A Randomized, Placebo Controlled, Clinical Efficacy Trial of Mexiletine for Myotonic Dystrophy Type-1 (DM1)
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 42
- 试验地点
- 1
- 主要终点
- Mean Change From Baseline in Ambulation Using the 6 Minute Walk Distance
研究概览
简要总结
The purpose of this study is to investigate the effects of mexiletine treatment for 6 months on ambulation, myotonia, muscle function and strength, pain, gastrointestinal functioning, cardiac conduction, and quality of life in myotonic dystrophy type 1 (DM1).
详细描述
This study will provide data on the long term (6 months) safety and efficacy of mexiletine in:
- improving the distance participants are able to walk in six minutes;
- reducing myotonia;
- improving muscle strength;
- increasing lean muscle mass;
- decreasing musculoskeletal pain;
- improving gastrointestinal function and swallowing);
- improving functional abilities;
- decreasing cardiac arrhythmias; and
- improving disease-specific health related quality-of-life.
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
- •A diagnosis of DM1, confirmed by DM1 genetic mutation
- •Ability to walk 30 feet (assistance with cane and/or leg bracing permitted)
- •Presence of grip myotonia
排除标准
- •Congenital DM1
- •Treatment with Mexiletine within past 8 weeks
- •Second or third degree heart block, atrial flutter, atrial fibrillation, ventricular arrhythmias, or is receiving medication for treatment of a cardiac arrhythmia
- •Receiving another antimyotonia drug
- •Liver or kidney disease requiring ongoing treatment
- •Has a seizure disorder
- •Is pregnant or lactating
- •Had severe depression within 3 months or a history of suicide ideation
- •Has any one of the following medical conditions: uncontrolled diabetes mellitus, congestive heart failure, symptomatic cardiomyopathy, symptomatic coronary artery disease, cancer (other than skin cancer) less than five years previously, multiple sclerosis, or other serious medical illness.
- •Drug or alcohol abuse within 3 months
- •Coexistence of another neuromuscular disease
- •Is unable to give informed consent
- •Severe arthritis or other medical condition (besides DM1) that would significantly impact ambulation
研究组 & 干预措施
Mexiletine
20 subjects will be randomized (assigned) to receive Mexiletine. Mexiletine is available on the market for the treatment of cardiac arrhythmias, but it is not currently approved for the treatment of myotonia or myotonic dystrophy.
干预措施: Mexiletine (Drug)
Sugar pill
20 subjects will be randomized (assigned) to receive placebo (sugar pill). This control group is necessary to definitely establish the antimyotonic efficacy and safety of mexiletine.
干预措施: Placebo (Drug)
结局指标
主要结局
Mean Change From Baseline in Ambulation Using the 6 Minute Walk Distance
时间窗: Baseline to 6 months
During this assessment, participants were asked to walk as far as they could back and forth on a fixed 20 meter route for 6 minutes. The total distance walked during the 6 minutes was recorded in meters. Change from baseline was defined as the difference between the average 6 minute walk distance at baseline and the average 6 minute walk distance at 6 months.
次要结局
- Mean Change From Baseline in Quantitative Measure of Hand Grip Myotonia(Baseline to 6 months)
- Mean Change From Baseline in Manual Muscle Testing (MMT) Score(Baseline to 6 months)
- Mean Change From Baseline in Patient-Reported Disease Burden and Quality of Life(Baseline to 6 months)
- Mean Change From Baseline in PR, QRS, and QTc Intervals, and Average Minimum Heart Rate (HR) Via Electrocardiogram (ECG) Monitoring(Baseline to 6 Months)
- Percentage of Participants That Had a Dose Reduction or a Study Drug Withdrawal or Suspension Over 6 Months(6 months)
研究者
Chad Heatwole
Associate Professor of Neurology
University of Rochester