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

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Alexion Pharmaceuticals
ClinicalTrials.gov Identifier:
NCT00727194
First received: July 30, 2008
Last updated: November 17, 2011
Last verified: July 2010
  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 2

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Crossover Assignment
Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
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:


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 ]

Enrollment: 14
Study Start Date: October 2008
Study Completion Date: July 2011
Primary Completion Date: March 2011 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: 1
eculizumab
Drug: eculizumab
eculizumab 600 mg IV weekly for 4 doses followed by eculizumab 900 my IV every two weeks for 7 doses
Other Name: Soliris
Placebo Comparator: 2
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

  Show 25 Study Locations
Sponsors and Collaborators
Alexion Pharmaceuticals
  More Information

No publications provided

Responsible Party: Alexion Pharmaceuticals
ClinicalTrials.gov Identifier: NCT00727194     History of Changes
Other Study ID Numbers: C08-001
Study First Received: July 30, 2008
Last Updated: November 17, 2011
Health Authority: United States: Food and Drug Administration

Additional relevant MeSH terms:
Myasthenia Gravis
Muscle Weakness
Autoimmune Diseases of the Nervous System
Nervous System Diseases
Neuromuscular Junction Diseases
Neuromuscular Diseases
Autoimmune Diseases
Immune System Diseases
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Manifestations
Neurologic Manifestations
Pathologic Processes
Signs and Symptoms
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Pharmacologic Actions

ClinicalTrials.gov processed this record on April 16, 2014