Text Size

Clinical Efficacy Trial of Mexiletine for Myotonic Dystrophy Type 1

This study is currently recruiting participants.
Verified May 2013 by University of Rochester
Sponsor:
Information provided by (Responsible Party):
Richard T Moxley, University of Rochester
ClinicalTrials.gov Identifier:
NCT01406873
First received: July 20, 2011
Last updated: May 17, 2013
Last verified: May 2013
History of Changes
  Purpose

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).


Condition Intervention Phase
Myotonic Dystrophy
 Drug: Mexiletine
Drug: Placebo
 Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Randomized, Placebo Controlled, Clinical Efficacy Trial of Mexiletine for Myotonic Dystrophy Type-1 (DM1)

Resource links provided by NLM:

U.S. FDA Resources

Further study details as provided by University of Rochester:

Primary Outcome Measures:
  • Ambulation [ Time Frame: 6 months ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • The Number of Study Participants Who Safely Tolerated mexiletine [ Time Frame: 6 months ] [ Designated as safety issue: Yes ]
    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 ] [ Designated as safety issue: No ]
  • Muscle function and strength [ Time Frame: 6 months ] [ Designated as safety issue: No ]
  • Cardiac conduction [ Time Frame: 6 months ] [ Designated as safety issue: No ]
  • Quality of Life [ Time Frame: 6 months ] [ Designated as safety issue: No ]

Estimated Enrollment: 40
Study Start Date: June 2011
Estimated Study Completion Date: October 2014
Estimated Primary Completion Date: October 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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.
 Drug: Mexiletine
150 mg/kg Mexiletine capsules taken by mouth, three times daily for 6 months
Other Name: Generic name: mexiletine hydrochloride
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.
 Drug: Placebo
150 mg/kg placebo capsules taken by mouth, three times daily for 6 months

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.
  Eligibility

Ages Eligible for Study:   18 Years to 80 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01406873

Contacts
Contact: Liz Luebbe 585-275-7867 elizabeth_luebbe@urmc.rochester.edu

Locations
United States, New York
University of Rochester Medical Center, Department of Neurology Recruiting
Rochester, New York, United States, 14642
Contact: Liz Luebbe     585-275-7867     elizabeth_luebbe@urmc.rochester.edu    
Principal Investigator: Richard T. Moxley, III, MD            
Sub-Investigator: Chad Heatwole, MD            
Sub-Investigator: Eric Logigian, MD            
Sub-Investigator: Charles Thornton, MD            
Sponsors and Collaborators
University of Rochester
Investigators
Principal Investigator: Richard T. Moxley, III, MD University of Rochester
  More Information

Publications:

Responsible Party: Richard T Moxley, Professor of Neurology, University of Rochester
ClinicalTrials.gov Identifier: NCT01406873     History of Changes
Other Study ID Numbers: 3716, Funding Source: FDA/OOPD
Study First Received: July 20, 2011
Last Updated: May 17, 2013
Health Authority: United States: Food and Drug Administration

Keywords provided by University of Rochester:
Myotonic Dystrophy
Myotonic Dystrophy Type 1 (DM1)
Muscular Dystrophy
Mexiletine

Additional relevant MeSH terms:
Myotonic Dystrophy
Muscular Dystrophies
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Myotonic Disorders
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
 Nervous System Diseases
Neuromuscular Diseases
Genetic Diseases, Inborn
Mexiletine
Anti-Arrhythmia Agents
Cardiovascular Agents
Therapeutic Uses
Pharmacologic Actions

ClinicalTrials.gov processed this record on May 22, 2013