Evidence map›Paper›PMID 20439846›Full record

Trial reportNeurology2010

Mexiletine is an effective antimyotonia treatment in myotonic dystrophy type 1.

E L Logigian, W B Martens, R T Moxley, M P McDermott, N Dilek, A W Wiegner, A T Pearson, C A Barbieri, C L Annis, C A Thornton and 1 more

Registry-linked trialOpen access · bronzeAbstract readRandomized Controlled Trial
In one paragraph

Trial report in Neurology, 2010. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT01406873 (A Randomized, Placebo Controlled, Clinical Efficacy Trial of Mexiletine for Myotonic Dystrophy Type-1), which is not on this map. Cited by 60 papers, 4 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
60citing papers in PubMed, 4 pooled it
4.5field-weighted citation impact, top 4% of its field
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

NCT01406873 phase2completednot on this mapstarted 2011, after this paper: background citation

A Randomized, Placebo Controlled, Clinical Efficacy Trial of Mexiletine for Myotonic Dystrophy Type-1 (DM1)

TypeinterventionalSponsorUniversity of RochesterRan2011 to 2017Enrolled42ConditionsMyotonic DystrophyArmsMexiletine, Placebo
3 · Its place in the literature

Who cites it

60 citing papers in PubMed, 4 syntheses or guidelines pooled it, 195 citations in OpenAlex.

  1. Drug treatment for myotonia.The Cochrane database of systematic reviews · 2025
    Pooled it
  2. Effectiveness and safety of mexiletine versus placebo in patients with myotonia: a systematic review and meta-analysis.Neurological sciences : official journal of the Italian Neurological Society and of the Italian Society of Clinical Neurophysiology · 2024
    Pooled it
  3. Guideline
  4. Drug treatment for myotonia.The Cochrane database of systematic reviews · 2006
    Pooled it
  5. Trial
  6. Trial
  7. Trial
  8. Trial
  9. Review
  10. Article
  11. Review
  12. Article
  13. Review
  14. Article
  15. Molecular mechanisms and therapeutic strategies for neuromuscular diseases.Cellular and molecular life sciences : CMLS · 2024
    Review
  16. Article
  17. Review
  18. The role of sleep in neuromuscular disorders.Frontiers in neurology · 2023
    Review
  19. Article
  20. Article
4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

11 authors at 1 institution in 1 country.

E L LogigianDepartment of Neurology, University of Rochester, Rochester, NY, USA. eric_logigian@urmc.rochester.edu
W B Martens
R T Moxley
M P McDermott
N Dilek
A W Wiegner
A T Pearson
C A Barbieri
C L Annis
C A Thornton
R T Moxley
MACOM (United States) · US

