Mexiletine for Muscle Cramps in Charcot Marie Tooth Disease

• Status: Completed
• Trial end date: 2016-12-31
• Target enrollment: 0
• Participant gender: All

Summary

Charcot Marie Tooth Disease is a family of inherited peripheral neuropathies, with over 70 causative genes identified to date.1-4 Muscle cramps are frequent in CMT, affecting up to 85% of patients with some subtypes of CMT. These cramps impact quality of life and have been identified as an important therapeutic target for clinical trials in CMT.1-4 There is no FDA approved treatment for muscle cramps.5 Mexiletine is a sodium channel blocker approved for treatment of arrhythmias. As a sodium channel blocker, mexiletine offers the promise of effective therapy for muscle cramps.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: University of Rochester
• Treatments: Mexiletine

Criteria

Inclusion Criteria:

• The subject has known CMT, previously diagnosed by a neurologist with subspeciality expertise in neuromuscular disorders/peripheral neuropathy. The diagnosis of CMT relies on a combination of clinical history (including family history), neurological examination electrophysiological features, and prior genetic testing results.

• The subject is at least 18 years old, and has signed the Informed Consent Form.

• The subject is ambulatory (cane, walker, orthoses allowed).

• The subject has experienced muscle cramps and has known provokable hamstring or calf muscle cramps on MVC.

• The subject has a reliable method of birth control (if a female subject of child bearing potential). Reliable birth control is defined as Hormonal contraception Intrauterine contraception/device Any two barrier methods (combination of male or female condom with diaphragm sponge or cervical cap) together with spermicidal foam/gel/film/cream/suppository True abstinence

Exclusion Criteria:

• The subject has a known neuropathy from another source (e.g., diabetes, drug induced, alcohol, etc.).

• The subject has an untreated medical disorder known to predispose to muscle cramps

• The subject is pregnant or nursing, has a known mexiletine allergy or has taken mexiletine in the past 12 weeks.

• The subject is participating in another therapeutic trial.

• The subject has second or 3rd degree heart block, atrial flutter/fibrillation, ventricular arrhythmias, or is receiving treatment of a cardiac arrhythmia.

• The subject is currently taking another agent for muscle cramps or a muscle relaxant (e.g., benzodiazepine, baclofen, tizanidine, soma, meprobromate).

• The subject is on another sodium channel blocker or medication that precludes administration of mexiletine.

• The subject has known diabetes mellitus, liver or kidney disease requiring ongoing treatment, untreated thyroid dysfunction, symptomatic cardiomyopathy, or symptomatic coronary artery disease.

• The subject, in the opinion of the investigator, is unsuitable for enrollment for any other reason