Myasthenia Graves Research Group


MG Research

Safety And Efficacy Study Of Eculizumab In Patients With Refractory Generalized Myasthenia Gravis (Eculizumab)

Sponsor Alexion Pharmaceuticals, Inc
Site-PI Michael Benatar, MD, PhD
Summary The purpose of this study is to determine whether eculizumab is safe and effective in the treatment of patients with generalized myasthenia gravis despite treatment with various immune suppressive agents. Eculizumab is a monoclonal antibody that inhibits the complement pathway. It has previously been shown to be effective in treating patients with paroxysmal nocturnal hemoglobinuria.
Eligibility criteria
  • Generalized myasthenia gravis, onset within last 10 years
  • Positive acetylcholine receptor binding antibodies
  • Age 18-80
  • MGFA class II-IVa
  • QMG Score ≥ 12 (with minimum score of 2 in at least 4 items)
  • Failed at least two immune suppressive agents after one year of treatment
  • No thymoma
  • No current or chronic use of plasma exchange
  • No IVIg within 8 weeks prior to screening
Funding agency Alexion Pharmaceuticals
Project manager
Contact information
Presentations / Publications
  • Howard JF Jr, Barohn RJ, Cutter GR, Freimer M, Juel VC, Mozaffar T, Mellion ML, Benatar MG, Farrugia ME, Wang JJ, Malhotra SS, Kissel JT; MG Study Group. A randomized, double-blind, placebo-controlled phase II study of eculizumab in patients with refractory generalized myasthenia gravis. Muscle Nerve. 2013;48(1):76-84.