Myasthenia Graves Research Group

 
MG Research

A Randomized, Double-Blind, Placebo-Controlled, Multi-Center Study to Evaluate the Safety and Efficacy of Eculizumab in Subjects With Refractory Generalized Myasthenia Gravis

Sponsor Alexion Pharmaceuticals, Inc.
Site-PI Michael Benatar, MD, PhD
Summary Eculizumab is a monoclonal antibody that inhibits the complement pathway. A phase II trial of eculizumab in myasthenia gravis (MG) patients indicated that eculizumab may be effective in treating refractory generalized MG. The goal of this international phase III trial is to determine whether eculizumab is a safe and effective therapy for patients with generalized MG and whose symptoms have proven refractory to other treatments. Patients will be randomized to receive intravenous infusions of either eculizumab or placebo. Study visits will be performed at the University of Miami and study participation will extend over a period of about 34 weeks.
Eligibility criteria
  • Male or female ≥ 18 years old
  • Diagnosis of generalized myasthenia gravis
  • Positive serologic test for anti-acetylcholine receptor antibodies
  • Confirmatory test (e.g. acetylcholine receptor antibodies, repetitive nerve stimulation, single fiber electromyography, history of positive anticholinesterase test or improvement in MG signs on oral cholinesterase inhibitors)
  • Failed treatment with at least 2 immune suppressive therapies OR with one immune suppressive therapy and chronic plasma exchange or IVIg over the last 12 months
  • No previous treatment with eculizumab
  • No plasma exchange or IVIg in the last 4 weeks
  • No use of rituximab in the last 6 months
  • No history of thymoma
  • No history of thymectomy in the last 12 months
  • No pregnancy or breast-feeding
  • No exposure to other investigational agent/device/procedure in the last 30 days
Funding agency Alexion Pharmaceuticals, Inc.
Project manager Sara-Claude Michon
Contact information Email:
Phone: (305) 243-6481
Links http://clinicaltrials.gov/ct2/show/NCT01997229?term=eculizumab+and+myasthenia&rank=2
Presentations / Publications