Safety and Efficacy of Eculizumab in Refractory Generalized Myasthenia Gravis (REGAIN Study)
- Conditions
- Refractory Generalized Myasthenia Gravis
- Interventions
- Biological: EculizumabDrug: Placebo
- Registration Number
- NCT01997229
- Lead Sponsor
- Alexion Pharmaceuticals, Inc.
- Brief Summary
The purpose of this study is to determine if eculizumab is safe and effective for the treatment of refractory generalized Myasthenia Gravis.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 125
-
Male or female patients ≥18 years old
-
Diagnosis of MG made by the following tests:
-
Positive serologic test for anti-AChR Abs as confirmed at screening, and
-
One of the following:
- History of abnormal neuromuscular transmission test demonstrated by single-fiber electromyography (SFEMG) or repetitive nerve stimulation, or
- History of positive anticholinesterase test, e.g. edrophonium chloride test, or
- 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:
- Failed treatment with at least two immunosuppressive agents. Or,
- Failed treatment with at least one immunosuppressive agent and require chronic plasma exchange or IVIg
Key
- 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 & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Eculizumab 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). Placebo 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).
- Primary Outcome Measures
Name Time Method 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) 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).
- Secondary Outcome Measures
Name Time Method
Trial Locations
- Locations (114)
University of Alabama
🇺🇸Birmingham, Alabama, United States
University of California San Francisco-Fresno
🇺🇸Fresno, California, United States
University of California-Irvine
🇺🇸Orange, California, United States
University of Florida Health Science Center
🇺🇸Jacksonville, Florida, United States
University of California Davis Health System
🇺🇸Sacramento, California, United States
Yale New Haven Hospital
🇺🇸New Haven, Connecticut, United States
Stanford University School of Medicine
🇺🇸Stanford, California, United States
University of South Florida
🇺🇸Tampa, Florida, United States
Georgia Regents University
🇺🇸Augusta, Georgia, United States
Medical Associates of North Georgia
🇺🇸Canton, Georgia, United States
University of Iowa College of Medicine
🇺🇸Iowa City, Iowa, United States
Southern Illinois University School of Medicine
🇺🇸Springfield, Illinois, United States
University of Maryland Medical Center
🇺🇸Baltimore, Maryland, United States
Johns Hopkins University School Of Medicine
🇺🇸Baltimore, Maryland, United States
Brigham and Women's Hospital
🇺🇸Boston, Massachusetts, United States
Lahey Clinic Inc.
🇺🇸Burlington, Massachusetts, United States
Wayne State University School of Medicine
🇺🇸Detroit, Michigan, United States
Buffalo General Hospital
🇺🇸Buffalo, New York, United States
Hospital For Special Surgery/New York Presbyterian Hospital Cornell Campus
🇺🇸New York, New York, United States
Mount Sinai School of Medicine
🇺🇸New York, New York, United States
University of North Carolina at Chapel Hill
🇺🇸Chapel Hill, North Carolina, United States
Carolinas Healthcare System
🇺🇸Charlotte, North Carolina, United States
Ohio State University Medical Center
🇺🇸Columbus, Ohio, United States
Oregon Health & Science University
🇺🇸Portland, Oregon, United States
University of Pennsylvania
🇺🇸Philadelphia, Pennsylvania, United States
Wesley Neurology Clinic
🇺🇸Cordova, Tennessee, United States
UT Southwestern
🇺🇸Dallas, Texas, United States
University of Vermont Medical Center
🇺🇸Burlington, Vermont, United States
University of Southern California
🇺🇸Los Angeles, California, United States
Hospital Italiano
🇦🇷Ciudad Autonoma, Buenos Aires, Argentina
Instituto de Investigaciones Neurologicas Raul Carrea, FLENI
🇦🇷Ciudad Autonoma, Buenos Aires, Argentina
Toho University Ohashi Medical Center
🇯🇵Meguro-ku, Tokyo-To, Japan
UZ Antwerpen
🇧🇪Edegem, Belgium
St Vincent's Hospital Melbourne
🇦🇺Fitzroy, Victoria, Australia
AZ Sint-Lucas - Campus Sint-Lucas
🇧🇪Gent, Belgium
Faculdade de Medicina do ABC
🇧🇷Santo André, Sao Paulo, Brazil
UZ Leuven
🇧🇪Leuven, Belgium
HUCFF-UFRJ - Hospital Universitário Clementino Fraga Filho - Universidade Federal do Rio de Janeiro
🇧🇷Rio De Janeiro, Brazil
Fundação Faculdade Regional de Medicina de São José do Rio Preto
🇧🇷São José do Rio Preto, Sao Paulo, Brazil
University of Alberta Hospital
🇨🇦Edmonton, Alberta, Canada
UNIFESP - Universidade Federal de São Paulo
🇧🇷São Paulo, Brazil
Toronto General Hospital
🇨🇦Toronto, Ontario, Canada
Vseobecna fakultni nemocnice v Praze
🇨🇿Praha, Prague, Czechia
Montreal Neurological Institute
🇨🇦Montreal, Quebec, Canada
Fakultni nemocnice Ostrava
🇨🇿Ostrava - Poruba, Czechia
CHU de Nice Hôpital Pasteur 2
🇫🇷Nice Cedex 3, Alpes Maritimes, France
Helsingin yliopistollinen keskussairaala
🇫🇮Helsinki, Finland
Neuro NEO Oy
🇫🇮Turku, Finland
Groupe Hospitalier Pellegrin - Hôpital Pellegrin
🇫🇷Bordeaux Cedex, Gironde, France
Medizinische Hochschule Hannover
🇩🇪Hannover, Niedersachsen, Germany
Hopital Roger Salengro - CHU Lille
🇫🇷Lille Cedex, Nord, France
Hopital Neurologique Pierre Wertheimer
🇫🇷Bron cedex, Rhone, France
LMU-Campus Innenstadt
🇩🇪Muenchen, Bayern, Germany
Navy Hospital of Athens
🇬🇷Athens, Greece
Jahn Ferenc Del-Pesti Korhaz
🇭🇺Budapest, Hungary
General Hospital of Thessaloniki "G. Papanikolaou"
🇬🇷Thessaloniki, Greece
Ospedale San Camillo IRCCS
🇮🇹Padova, Italy
Szegedi Tud.Egyetem Szent-Gyorgyi Albert Klin. Közp.
