search
Back to results

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

Primary Purpose

Refractory Generalized Myasthenia Gravis

Status
Completed
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
Eculizumab
Placebo
Sponsored by
Alexion
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Refractory Generalized Myasthenia Gravis focused on measuring Myasthenia Gravis, Eculizumab, safety, efficacy

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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)

Sites / Locations

  • University of Alabama
  • University of California San Francisco-Fresno
  • University of Southern California
  • University of California-Irvine
  • University of California Davis Health System
  • California Pacific Medical Center
  • Stanford University School of Medicine
  • Yale New Haven Hospital
  • University of Florida Health Science Center
  • University of Miami School of Medicine
  • University of South Florida
  • Georgia Regents University
  • Medical Associates of North Georgia
  • Southern Illinois University School of Medicine
  • Indiana University
  • University of Iowa College of Medicine
  • University of Kansas Medical Center
  • University of Maryland Medical Center
  • Johns Hopkins University School Of Medicine
  • Massachusetts General Hospital
  • Brigham and Women's Hospital
  • Lahey Clinic Inc.
  • Wayne State University School of Medicine
  • Las Vegas Clinic
  • Buffalo General Hospital
  • Hospital For Special Surgery/New York Presbyterian Hospital Cornell Campus
  • Mount Sinai School of Medicine
  • University of North Carolina at Chapel Hill
  • Carolinas Healthcare System
  • Duke University Health System
  • Ohio State University Medical Center
  • Oregon Health & Science University
  • University of Pennsylvania
  • Rhode Island Hospital
  • Wesley Neurology Clinic
  • Vanderbilt Medical Center
  • UT Southwestern
  • Methodist Neurological Institute
  • University of Texas Health Science Center at San Antonio
  • University of Vermont Medical Center
  • University of Washington
  • Hospital Italiano
  • Instituto de Investigaciones Neurologicas Raul Carrea, FLENI
  • St Vincent's Hospital Melbourne
  • Royal Perth Hospital
  • UZ Antwerpen
  • AZ Sint-Lucas - Campus Sint-Lucas
  • UZ Leuven
  • Hospital Mãe de Deus
  • Faculdade de Medicina do ABC
  • Fundação Faculdade Regional de Medicina de São José do Rio Preto
  • HUCFF-UFRJ - Hospital Universitário Clementino Fraga Filho - Universidade Federal do Rio de Janeiro
  • UNIFESP - Universidade Federal de São Paulo
  • University of Alberta Hospital
  • Toronto General Hospital
  • Montreal Neurological Institute
  • Vseobecna fakultni nemocnice v Praze
  • Fakultni nemocnice Brno
  • Fakultni nemocnice Ostrava
  • Århus Universitetshospital
  • Rigshospitalet
  • Helsingin yliopistollinen keskussairaala
  • Neuro NEO Oy
  • CHU de Nice Hôpital Pasteur 2
  • Groupe Hospitalier Pellegrin - Hôpital Pellegrin
  • Hopital Roger Salengro - CHU Lille
  • Hopital Neurologique Pierre Wertheimer
  • LMU-Campus Innenstadt
  • Medizinische Hochschule Hannover
  • Navy Hospital of Athens
  • General Hospital of Thessaloniki "G. Papanikolaou"
  • Jahn Ferenc Del-Pesti Korhaz
  • Szegedi Tud.Egyetem Szent-Gyorgyi Albert Klin. Közp.
  • Policlinico di Catania
  • Fondazione IRCCS Istituto Neurologico Carlo Besta
  • Azienda Ospedaliera Universitaria "Federico II"
  • Ospedale San Camillo IRCCS
  • Azienda Ospedaliero Universitaria Pisana
  • Umberto I Pol. di Roma-Università di Roma La Sapienza
  • Policlinico Universitario Agostino Gemelli
  • Azienda Ospedaliera Sant'Andrea - Università di Roma La Sapienza
  • Ospedale Santa Chiara
  • Chiba University Hospital
  • Kyushu University Hospital
  • Sapporo Medical University Hospital
  • Hanamaki General Hospitals
  • NHO Sendai Medical Center
  • NHO Nagasaki Kawatana Medical Center
  • Nagasaki University Hospital
  • Kinki University Hospital
  • Osaka University Hospital
  • Toho University Ohashi Medical Center
  • Yamaguchi University Hospital
  • Severance Hospital, Yonsei University Health System
  • Samsung Medical Center
  • Korea University Anam Hospital
  • Seoul Metropolitan Government Seoul National University
  • Academisch Medisch Centrum
  • Leiden Universitair Medisch Centrum
  • Maastricht University Medical Center
  • Hospital Universitari Vall d'Hebron
  • Hospital de la Santa Creu i Sant Pau
  • Hospital Universitari de Bellvitge
  • Hospital Universitario La Paz
  • Sahlgrenska Sjukhuset
  • Karolinska Universitetssjukhuset - Solna
  • Hacettepe University Medical Faculty
  • Karadeniz Tecnical Uni. Med. Fac.
  • Dokuz Eylul University Medicine Faculty
  • Kocaeli University Medical Faculty
  • Ondokuz Mayıs University of Medicine
  • The Walton Centre
  • Queen Elizabeth Hospital
  • King's College Hospital

