Safety, Tolerability, and Efficacy of Zilucoplan in Subjects With Generalized Myasthenia Gravis
NCT ID: NCT04115293
Last Updated: 2025-07-16
Study Results
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View full resultsBasic Information
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COMPLETED
PHASE3
174 participants
INTERVENTIONAL
2019-09-17
2021-12-30
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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0.3 mg/kg zilucoplan (RA101495)
zilucoplan (RA101495)
Daily subcutaneous (SC) injection
Placebo
Placebo
Daily subcutaneous (SC) injection
Interventions
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zilucoplan (RA101495)
Daily subcutaneous (SC) injection
Placebo
Daily subcutaneous (SC) injection
Eligibility Criteria
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Inclusion Criteria
* Positive serology for acetylcholine receptor (AChR) autoantibodies
* MG-ADL Score of ≥ 6 at Screening and Baseline
* QMG score ≥ 12 at Screening and Baseline
* No change in corticosteroid dose for at least 30 days prior to Baseline or anticipated to occur during the 12-week Treatment Period
* No change in immunosuppressive therapy, including dose, for at least 30 days prior to Baseline or anticipated to occur during the 12-week Treatment Period
Exclusion Criteria
* History of meningococcal disease
* Current or recent systemic infection within 2 weeks prior to Baseline or injection requiring intravenous (IV) antibiotics within 4 weeks prior to Baseline
18 Years
74 Years
ALL
No
Sponsors
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Ra Pharmaceuticals, Inc.
INDUSTRY
Responsible Party
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Principal Investigators
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UCB Cares
Role: STUDY_DIRECTOR
0018445992273 (UCB)
Locations
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Site 41: Diagnostic and Medical Clinic
Mobile, Alabama, United States
Site 116: Neuromuscular Clinic and Research Center
Phoenix, Arizona, United States
Site 4: University of Southern California
Los Angeles, California, United States
Site 31: University of California Irvine
Orange, California, United States
Site 220: Investigator Site
Pasadena, California, United States
Site 160: Forbes Norris MDA/ALS Research and Treatment Center
San Francisco, California, United States
Site 24: Yale University
New Haven, Connecticut, United States
Site 27: George Washington University
Washington D.C., District of Columbia, United States
Site 182: Gelasio Baras Neurology
Miami, Florida, United States
Site 25: University of South Florida
Tampa, Florida, United States
Site 135: Augusta University Medical Center
Augusta, Georgia, United States
Site 176: Hawaii Pacific Neuroscience
Honolulu, Hawaii, United States
Site 188: North Shore Medical Group - Glenview
Glenview, Illinois, United States
Site 156: Indiana University Health Neuroscience Center
Indianapolis, Indiana, United States
Site 32: Kansas University Medical Center Research Institute
Kansas City, Kansas, United States
Site 221: Neurology Center of New England
Foxborough, Massachusetts, United States
Site 33: Detroit medical Center - University Health Center
Detroit, Michigan, United States
Site 49: Michigan State University
East Lansing, Michigan, United States
Site 127: University of Minnesota
Minneapolis, Minnesota, United States
Site 134: Neurology and Sleep Disorders Clinic
Columbia, Missouri, United States
Site 117: Las Vegas Clinic
Las Vegas, Nevada, United States
Site 123: Northwell Health Neuroscience Institute
Great Neck, New York, United States
Site 23: Hospital for Special Surgery
New York, New York, United States
Site 47: Mount Sinai Hospital
New York, New York, United States
Site 22: University of North Carolina
Chapel Hill, North Carolina, United States
Site 15: Duke University
Durham, North Carolina, United States
Site 122: Cleveland Clinic
Cleveland, Ohio, United States
Site 38: Ohio State University
Columbus, Ohio, United States
Site 40: Allegheny Neurological Associates
Pittsburgh, Pennsylvania, United States
Site 128: Medical University of South Carolina
Charleston, South Carolina, United States
Site 185: Neurology Clinic Cordova
Cordova, Tennessee, United States
Site 131: Austin Neuromuscular Center
Austin, Texas, United States
Site 19: University of Texas Southwestern
Dallas, Texas, United States
Site 39: University of Utah
Salt Lake City, Utah, United States
Site 164: University of Virginia Health System
Charlottesville, Virginia, United States
Site 154: University of Washington
Seattle, Washington, United States
Site 45: Center for Neurological Disorders
Milwaukee, Wisconsin, United States
Site 44: London Health Sciences Centre University Hospital
London, Ontario, Canada
