Safety and Efficacy Study of Eculizumab in Patients With Refractory Generalized Myasthenia Gravis - Full Text View

Sponsor:	Alexion Pharmaceuticals
Information provided by:	Alexion Pharmaceuticals
ClinicalTrials.gov Identifier:	NCT00727194

Purpose

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.

Condition	Intervention	Phase
Myasthenia Gravis	Drug: eculizumab Drug: Placebo	Phase II

Study Type:	Interventional
Study Design:	Treatment, Randomized, Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Crossover Assignment, Safety/Efficacy Study
Official Title:	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

Resource links provided by NLM:

MedlinePlus related topics: Myasthenia Gravis

Drug Information available for: Eculizumab

U.S. FDA Resources

Further study details as provided by Alexion Pharmaceuticals:

Primary Outcome Measures:

Adverse event [ Time Frame: every visit ] [ Designated as safety issue: No ]
Quantitative Myasthenia Gravis (QMG) [ Time Frame: Every visit ] [ Designated as safety issue: No ]

Secondary Outcome Measures:

Quantitative Myasthenia Gravis (QMG) [ Time Frame: every visit ] [ Designated as safety issue: No ]
MGFA Post-Intervention Status [ Time Frame: every visit ] [ Designated as safety issue: No ]
MG-Activity of Daily Living Profile (MG-ADL) [ Time Frame: every visit ] [ Designated as safety issue: No ]
SF-36 [ Time Frame: every 4 weeks ] [ Designated as safety issue: No ]
Forced vital capacity and negative inspiratory force [ Time Frame: every visit ] [ Designated as safety issue: No ]

Estimated Enrollment:	24
Study Start Date:	October 2008
Estimated Primary Completion Date:	July 2010 (Final data collection date for primary outcome measure)

Arms	Assigned Interventions
1: Experimental eculizumab	Drug: eculizumab eculizumab 600 mg IV weekly for 4 doses followed by eculizumab 900 my IV every two weeks for 7 doses
2: Placebo Comparator Placebo	Drug: Placebo Placebo IV weekly for 4 doses then every two weeks for 7 doses

Eligibility

Ages Eligible for Study:	18 Years to 80 Years
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

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

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 onic 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

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00727194

Contacts

Contact: Sharon K Wilson

203-439-9602

wilsons@alxn.com

Show 21 Study Locations

Sponsors and Collaborators

Alexion Pharmaceuticals

More Information

No publications provided

Responsible Party:	Alexion Pharmaceuticals ( Jing Jing Wang, MD. Associate Medical Director )
Study ID Numbers:	C08-001
Study First Received:	July 30, 2008
Last Updated:	February 1, 2010
ClinicalTrials.gov Identifier:	NCT00727194 History of Changes
Health Authority:	United States: Food and Drug Administration

Additional relevant MeSH terms:

Autoimmune Diseases
Immune System Diseases
Neuromuscular Diseases
Nervous System Diseases

Neuromuscular Junction Diseases
Autoimmune Diseases of the Nervous System
Myasthenia Gravis

ClinicalTrials.gov processed this record on February 08, 2010