Back to resultsMexiletine for Muscle Cramps in Charcot Marie Tooth Disease
Primary Purpose
Charcot Marie Tooth Disease
Status
Completed
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
Placebo
Mexiletine
Sponsored by
About this trial
This is an interventional treatment trial for Charcot Marie Tooth Disease
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
Sites / Locations
- University of Rochester
Arms of the Study
Arm 1
Arm 2
Arm Type
Experimental
Experimental
Arm Label
Mexiletine first/placebo second
placebo first/mexiletine second
Arm Description
Participants will receive 150 mg of mexiletine by mouth 3 times daily for 5-7 days followed by 7 day washout period and 5-7 days of placebo (240 mg of lactose powder) taken by mouth 3 times daily
Participants will receive placebo (240 mg of lactose powder) taken by mouth 3 times daily for 5-7 days followed by 7 day washout period and 5-7 days of 150 mg of mexiletine by mouth 3 times daily
Outcomes
Primary Outcome Measures
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.
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.
Secondary Outcome Measures
Full Information
NCT ID
NCT02561702
First Posted
September 24, 2015
Last Updated
February 23, 2018
Sponsor
University of Rochester
1. Study Identification
Unique Protocol Identification Number
NCT02561702
Brief Title
Mexiletine for Muscle Cramps in Charcot Marie Tooth Disease
Official Title
Mexiletine for Muscle Cramps in Charcot Marie Tooth Disease
Study Type
Interventional
2. Study Status
Record Verification Date
February 2018
Overall Recruitment Status
Completed
Study Start Date
September 2015 (undefined)
Primary Completion Date
December 31, 2016 (Actual)
Study Completion Date
December 31, 2016 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
University of Rochester
4. Oversight
Data Monitoring Committee
No
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Charcot Marie Tooth Disease
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Crossover Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
4 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Mexiletine first/placebo second
Arm Type
Experimental
Arm Description
Participants will receive 150 mg of mexiletine by mouth 3 times daily for 5-7 days followed by 7 day washout period and 5-7 days of placebo (240 mg of lactose powder) taken by mouth 3 times daily
Arm Title
placebo first/mexiletine second
Arm Type
Experimental
Arm Description
Participants will receive placebo (240 mg of lactose powder) taken by mouth 3 times daily for 5-7 days followed by 7 day washout period and 5-7 days of 150 mg of mexiletine by mouth 3 times daily
Intervention Type
Other
Intervention Name(s)
Placebo
Other Intervention Name(s)
lactose powder
Intervention Description
240 mg lactose powder in a size 3 capsule taken by mouth 3 times daily for 5-7 days
Intervention Type
Drug
Intervention Name(s)
Mexiletine
Intervention Description
150 mg Mexiletine taken by mouth in capsule form 3 times daily for 5-7 days
Primary Outcome Measure Information:
Title
Number of Participants With a Decrease in Cramp Duration
Description
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.
Time Frame
120 minutes
Title
Number of Participants With a Decrease in Cramp Intensity
Description
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.
Time Frame
120 minutes
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
David Herrmann, MD
Organizational Affiliation
University of Rochester
Official's Role
Principal Investigator
Facility Information:
Facility Name
University of Rochester
City
Rochester
State/Province
New York
ZIP/Postal Code
14642
Country
United States
12. IPD Sharing Statement
Learn more about this trial
Mexiletine for Muscle Cramps in Charcot Marie Tooth Disease
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