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).
Safety and efficacy of chronic weekly rozanolixizumab in generalized myasthenia gravis: the randomized open-label extension MG0004 study / Bril, Vera; Drużdż, Artur; Grosskreutz, Julian; Habib, Ali A.; Kaminski, Henry J.; Mantegazza, Renato; Sacconi, Sabrina; Utsugisawa, Kimiaki; Vu, Tuan; Boehnlein, Marion; Gayfieva, Maryam; Greve, Bernhard; Woltering, Franz; Vissing, John; Null, Null; Álvarez-Velasco, Rodrigo; Aly, Radwa; Andersen, Henning; Antonini, Giovanni; Balogun, Aramide; Barnabei, Ruggero; Beydoun, Said; Blaes, Franz; Bonarino, Silvia; Soevang, Anna Boss; Botchorishvili, Nazibrola; Botez, Stephan A.; Bozovic, Ivo; Budzinska, Paulina; Businaro, Pietro; Campetella, Lucia; Cánovas, Ana Belen; Casasnovas, Carlos; Chiu, Hou-Chang; Chou, His-Chieh; Comer, Adam; Vicente, Elena Cortés; D'Angelo, Roberto; Daniyal, Lubna; Dionne, Annie; Diószeghy, Péter; Fionda, Laura; Flemm, Denis; Frangiamore, Rita; Gambella, Manuela; Mehta, Rachana K. Gandhi; Garibaldi, Matteo; Gastaldi, Matteo; Geis, Christian; George, Hannah; Gingele, Stefan; Martin, Monica Grau; Guo, Yuh-Cherng; Gutiérrez Gutiérrez, Gerardo; Habetswallner, Francesco; Hassoun, Lina; Holm-Yildiz, Sonja; Hussain, Faraz; Iniesta, Francisca; Irodenko, Viktoriya; Janelidze, Marina; Kang, Min; Karam, Chafic; Korobko, Denis; Kotov, Sergey; Kretkowski, Michal; Kvirkvelia, Nana; Lauletta, Antonio; Lee, Yi-Chung; Leonardi, Luca; Liow, Kore; Gayete, Arnau Llauradó; Llufriu, Sara; Lomen-Hoerth, Catherine; Lünemann, Jan D.; Maggi, Lorenzo; Hernández, Eugenia Martínez; Masi, Gianvito; Masingue, Marion; Massie, Rami; Masullo, Marco; Mazzacane, Federico; Möhn, Nora; Morino, Stefania; Moulton, Kelsey; Mozaffar, Tahseen; Nebadze, Elene; Nedkova-Hristova, Velina; Ng, Eduardo; Novikova, Ekaterina; Obál, Izabella; Palsgård, Anita; Papi, Claudia; Pérez, Lorena; Peric, Stojan; Petrov, Mikhail; Preisler, Nicolai Rasmus; Querin, Giorgia; Rejdak, Konrad; Rezania, Kourosh; Rinaldi, Elena; Rinaldi, Rita; Rivner, Michael H.; Roediger, Annekathrin; Rosow, Laura; Rossi, Simone; Rossini, Elena; Ryan, Stephen; Levison, Lotte Sahin; Saiz, Albert; Salvado, Maria; Sánchez-Tejerina, Daniel; Schwarz, Margret; Sepúlveda, María; Sharma, Khema R.; Shroff, Sheetal; Sidorova, Olga; Solé, Guilhem; Sotoca, Javier; Stemmerik, Mads; Stojanov, Aleksandar; Stojkovic, Tanya; Su, Kai; Szklener, Sebastian; Tsiskaridze, Alexander; Tufano, Laura; Tyblova, Michaela; Uenaka, Eiko; Unterlauft, Astrid; Valero, Gabriel; Vanoli, Fiammetta; Vashadze, Tamar; Fernández, Nuria Vidal; Violleau, Marie-Hélène; Weiss, Nicolas; Witting, Nanna; Yeh, Jiann-Horng; Zaidi, Leila; Zaslavskiy, Leonid; Zschüntzsch, Jana. - In: JOURNAL OF NEUROLOGY. - ISSN 0340-5354. - 272:4(2025). [10.1007/s00415-025-12958-9]
Safety and efficacy of chronic weekly rozanolixizumab in generalized myasthenia gravis: the randomized open-label extension MG0004 study
Antonini, Giovanni;Fionda, Laura;Garibaldi, Matteo;Lauletta, Antonio;Leonardi, Luca;Morino, Stefania;Rossini, Elena;Tufano, Laura;Vanoli, Fiammetta;
2025
Abstract
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).| File | Dimensione | Formato | |
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68. Safety and efficacy of chronic weekly rozanolixizumab in generalized myasthenia gravis- the randomized open-label extension MG0004 study.pdf
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