Trial record 1 of 1 for:    NCT01997229
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Safety and Efficacy of Eculizumab in Refractory Generalized Myasthenia Gravis (REGAIN Study)

This study is ongoing, but not recruiting participants.
Information provided by (Responsible Party):
Alexion Pharmaceuticals Identifier:
First received: November 18, 2013
Last updated: September 13, 2015
Last verified: September 2015
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


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

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 V)
  • Use of rituximab within 6 months prior to screening
  • Use of IVIg or PE within 4 weeks prior to screening
  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 identifier: NCT01997229

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Sponsors and Collaborators
Alexion Pharmaceuticals
  More Information

Additional Information:
Responsible Party: Alexion Pharmaceuticals Identifier: NCT01997229     History of Changes
Other Study ID Numbers: ECU-MG-301  2013-003589-15 
Study First Received: November 18, 2013
Last Updated: September 13, 2015
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

Additional relevant MeSH terms:
Myasthenia Gravis
Autoimmune Diseases
Autoimmune Diseases of the Nervous System
Immune System Diseases
Nervous System Diseases
Neuromuscular Diseases
Neuromuscular Junction Diseases processed this record on May 25, 2016