Effectiveness of Mexiletine for Treating People With Non-Dystrophic Myotonia

NCT00832000 ↗

Phase 2 Completed 59 enrolled Richard Barohn, MD

RandomizedCrossoverDouble-blindTreatment

Summary

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.

Timeline

Start: 2008-12
Primary completion: 2011-03
Completion: 2011-03

Outcome

Met primary endpoint

paper Mexiletine significantly improved patient-reported severity score stiffness on the IVR diary. PMID 23032552 ↗

paper period 1 means were 2.53 for mexiletine and 4.21 for placebo; difference, -1.68; 95% CI, -2.66 to -0.706; P < .001; PMID 23032552 ↗

paper period 2 means were 1.60 for mexiletine and 5.27 for placebo; difference, -3.68; 95% CI, -3.85 to -0.139; P = .04 PMID 23032552 ↗

Publications

Statland JM, Bundy BN, Wang Y, Rayan DR, Trivedi JR, Sansone VA, Salajegheh MK, Venance SL, Ciafaloni E, Matthews E, Meola G, Herbelin L, Griggs RC, Barohn RJ, Hanna MG; Consortium for Clinical Investigation of Neurologic Channelopathies. Mexiletine for symptoms and signs of myotonia in nondystrophic myotonia: a randomized controlled trial. JAMA. 2012 Oct 3;308(13):1357-65. doi: 10.1001/jama.2012.12607.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Mexiletine	Small molecule	200 mg	Oral