Multicenter Trial to Test Safety of Mexiletine in Sporadic ALS

by Amy Madsen on Mon, 2013-07-15 05:00

Enrollment is open for a phase 2 trial of a drug normally used to treat abnormal heart rhythms that showed promise in ALS mouse studies

Article Highlights:
  • Researchers are seeking approximately 60 people with sporadic amyotrophic lateral sclerosis (ALS) to participate in a phase 2 trial of mexiletine.
  • The trial, which is being conducted at 10 sites in the United States, is designed to evaluate the safety and tolerability of the drug in people with sporadic ALS (in which there is no known family history of the disease).
  • Mexiletine blocks sodium channels and is approved by the U.S. Food and Drug Administration to treat abnormal heart rhythms (arrhythmias).
  • In preclinical studies, treatment with mexiletine prolonged survival in a mouse model of ALS.

Update (June 16, 2014): This trial is now closed to new participants.

Update (Jan. 28, 2014): This story has been updated with new contact information for the Boston and St. Louis centers.

original story:

The Northeast Amyotrophic Lateral Sclerosis Consortium (NEALS) is actively seeking participants for a phase 2 clinical trial that will evaluate the safety and tolerability of mexiletine in people with the sporadic form of amyotrophic lateral sclerosis (ALS).

Approximately 90 to 95 percent of ALS is sporadic, in which there is no known family history of the disease.

Mexiletine is approved by the U.S. Food and Drug Administration for the treatment of heart rhythm abnormalities (arrhythmias). It also has been used to treat nerve damage related to diabetes (diabetic neuropathy) and prolonged muscle contraction (myotonia) associated with neuromuscular disorders. It blocks sodium channels, which are cellular pores through which sodium flows.

According to the study summary on the NEALS website, studies have shown that mexiletine prolongs survival in animal models of ALS.

Although mexiletine is available by prescription, its effects in ALS are unknown. MDA advises individuals not to use mexiletine to treat ALS until its safety and efficacy have been proven in clinical trials. (Anti-arrhythmic drugs similar to mexiletine have been reported to increase the risk of death or heart attack, and mexiletine may increase the chance of having irregular heartbeats.)

10 US trial sites

Study investigators expect to enroll approximately 60 people with sporadic ALS for the 12-week study, which will be conducted at 10 trial sites in the United States. Michael Weiss, who is at the University of Washington Medical Center and co-director of the MDA/ALS center at that institution, is the principal investigator for the trial. MDA is not funding this trial.

Participants will be randomly assigned to one of three groups. One group will receive a low dose of mexiletine; the second will receive a higher dose; and the third will receive a placebo.

For more information about the study, see A Safety and Tolerability Study of Mexiletine in Patients with Sporadic Amyotrophic Lateral Sclerosis (SALS) (MX-ALS-001) or enter NCT01849770 in the search box at

To participate

Prospective participants must:

  • be 18 years or older;
  • have a diagnosis of possible, probable or definite ALS according to El Escorial criteria; and
  • have the sporadic form of ALS (no known family history of the disease).

To participate in the study, contact the site near you, or contact Angela S. Knox at the Massachusetts General Hospital Coordination Center: (617) 724-3314 or

University of California
Neuromuscular Research Center
Los Angeles
Contact: Rebecca Alvarez at (310) 825-5232, or

University of Iowa
Iowa City, Iowa
Contact: Heena Olalde at (319) 356-8323, or

University of Kansas Medical Center
Kansas City
Contact: Maureen Walsh at (913) 588-0645, or

Massachusetts General Hospital
Contact: Ashley Fetterman at (617) 643-7290 or

University of Massachusetts, Worcester
Memorial Medical Center
Worcester, Mass.
Contact: Diane McKenna-Yasek at (508) 856-4697, or

Washington University Medical School
St. Louis, Mo.
Contact: Julaine Florence at (314) 362-6983 or

SUNY Upstate Medical Center
Syracuse, N.Y.
Contact: Jennifer Moore at (315) 464-4619, or

Penn State Hershey Medical Center
Hershey, Pa.
Contact: Beth Stephens at (717) 531-0003 x283395, or

University of Texas Southwestern Medical Center at Dallas
Contact: Nina Gorham at (214) 648-0462, or

University of Washington Medical Center
Contact: Sharon Downing at (206) 543-0081, or


About Clinical Trials

About Clinical Trials

A clinical trial is a test, in humans, of an experimental treatment. Although it's possible that benefit may be derived from participating in a clinical trial, it's also possible that no benefit, or even harm, may occur.

MDA has no ability to influence who is chosen to participate in a clinical trial.

To learn more about clinical trials, read Being a Co-Adventurer. For a more refined list of ALS clinical trials, visit, a registry of federally and privately supported clinical trials in the United States and around the world. Select the "Find Studies" tab, and follow the instructions to narrow down your search results.

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