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

July 12, 2019 updated by: Alexion Pharmaceuticals

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)

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

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

125

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Argentina
    • Buenos Aires
      • Ciudad Autonoma, Buenos Aires, Argentina, 1181
        • Hospital Italiano
      • Ciudad Autonoma, Buenos Aires, Argentina, C1428AQK
        • Instituto de Investigaciones Neurologicas Raul Carrea, FLENI
  • Australia
    • Victoria
      • Fitzroy, Victoria, Australia, 3065
        • St Vincent's Hospital Melbourne
    • Western Australia
      • Perth, Western Australia, Australia, 6000
        • Royal Perth Hospital
  • Belgium
      • Edegem, Belgium, 2650
        • UZ Antwerpen
      • Gent, Belgium, 9000
        • AZ Sint-Lucas - Campus Sint-Lucas
      • Leuven, Belgium, 3000
        • UZ Leuven
  • Brazil
      • Rio De Janeiro, Brazil, 21941-913
        • HUCFF-UFRJ - Hospital Universitario Clementino Fraga Filho - Universidade Federal do Rio de Janeiro
      • São Paulo, Brazil, 04037-002
        • UNIFESP - Universidade Federal de São Paulo
    • Rio Grande Do Sul
      • Porto Alegre, Rio Grande Do Sul, Brazil, 90110-270
        • Hospital Mãe de Deus
    • Sao Paulo
      • Santo André, Sao Paulo, Brazil, Postal Code: 09060-650
        • Faculdade de Medicina do ABC
      • São José do Rio Preto, Sao Paulo, Brazil, 15090-000
        • Fundacao Faculdade Regional de Medicina de Sao Jose Do Rio Preto
  • Canada
    • Alberta
      • Edmonton, Alberta, Canada, T6G 2B7
        • University of Alberta Hospital
    • Ontario
      • Toronto, Ontario, Canada, M5G 2C4
        • Toronto General Hospital
    • Quebec
      • Montreal, Quebec, Canada, H3A 2B4
        • Montreal Neurological Institute
  • Czechia
      • Brno, Czechia, 625 00
        • Fakultni Nemocnice Brno
      • Ostrava - Poruba, Czechia, 708 52
        • Fakultni Nemocnice Ostrava
    • Prague
      • Praha, Prague, Czechia, 120 00
        • Vseobecna fakultni nemocnice v Praze
  • Denmark
      • Aarhus C, Denmark, 8000
        • Århus Universitetshospital
      • København Ø, Denmark, 2100
        • Rigshospitalet
  • Finland
      • Helsinki, Finland, 00290
        • Helsingin Yliopistollinen Keskussairaala
      • Turku, Finland, 20520
        • Neuro NEO Oy
  • France
    • Alpes Maritimes
      • Nice Cedex 3, Alpes Maritimes, France, 06202
        • CHU de Nice Hôpital Pasteur 2
    • Gironde
      • Bordeaux Cedex, Gironde, France, 33000
        • Groupe Hospitalier Pellegrin - Hôpital Pellegrin
    • Nord
      • Lille Cedex, Nord, France, 59037
        • Hopital Roger Salengro - CHU Lille
    • Rhone
      • Bron cedex, Rhone, France, 69677
        • Hôpital Neurologique Pierre Wertheimer
  • Germany
    • Bayern
      • Muenchen, Bayern, Germany, 80336
        • LMU-Campus Innenstadt
    • Niedersachsen
      • Hannover, Niedersachsen, Germany, 30625
        • Medizinische Hochschule Hannover
  • Greece
      • Athens, Greece, 11521
        • Navy Hospital of Athens
      • Thessaloniki, Greece, 57010
        • General Hospital of Thessaloniki "G. Papanikolaou"
  • Hungary
      • Budapest, Hungary, 1204
        • Jahn Ferenc Dél-Pesti Kórház
      • Szeged, Hungary, 6725
        • Szegedi Tud.Egyetem Szent-Gyorgyi Albert Klin. Közp.
  • Italy
      • Catania, Italy, 95123
        • Policlinico di Catania
      • Milano, Italy, 20133
        • Fondazione IRCCS Istituto Neurologico Carlo Besta
      • Napoli, Italy, 80131
        • Azienda Ospedaliera Universitaria "Federico II"
      • Padova, Italy, 35128
        • Ospedale San Camillo IRCCS
      • Pisa, Italy, 56126
        • Azienda Ospedaliero Universitaria Pisana
      • Roma, Italy, 00168
        • Policlinico Universitario Agostino Gemelli
      • Roma, Italy, 00161
        • Umberto I Pol. di Roma-Università di Roma La Sapienza
      • Roma, Italy, 00189
        • Azienda Ospedaliera Sant'Andrea - Università di Roma La Sapienza
      • Trento, Italy, 38122
        • Ospedale Santa Chiara
  • Japan
    • Chiba-Ken
      • Chiba-shi, Chiba-Ken, Japan, 260-8677
        • Chiba University Hospital
    • Fukuoka-Ken
      • Fukuoka-shi, Fukuoka-Ken, Japan, 812-8582
        • Kyushu University Hospital
    • Hokkaido
      • Sapporo-shi, Hokkaido, Japan, 060-8543
        • Sapporo Medical University Hospital
    • Iwate-Ken
      • Hanamaki-shi, Iwate-Ken, Japan, 025-0075
        • Hanamaki General Hospitals
    • Miyagi-Ken
      • Sendai-shi, Miyagi-Ken, Japan, 983-8520
        • NHO Sendai Medical Center
    • Nagasaki-Ken
      • Kawatana, Nagasaki-Ken, Japan, 859-3615
        • NHO Nagasaki Kawatana Medical Center
      • Nagasaki-shi, Nagasaki-Ken, Japan, 852-8501
        • Nagasaki University Hospital
    • Osaka-Fu
      • Osakasayama-shi, Osaka-Fu, Japan, 589-8511
        • Kinki University Hospital
      • Suita-shi, Osaka-Fu, Japan, 565-0871
        • Osaka University Hospital
    • Tokyo-To
      • Meguro-ku, Tokyo-To, Japan, 153-8515
        • Toho University Ohashi Medical Center
    • Yamaguchi-Ken
      • Ube-shi, Yamaguchi-Ken, Japan, 755-8505
        • Yamaguchi University Hospital
  • Korea, Republic of
      • Seoul, Korea, Republic of, 135-710
        • Samsung Medical Center
      • Seoul, Korea, Republic of, 136-705
        • Korea University Anam Hospital
      • Seoul, Korea, Republic of, 120-752
        • Severance Hospital, Yonsei University Health System
      • Seoul, Korea, Republic of, 156707
        • Seoul Metropolitan Government Seoul National University
