Clinical trial NCT02561702: Mexiletine for Muscle Cramps in Charcot Marie Tooth Disease
Official title: Mexiletine for Muscle Cramps in Charcot Marie Tooth Disease
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Charcot Marie Tooth Disease
Lead sponsor: University of Rochester
Sponsor class: OTHER
First posted: 2015-09-28
Enrollment: 4
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: CROSSOVER
Primary purpose: TREATMENT

Brief 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.

Detailed description: This study will provide data on the short term efficacy of oral mexiletine in helping to prevent muscle cramps in adults with CMT. The study will also assess the short-term safety and tolerability of low dose mexiletine in adults with CMT.

Eligibility 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

Primary outcomes:
measure: Number of Participants With a Decrease in Cramp Duration
timeFrame: 120 minutes
measure: Number of Participants With a Decrease in Cramp Intensity
timeFrame: 120 minutes
Secondary outcomes:
Other outcomes:
Locations:
city: Rochester
country: United States
facility: University of Rochester
state: New York
Study officials:
affiliation: University of Rochester
name: David Herrmann, MD
role: PRINCIPAL_INVESTIGATOR
References: