Extension Study of ECU-MG-301 to Evaluate Safety and Efficacy of Eculizumab in Refractory Generalized Myasthenia Gravis
A Phase III, Open-label Extension Trial of ECU-MG-301 to Evaluate the Safety and Efficacy of Eculizumab in Subjects With Refractory Generalized Myasthenia Gravis (gMG)
2 other identifiers
interventional
117
17 countries
60
Brief Summary
To evaluate the safety and efficacy of eculizumab in the treatment of refractory generalized myasthenia gravis (gMG) as an extension study for the participants who previously completed Study ECU-MG-301(NCT01997229).
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P25-P50 for phase_3
Started Nov 2014
Typical duration for phase_3
60 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
Study Start
First participant enrolled
November 12, 2014
CompletedFirst Submitted
Initial submission to the registry
November 22, 2014
CompletedFirst Posted
Study publicly available on registry
November 26, 2014
CompletedPrimary Completion
Last participant's last visit for primary outcome
January 15, 2019
CompletedStudy Completion
Last participant's last visit for all outcomes
January 15, 2019
CompletedResults Posted
Study results publicly available
February 5, 2020
CompletedFebruary 5, 2020
January 1, 2020
4.2 years
November 22, 2014
December 9, 2019
January 27, 2020
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Count Of Participants With Treatment-Emergent Adverse Events
Treatment-emergent adverse events (TEAEs) are adverse events with onset on or after the first study drug dose in Study ECU-MG-302. Likewise, treatment-emergent serious adverse events (TESAEs) are serious adverse events that onset on or after the first study drug dose in Study ECU-MG-302. A summary of serious and all other non-serious adverse events, regardless of causality, is located in the Reported Adverse Events module.
Day 1 (after dosing) through End of Study (Week 208)
Secondary Outcomes (1)
Change From Baseline In Myasthenia Gravis Activities Of Daily Living Profile (MG-ADL) Total Score At Week 4 And Week 130
Baseline, Week 4 and Week 130
Study Arms (2)
Eculizumab/Eculizumab
EXPERIMENTALBlind Induction Phase: Participants who had received blinded treatment with eculizumab in Study ECU-MG-301 were administered eculizumab (4 vials/1200 milligrams \[mg\]) on Day 1 and Week 2 and placebo (4 vials/0 mg) at Weeks 1 and 3. Open-Label Maintenance Phase: Participants received open-label eculizumab (4 vials/1200 mg) every 2 weeks starting at Week 4 and continued throughout the study. Eculizumab 1200 mg was administered for up to 4 years in this extension study.
Placebo/Eculizumab
EXPERIMENTALBlind Induction Phase: Participants who had received blinded treatment with placebo in Study ECU-MG-301 were administered eculizumab/placebo (3 vials/900 mg, plus 1 vial/0 mg, respectively) on Day 1 and Weeks 1 through 3. Open-Label Maintenance Phase: Participants received open-label eculizumab (4 vials/1200 mg) every 2 weeks starting at Week 4 and continued throughout the study. Eculizumab 1200 mg was administered for up to 4 years in this extension study.
Interventions
Intravenous administration of eculizumab.
Intravenous administration of matching placebo. Participants received placebo only during the Blind Induction Phase.
Eligibility Criteria
You may qualify if:
- Participant has completed Study ECU-MG-301.
- Participant has given written informed consent.
- Participant was willing and able to comply with the protocol requirements for the duration of the study.
- Female participant of childbearing potential must have had a negative pregnancy test (serum human chorionic gonadotropin). All participants were required to practice an effective, reliable, and medically approved contraceptive regimen during the study and for up to 5 months following discontinuation of treatment.
You may not qualify if:
- Participants who withdrew from Study ECU-MG-301 as a result of an adverse event related to study drug.
- Female participants who were pregnant, breastfeeding, or intended to conceive during the course of the study.