  • Netherlands
      • Amsterdam, Netherlands, 1105 AZ
        • Academisch Medisch Centrum
      • Leiden, Netherlands, 2333 ZA
        • Leiden Universitair Medisch Centrum
      • Maastricht, Netherlands, 6229 HX
        • Maastricht University Medical Center
  • Spain
      • Barcelona, Spain, 08035
        • Hospital Universitari Vall d'Hebron
      • Barcelona, Spain, 08041
        • Hospital de La Santa Creu I Sant Pau
      • Barcelona, Spain, 08907
        • Hospital Universitari de Bellvitge
      • Madrid, Spain, 28046
        • Hospital Universitario La Paz
  • Sweden
      • Göteborg, Sweden, 41345
        • Sahlgrenska Sjukhuset
      • Stockholm, Sweden, 17176
        • Karolinska Universitetssjukhuset - Solna
  • Turkey
      • Ankara, Turkey, 06100
        • Hacettepe University Medical Faculty
      • Istanbul, Turkey, 61080
        • Karadeniz Tecnical Uni. Med. Fac.
      • Izmir, Turkey, 35340
        • Dokuz Eylul University Medicine Faculty
      • Kocaeli, Turkey, 41380
        • Kocaeli University Medical Faculty
      • Samsun, Turkey, 55139
        • Ondokuz Mayıs University of Medicine
  • United Kingdom
      • London, United Kingdom, SE5 9NU
        • King's College Hospital
    • Merseyside
      • Liverpool, Merseyside, United Kingdom, L9 7LJ
        • The Walton Centre
    • West Midlands
      • Birmingham, West Midlands, United Kingdom, B15 2WB
        • Queen Elizabeth Hospital
  • United States
    • Alabama
      • Birmingham, Alabama, United States, 35233
        • University of Alabama
    • California
      • Fresno, California, United States, 93720
        • University of California San Francisco-Fresno
      • Los Angeles, California, United States, 90033
        • University of Southern California
      • Orange, California, United States, 92868
        • University of California-Irvine
      • Sacramento, California, United States, 95765
        • University of California Davis Health System
      • San Francisco, California, United States, 94115
        • California Pacific Medical Center
      • Stanford, California, United States, 94305
        • Stanford University School of Medicine
    • Connecticut
      • New Haven, Connecticut, United States, 06510
        • Yale New Haven Hospital
    • Florida
      • Jacksonville, Florida, United States, 32209
        • University of Florida Health Science Center
      • Miami, Florida, United States, 33136
        • University of Miami School of Medicine
      • Tampa, Florida, United States, 33612
        • University of South Florida
    • Georgia
      • Augusta, Georgia, United States, 30912
        • Georgia Regents University
      • Canton, Georgia, United States, 30114
        • Medical Associates of North Georgia
    • Illinois
      • Springfield, Illinois, United States, 62702
        • Southern Illinois University School of Medicine
    • Indiana
      • Indianapolis, Indiana, United States, 46202
        • Indiana University
    • Iowa
      • Iowa City, Iowa, United States, 52242
        • University of Iowa College of Medicine
    • Kansas
      • Kansas City, Kansas, United States, 66160
        • University of Kansas Medical Center
    • Maryland
      • Baltimore, Maryland, United States, 21201
        • University of Maryland Medical Center
      • Baltimore, Maryland, United States, 21287-0876
        • Johns Hopkins University School of Medicine
    • Massachusetts
      • Boston, Massachusetts, United States, 02115
        • Brigham and Women's Hospital
      • Boston, Massachusetts, United States, 02114
        • Massachusetts General Hospital
      • Burlington, Massachusetts, United States, 01805
        • Lahey Clinic Inc.
    • Michigan
      • Detroit, Michigan, United States, 48201
        • Wayne State University School of Medicine
    • Nevada
      • Las Vegas, Nevada, United States, 89145
        • Las Vegas Clinic
    • New York
      • Buffalo, New York, United States, 14203
        • Buffalo General Hospital
      • New York, New York, United States, 10029
        • Mount Sinai School of Medicine
      • New York, New York, United States, 10021
        • Hospital For Special Surgery/New York Presbyterian Hospital Cornell Campus
    • North Carolina
      • Chapel Hill, North Carolina, United States, 27599
        • University of North Carolina at Chapel Hill
      • Charlotte, North Carolina, United States, 28207
        • Carolinas Healthcare System
      • Durham, North Carolina, United States, 27705
        • Duke University Health System
    • Ohio
      • Columbus, Ohio, United States, 43210
        • Ohio State University Medical Center
    • Oregon
      • Portland, Oregon, United States, 97239
        • Oregon Health & Science University
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19104
        • University of Pennsylvania
    • Rhode Island
      • Providence, Rhode Island, United States, 02905
        • Rhode Island Hospital
    • Tennessee
      • Cordova, Tennessee, United States, 38018
        • Wesley Neurology Clinic
      • Nashville, Tennessee, United States, 37232
        • Vanderbilt Medical Center
    • Texas
      • Dallas, Texas, United States, 75390
        • UT Southwestern
      • Houston, Texas, United States, 77030
        • Methodist Neurological Institute
      • San Antonio, Texas, United States, 78229
        • University of Texas Health Science Center at San Antonio
    • Vermont
      • Burlington, Vermont, United States, 05405
        • University of Vermont Medical Center
    • Washington
      • Seattle, Washington, United States, 98195
        • University of Washington

