Efficacy and Safety of Mexiletine in Non-dystrophic Myotonias
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 24
- 试验地点
- 1
- 主要终点
- score of stiffness severity on a self-assessment scale (100 mm VAS)
研究概览
简要总结
Treatment strategies in non-dystrophic myotonias are based on selective case reports, clinical experience and theoretical benefit. Presently, the most promising antimyotonic medication is mexiletine (MEX) but its manufacturing was stopped. The proposed randomized, double-blind, placebo-controlled, crossover trial is designed to:
- study the safety and efficacy of mexiletine for the treatment of non-dystrophic myotonias
- validate electromyographic tests as a standardized outcome measure of myotonia
- assess the reliability and validity of a new clinical rating scale for myotonia
详细描述
A. Specific aims
Treatment strategies in non-dystrophic myotonias are based on selective case reports, clinical experience and theoretical benefit. Presently, the most promising antimyotonic medication is mexiletine (MEX) but its manufacturing was stopped. The proposed randomized, double-blind, placebo-controlled, crossover with wash-out trial is designed to:
- study the safety and efficacy of mexiletine for the treatment of non-dystrophic myotonias
- validate electromyographic tests as a standardized outcome measure of myotonia
- assess the reliability and validity of a new clinical rating scale for myotonia
B. Research design Because of their differing phenotypes, 12 Paramyotonia Congenita and 12 Myotonia Congenita subjects will be enrolled in a stratified trial
C. Outcome variables
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Crossover
- 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 65 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- 未提供
排除标准
- 未提供
研究组 & 干预措施
1
Mexiletine / Placebo
干预措施: Mexiletine (Drug)
1
Mexiletine / Placebo
干预措施: placebo (Drug)
2
Placebo / Mexiletine
干预措施: Mexiletine (Drug)
2
Placebo / Mexiletine
干预措施: placebo (Drug)
结局指标
主要结局
score of stiffness severity on a self-assessment scale (100 mm VAS)
时间窗: 18 days
次要结局
- CGI efficacy (Clinical Global Impression- Efficacy index)(18 days)
- standardized EMG measures after repetitive short exercise test at cold and long exercise test(18 days)
- chair test: time needed to stand up from a chair, walk around it and sit down again(18 days)
- severity and disability scale of myotonia to be validated(18 days)
- quality of life scale (INQOL)(18 days)