🇭🇺Szeged, Hungary
Policlinico di Catania
🇮🇹Catania, Italy
Umberto I Pol. di Roma-Università di Roma La Sapienza
🇮🇹Roma, Italy
Azienda Ospedaliero Universitaria Pisana
🇮🇹Pisa, Italy
Fondazione IRCCS Istituto Neurologico Carlo Besta
🇮🇹Milano, Italy
Azienda Ospedaliera Universitaria "Federico II"
🇮🇹Napoli, Italy
Policlinico Universitario Agostino Gemelli
🇮🇹Roma, Italy
Azienda Ospedaliera Sant'Andrea - Università di Roma La Sapienza
🇮🇹Roma, Italy
Chiba University Hospital
🇯🇵Chiba-shi, Chiba-Ken, Japan
Kyushu University Hospital
🇯🇵Fukuoka-shi, Fukuoka-Ken, Japan
Ospedale Santa Chiara
🇮🇹Trento, Italy
Osaka University Hospital
🇯🇵Suita-shi, Osaka-Fu, Japan
Hanamaki General Hospitals
🇯🇵Hanamaki-shi, Iwate-Ken, Japan
NHO Sendai Medical Center
🇯🇵Sendai-shi, Miyagi-Ken, Japan
Kinki University Hospital
🇯🇵Osakasayama-shi, Osaka-Fu, Japan
Sapporo Medical University Hospital
🇯🇵Sapporo-shi, Hokkaido, Japan
NHO Nagasaki Kawatana Medical Center
🇯🇵Kawatana, Nagasaki-Ken, Japan
Nagasaki University Hospital
🇯🇵Nagasaki-shi, Nagasaki-Ken, Japan
Yamaguchi University Hospital
🇯🇵Ube-shi, Yamaguchi-Ken, Japan
Samsung Medical Center
🇰🇷Seoul, Korea, Republic of
Korea University Anam Hospital
🇰🇷Seoul, Korea, Republic of
Seoul Metropolitan Government Seoul National University
🇰🇷Seoul, Korea, Republic of
Severance Hospital, Yonsei University Health System
🇰🇷Seoul, Korea, Republic of
Academisch Medisch Centrum
🇳🇱Amsterdam, Netherlands
Hospital Universitario La Paz
🇪🇸Madrid, Spain
Leiden Universitair Medisch Centrum
🇳🇱Leiden, Netherlands
Hospital Universitari de Bellvitge
🇪🇸Barcelona, Spain
Hospital Universitari Vall d'Hebron
🇪🇸Barcelona, Spain
Sahlgrenska Sjukhuset
🇸🇪Göteborg, Sweden
Maastricht University Medical Center
🇳🇱Maastricht, Netherlands
Karolinska Universitetssjukhuset - Solna
🇸🇪Stockholm, Sweden
Hospital de la Santa Creu i Sant Pau
🇪🇸Barcelona, Spain
Dokuz Eylul University Medicine Faculty
🇹🇷Izmir, Turkey
King's College Hospital
🇬🇧London, United Kingdom
Hacettepe University Medical Faculty
🇹🇷Ankara, Turkey
Karadeniz Tecnical Uni. Med. Fac.
🇹🇷Istanbul, Turkey
Kocaeli University Medical Faculty
🇹🇷Kocaeli, Turkey
Ondokuz Mayıs University of Medicine
🇹🇷Samsun, Turkey
The Walton Centre
🇬🇧Liverpool, Merseyside, United Kingdom
Queen Elizabeth Hospital
🇬🇧Birmingham, West Midlands, United Kingdom
Massachusetts General Hospital
🇺🇸Boston, Massachusetts, United States
Methodist Neurological Institute
🇺🇸Houston, Texas, United States
University of Washington
🇺🇸Seattle, Washington, United States
University of Miami School of Medicine
🇺🇸Miami, Florida, United States
Indiana University
🇺🇸Indianapolis, Indiana, United States
Las Vegas Clinic
🇺🇸Las Vegas, Nevada, United States
Fakultni nemocnice Brno
🇨🇿Brno, Czechia
Hospital Mãe de Deus
🇧🇷Porto Alegre, Rio Grande Do Sul, Brazil
Royal Perth Hospital
🇦🇺Perth, Western Australia, Australia
Vanderbilt Medical Center
🇺🇸Nashville, Tennessee, United States
California Pacific Medical Center
🇺🇸San Francisco, California, United States
Duke University Health System
🇺🇸Durham, North Carolina, United States
University of Texas Health Science Center at San Antonio
🇺🇸San Antonio, Texas, United States
Århus Universitetshospital
🇩🇰Aarhus C, Denmark
Rigshospitalet
🇩🇰København Ø, Denmark
University of Kansas Medical Center
🇺🇸Kansas City, Kansas, United States
Rhode Island Hospital
🇺🇸Providence, Rhode Island, United States