NCT01406873

Clinical Efficacy Trial of Mexiletine for Myotonic Dystrophy Type 1

A Randomized, Placebo Controlled, Clinical Efficacy Trial of Mexiletine for Myotonic Dystrophy Type-1 (DM1)

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 42 (actual)
• Sponsor: University of Rochester · Academic / Other
• Sex: All
• Age: 18 Years – 80 Years
• Healthy volunteers: Not accepted

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 description

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.

Conditions

• Myotonic Dystrophy

Interventions

Type	Name	Description
DRUG	Mexiletine	150 mg/kg Mexiletine capsules taken by mouth, three times daily for 6 months
DRUG	Placebo	150 mg/kg placebo capsules taken by mouth, three times daily for 6 months

Timeline

• Start date: 2011-06-01
• Primary completion: 2017-02-01
• Completion: 2017-03-01
• First posted: 2011-08-01
• Last updated: 2018-06-19
• Results posted: 2018-03-29

Locations

1 site across 1 country: United States