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Placebo Comparator

Arm Label

Eculizumab

Placebo

Arm Description

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 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).

Outcomes

Primary Outcome Measures

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)
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

Full Information

First Posted
November 18, 2013
Last Updated
July 12, 2019
Sponsor
Alexion
search

1. Study Identification

Unique Protocol Identification Number
NCT01997229
Brief Title
Safety and Efficacy of Eculizumab in Refractory Generalized Myasthenia Gravis (REGAIN Study)
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)
Study Type
Interventional

2. Study Status

Record Verification Date
July 2019
Overall Recruitment Status
Completed
Study Start Date
December 2013 (undefined)
Primary Completion Date
February 2016 (Actual)
Study Completion Date
June 2016 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Alexion

4. Oversight

Data Monitoring Committee
No

5. Study Description

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

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Refractory Generalized Myasthenia Gravis
Keywords
Myasthenia Gravis, Eculizumab, safety, efficacy

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
125 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Eculizumab
Arm Type
Experimental
Arm Description
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).
Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Description
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).
Intervention Type
Biological
Intervention Name(s)
Eculizumab
Intervention Description
Induction Phase: 900 mg IV weekly X 4 Maintenance Phase: 1200 mg IV every 2 weeks during Weeks 4-26
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
Placebo
Primary Outcome Measure Information:
Title
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)
Description
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).
Time Frame
End of study (Week 26)

10. Eligibility

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

12. IPD Sharing Statement

Plan to Share IPD
No
Citations:
PubMed Identifier
23512355
Citation
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.
Results Reference
background
PubMed Identifier
29066163
Citation
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. Erratum In: Lancet Neurol. 2017 Dec;16(12 ):954.
Results Reference
result
PubMed Identifier
34795626
Citation
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.
Results Reference
derived
PubMed Identifier
34590717
Citation
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.
Results Reference
derived
PubMed Identifier
33329303
Citation
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.
Results Reference
derived
PubMed Identifier
33229455
Citation
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.
Results Reference
derived
PubMed Identifier
32700461
Citation
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.
Results Reference
derived
PubMed Identifier
32426038
Citation
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.
Results Reference
derived
PubMed Identifier
32189108
Citation
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.
Results Reference
derived
PubMed Identifier
30905021
Citation
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. Erratum In: Qual Life Res. 2019 May 21;:
Results Reference
derived
Links:
URL
http://www.alexionclinicaltrials.com
Description
Alexion Clinical Trials Webpage

Learn more about this trial

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

We'll reach out to this number within 24 hrs