- Unresolved meningococcal infection
- Hypersensitivity to murine proteins or to one of the excipients of eculizumab
- Any medical condition or circumstances that, in the opinion of the investigator, might have interfered with the participant's participation in the study, posed any added risk for the participant, or confounded the assessment of the participants.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (60)
Unknown Facility
Birmingham, Alabama, 35233, United States
Unknown Facility
Los Angeles, California, 90033, United States
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Orange, California, 92868, United States
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Palo Alto, California, 94304, United States
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San Francisco, California, 94115, United States
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New Haven, Connecticut, 06519, United States
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Jacksonville, Florida, 32209, United States
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Miami, Florida, 33136, United States
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Tampa, Florida, 33612, United States
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Springfield, Illinois, 62702, United States
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Indianapolis, Indiana, 46202, United States
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Iowa City, Iowa, 52242, United States
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Kansas City, Kansas, 66160, United States
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Baltimore, Maryland, 21201-1595, United States
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Baltimore, Maryland, 21287-0876, United States
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Boston, Massachusetts, 02115, United States
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Burlington, Massachusetts, 01805, United States
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Las Vegas, Nevada, 89145, United States
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Chapel Hill, North Carolina, 27599, United States
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Charlotte, North Carolina, 28207, United States
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Durham, North Carolina, 27710, United States
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Columbus, Ohio, 43210, United States
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Portland, Oregon, 97239, United States
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San Antonio, Texas, 78229, United States
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Burlington, Vermont, 05401, United States
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Seattle, Washington, 98195, United States
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Buenos Aires, Argentina
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Edegem, Belgium
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Ghent, Belgium
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Leuven, Belgium
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São Paulo, Brazil
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Edmonton, Canada
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Ostrava - Poruba, Czechia
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Prague, Czechia
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Arhus C, Denmark
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Copenhagen, Denmark
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Turku, Finland
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Szeged, Hungary
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Milan, Italy
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Napoli, Italy
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Roma, Italy
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Chiba, Japan
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Fukuoka, Japan
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Hanamaki, Japan
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Hokkaido, Japan
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Miyagi, Japan
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Nagasaki, Japan
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Osaka, Japan
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Osaka-Fu, Japan
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Amsterdam, Netherlands
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Seoul, South Korea
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Barcelona, Spain
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Madrid, Spain
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Stockholm, Sweden
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Ankara, Turkey (Türkiye)
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Izmir, Turkey (Türkiye)
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Kocaeli, Turkey (Türkiye)
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Birmingham, United Kingdom
Unknown Facility
Liverpool, United Kingdom
Unknown Facility
London, United Kingdom
Related Publications (11)
Muppidi S, Utsugisawa K, Benatar M, Murai H, Barohn RJ, Illa I, Jacob S, Vissing J, Burns TM, Kissel JT, Nowak RJ, Andersen H, Casasnovas C, de Bleecker JL, Vu TH, Mantegazza R, O'Brien FL, Wang JJ, Fujita KP, Howard JF Jr; Regain Study Group. Long-term safety and efficacy of eculizumab in generalized myasthenia gravis. Muscle Nerve. 2019 Jul;60(1):14-24. doi: 10.1002/mus.26447. Epub 2019 Mar 8.
PMID: 30767274RESULTAndersen H, Mantegazza R, Wang JJ, O'Brien F, Patra K, Howard JF Jr; REGAIN Study Group. Correction to: Eculizumab improves fatigue in refractory generalized myasthenia gravis. Qual Life Res. 2019 Aug;28(8):2255. doi: 10.1007/s11136-019-02204-x.
PMID: 31115842RESULTAndersen 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.
PMID: 30905021RESULTMurai H, Uzawa A, Suzuki Y, Imai T, Shiraishi H, Suzuki H, Okumura M, O'Brien F, Wang JJ, Fujita KP, Utsugisawa K; REGAIN Study Group. Long-term efficacy and safety of eculizumab in Japanese patients with generalized myasthenia gravis: A subgroup analysis of the REGAIN open-label extension study. J Neurol Sci. 2019 Dec 15;407:116419. doi: 10.1016/j.jns.2019.08.004. Epub 2019 Aug 3.
PMID: 31698177RESULTSiddiqi 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.
PMID: 34590717DERIVEDMurai H, Suzuki S, Hasebe M, Fukamizu Y, Rodrigues E, Utsugisawa K. Safety and effectiveness of eculizumab in Japanese patients with generalized myasthenia gravis: interim analysis of post-marketing surveillance. Ther Adv Neurol Disord. 2021 Mar 16;14:17562864211001995. doi: 10.1177/17562864211001995. eCollection 2021.
PMID: 33796147DERIVEDNowak 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.
PMID: 33329303DERIVEDMantegazza 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.
PMID: 33229455DERIVEDMantegazza 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.
PMID: 32700461DERIVEDJacob 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.
PMID: 32426038DERIVEDVissing 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.
PMID: 32189108DERIVED
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Results Point of Contact
- Title
- Alexion Pharmaceuticals Inc.
- Organization
- Alexion Pharmaceuticals Inc.
Study Officials
- STUDY DIRECTOR
Marcus Yountz, MD
Alexion Pharmaceuticals, Inc.
Publication Agreements
- PI is Sponsor Employee
- No
- Restriction Type
- OTHER
- Restrictive Agreement
- Yes
Study Design
- Study Type
- interventional
- Phase
- phase 3
- Allocation
- NON RANDOMIZED
- Masking
- NONE
- Masking Details
- The study consisted of a 4-week Blind Induction Phase to preserve the blinded nature of Study ECU-MG-301, followed by an Open-Label Maintenance Phase.
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
November 22, 2014
First Posted
November 26, 2014
Study Start
November 12, 2014
Primary Completion
January 15, 2019
Study Completion
January 15, 2019
Last Updated
February 5, 2020
Results First Posted
February 5, 2020
Record last verified: 2020-01