Phase 2/3 Study of Efficacy and Tolerability of Methylphenidate in the Treatment of Excessive Daytime Sleepiness in Myotonic Dystrophy Type 1
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 28
- 试验地点
- 1
- 主要终点
- Change from baseline of excessive daytime sleepiness
研究概览
简要总结
The purpose of this study is to determine whether methylphenidate is effective in the treatment of excessive daytime sleepiness due to myotonic dystrophy type 1 (DM1).
详细描述
Myotonic dystrophy type 1 (DM1) is a multisystemic disorder characterized by muscle weakness, myotonia, and the involvement of several systems. Hypersomnolence is one of the most frequently reported symptoms in patients with DM1 and often lead to handicap such as cessation of employment and withdrawal from social activities.The current investigation represents a prospective, double-blind, randomized, crossover, placebo-controlled study designed to evaluate the efficacy of methylphenidate for the treatment of excessive daytime sleepiness (EDS) in adults with DM1.
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
- •Epworth score ≥ 10
- •Diagnosis of myotonic dystrophy type 1
排除标准
- •hypersensibility to methylphenidate
- •Pregnancy
- •Patients who receive drugs that interfere with methylphenidate
- •Cognitive impairment
- •Sleep apnea
研究组 & 干预措施
Arm 1: Methylphenidate versus baseline
干预措施: Methylphenidate (Drug)
Arm 2: Placebo versus baseline
One table placebo per day during 3 week
干预措施: Placebo (Drug)
结局指标
主要结局
Change from baseline of excessive daytime sleepiness
时间窗: 3 weeks after treatment
次要结局
- Change from baseline of POMS, Rand36-Item Health survey and mean sleep latency(3 weeks after treatment)