Status:

COMPLETED

Clinical Efficacy Trial of Mexiletine for Myotonic Dystrophy Type 1

Lead Sponsor:

University of Rochester

Conditions:

Myotonic Dystrophy

Eligibility:

All Genders

18-80 years

Phase:

PHASE2

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 conducti...

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 ...

Eligibility Criteria

Inclusion

  • 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

  • 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

Key Trial Info

Start Date :

June 1 2011

Trial Type :

INTERVENTIONAL

Allocation :

ACTUAL

End Date :

March 1 2017

Estimated Enrollment :

42 Patients enrolled

Trial Details

Trial ID

NCT01406873

Start Date

June 1 2011

End Date

March 1 2017

Last Update

June 19 2018

Active Locations (1)

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1

University of Rochester Medical Center, Department of Neurology

Rochester, New York, United States, 14642