Safety and Efficacy Study of Eculizumab in Patients With Refractory Generalized Myasthenia Gravis
A Randomized, Double-Blind, Placebo-Controlled, Cross-Over, Multi-Center Study of Eculizumab in Patients With Generalized Myasthenia Gravis (gMG) Who Have Moderate to Severe Muscle Weakness Despite Treatment With Immunosuppressants
1 other identifier
interventional
14
3 countries
25
Brief 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 Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_2
Started Oct 2008
25 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
July 30, 2008
CompletedFirst Posted
Study publicly available on registry
August 1, 2008
CompletedStudy Start
First participant enrolled
October 1, 2008
CompletedPrimary Completion
Last participant's last visit for primary outcome
March 1, 2011
CompletedStudy Completion
Last participant's last visit for all outcomes
July 1, 2011
CompletedResults Posted
Study results publicly available
December 12, 2016
CompletedSeptember 24, 2019
September 1, 2019
2.4 years
July 30, 2008
March 22, 2016
September 13, 2019
Conditions
Outcome Measures
Primary Outcomes (1)
Quantitative Myasthenia Gravis (QMG): The Primary Efficacy Endpoint in This Study Was the Percentage of Patients With a 3-point Reduction From Baseline in the QMG Total Score for Disease Severity.
The QMG scoring system is considered to be an objective evaluation of muscle strength based on quantitative testing of sentinel muscle groups. The MGFA task force has recommended that the QMG score be used in prospective studies of therapy for MG.
16 weeks
Secondary Outcomes (8)
Mean Change From Baseline in QMG Total Score
16 weeks
Change From Baseline in the MGFA Post-Intervention Status (PIS)
16 weeks
Change From Baseline in the MG-Activity of Daily Living Profile (MG-ADL)
16 weeks
Change From Baseline in the QoL Instrument, SF-36.
16 weeks
Change From Baseline in Respiratory Function Tests to Characterize the Degree of Involvement of Respiratory Muscles.
16 weeks
- +3 more secondary outcomes
Study Arms (2)
1
EXPERIMENTALeculizumab
2
PLACEBO COMPARATORPlacebo
Interventions
eculizumab 600 mg IV weekly for 4 doses followed by eculizumab 900 mg IV every two weeks for 7 doses
Eligibility Criteria
You may qualify if:
- 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.
You may not qualify if:
- 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (25)
University of Alabama at Birmingham
Birmingham, Alabama, 35233, United States
University of California, Irvine
Orange, California, 92868, United States
University of California - Davis
Sacramento, California, 95817, United States
University of Florida & Shands Neuroscience Institute
Jacksonville, Florida, 32209, United States
Emory University
Atlanta, Georgia, 30322, United States
Wishard Hospital
Indianapolis, Indiana, 46202, United States
University of Kansas Medical Center
Kansas City, Kansas, 66160, United States
Johns Hopkins University School of Medicine
Baltimore, Maryland, 21287, United States
Massachusetts General Hospital
Boston, Massachusetts, 02114, United States
Caritas St. Elizabeths' Medical Center
Boston, Massachusetts, 02135, United States
Wayne State University
Detroit, Michigan, 48201, United States
Mount Sinai School of Medicine
New York, New York, 10029-6574, United States
University of North Carolina at Chapel Hill
Chapel Hill, North Carolina, 27599-1651, United States
Duke University Medical Center
Durham, North Carolina, 27710, United States
University Hospitals - Case Medical Center
Cleveland, Ohio, 44106-5098, United States
The Ohio State University
Columbus, Ohio, 43210, United States
The Warren Alpert Medical School of Brown University
Providence, Rhode Island, 02905, United States
Vanderbilt University Medical Center
Nashville, Tennessee, 37232, United States
University of Texas Southwestern Medical School
Dallas, Texas, 75390, United States
The University of Vermont College of Medicine
Burlington, Vermont, 05405, United States
University of Virginia
Charlottesville, Virginia, 22908-0394, United States
The Northern Alberta Clinical trials and Research Centre
Edmonton, Alberta, Canada
Institute of Neurological Sciences, Department of Neurology, Southern General Hospital,
Glasgow, United Kingdom
Institute of Neurology
London, United Kingdom
Department of Clinical Neurology, West Wing, John Radcliffe Hospital
Oxford, United Kingdom
Related Publications (1)
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 Jul;48(1):76-84. doi: 10.1002/mus.23839. Epub 2013 Apr 30.
PMID: 23512355RESULT
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Limitations and Caveats
Small sample size limited ability to detect intercohort differences. Carryover effect from Treatment Period (TP) 1 warrants cautious interpretation of TP 2 data. Study terminated early; 13 patients received eculizumab or placebo in TP2.
Results Point of Contact
- Title
- Alexion Pharmaceuticals
- Organization
- Alexion Pharmaceuticals
Publication Agreements
- PI is Sponsor Employee
- No
- Restrictive Agreement
- No
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- RANDOMIZED
- Masking
- QUADRUPLE
- Who Masked
- PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
- Purpose
- TREATMENT
- Intervention Model
- CROSSOVER
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
July 30, 2008
First Posted
August 1, 2008
Study Start
October 1, 2008
Primary Completion
March 1, 2011
Study Completion
July 1, 2011
Last Updated
September 24, 2019
Results First Posted
December 12, 2016
Record last verified: 2019-09