Queen Square Centre for Neuromuscular Diseases


A Study of Eculizumab in Patients with Generalised Myasthenia Gravis (MG) who have Moderate to Severe Muscle Weakness despite Treatment with Immunosupressants

Sponsor / Funder Alexion Pharmaceuticals, Inc.
CI Dimitri Kullmann
UK sites London Queen square
More information

The National Institute for Neurological Disorders and Stroke has a wealth of resources related to Mayasthenia Gravis.


MG is an acquired autoimmune syndrome caused by faults in the transmission of nerve impulses to muscles. This results from the binding of autoantibodies to signalling proteins at the neuromuscular junction (NMJ)*.

*The NMJ is the place where nerve cells connect with the muscles they control.

Current available treatments for MG aim to modulate neuromuscular transmission to:

  • inhibit the production or effects of abnormal antibodies
  • inhibit inflammatory cytokines.

There is no specific treatment that corrects the autoimmune defect in MG.

Trial information

This was a randomised, double-blind, placebo-controlled, cross-over, multicentre study testing an antibody: Eculizumab.

Primary objective

to evaluate the safety and efficacy of Eculizumab for the treatment of patients with MG. 

Patients received 22 infusions including 11 of the drug and 11 of placebo. Patients participated in the study for approximately 41 weeks.