Mexiletine in Non-Dystrophic Myotonia

  • Research type

    Research Study

  • Full title

    A Phase II Randomised, Double-Blind, Placebo-Controlled, Cross-Over Study to Investigate the Efficacy of Mexiletine in Patients with Non-Dystrophic Myotonia.

  • IRAS ID

    9648

  • Sponsor organisation

    UCL - University College London

  • Eudract number

    2009-011184-36

  • Clinicaltrials.gov Identifier

    NCT00832000

  • Research summary

    This study aims to test the efficacy of the drug mexiletine in the treatment of the non-dystrophic myotonias. 15 participants will be enrolled in the UK at the National Hospital for Neurology and Neurosurgery.The non-dystrophic myotonias are a group of rare neuromuscular disorders that cause episodes of muscle stiffness (known as myotonia) and paralysis. Predominantly the muscles of the face, hands and legs are affected. In addition to these episodes a permanent and debilitating muscle weakness can develop. The optimal treatment for these disorders is unknown.The non-dystrophic myotonias are due to abnormalities of ion channels present in skeletal muscle membranes. There is experimental evidence that drugs like mexiletine which block the abnormal function of these ion channels allow the muscle to perform normally. In addition there are case reports of individuals or small numbers of patients which show benefit from taking mexiletine. Mexiletine is also used in the treatment of abnormal heart rhythms and from this experience it is well tolerated by patients.Participants will be randomised to receive either mexiletine or a placebo for a four week treatment block followed by a one week washout period and then cross-over to receive the other treatment for a second four week treatment block. Both investigators and the patient will be "blinded" to which drug is given.During the study the patients will have four daily visits to the National Hospital for Neurology and Neurosurgery. During these visits they will undergo a number of clinical measures to assess the efficacy of mexiletine. These will include 2 quality of life questionnaires. Patients will also be asked to keep a daily telephone diary of their symptoms.A study obeying an identical protocol will run simultaneously at 5 international sites.

  • REC name

    London - Harrow Research Ethics Committee

  • REC reference

    09/H0714/21

  • Date of REC Opinion

    6 Apr 2009

  • REC opinion

    Favourable Opinion