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Background <p>In the Phase 3 MycarinG study (NCT03971422), six once-weekly subcutaneous infusions of rozanolixizumab significantly improved myasthenia gravis (MG)-specific outcomes versus placebo in patients with acetylcholine receptor or muscle-specific tyrosine kinase autoantibody-positive generalized MG (gMG). Following completion of MycarinG, patients could enroll in the open-label extension MG0004 study (NCT04124965) to receive chronic weekly rozanolixizumab.</p> Methods <p>Patients were re-randomized 1:1 to once-weekly rozanolixizumab 7 or 10&#xa0;mg/kg for up to 52 infusions. The primary endpoints were the occurrence of treatment-emergent adverse events (TEAEs) and TEAEs leading to rozanolixizumab discontinuation. After ≥6 visits/infusions patients could switch to the MG0007 study (NCT04650854) to receive cyclic rozanolixizumab treatment.</p> Results <p>In MG0004, 70 patients received rozanolixizumab 7&#xa0;mg/kg (<i>n</i> = 35) or 10&#xa0;mg/kg (<i>n</i> = 35). Mean treatment duration was 22.9 and 23.7&#xa0;weeks, respectively, due to rollover into MG0007. TEAEs were reported in 60/70 (85.7%) patients; most were mild/moderate. The most frequently reported TEAEs were headache (25/70 [35.7%]), diarrhea (13/70 [18.6%]) and decreased blood immunoglobulin G (11/70 [15.7%]). There were no opportunistic, serious or severe infections, serious or severe hypersensitivity or injection-site reactions, any anaphylactic reactions or albumin or lipid abnormalities. Maximum mean reduction from baseline in MG Activities of Daily Living score was 3.1 in the 7&#xa0;mg/kg group and 4.1 in the 10&#xa0;mg/kg group.</p> Conclusion <p>Chronic weekly rozanolixizumab for up to 52 infusions was generally well tolerated, and clinically relevant improvements across MG-specific outcomes were maintained, supporting the long-term use of rozanolixizumab in patients with gMG.</p> Trial registration <p>NCT04124965 (registered October 11, 2019).</p>

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Safety and efficacy of chronic weekly rozanolixizumab in generalized myasthenia gravis: the randomized open-label extension MG0004 study

