Safety and Efficacy Study of Eculizumab in Patients With Refractory Generalized Myasthenia Gravis

Study Identifier:
C08-001
ClinicalTrials.gov Identifier:
NCT00727194
EudraCT Identifier:
N/A
EU CT ID:
N/A
Sponsor:
Alexion Pharmaceuticals, Inc.
Study Contact Information:
N/A
Other

Study Details

Medical Condition
  • Myasthenia Gravis
Study Drug
  • Drug: eculizumab
  • Drug: Placebo
Date
Oct 2008 - Mar 2011
Phase 1
Phase 2
Phase 3
Phase 4
N/A
Patient Requirements
Sex: Female & Male
Age: 18 - 80 Years
Requirements Information
Inclusion and Exclusion Criteria
Inclusion Criteria
  • Generalized MG
  • MGFA Clinical Classification Class II, III or IVa.
  • QMG total score ≥12
  • Minimum score of two (2) in four (4) or more test items in the QMG
  • Able to give informed consent.
  • Have failed at least two immunosuppressants after one year of treatment
  • A positive serologic test for binding anti-acetylcholine receptor Abs at Screening and one of the following a) history of abnormal neuromuscular transmission test demonstrated by single-fiber electromyography or repetitive nerve stimulation, or b) history of positive anticholinesterase test, eg, edrophonium chloride test, or c) patient has demonstrated improvement in MG signs on acetylcholinesterase inhibitors as assessed by treating physician.
Exclusion Criteria
  • History of thymoma or other neoplasms of the thymus.
  • History of thymectomy within 12 months prior to screening.
  • Pregnancy or lactation
  • Current or chronic use of plasmapheresis/plasma exchange
  • IVIG treatment within 8 weeks prior to screening.
  • Use of etanercept within 2 months prior to screening.
  • Use of rituximab (RITUXAN®) within 6 months prior to screening.
  • MGFA Class I, IVb, and V
  • Crisis or impending crisis
Sex
Female & Male
Age
18 - 80 Years

Study Details

Medical Condition
  • Myasthenia Gravis
Study Drug
  • Drug: eculizumab
  • Drug: Placebo
Date
Oct 2008 - Mar 2011
Phase 1
Phase 2
Phase 3
Phase 4
N/A
Patient Requirements
Sex: Female & Male
Age: 18 - 80 Years years
Requirements Information
Inclusion and Exclusion Criteria
Inclusion Criteria
  • Generalized MG
  • MGFA Clinical Classification Class II, III or IVa.
  • QMG total score ≥12
  • Minimum score of two (2) in four (4) or more test items in the QMG
  • Able to give informed consent.
  • Have failed at least two immunosuppressants after one year of treatment
  • A positive serologic test for binding anti-acetylcholine receptor Abs at Screening and one of the following a) history of abnormal neuromuscular transmission test demonstrated by single-fiber electromyography or repetitive nerve stimulation, or b) history of positive anticholinesterase test, eg, edrophonium chloride test, or c) patient has demonstrated improvement in MG signs on acetylcholinesterase inhibitors as assessed by treating physician.
Exclusion Criteria
  • History of thymoma or other neoplasms of the thymus.
  • History of thymectomy within 12 months prior to screening.
  • Pregnancy or lactation
  • Current or chronic use of plasmapheresis/plasma exchange
  • IVIG treatment within 8 weeks prior to screening.
  • Use of etanercept within 2 months prior to screening.
  • Use of rituximab (RITUXAN®) within 6 months prior to screening.
  • MGFA Class I, IVb, and V
  • Crisis or impending crisis

Protocol 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 immunosuppressants, such as prednisone, methotrexate, Cellcept, cyclosporine, and cyclophosphamide, that are currently available.

Trial Locations

Location
Status
Location
University of Alabama at Birmingham
Birmingham, Alabama, United States, 35233
Status
N/A
Location
University of California, Irvine
Orange, California, United States, 92868
Status
N/A
Location
University of California - Davis
Sacramento, California, United States, 95817
Status
N/A
Location
University of Florida & Shands Neuroscience Institute
Jacksonville, Florida, United States, 32209
Status
N/A
Location
Emory University
Atlanta, Georgia, United States, 30322
Status
N/A
Location
Wishard Hospital
Indianapolis, Indiana, United States, 46202
Status
N/A
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