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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)

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Eculizumab
Biological/Vaccine: Eculizumab; Induction phase: 3 vials of study drug (equivalent to 900 mg of eculizumab) weekly for 4 doses (every 7 days ± 2 days) followed by 4 vials of study drug (equivalent to 1200 mg of eculizumab) 1 week later for the fifth dose (Week 4); Maintenance phase: 4 vials of study drug (equivalent to 1200 mg of eculizumab) every 2 weeks (14 days ± 2 days) from the fifth dose onwards (Week 6 through Week 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; Induction phase: 3 vials of study drug (placebo) weekly for 4 doses (every 7 days ± 2 days) followed by 4 vials of study drug (placebo) 1 week later for the fifth dose (Week 4); Maintenance phase: 4 vials of study drug (placebo) every 2 weeks (14 days ± 2 days) from the fifth dose onwards (Week 6 through Week 26).
Drug: Placebo
Placebo

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Myasthenia Gravis Activities of Daily Living Profile (MG-ADL): Change From Baseline in MG-ADL Total Score at Week 26 by Worst-Rank Analysis of Covariance (ANCOVA)
Time Frame: End of study (Week 26)
In the Worst-Rank analysis, the 125 total patients were ranked from best outcome (rank/score of 1) to worst outcome (rank/score of 125).
End of study (Week 26)

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Alexion Pharmaceuticals

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

December 1, 2013

Primary Completion (Actual)

February 1, 2016

Study Completion (Actual)

June 1, 2016

Study Registration Dates

First Submitted

November 18, 2013

First Submitted That Met QC Criteria

November 22, 2013

First Posted (Estimate)

November 28, 2013

Study Record Updates

Last Update Posted (Actual)

July 16, 2019

Last Update Submitted That Met QC Criteria

July 12, 2019

Last Verified

July 1, 2019

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • ECU-MG-301
  • 2013-003589-15 (EudraCT Number)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

Clinical Trials on Refractory Generalized Myasthenia Gravis

Clinical Trials on Placebo

Search Similar Trials

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Croatia

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations

3
Subscribe