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  • 发表日期：

    2025年3月19日

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  • Vera Bril,
  • Artur Drużdż,
  • Julian Grosskreutz,
  • Ali A. Habib,
  • Henry J. Kaminski,
  • Renato Mantegazza,
  • Sabrina Sacconi,
  • Kimiaki Utsugisawa,
  • Tuan Vu,
  • Marion Boehnlein,
  • Maryam Gayfieva,
  • Bernhard Greve,
  • Franz Woltering,
  • John Vissing,
  • Rodrigo Álvarez-Velasco,
  • Radwa Aly,
  • Henning Andersen,
  • Giovanni Antonini,
  • Aramide Balogun,
  • Ruggero Barnabei,
  • Said Beydoun,
  • Franz Blaes,
  • Silvia Bonarino,
  • Anna Boss Soevang,
  • Nazibrola Botchorishvili,
  • Stephan A. Botez,
  • Ivo Bozovic,
  • Paulina Budzinska,
  • Pietro Businaro,
  • Lucia Campetella,
  • Ana Belen Cánovas,
  • Carlos Casasnovas,
  • Hou-Chang Chiu,
  • His-Chieh Chou,
  • Adam Comer,
  • Elena Cortés Vicente,
  • Roberto D’Angelo,
  • Lubna Daniyal,
  • Annie Dionne,
  • Péter Diószeghy,
  • Laura Fionda,
  • Denis Flemm,
  • Rita Frangiamore,
  • Manuela Gambella,
  • Rachana K. Gandhi Mehta,
  • Matteo Garibaldi,
  • Matteo Gastaldi,
  • Christian Geis,
  • Hannah George,
  • Stefan Gingele,
  • Monica Grau Martin,
  • Yuh-Cherng Guo,
  • Gerardo Gutiérrez Gutiérrez,
  • Francesco Habetswallner,
  • Lina Hassoun,
  • Sonja Holm-Yildiz,
  • Faraz Hussain,
  • Francisca Iniesta,
  • Viktoriya Irodenko,
  • Marina Janelidze,
  • Min Kang,
  • Chafic Karam,
  • Denis Korobko,
  • Sergey Kotov,
  • Michal Kretkowski,
  • Nana Kvirkvelia,
  • Antonio Lauletta,
  • Yi-Chung Lee,
  • Luca Leonardi,
  • Kore Liow,
  • Arnau Llauradó Gayete,
  • Sara Llufriu,
  • Catherine Lomen-Hoerth,
  • Jan D. Lünemann,
  • Lorenzo Maggi,
  • Eugenia Martínez Hernández,
  • Gianvito Masi,
  • Marion Masingue,
  • Rami Massie,
  • Marco Masullo,
  • Federico Mazzacane,
  • Nora Möhn,
  • Stefania Morino,
  • Kelsey Moulton,
  • Tahseen Mozaffar,
  • Elene Nebadze,
  • Velina Nedkova-Hristova,
  • Eduardo Ng,
  • Ekaterina Novikova,
  • Izabella Obál,
  • Anita Palsgård,
  • Claudia Papi,
  • Lorena Pérez,
  • Stojan Peric,
  • Mikhail Petrov,
  • Nicolai Rasmus Preisler,
  • Giorgia Querin,
  • Konrad Rejdak,
  • Kourosh Rezania,
  • Elena Rinaldi,
  • Rita Rinaldi,
  • Michael H. Rivner,
  • Annekathrin Roediger,
  • Laura Rosow,
  • Simone Rossi,
  • Elena Rossini,
  • Stephen Ryan,
  • Lotte Sahin Levison,
  • Albert Saiz,
  • Maria Salvado,
  • Daniel Sánchez-Tejerina,
  • Margret Schwarz,
  • María Sepúlveda,
  • Khema R. Sharma,
  • Sheetal Shroff,
  • Olga Sidorova,
  • Guilhem Solé,
  • Javier Sotoca,
  • Mads Stemmerik,
  • Aleksandar Stojanov,
  • Tanya Stojkovic,
  • Kai Su,
  • Sebastian Szklener,
  • Alexander Tsiskaridze,
  • Laura Tufano,
  • Michaela Tyblova,
  • Eiko Uenaka,
  • Astrid Unterlauft,
  • Gabriel Valero,
  • Fiammetta Vanoli,
  • Tamar Vashadze,
  • Nuria Vidal Fernández,
  • Marie-Hélène Violleau,
  • Nicolas Weiss,
  • Nanna Witting,
  • Jiann-Horng Yeh,
  • Leila Zaidi,
  • Leonid Zaslavskiy,
  • Jana Zschüntzsch

摘要

Background

In the Phase 3 MycarinG study (NCT03971422), six once-weekly subcutaneous infusions of rozanolixizumab significantly improved myasthenia gravis (MG)-specific outcomes versus placebo in patients with acetylcholine receptor or muscle-specific tyrosine kinase autoantibody-positive generalized MG (gMG). Following completion of MycarinG, patients could enroll in the open-label extension MG0004 study (NCT04124965) to receive chronic weekly rozanolixizumab.

Methods

Patients were re-randomized 1:1 to once-weekly rozanolixizumab 7 or 10 mg/kg for up to 52 infusions. The primary endpoints were the occurrence of treatment-emergent adverse events (TEAEs) and TEAEs leading to rozanolixizumab discontinuation. After ≥6 visits/infusions patients could switch to the MG0007 study (NCT04650854) to receive cyclic rozanolixizumab treatment.

Results

In MG0004, 70 patients received rozanolixizumab 7 mg/kg (n = 35) or 10 mg/kg (n = 35). Mean treatment duration was 22.9 and 23.7 weeks, respectively, due to rollover into MG0007. TEAEs were reported in 60/70 (85.7%) patients; most were mild/moderate. The most frequently reported TEAEs were headache (25/70 [35.7%]), diarrhea (13/70 [18.6%]) and decreased blood immunoglobulin G (11/70 [15.7%]). There were no opportunistic, serious or severe infections, serious or severe hypersensitivity or injection-site reactions, any anaphylactic reactions or albumin or lipid abnormalities. Maximum mean reduction from baseline in MG Activities of Daily Living score was 3.1 in the 7 mg/kg group and 4.1 in the 10 mg/kg group.

Conclusion

Chronic weekly rozanolixizumab for up to 52 infusions was generally well tolerated, and clinically relevant improvements across MG-specific outcomes were maintained, supporting the long-term use of rozanolixizumab in patients with gMG.

Trial registration

NCT04124965 (registered October 11, 2019).