Safety and Efficacy of Eculizumab in Refractory Generalized Myasthenia Gravis

This study is currently recruiting participants.
Verified March 2014 by Alexion Pharmaceuticals
Sponsor:
Information provided by (Responsible Party):
Alexion Pharmaceuticals
ClinicalTrials.gov Identifier:
NCT01997229
First received: November 18, 2013
Last updated: March 20, 2014
Last verified: March 2014
  Purpose

The purpose of this study is to determine if eculizumab is safe and effective for the treatment of refractory generalized Myasthenia Gravis.


Condition Intervention Phase
Refractory Generalized Myasthenia Gravis
Biological: Eculizumab
Drug: Placebo
Phase 3

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Randomized, Double-Blind, Placebo-Controlled, Multi-Center Study to Evaluate the Safety and Efficacy of Eculizumab in Subjects With Refractory Generalized Myasthenia Gravis (gMG)

Resource links provided by NLM:


Further study details as provided by Alexion Pharmaceuticals:

Primary Outcome Measures:
  • change in total MG-ADL score [ Time Frame: End of study (Week 26/ Visit 17) ] [ Designated as safety issue: No ]
    change from baseline in MG-ADL total score @Week 26 for eculizumab as compared to placebo.


Secondary Outcome Measures:
  • Overall safety and tolerability of eculizumab [ Time Frame: end of study (week 26) ] [ Designated as safety issue: Yes ]
    Adverse events, vital signs, laboratory assessments, Physical Exams, and ECGs.


Estimated Enrollment: 92
Study Start Date: December 2013
Estimated Study Completion Date: February 2016
Estimated Primary Completion Date: January 2016 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Eculizumab
Biological/Vaccine: Eculizumab Induction Phase: 900 mg IV weekly X 4 Maintenance Phase: 1200 mg IV every 2 weeks 5-26
Biological: Eculizumab
Induction Phase: 900 mg IV weekly X 4 Maintenance Phase: 1200 mg IV every 2 weeks 5-26
Placebo Comparator: Placebo
Placebo contains the same buffer components without the active ingredient
Drug: Placebo
Placebo
Other Name: Placebo

  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Key Inclusion Criteria:

  • Male or female patients ≥18 years old
  • Diagnosis of MG made by the following tests:

    1. Positive serologic test for anti-AChR Abs as confirmed at screening, and
    2. One of the following:

      1. History of abnormal neuromuscular transmission test demonstrated by single-fiber electromyography (SFEMG) or repetitive nerve stimulation, or
      2. History of positive anticholinesterase test, e.g. edrophonium chloride test, or
      3. Subject has demonstrated improvement in MG signs on oral cholinesterase inhibitors, as assessed by the treating physician.
  • MGFA Clinical Classification Class II to IV at screening.
  • MG-ADL total score must be ≥6 at screening
  • Subjects who have:

    1. Failed treatment with at least two immunosuppressive agents. Or,
    2. Failed treatment with at least one immunosuppressive agent and require chronic plasma exchange or IVIg

Key Exclusion Criteria:

  • History of thymoma or other neoplasms of the thymus
  • History of thymectomy within 12 months prior to screening
  • MGFA Class I or MG crisis at screening (MGFA Class IV)
  • Use of rituximab within 6 months prior to screening
  • Use of IVIg or PE within 4 weeks prior to screening
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01997229

Contacts
Contact: Alexion Pharmaceuticals (Sponsor) 203 699-7839 clinicaltrials@alxn.com

Locations
United States, Florida
University of Florida Health Science Center Recruiting
Jacksonville, Florida, United States, 32209
Contact: Alexion pharmaceuticals, Inc       clinicaltrials@alxn.com   
United States, Kansas
University of Kansas Medical Center Recruiting
Kansas City, Kansas, United States, 66160
Contact: Alexion Pharmaceuticals Inc       clinicaltrials@alxn.com   
United States, Ohio
Ohio State University Medical Center Recruiting
Columbus, Ohio, United States, 43221
Contact: Alexion Pharmaceuticals Inc       clinicaltrials@alxn.com   
Sponsors and Collaborators
Alexion Pharmaceuticals
  More Information

Publications:
Responsible Party: Alexion Pharmaceuticals
ClinicalTrials.gov Identifier: NCT01997229     History of Changes
Other Study ID Numbers: ECU-MG-301, 2013-003589-15
Study First Received: November 18, 2013
Last Updated: March 20, 2014
Health Authority: United States: Food and Drug Administration
Argentina: Administracion Nacional de Medicamentos, Alimentos y Tecnologia Medica
Australia: Department of Health and Ageing Therapeutic Goods Administration
Brazil: National Health Surveillance Agency
Belgium: Federal Agency for Medicines and Health Products, FAMHP
Canada: Health Canada
Denmark: Danish Health and Medicines Authority
Finland: Finnish Medicines Agency
France: Agence Nationale de Sécurité du Médicament et des produits de santé
Germany: Paul-Ehrlich-Institut
Greece: National Organization of Medicines
Japan: Pharmaceuticals and Medical Devices Agency
Italy: The Italian Medicines Agency
Netherlands: The Central Committee on Research Involving Human Subjects (CCMO)
Spain: Agencia Española de Medicamentos y Productos Sanitarios
Sweden: Medical Products Agency
Turkey: Turkish Pharmaceuticals and Medical Devices Institution
United Kingdom: Medicines and Healthcare Products Regulatory Agency

Keywords provided by Alexion Pharmaceuticals:
Myasthenia Gravis
Eculizumab
safety
efficacy

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

ClinicalTrials.gov processed this record on April 22, 2014