Extension Study of ECU-MG-301 to Evaluate Safety and Efficacy of Eculizumab in Refractory Generalized Myasthenia Gravis
NCT ID: NCT02301624
Last Updated: 2020-02-05
Study Results
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View full resultsBasic Information
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COMPLETED
PHASE3
117 participants
INTERVENTIONAL
2014-11-12
2019-01-15
Brief Summary
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Detailed Description
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After receiving blinded study treatment (eculizumab or placebo) in Study ECU-MG-301 for 26 weeks, participants were eligible to enroll in the ECU-MG-302 extension study. Participants were to enter Study ECU-MG-302 within 2 weeks after completing their Week 26 visit in Study ECU-MG-301.
Study ECU-MG-302 consisted of a 4-week Blind Induction Phase to preserve the blinded nature of Study ECU-MG-301, an Open-Label Maintenance Phase (up to 4 years), and a Safety Follow-up visit 8 weeks after the last dose for participants who withdrew from the study or discontinued eculizumab treatment at any time and for any reason after receiving any amount of eculizumab.
Conditions
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Study Design
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NON_RANDOMIZED
PARALLEL
TREATMENT
NONE
Study Groups
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Eculizumab/Eculizumab
Blind 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.
Eculizumab
Intravenous administration of eculizumab.
Placebo
Intravenous administration of matching placebo. Participants received placebo only during the Blind Induction Phase.
Placebo/Eculizumab
Blind 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.
Eculizumab
Intravenous administration of eculizumab.
Placebo
Intravenous administration of matching placebo. Participants received placebo only during the Blind Induction Phase.
Interventions
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Eculizumab
Intravenous administration of eculizumab.
Placebo
Intravenous administration of matching placebo. Participants received placebo only during the Blind Induction Phase.
Eligibility Criteria
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Inclusion Criteria
2. Participant has given written informed consent.
3. Participant was willing and able to comply with the protocol requirements for the duration of the study.
4. 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.
Exclusion Criteria
2. Female participants who were pregnant, breastfeeding, or intended to conceive during the course of the study.
3. Unresolved meningococcal infection
4. Hypersensitivity to murine proteins or to one of the excipients of eculizumab
5. 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.
18 Years
ALL
No
Sponsors
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Alexion Pharmaceuticals, Inc.
INDUSTRY
Responsible Party
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Principal Investigators
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Marcus Yountz, MD
Role: STUDY_DIRECTOR
Alexion Pharmaceuticals, Inc.
Locations
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Birmingham, Alabama, United States
Los Angeles, California, United States
Orange, California, United States
Palo Alto, California, United States
San Francisco, California, United States
New Haven, Connecticut, United States
Jacksonville, Florida, United States
Miami, Florida, United States
Tampa, Florida, United States
Springfield, Illinois, United States
Indianapolis, Indiana, United States
Iowa City, Iowa, United States
Kansas City, Kansas, United States
Baltimore, Maryland, United States
Baltimore, Maryland, United States
Boston, Massachusetts, United States
Burlington, Massachusetts, United States
Las Vegas, Nevada, United States
Chapel Hill, North Carolina, United States
Charlotte, North Carolina, United States
Durham, North Carolina, United States
Columbus, Ohio, United States
Portland, Oregon, United States
San Antonio, Texas, United States
Burlington, Vermont, United States
Seattle, Washington, United States
Buenos Aires, , Argentina
Edegem, , Belgium
Ghent, , Belgium
Leuven, , Belgium
São Paulo, , Brazil
Edmonton, , Canada
Ostrava - Poruba, , Czechia
Prague, , Czechia
Arhus C, , Denmark
Copenhagen, , Denmark
Turku, , Finland
Szeged, , Hungary
Milan, , Italy
Napoli, , Italy
Roma, , Italy
Chiba, , Japan
Fukuoka, , Japan
Hanamaki, , Japan
Hokkaido, , Japan
Miyagi, , Japan
Nagasaki, , Japan
Osaka, , Japan
Osaka-Fu, , Japan
Amsterdam, , Netherlands
Seoul, , South Korea
Barcelona, , Spain
Madrid, , Spain
Stockholm, , Sweden
Ankara, , Turkey (Türkiye)
Izmir, , Turkey (Türkiye)
Kocaeli, , Turkey (Türkiye)
Birmingham, , United Kingdom
Liverpool, , United Kingdom
London, , United Kingdom
Countries
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References
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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.
Andersen 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.
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.
Murai 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.
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.
Murai 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.
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.
Provided Documents
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Document Type: Study Protocol
Document Type: Statistical Analysis Plan
Other Identifiers
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2013-002191-41
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
ECU-MG-302
Identifier Type: -
Identifier Source: org_study_id
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