Site 11: Montreal Neurological Institute and Hospital (McGill University)
Montreal, Quebec, Canada
Site 191: Centre Hosptitalier Universitaire d'Angers
Angers, , France
Site 204: Centre Hospitalier Régional Universitaire de Lille
Lille, , France
Site 118: Hôpital Pasteur
Nice, , France
Site 105: Pitié-Salpêtrière University Hospital
Paris, , France
Site 137: Les Hôpitaux Universitaires de Strasbourg
Strasbourg, , France
Site 150: Universitätsmedizin Göttingen
Göttingen, , Germany
Site 129: Universitätsklinikum Tübingen
Tübingen, , Germany
Site 126: Fondazione IRCCS Istituto Neurologico Carlo Besta
Milan, , Italy
Site 132: Università Cattolica del Sacro Cuore - Campus di Milano
Roma, , Italy
Site 169: International University of Health and Welfare Narita Hospital
Narita, Chiba, Japan
Site 151: Chiba University Hospital
Chiba, , Japan
Site 136: General Hanamaki Hospital
Iwata, , Japan
Site 179: Kagawa University Hospital - Collagen disease/Rheumatic int
Kita-gun, , Japan
Site 146: Nagasaki University Hospital
Nagasaki, , Japan
Site 152: Hokkaido Medical Center
Sapporo, , Japan
Site 144: Sendai Medical Center
Sendai, , Japan
Site 153: Toho University Ohashi Medical Center
Tokyo, , Japan
Site 163: Tokyo Medical University Hospital
Tokyo, , Japan
Site 141: Keio University Hospital
Tokyo, , Japan
Site 165: Osaka University Hospital
Tokyo, , Japan
Site 140: Haukeland University Hospital / Health Bergen
Bergen, , Norway
Site 143: Oslo Universitetssykehus
Oslo, , Norway
Site 209: Niepubliczny Zakład Opieki Zdrowotnej NEURO - KARD
Poznan, Greater Poland Voivodeship, Poland
Site 192: Krakowski Szpital Specjalistyczny im. Jana Pawła II
Krakow, Lesser Poland Voivodeship, Poland
Site 205: Prywatny Gabinet Lekarski Urszula Chyrchel-Paszkiewicz
Lublin, Lublin Voivodeship, Poland
Site 194: Twoja Przychodnia - Centrum Medyczne Nowa Sól
Nowa Sól, Lubusz Voivodeship, Poland
Site 214: AmiCare Centrum Medyczne
Lodz, Lódzkie, Poland
Site 201: Centrum Medyczne Pratia - Warszawa
Warsaw, Masovian Voivodeship, Poland
Site 195: Wielospecjalistyczna Poradnia Lekarska Synapsis
Katowice, Silesian Voivodeship, Poland
Site 213: Niepubliczny Zakład Opieki Zdrowotnej NOVO-MED
Katowice, Silesian Voivodeship, Poland
Site 193: Krakowska Akademia Neurologii - Centrum Neurologii Kliniczne
Krakow, , Poland
Site 211: Specjalistyczne Gabinety Sp. z o.o.
Krakow, , Poland
Site 210: Clinhouse Centrum Medyczne
Lublin, , Poland
Site 133: Hospital Universitari Vall d'Hebrón
Barcelona, , Spain
Site 168: Hospital de la Santa Creu i Sant Pau
Barcelona, , Spain
Site 138: Hospital Universitario de Basurto
Bilbao, , Spain
Site 119: Oxford University Hospitals NHS Foundation Trust
Oxford, , United Kingdom
Site 130: Royal Hallamshire Hospital
Sheffield, , United Kingdom
Countries
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References
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Howard JF Jr, Bresch S, Genge A, Hewamadduma C, Hinton J, Hussain Y, Juntas-Morales R, Kaminski HJ, Maniaol A, Mantegazza R, Masuda M, Sivakumar K, Smilowski M, Utsugisawa K, Vu T, Weiss MD, Zajda M, Boroojerdi B, Brock M, de la Borderie G, Duda PW, Lowcock R, Vanderkelen M, Leite MI; RAISE Study Team. Safety and efficacy of zilucoplan in patients with generalised myasthenia gravis (RAISE): a randomised, double-blind, placebo-controlled, phase 3 study. Lancet Neurol. 2023 May;22(5):395-406. doi: 10.1016/S1474-4422(23)00080-7.
Weiss MD, Freimer M, Leite MI, Maniaol A, Utsugisawa K, Bloemers J, Boroojerdi B, Howard E, Savic N, Howard JF Jr. Improvement of fatigue in generalised myasthenia gravis with zilucoplan. J Neurol. 2024 May;271(5):2758-2767. doi: 10.1007/s00415-024-12209-3. Epub 2024 Feb 24.
de la Borderie G, Chimits D, Boroojerdi B, Brock M, Duda PW, Grimson F, Mahoney P, Strimenopoulou F, Cutter G, Aban I, Brauner S, Petersson M, Howard JF Jr, Bennett N. Maintenance of zilucoplan efficacy in patients with generalised myasthenia gravis up to 24 weeks: a model-informed analysis. Ther Adv Neurol Disord. 2024 Sep 21;17:17562864241279125. doi: 10.1177/17562864241279125. eCollection 2024.
Hewamadduma C, Freimer M, Genge A, Leite MI, Utsugisawa K, Vu T, Boroojerdi B, Grimson F, Savic N, Vanderkelen M, Howard JF Jr; RAISE-XT study team. Changes in corticosteroid and non-steroidal immunosuppressive therapy with long-term zilucoplan treatment in generalized myasthenia gravis. J Neurol. 2025 Jun 12;272(7):457. doi: 10.1007/s00415-025-13113-0.
Provided Documents
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Document Type: Study Protocol
Document Type: Statistical Analysis Plan
Other Identifiers
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RA101495-02.301
Identifier Type: -
Identifier Source: org_study_id
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