be orally administered to study participants
Amifampridine will be orally administered to study participants following completion of the baseline SFEMG. Post-dose SFEMG will commence at 30 minutes following dosing and will be completed within 30 minutes. The participant will remain under observation in the Diagnostic Neurology suite for 2 hours after dosing so it is estimated that the entire protocol including monitoring will be completed within 2-3 hours.
干预措施: Amifampridine (Drug)
结局指标
主要结局
Percentage of abnormal pairs
时间窗: Hour 3
Wilcoxon's test for paired data will be used to analyze baseline and post-treatment percent abnormal pairs
Percentage of Jitter
时间窗: Hour 3
Wilcoxon's test for paired data will be used to analyze baseline and post-treatment mean jitter
Percentage of pairs that show blocking
时间窗: Hour 3
Wilcoxon's test for paired data will be used to analyze baseline and post-treatment percent blocking
次要结局
未报告次要终点