Charcot Marie Tooth Disease Clinical Trial
Official title:
Mexiletine for Muscle Cramps in Charcot Marie Tooth Disease
Verified date | February 2018 |
Source | University of Rochester |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
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.
Status | Completed |
Enrollment | 4 |
Est. completion date | December 31, 2016 |
Est. primary completion date | December 31, 2016 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 18 Years and older |
Eligibility |
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 |
Country | Name | City | State |
---|---|---|---|
United States | University of Rochester | Rochester | New York |
Lead Sponsor | Collaborator |
---|---|
University of Rochester |
United States,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Number of Participants With a Decrease in Cramp Duration | Participant will be evaluated for muscle cramps 120 minutes following a single dose of oral mexiletine. The Clinical Evaluator applied pressure to provoke hamstring cramps bilaterally, one at a time 2 hours after dose. The cramp duration in seconds of the right hamstring was used. | 120 minutes | |
Primary | Number of Participants With a Decrease in Cramp Intensity | Participant will be evaluated for muscle cramps 120 minutes following a single dose of oral mexiletine. The Clinical Evaluator applied pressure to provoke hamstring cramps bilaterally, one at a time 2 hours after dose. The cramp intensity of the right hamstring was reported by the subject on a scale of 1-10 with 1 being weak and 10 being severe. | 120 minutes |
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