Phase 2
N=4
Mexiletine for Muscle Cramps in Charcot Marie Tooth Disease
Charcot Marie Tooth Disease
Bottom Line
View on ClinicalTrials.gov: NCT02561702 ↗Enrolled (actual)
4
Serious AEs
0.0%
Results posted
Mar 2018
Primary outcome: Primary: Number of Participants With a Decrease in Cramp Duration — 2; 0 participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Placebo (Other); Mexiletine (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- University of Rochester
- Primary completion
- Dec 2016
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants With a Decrease in Cramp Duration |
2; 0 | — |
| PRIMARY Number of Participants With a Decrease in Cramp Intensity |
1; 2 | — |
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.
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
Data sourced from ClinicalTrials.gov (NCT02561702). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.