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Safety and Efficacy of Eculizumab in Refractory Generalized Myasthenia Gravis (REGAIN Study)

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Alexion Pharmaceuticals
ClinicalTrials.gov Identifier:
NCT01997229
First received: November 18, 2013
Last updated: March 1, 2017
Last verified: March 2017
  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
Intervention Model: Parallel Assignment
Masking: Participant, Care Provider, 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) ]
    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) ]
    Adverse events, vital signs, laboratory assessments, Physical Exams, and ECGs.


Enrollment: 125
Study Start Date: December 2013
Study Completion Date: June 2016
Primary Completion Date: February 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 during Weeks 4-26
Biological: Eculizumab
Induction Phase: 900 mg IV weekly X 4 Maintenance Phase: 1200 mg IV every 2 weeks during Weeks 4-26
Placebo Comparator: Placebo
Placebo contains the same buffer components without the active ingredient
Drug: Placebo
Placebo

  Eligibility

Ages Eligible for Study:   18 Years and older   (Adult, Senior)
Sexes Eligible for Study:   All
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 and Randomization (Day 1).
  • 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 V)
  • Use of rituximab within 6 months prior to screening
  • Use of IVIg or PE within 4 weeks prior to Randomization (Day 1)
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01997229

  Show 114 Study Locations
Sponsors and Collaborators
Alexion Pharmaceuticals
  More Information

Additional Information:
Publications:
Responsible Party: Alexion Pharmaceuticals
ClinicalTrials.gov Identifier: NCT01997229     History of Changes
Other Study ID Numbers: ECU-MG-301
2013-003589-15 ( EudraCT Number )
Study First Received: November 18, 2013
Last Updated: March 1, 2017
Individual Participant Data  
Plan to Share IPD: No

Keywords provided by Alexion Pharmaceuticals:
Myasthenia Gravis
Eculizumab
safety
efficacy

Additional relevant MeSH terms:
Myasthenia Gravis
Muscle Weakness
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Manifestations
Neurologic Manifestations
Nervous System Diseases
Pathologic Processes
Signs and Symptoms
Autoimmune Diseases of the Nervous System
Neuromuscular Junction Diseases
Neuromuscular Diseases
Autoimmune Diseases
Immune System Diseases

ClinicalTrials.gov processed this record on April 28, 2017