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

Status

Indicates the current recruitment status or the expanded access status.
Completed

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
125

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • 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 Mae 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
        • Fundação Faculdade Regional de Medicina de São José 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
        • Fakultní 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. Kozp.
  • 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 - Universita 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

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

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

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
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

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
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

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
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

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
December 1, 2013

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
February 1, 2016

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
June 1, 2016

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
November 18, 2013

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
November 22, 2013

First Posted (Estimate)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
November 28, 2013

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
July 16, 2019

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
July 12, 2019

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
July 1, 2019

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant 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.

Cookie Settings

We use cookies to ensure that we give you the best experience on our website. For more details carefully read our Privacy and Cookies Policy. ...>>>You can change your preferences or withdraw your consent at any time by deleting the cookies from your website or computer as described in the policy. Please accept it and choose your preferences by ticking the corresponding boxes in the "Manage Settings" section below. Please carefully read our Terms of Service before you proceed with using any part of this website.
«Manage Settings