A Safety and Tolerability Study of Mexiletine in Patients with Sporadic ALS (SALS)
Study Purpose:
The purpose of this study is to determine if mexiletine is safe and tolerable in volunteers with sporadic ALS (SALS).Disease:
Amyotrophic Lateral Sclerosis (ALS), Sporadic ALSStudy Type:
Interventional TrialStudy Category:
Drug TrialStudy Status:
Not enrollingPhase:
Phase IIStudy Chair(s)/Principal Investigator(s):
Michael Weiss, MD (University of Washington School of Medicine, Seattle, WA)Merit Cudkowicz, MD, MSc (Massachusetts General Hospital)
Clinicaltrials.gov ID (11 digit #):
NCT01849770Neals Affiliated?
YesCoordinating Center Contact Information
Mass GeneralDaniela Grasso / .(JavaScript must be enabled to view this email address) / 617-726-0842
.(JavaScript must be enabled to view this email address) 165 Cambridge St.
Boston, Massachusetts 02109 United States
Full Study Summary:
Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disorder affecting primarily motor neurons, for which treatment designed to slow or arrest progression remains lacking. Mexiletine is a use-dependent sodium channel blocker that has been FDA-approved for decades for the treatment of cardiac arrhythmias and more recently to treat neuropathic pain in diabetic polyneuropathy. Mexiletine has been shown to be protective of neurons following spinal cord, head injury, and cerebral ischemia, largely by blocking excitotoxicity. Recent unpublished studies have shown that mexiletine prolongs survival in animal models of ALS. Since mexiletine has not been rigorously evaluated in ALS populations, a clinical trial has been designed to scientifically determine the safety and tolerability, as well as the effect of mexiletine on ALS patients.Approximately 60 eligible subjects with sporadic ALS will be recruited from multiple centers in the US. Subjects will be treated for 12 weeks with either mexiletine (300 mg/day or 900 mg/day) or placebo. A follow-up telephone call will occur approximately 4 weeks after completion of the study.