Clinical Efficacy Trial of Mexiletine for Myotonic Dystrophy Type 1

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Brief Title

Clinical Efficacy Trial of Mexiletine for Myotonic Dystrophy Type 1

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 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.
    

Study Phase

Phase 2

Study Type

Interventional


Primary Outcome

Mean Change From Baseline in Ambulation Using the 6 Minute Walk Distance

Secondary Outcome

 Percentage of Participants That Had a Dose Reduction or a Study Drug Withdrawal or Suspension Over 6 Months

Condition

Myotonic Dystrophy

Intervention

Mexiletine

Study Arms / Comparison Groups

 Mexiletine
Description:  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.

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Drug

Estimated Enrollment

42

Start Date

June 2011

Completion Date

March 2017

Primary Completion Date

February 2017

Eligibility 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
      

Gender

All

Ages

18 Years - 80 Years

Accepts Healthy Volunteers

No

Contacts

Richard T. Moxley, III, MD, , 

Location Countries

United States

Location Countries

United States

Administrative Informations


NCT ID

NCT01406873

Organization ID

3716

Secondary IDs

Funding Source: FDA/OOPD

Responsible Party

Principal Investigator

Study Sponsor

University of Rochester


Study Sponsor

Richard T. Moxley, III, MD, Principal Investigator, University of Rochester


Verification Date

May 2018