Clinical Trial: Clinical Efficacy Trial of Mexiletine for Myotonic Dystrophy Type 1
Study Status: Completed
Recruit Status: Completed
Study Type: Interventional
Official Title: A Randomized, Placebo Controlled, Clinical Efficacy Trial of Mexiletine for Myotonic Dystrophy Type-1 (DM1)
Brief Summary: 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).
Detailed Summary:
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.
Sponsor: University of Rochester
Current Primary Outcome: Ambulation [ Time Frame: 6 months ]
Original Primary Outcome: Same as current
Current Secondary Outcome:
- The Number of Study Participants Who Safely Tolerated mexiletine [ Time Frame: 6 months ]Safety and tolerability are monitored by interval laboratory studies, Holter monitoring, resting echocardiograms, dula energy x-ray absorptiometry (DEXA)studies, and physical examinations. The participants have three in-person evaluations at the University of Rochester Clinical Research Center (Months 0, 3, and 6) and telephone evaluations every 2 weeks. Patients complete side effects diaries to record any adverse events in the interval time between the in-person evaluations.
- Myotonia [ Time Frame: 6 months ]
- Muscle function and strength [ Time Frame: 6 months ]
- Cardiac conduction [ Time Frame: 6 months ]
- Quality of Life [ Time Frame: 6 months ]
Original Secondary Outcome: Same as current
Information By: University of Rochester
Dates:
Date Received: July 20, 2011
Date Started: June 2011
Date Completion:
Last Updated: April 25, 2017
Last Verified: April 2017