Funding

UNIVERSITY OF ROCHESTER'S RESEARCH ASSOCIATE ORGANIZATIONUL1RR024160 · NCRR · UNIVERSITY OF ROCHESTER · PI PEARSON, THOMAS ARTHUR · 2006 to 2011
$38.7M
Training and Educational CoreU54NS048843 · NINDS · UNIVERSITY OF ROCHESTER · PI MOXLEY, RICHARD T · 2003 to 2017
$21.9M
YOHIMBINE CHALLENGE IN PARKINSONIAN PATIENTS WITH PANIC DISORDERM01RR000044 · NCRR · UNIVERSITY OF ROCHESTER · PI GUZICK, DAVID S · 1985 to 2006
$13.8M
Coordination and Statistics for CoQ10 in HD [2CARE]R01NS052619 · NINDS · UNIVERSITY OF ROCHESTER · PI MCDERMOTT, MICHAEL P · 2005 to 2014
$13.5M
Early Randomized Surgical Epilepsy TrialU01NS042372 · NINDS · UNIVERSITY OF CALIFORNIA LOS ANGELES · PI ENGEL, JEROME NONE · 2002 to 2007
$9.8M
Parkinson's Disease Data Organizing CenterU01NS050095 · NINDS · UNIVERSITY OF ROCHESTER · PI KURLAN, ROGER M · 2004 to 2010
$7.3M
Dichlorphenamide vs Acetazolamide for Periodic ParalysisR01NS045686 · NINDS · UNIVERSITY OF ROCHESTER · PI GRIGGS, ROBERT C · 2004 to 2010
$5.7M
Coordination and Statistics for CoQ10 in PD in Phase 3U01NS050573 · NINDS · UNIVERSITY OF ROCHESTER · PI OAKES, DAVID · 2005 to 2009
$4.7M
Study of Antidepressants in Parkinson's Disease (SAD PD)R01NS046487 · NINDS · UNIVERSITY OF ROCHESTER · PI RICHARD, IRENE H · 2004 to 2008
$3.7M
Pathogenesis of Myopathy in Models of Myotonic DystrophyR01AR046806 · NIAMS · UNIVERSITY OF ROCHESTER · PI THORNTON, CHARLES A · 2001 to 2009
$3.1M
Congenital HHV6 Infection: Characteristics and OutcomeR01HD044430 · NICHD · UNIVERSITY OF ROCHESTER · PI HALL, CAROLINE BREESE · 2003 to 2007
$3.1M
RNA-mediated Mechanisms in the Myotonic DystrophiesR01AR049077 · NIAMS · UNIVERSITY OF ROCHESTER · PI THORNTON, CHARLES A · 2002 to 2011
$3.0M
FDA HHS FD-R-001662NCRR NIH HHS 1UL 1RR02416002NCRR NIH HHS 5 MO1 RR00044NCRR NIH HHS M01 RR00044NCRR NIH HHS RR24160NCRR NIH HHS UL1 RR024160NHLBI NIH HHS HL80107NIAMS NIH HHS 2 R01 AR049077NIAMS NIH HHS AR046806NIAMS NIH HHS AR049077NIAMS NIH HHS AR48143NIAMS NIH HHS AR49077NIAMS NIH HHS AR52274NIAMS NIH HHS K24 AR048143NIAMS NIH HHS N01-AR-0-2250NIAMS NIH HHS N01-AR-5-2274NIAMS NIH HHS R01 AR49077NICHD NIH HHS HD44430NIDCR NIH HHS DE16280NINDS NIH HHS 1 R13 NS066630-01NINDS NIH HHS 9 U54NS48843NINDS NIH HHS NS048843NINDS NIH HHS NS42372NINDS NIH HHS NS45686NINDS NIH HHS NS46487NINDS NIH HHS NS48843NINDS NIH HHS NS49639NINDS NIH HHS NS50095NINDS NIH HHS NS50573NINDS NIH HHS NS52619NINDS NIH HHS NS58259NINDS NIH HHS U54NS48843
6 · The paper itself

Abstract

objectiveTo determine if mexiletine is safe and effective in reducing myotonia in myotonic dystrophy type 1 (DM1).

backgroundMyotonia is an early, prominent symptom in DM1 and contributes to decreased dexterity, gait instability, difficulty with speech/swallowing, and muscle pain. A few preliminary trials have suggested that the antiarrhythmic drug mexiletine is useful, symptomatic treatment for nondystrophic myotonic disorders and DM1.

methodsWe performed 2 randomized, double-blind, placebo-controlled crossover trials, each involving 20 ambulatory DM1 participants with grip or percussion myotonia on examination. The initial trial compared 150 mg of mexiletine 3 times daily to placebo, and the second trial compared 200 mg of mexiletine 3 times daily to placebo. Treatment periods were 7 weeks in duration separated by a 4- to 8-week washout period. The primary measure of myotonia was time for isometric grip force to relax from 90% to 5% of peak force after a 3-second maximum grip contraction. EKG measurements and adverse events were monitored in both trials.

resultsThere was a significant reduction in grip relaxation time with both 150 and 200 mg dosages of mexiletine. Treatment with mexiletine at either dosage was not associated with any serious adverse events, or with prolongation of the PR or QTc intervals or of QRS duration. Mild adverse events were observed with both placebo and mexiletine treatment.

conclusionsMexiletine at dosages of 150 and 200 mg 3 times daily is effective, safe, and well-tolerated over 7 weeks as an antimyotonia treatment in DM1. CLASSIFICATION OF EVIDENCE: This study provides Class I evidence that mexiletine at dosages of 150 and 200 mg 3 times daily over 7 weeks is well-tolerated and effective in reducing handgrip relaxation time in DM1.

Indexed as

AdultAnti-Arrhythmia AgentsCross-Over StudiesDouble-Blind MethodFemaleHumansMaleMexiletineMiddle AgedMyotoniaMyotonic DystrophyPatient SelectionPlacebosTreatment OutcomeAnti-Arrhythmia AgentsMexiletinePlacebos

Identifiers

PMID20439846
PMCPMC2871004
OpenAlexW1983101498

What OpenQuestion holds

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Registered trials

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.