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

NCT ID: NCT00727194

Last Updated: 2019-09-24

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

View full results

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

TERMINATED

Clinical Phase

PHASE2

Total Enrollment

14 participants

Study Classification

INTERVENTIONAL

Study Start Date

2008-10-31

Study Completion Date

2011-07-31

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

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.

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Myasthenia Gravis

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Allocation Method

RANDOMIZED

Intervention Model

CROSSOVER

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

1

eculizumab

Group Type EXPERIMENTAL

eculizumab

Intervention Type DRUG

eculizumab 600 mg IV weekly for 4 doses followed by eculizumab 900 mg IV every two weeks for 7 doses

2

Placebo

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

Placebo IV weekly for 4 doses then every two weeks for 7 doses

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

eculizumab

eculizumab 600 mg IV weekly for 4 doses followed by eculizumab 900 mg IV every two weeks for 7 doses

Intervention Type DRUG

Placebo

Placebo IV weekly for 4 doses then every two weeks for 7 doses

Intervention Type DRUG

Other Intervention Names

Discover alternative or legacy names that may be used to describe the listed interventions across different sources.

Soliris

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

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
Minimum Eligible Age

18 Years

Maximum Eligible Age

80 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

Meet the organizations funding or collaborating on the study and learn about their roles.

Alexion Pharmaceuticals, Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.

Responsibility Role SPONSOR

Locations

Explore where the study is taking place and check the recruitment status at each participating site.

University of Alabama at Birmingham

Birmingham, Alabama, United States

Site Status

University of California, Irvine

Orange, California, United States

Site Status

University of California - Davis

Sacramento, California, United States

Site Status

University of Florida & Shands Neuroscience Institute

Jacksonville, Florida, United States

Site Status

Emory University

Atlanta, Georgia, United States

Site Status

Wishard Hospital

Indianapolis, Indiana, United States

Site Status

University of Kansas Medical Center

Kansas City, Kansas, United States

Site Status

Johns Hopkins University School of Medicine

Baltimore, Maryland, United States

Site Status

Massachusetts General Hospital

Boston, Massachusetts, United States

Site Status

Caritas St. Elizabeths' Medical Center

Boston, Massachusetts, United States

Site Status

Wayne State University

Detroit, Michigan, United States

Site Status

Mount Sinai School of Medicine

New York, New York, United States

Site Status

University of North Carolina at Chapel Hill

Chapel Hill, North Carolina, United States

Site Status

Duke University Medical Center

Durham, North Carolina, United States

Site Status

University Hospitals - Case Medical Center

Cleveland, Ohio, United States

Site Status

The Ohio State University

Columbus, Ohio, United States

Site Status

The Warren Alpert Medical School of Brown University

Providence, Rhode Island, United States

Site Status

Vanderbilt University Medical Center

Nashville, Tennessee, United States

Site Status

University of Texas Southwestern Medical School

Dallas, Texas, United States

Site Status

The University of Vermont College of Medicine

Burlington, Vermont, United States

Site Status

University of Virginia

Charlottesville, Virginia, United States

Site Status

The Northern Alberta Clinical trials and Research Centre

Edmonton, Alberta, Canada

Site Status

Institute of Neurological Sciences, Department of Neurology, Southern General Hospital,

Glasgow, , United Kingdom

Site Status

Institute of Neurology

London, , United Kingdom

Site Status

Department of Clinical Neurology, West Wing, John Radcliffe Hospital

Oxford, , United Kingdom

Site Status

Countries

Review the countries where the study has at least one active or historical site.

United States Canada United Kingdom

References

Explore related publications, articles, or registry entries linked to this study.

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.

Reference Type RESULT
PMID: 23512355 (View on PubMed)

Other Identifiers

Review additional registry numbers or institutional identifiers associated with this trial.

C08-001

Identifier Type: -

Identifier Source: org_study_id

More Related Trials

Additional clinical trials that may be relevant based on similarity analysis.

Myasthenia Gravis Inebilizumab Trial
NCT04524273 ACTIVE_NOT_RECRUITING PHASE3