Safety and Efficacy of Eculizumab in Refractory Generalized Myasthenia Gravis (REGAIN Study)
NCT ID: NCT01997229
Last Updated: 2019-07-16
Study Results
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View full resultsBasic Information
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COMPLETED
PHASE3
125 participants
INTERVENTIONAL
2013-12-31
2016-06-30
Brief Summary
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Detailed Description
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Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
QUADRUPLE
Study Groups
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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).
Eculizumab
Induction Phase: 900 mg IV weekly X 4 Maintenance Phase: 1200 mg IV every 2 weeks during Weeks 4-26
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).
Placebo
Placebo
Interventions
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Eculizumab
Induction Phase: 900 mg IV weekly X 4 Maintenance Phase: 1200 mg IV every 2 weeks during Weeks 4-26
Placebo
Placebo
Eligibility Criteria
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Inclusion Criteria
* 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
Exclusion Criteria
* 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)
18 Years
ALL
No
Sponsors
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Alexion Pharmaceuticals, Inc.
INDUSTRY
Responsible Party
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Locations
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University of Alabama
Birmingham, Alabama, United States
University of California San Francisco-Fresno
Fresno, California, United States
University of Southern California
Los Angeles, California, United States
University of California-Irvine
Orange, California, United States
University of California Davis Health System
Sacramento, California, United States
California Pacific Medical Center
San Francisco, California, United States
Stanford University School of Medicine
Stanford, California, United States
Yale New Haven Hospital
New Haven, Connecticut, United States
University of Florida Health Science Center
Jacksonville, Florida, United States
University of Miami School of Medicine
Miami, Florida, 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
Southern Illinois University School of Medicine
Springfield, Illinois, United States
Indiana University
Indianapolis, Indiana, United States
University of Iowa College of Medicine
Iowa City, Iowa, United States
University of Kansas Medical Center
Kansas City, Kansas, United States
University of Maryland Medical Center
Baltimore, Maryland, United States
Johns Hopkins University School Of Medicine
Baltimore, Maryland, United States
Massachusetts General Hospital
Boston, Massachusetts, 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
Las Vegas Clinic
Las Vegas, Nevada, 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
Duke University Health System
Durham, 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
Rhode Island Hospital
Providence, Rhode Island, United States
Wesley Neurology Clinic
Cordova, Tennessee, United States
Vanderbilt Medical Center
Nashville, Tennessee, United States
UT Southwestern
Dallas, Texas, United States
Methodist Neurological Institute
Houston, Texas, United States
University of Texas Health Science Center at San Antonio
San Antonio, Texas, United States
University of Vermont Medical Center
Burlington, Vermont, United States
University of Washington
Seattle, Washington, United States
Hospital Italiano
Ciudad Autonoma, Buenos Aires, Argentina
Instituto de Investigaciones Neurologicas Raul Carrea, FLENI
Ciudad Autonoma, Buenos Aires, Argentina
St Vincent's Hospital Melbourne
Fitzroy, Victoria, Australia
Royal Perth Hospital
Perth, Western Australia, Australia
UZ Antwerpen
Edegem, , Belgium
AZ Sint-Lucas - Campus Sint-Lucas
Ghent, , Belgium
UZ Leuven
Leuven, , Belgium
Hospital Mãe de Deus
Porto Alegre, Rio Grande do Sul, Brazil
Faculdade de Medicina do ABC
Santo André, São Paulo, Brazil
Fundação Faculdade Regional de Medicina de São José do Rio Preto
São José do Rio Preto, São Paulo, Brazil
HUCFF-UFRJ - Hospital Universitário Clementino Fraga Filho - Universidade Federal do Rio de Janeiro
Rio de Janeiro, , Brazil
UNIFESP - Universidade Federal de São Paulo
São Paulo, , Brazil
University of Alberta Hospital
Edmonton, Alberta, Canada
Toronto General Hospital
Toronto, Ontario, Canada
Montreal Neurological Institute
Montreal, Quebec, Canada
Vseobecna fakultni nemocnice v Praze
Prague, Prague, Czechia
Fakultni nemocnice Brno
Brno, , Czechia
Fakultni nemocnice Ostrava
Ostrava - Poruba, , Czechia
Århus Universitetshospital
Aarhus C, , Denmark
Rigshospitalet
København Ø, , Denmark
Helsingin yliopistollinen keskussairaala
Helsinki, , Finland
Neuro NEO Oy
Turku, , Finland
CHU de Nice Hôpital Pasteur 2
Nice, Alpes Maritimes, France
Groupe Hospitalier Pellegrin - Hôpital Pellegrin
Bordeaux, Gironde, France
Hopital Roger Salengro - CHU Lille
Lille, Nord, France
Hopital Neurologique Pierre Wertheimer
Bron, Rhone, France
LMU-Campus Innenstadt
Munich, Bavaria, Germany
Medizinische Hochschule Hannover
Hanover, Lower Saxony, Germany
Navy Hospital of Athens
Athens, , Greece
General Hospital of Thessaloniki "G. Papanikolaou"
Thessaloniki, , Greece
Jahn Ferenc Del-Pesti Korhaz
Budapest, , Hungary
Szegedi Tud.Egyetem Szent-Gyorgyi Albert Klin. Közp.
