Conditions: Charcot Marie Tooth Disease
Allocation: RANDOMIZED | Intervention model: CROSSOVER
Enrollment: 4
Healthy volunteers: no

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.

Arms:
arm 1: 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 2: 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

Interventions: intervention 1: Placebo intervention 2: Mexiletine
Intervention descriptions: intervention 1: 240 mg lactose powder in a size 3 capsule taken by mouth 3 times daily for 5-7 days intervention 2: 150 mg Mexiletine taken by mouth in capsule form 3 times daily for 5-7 days

Protocol document: