Register a trial

The ANZCTR website will be unavailable from 1pm until 3pm (AEDT) on Wednesday the 30th of October for website maintenance. Please be sure to log out of the system in order to avoid any loss of data.

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been endorsed by the ANZCTR. Before participating in a study, talk to your health care provider and refer to this information for consumers
Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT01997229




Registration number
NCT01997229
Ethics application status
Date submitted
18/11/2013
Date registered
28/11/2013

Titles & IDs
Public title
Safety and Efficacy of Eculizumab in Refractory Generalized Myasthenia Gravis (REGAIN Study)
Scientific 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)
Secondary ID [1] 0 0
2013-003589-15
Secondary ID [2] 0 0
ECU-MG-301
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Refractory Generalized Myasthenia Gravis 0 0
Condition category
Condition code
Inflammatory and Immune System 0 0 0 0
Autoimmune diseases
Neurological 0 0 0 0
Other neurological disorders
Musculoskeletal 0 0 0 0
Other muscular and skeletal disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Other - Eculizumab
Treatment: Drugs - Placebo

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).

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).


Treatment: Other: Eculizumab
Induction Phase: 900 mg IV weekly X 4 Maintenance Phase: 1200 mg IV every 2 weeks during Weeks 4-26

Treatment: Drugs: Placebo
Placebo
Intervention code [1] 0 0
Treatment: Other
Intervention code [2] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
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)
Timepoint [1] 0 0
End of study (Week 26)

Eligibility
Key inclusion criteria
Key

* 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
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
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 design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
1/12/2013
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
1/06/2016
Sample size
Target
Accrual to date
Final
125
Recruitment in Australia
Recruitment state(s)
VIC,WA
Recruitment hospital [1] 0 0
St Vincent's Hospital Melbourne - Fitzroy
Recruitment hospital [2] 0 0
Royal Perth Hospital - Perth
Recruitment postcode(s) [1] 0 0
3065 - Fitzroy
Recruitment postcode(s) [2] 0 0
6000 - Perth
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Alabama
Country [2] 0 0
United States of America
State/province [2] 0 0
California
Country [3] 0 0
United States of America
State/province [3] 0 0
Connecticut
Country [4] 0 0
United States of America
State/province [4] 0 0
Florida
Country [5] 0 0
United States of America
State/province [5] 0 0
Georgia
Country [6] 0 0
United States of America
State/province [6] 0 0
Illinois
Country [7] 0 0
United States of America
State/province [7] 0 0
Indiana
Country [8] 0 0
United States of America
State/province [8] 0 0
Iowa
Country [9] 0 0
United States of America
State/province [9] 0 0
Kansas
Country [10] 0 0
United States of America
State/province [10] 0 0
Maryland
Country [11] 0 0
United States of America
State/province [11] 0 0
Massachusetts
Country [12] 0 0
United States of America
State/province [12] 0 0
Michigan
Country [13] 0 0
United States of America
State/province [13] 0 0
Nevada
Country [14] 0 0
United States of America
State/province [14] 0 0
New York
Country [15] 0 0
United States of America
State/province [15] 0 0
North Carolina
Country [16] 0 0
United States of America
State/province [16] 0 0
Ohio
Country [17] 0 0
United States of America
State/province [17] 0 0
Oregon
Country [18] 0 0
United States of America
State/province [18] 0 0
Pennsylvania
Country [19] 0 0
United States of America
State/province [19] 0 0
Rhode Island
Country [20] 0 0
United States of America
State/province [20] 0 0
Tennessee
Country [21] 0 0
United States of America
State/province [21] 0 0
Texas
Country [22] 0 0
United States of America
State/province [22] 0 0
Vermont
Country [23] 0 0
United States of America
State/province [23] 0 0
Washington
Country [24] 0 0
Argentina
State/province [24] 0 0
Buenos Aires
Country [25] 0 0
Belgium
State/province [25] 0 0
Edegem
Country [26] 0 0
Belgium
State/province [26] 0 0
Gent
Country [27] 0 0
Belgium
State/province [27] 0 0
Leuven
Country [28] 0 0
Brazil
State/province [28] 0 0
Rio Grande Do Sul
Country [29] 0 0
Brazil
State/province [29] 0 0
Sao Paulo
Country [30] 0 0
Brazil
State/province [30] 0 0
Rio De Janeiro
Country [31] 0 0
Brazil
State/province [31] 0 0
São Paulo
Country [32] 0 0
Canada
State/province [32] 0 0
Alberta
Country [33] 0 0
Canada
State/province [33] 0 0
Ontario
Country [34] 0 0
Canada
State/province [34] 0 0
Quebec
Country [35] 0 0
Czechia
State/province [35] 0 0
Prague
Country [36] 0 0
Czechia
State/province [36] 0 0
Brno
Country [37] 0 0
Czechia
State/province [37] 0 0
Ostrava - Poruba
Country [38] 0 0
Denmark
State/province [38] 0 0
Aarhus C
Country [39] 0 0
Denmark
State/province [39] 0 0
København Ø
Country [40] 0 0
Finland
State/province [40] 0 0
Helsinki
Country [41] 0 0
Finland
State/province [41] 0 0
Turku
Country [42] 0 0
France
State/province [42] 0 0
Alpes Maritimes
Country [43] 0 0
France
State/province [43] 0 0
Gironde
Country [44] 0 0
France
State/province [44] 0 0
Nord
Country [45] 0 0
France
State/province [45] 0 0
Rhone
Country [46] 0 0
Germany
State/province [46] 0 0
Bayern
Country [47] 0 0
Germany
State/province [47] 0 0
Niedersachsen
Country [48] 0 0
Greece
State/province [48] 0 0
Athens
Country [49] 0 0
Greece
State/province [49] 0 0
Thessaloniki
Country [50] 0 0
Hungary
State/province [50] 0 0
Budapest
Country [51] 0 0
Hungary
State/province [51] 0 0
Szeged
Country [52] 0 0
Italy
State/province [52] 0 0
Catania
Country [53] 0 0
Italy
State/province [53] 0 0
Milano
Country [54] 0 0
Italy
State/province [54] 0 0
Napoli
Country [55] 0 0
Italy
State/province [55] 0 0
Padova
Country [56] 0 0
Italy
State/province [56] 0 0
Pisa
Country [57] 0 0
Italy
State/province [57] 0 0
Roma
Country [58] 0 0
Italy
State/province [58] 0 0
Trento
Country [59] 0 0
Japan
State/province [59] 0 0
Chiba-Ken
Country [60] 0 0
Japan
State/province [60] 0 0
Fukuoka-Ken
Country [61] 0 0
Japan
State/province [61] 0 0
Hokkaido
Country [62] 0 0
Japan
State/province [62] 0 0
Iwate-Ken
Country [63] 0 0
Japan
State/province [63] 0 0
Miyagi-Ken
Country [64] 0 0
Japan
State/province [64] 0 0
Nagasaki-Ken
Country [65] 0 0
Japan
State/province [65] 0 0
Osaka-Fu
Country [66] 0 0
Japan
State/province [66] 0 0
Tokyo-To
Country [67] 0 0
Japan
State/province [67] 0 0
Yamaguchi-Ken
Country [68] 0 0
Korea, Republic of
State/province [68] 0 0
Seoul
Country [69] 0 0
Netherlands
State/province [69] 0 0
Amsterdam
Country [70] 0 0
Netherlands
State/province [70] 0 0
Leiden
Country [71] 0 0
Netherlands
State/province [71] 0 0
Maastricht
Country [72] 0 0
Spain
State/province [72] 0 0
Barcelona
Country [73] 0 0
Spain
State/province [73] 0 0
Madrid
Country [74] 0 0
Sweden
State/province [74] 0 0
Göteborg
Country [75] 0 0
Sweden
State/province [75] 0 0
Stockholm
Country [76] 0 0
Turkey
State/province [76] 0 0
Ankara
Country [77] 0 0
Turkey
State/province [77] 0 0
Istanbul
Country [78] 0 0
Turkey
State/province [78] 0 0
Izmir
Country [79] 0 0
Turkey
State/province [79] 0 0
Kocaeli
Country [80] 0 0
Turkey
State/province [80] 0 0
Samsun
Country [81] 0 0
United Kingdom
State/province [81] 0 0
Merseyside
Country [82] 0 0
United Kingdom
State/province [82] 0 0
West Midlands
Country [83] 0 0
United Kingdom
State/province [83] 0 0
London

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Alexion Pharmaceuticals, Inc.
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
No/undecided IPD sharing reason/comment


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

TypeCitations or Other Details
Journal Howard JF Jr, Utsugisawa K, Benatar M, Murai H, Ba... [More Details]


Results are available at https://clinicaltrials.gov/study/NCT01997229