Szeged, , Hungary
Policlinico di Catania
Catania, , Italy
Fondazione IRCCS Istituto Neurologico Carlo Besta
Milan, , Italy
Azienda Ospedaliera Universitaria "Federico II"
Napoli, , Italy
Ospedale San Camillo IRCCS
Padua, , Italy
Azienda Ospedaliero Universitaria Pisana
Pisa, , Italy
Umberto I Pol. di Roma-Università di Roma La Sapienza
Roma, , Italy
Policlinico Universitario Agostino Gemelli
Roma, , Italy
Azienda Ospedaliera Sant'Andrea - Università di Roma La Sapienza
Roma, , Italy
Ospedale Santa Chiara
Trento, , Italy
Chiba University Hospital
Chiba, Chiba, Japan
Kyushu University Hospital
Fukuoka, Fukuoka, Japan
Sapporo Medical University Hospital
Sapporo, Hokkaido, Japan
Hanamaki General Hospitals
Hanamaki-shi, Iwate, Japan
NHO Sendai Medical Center
Sendai, Miyagi, Japan
NHO Nagasaki Kawatana Medical Center
Kawatana, Nagasaki, Japan
Nagasaki University Hospital
Nagasaki, Nagasaki, Japan
Kinki University Hospital
Osakasayama-shi, Osaka, Japan
Osaka University Hospital
Suita-shi, Osaka, Japan
Toho University Ohashi Medical Center
Meguro-ku, Tokyo-To, Japan
Yamaguchi University Hospital
Ube-shi, Yamaguchi, Japan
Academisch Medisch Centrum
Amsterdam, , Netherlands
Leiden Universitair Medisch Centrum
Leiden, , Netherlands
Maastricht University Medical Center
Maastricht, , Netherlands
Severance Hospital, Yonsei University Health System
Seoul, , South Korea
Samsung Medical Center
Seoul, , South Korea
Korea University Anam Hospital
Seoul, , South Korea
Seoul Metropolitan Government Seoul National University
Seoul, , South Korea
Hospital Universitari Vall d'Hebron
Barcelona, , Spain
Hospital de la Santa Creu i Sant Pau
Barcelona, , Spain
Hospital Universitari de Bellvitge
Barcelona, , Spain
Hospital Universitario La Paz
Madrid, , Spain
Sahlgrenska Sjukhuset
Gothenburg, , Sweden
Karolinska Universitetssjukhuset - Solna
Stockholm, , Sweden
Hacettepe University Medical Faculty
Ankara, , Turkey (Türkiye)
Karadeniz Tecnical Uni. Med. Fac.
Istanbul, , Turkey (Türkiye)
Dokuz Eylul University Medicine Faculty
Izmir, , Turkey (Türkiye)
Kocaeli University Medical Faculty
Kocaeli, , Turkey (Türkiye)
Ondokuz Mayıs University of Medicine
Samsun, , Turkey (Türkiye)
The Walton Centre
Liverpool, Merseyside, United Kingdom
Queen Elizabeth Hospital
Birmingham, West Midlands, United Kingdom
King's College Hospital
London, , United Kingdom
Countries
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References
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Howard JF Jr, Barohn RJ, Cutter GR, Freimer M, Juel VC, Mozaffar T, Mellion ML, Benatar MG, Farrugia ME, Wang JJ, Malhotra SS, Kissel JT; MG Study Group. A randomized, double-blind, placebo-controlled phase II study of eculizumab in patients with refractory generalized myasthenia gravis. Muscle Nerve. 2013 Jul;48(1):76-84. doi: 10.1002/mus.23839. Epub 2013 Apr 30.
