Clinical Efficacy Trial of Mexiletine for Myotonic Dystrophy Type 1

• Status: Completed
• Trial end date: 2017-03-01
• Target enrollment: 0
• Participant gender: All

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).

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: University of Rochester
• Treatments: Mexiletine

Criteria

Inclusion Criteria:

• 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

Exclusion Criteria:

• 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