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Clinical Efficacy Trial of Mexiletine for Myotonic Dystrophy Type 1

This study has been completed.
Sponsor:
ClinicalTrials.gov Identifier:
NCT01406873
First Posted: August 1, 2011
Last Update Posted: August 21, 2017
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
Information provided by (Responsible Party):
Chad Heatwole, University of Rochester
July 20, 2011
August 1, 2011
August 21, 2017
June 2011
February 2017   (Final data collection date for primary outcome measure)
Ambulation [ Time Frame: 6 months ]
Same as current
Complete list of historical versions of study NCT01406873 on ClinicalTrials.gov Archive Site
  • 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 ]
Same as current
Not Provided
Not Provided
 
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)
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).

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.
Interventional
Phase 2
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Myotonic Dystrophy
  • Drug: Mexiletine
    150 mg/kg Mexiletine capsules taken by mouth, three times daily for 6 months
    Other Name: Generic name: mexiletine hydrochloride
  • Drug: Placebo
    150 mg/kg placebo capsules taken by mouth, three times daily for 6 months
  • Active Comparator: Mexiletine
    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.
    Intervention: Drug: Mexiletine
  • Placebo Comparator: Sugar pill
    20 subjects will be randomized (assigned) to receive placebo (sugar pill). This control group is necessary to definitely establish the antimyotonic efficacy and safety of mexiletine.
    Intervention: Drug: Placebo

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Completed
42
March 2017
February 2017   (Final data collection date for primary outcome measure)

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
Sexes Eligible for Study: All
18 Years to 80 Years   (Adult, Senior)
No
Contact information is only displayed when the study is recruiting subjects
United States
 
 
NCT01406873
3716
Funding Source: FDA/OOPD ( Other Grant/Funding Number: R01FD003716 )
Yes
Not Provided
Not Provided
Chad Heatwole, University of Rochester
University of Rochester
Not Provided
Principal Investigator: Richard T. Moxley, III, MD University of Rochester
University of Rochester
August 2017

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP