Queen Square Centre for Neuromuscular Diseases


A Phase II Randomised, Double-Blind, Placebo controlled, Cross-Over Study

SponsorUniversity College London (UCL)
FunderFood and Drug Administration (FDA - USA)
CIProfessor Michael Hanna
sitesNewcastle and London GOSH
More information 


NDM is a group of rare neuromuscular disorders that causes periods of muscle stiffness (myotonias) and paralysis. NMD mainly affects the muscles of the face, hands and legs. A permanent and debilitating muscle weakness can also develop. There is no optimal treatment for these disorders.  NMDs occur due to abnormalities of ion channels in skeletal muscle walls. There is experimental evidence that drugs which block the abnormal function of these ion channels allow the muscle to function normally. Mexiletine is one such drug.

Primary objective

  • To test the efficacy of mexiletine in the treatment of NMDs.

This was a multi-centre, double-blind, placebo-controlled cross over trial lasting nine weeks.


15 patients in the UK at the MRC Centre.

This work has been completed and outcome data published in JAMA (Volume 308, No.13, pages 1357 - 1365, October 2012).

The European Commission has granted the MRC Centre Orphan Drug designation for Mexiletine for NMD.