Howard JF Jr, Utsugisawa K, Benatar M, Murai H, Barohn RJ, Illa I, Jacob S, Vissing J, Burns TM, Kissel JT, Muppidi S, Nowak RJ, O'Brien F, Wang JJ, Mantegazza R; REGAIN Study Group. Safety and efficacy of eculizumab in anti-acetylcholine receptor antibody-positive refractory generalised myasthenia gravis (REGAIN): a phase 3, randomised, double-blind, placebo-controlled, multicentre study. Lancet Neurol. 2017 Dec;16(12):976-986. doi: 10.1016/S1474-4422(17)30369-1. Epub 2017 Oct 20.
Monteleone JPR, Gao X, Kleijn HJ, Bellanti F, Pelto R. Eculizumab Pharmacokinetics and Pharmacodynamics in Patients With Generalized Myasthenia Gravis. Front Neurol. 2021 Nov 2;12:696385. doi: 10.3389/fneur.2021.696385. eCollection 2021.
Siddiqi ZA, Nowak RJ, Mozaffar T, O'Brien F, Yountz M, Patti F; REGAIN Study Group. Eculizumab in refractory generalized myasthenia gravis previously treated with rituximab: subgroup analysis of REGAIN and its extension study. Muscle Nerve. 2021 Dec;64(6):662-669. doi: 10.1002/mus.27422. Epub 2021 Oct 14.
Nowak RJ, Muppidi S, Beydoun SR, O'Brien FL, Yountz M, Howard JF Jr. Concomitant Immunosuppressive Therapy Use in Eculizumab-Treated Adults With Generalized Myasthenia Gravis During the REGAIN Open-Label Extension Study. Front Neurol. 2020 Nov 24;11:556104. doi: 10.3389/fneur.2020.556104. eCollection 2020.
Mantegazza R, Wolfe GI, Muppidi S, Wiendl H, Fujita KP, O'Brien FL, Booth HDE, Howard JF Jr; REGAIN Study Group. Post-intervention Status in Patients With Refractory Myasthenia Gravis Treated With Eculizumab During REGAIN and Its Open-Label Extension. Neurology. 2021 Jan 26;96(4):e610-e618. doi: 10.1212/WNL.0000000000011207. Epub 2020 Nov 23.
Mantegazza R, O'Brien FL, Yountz M, Howard JF Jr; REGAIN study group. Consistent improvement with eculizumab across muscle groups in myasthenia gravis. Ann Clin Transl Neurol. 2020 Aug;7(8):1327-1339. doi: 10.1002/acn3.51121. Epub 2020 Jul 22.
Jacob S, Murai H, Utsugisawa K, Nowak RJ, Wiendl H, Fujita KP, O'Brien F, Howard JF Jr. Response to eculizumab in patients with myasthenia gravis recently treated with chronic IVIg: a subgroup analysis of REGAIN and its open-label extension study. Ther Adv Neurol Disord. 2020 May 6;13:1756286420911784. doi: 10.1177/1756286420911784. eCollection 2020.
Vissing J, Jacob S, Fujita KP, O'Brien F, Howard JF; REGAIN study group. 'Minimal symptom expression' in patients with acetylcholine receptor antibody-positive refractory generalized myasthenia gravis treated with eculizumab. J Neurol. 2020 Jul;267(7):1991-2001. doi: 10.1007/s00415-020-09770-y. Epub 2020 Mar 18.
Andersen H, Mantegazza R, Wang JJ, O'Brien F, Patra K, Howard JF Jr; REGAIN Study Group. Eculizumab improves fatigue in refractory generalized myasthenia gravis. Qual Life Res. 2019 Aug;28(8):2247-2254. doi: 10.1007/s11136-019-02148-2. Epub 2019 Mar 23.
Related Links
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Alexion Clinical Trials Webpage
Other Identifiers
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2013-003589-15
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
ECU-MG-301
Identifier Type: -
Identifier Source: org_study_id
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