Mexiletine in Myotonic Dystrophy Type 1: A Randomized, Double-Blind, Placebo-Controlled Trial.

ABSTRACT:

Objective

To assess mexiletine's long-term safety and effect on 6-minute walk distance in a well-defined cohort of patients with myotonic dystrophy type 1 (DM1).

Methods

We performed a randomized, double-blind, placebo-controlled trial of mexiletine (150 mg 3 times daily) to evaluate its efficacy and safety in a homogenous cohort of adult ambulatory patients with DM1. The primary outcome was change in 6-minute walk distance at 6 months. Secondary outcomes included changes in hand grip myotonia, strength, swallowing, forced vital capacity, lean muscle mass, Myotonic Dystrophy Health Index scores, and 24-hour Holter and ECG results at 3 and 6 months.

Results

Forty-two participants were randomized and 40 completed the 6-month follow-up (n = 20 in both groups). No signifi

SUBMITTER: Heatwole C 

PROVIDER: S-EPMC7905778 | biostudies-literature | 2021 Jan

REPOSITORIES: biostudies-literature