Effectiveness of Mexiletine for Treating People With Non-Dystrophic Myotonia

Completed · Phase 2 · Has a placebo group

Conditions studied: Myotonia, Non-Dystrophic Myotonia

In brief

Nondystrophic myotonias (NDM) are neuromuscular disorders caused by genetic abnormalities in certain muscle cell membrane proteins. The proteins affect muscle contraction. Individuals with NDM experience limited muscle relaxation, which then can cause pain, weakness, incoordination, and impaired physical activity and function. Because NDM is very rare, information on the best way to treat people with the disorders is lacking, and there are no FDA-approved therapies. The purpose of this study is to determine the effectiveness of the medication mexiletine in treating people with NDM.

Key facts

Study ID

NCT00832000

Run by

Richard Barohn, MD

People needed

59

Starts

2008-12-01

Expected to finish

2011-03-01

Last updated by the study team

2013-08-23

Who can join

Age: 16 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Clinical symptoms or signs suggestive of myotonic disorders
• Presence of myotonic potentials on electromyography (EMG)
• Participant in the Non-Dystrophic Natural History study (RDCRN 5303) or a new patient with confirmed non-dystrophic myotonia

You may not qualify if…

• Other neurological condition that might affect the assessment of the study measurements
• Genetic confirmation of DM1 (more than 50 repeats of CTG) or DM2
• Existing cardiac conduction defects, as evidenced on EKG, including but not limited to the following conditions: malignant arrhythmia or cardiac conduction disturbances (e.g., second degree AV block, third degree AV block, or prolonged QT interval)
• Existing permanent pacemaker
• Current use of any of the following antiarrhythmic medications for a cardiac disorder: flecainide acetate, encainide, disopyramide, procainamide, quinidine, propafenone, or mexiletine
• Use of medications for myotonia, such as phenytoin and flecainide acetate, within 5 days of study entry; carbamazepine and mexiletine within 3 days of study entry; or propafenone, procainamide, disopyramide, quinidine, and encainide within 2 days of study entry
• Use of medications that produce myotonia, which may include fibrate acid derivatives, hydroxymethylglutaryl CoA reductase inhibitors, chloroquine, and colchicines
• Kidney or liver disease
• Heart failure
• Seizure disorder
• Pregnant or breastfeeding

Where it is running

• University of Kansas Medical Center — Kansas City, Kansas, United States
• Brigham & Women's Hospital — Boston, Massachusetts, United States
• University of Rochester School of Medicine & Dentistry — Rochester, New York, United States
• University of Texas Southwestern Medical Center — Dallas, Texas, United States
• London Health Sciences Center — London, Ontario, Canada
• University of Milan — Milan, Italy
• Institute of Neurology and National Hospital for Neurology — London, United Kingdom

Full record on ClinicalTrials.gov

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