Phase 2 N=42 Randomized Quadruple-blind Treatment

Clinical Efficacy Trial of Mexiletine for Myotonic Dystrophy Type 1

Myotonic Dystrophy

Bottom Line

View on ClinicalTrials.gov: NCT01406873 ↗

Enrolled (actual)

Serious AEs

4.8%

Results posted

Mar 2018

Primary outcome: Primary: Mean Change From Baseline in Ambulation Using the 6 Minute Walk Distance — 17.44; 7.25 Meters

Study Design & Population

Study type: Interventional
Phase: Phase 2
Interventions: Mexiletine (Drug); Placebo (Drug)
Age: Adult, Older Adult · 18+ yrs
Sex: All
Sponsor: University of Rochester
Primary completion: Feb 2017

Outcome Measures

Outcome	Result	p-value
PRIMARY Mean Change From Baseline in Ambulation Using the 6 Minute Walk Distance	17.44; 7.25	—
SECONDARY Percentage of Participants That Had a Dose Reduction or a Study Drug Withdrawal or Suspension Over 6 Months	2; 0; 1; 0; 1; 0	—
SECONDARY Mean Change From Baseline in Quantitative Measure of Hand Grip Myotonia	-1.01; 0.43	<0.01 sig
SECONDARY Mean Change From Baseline in Manual Muscle Testing (MMT) Score	0.05; -0.06	—
SECONDARY Mean Change From Baseline in PR, QRS, and QTc Intervals, and Average Minimum Heart Rate (HR) Via Electrocardiogram (ECG) Monitoring	11.35; 8.89; 0.70; 1.00; -4.40; -1.55	—
SECONDARY Mean Change From Baseline in Patient-Reported Disease Burden and Quality of Life	5.11; 2.33; 0.01; -1.10; -1.73; -1.35	—

Summary

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).

Eligibility 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

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT01406873). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.