Safety and efficacy of rozanolixizumab in patients with generalised myasthenia gravis (MycarinG): a randomised, double-blind, placebo-controlled, adaptive phase 3 study

Denny J. Padgett | Adam L. Comer | Yi-Chung Lee | J. Lünemann | S. Demeret | T. Hatano | H. Wiendl | M. Janelidze | T. Stojkovic | J. Vissing | Shigeaki Suzuki | R. Mantegazza | Yuka Komatsu | C. Karam | K. Lai | A. Saiz | N. Witting | K. Sharma | L. Maggi | Z. Siddiqi | A. Uzawa | Yumi Yokota | S. Sacconi | Elena Rinaldi | S. Szklener | K. Rejdak | V. Bril | B. Stolte | T. Hagenacker | A. Nadaj-Pakleza | N. Izawa | R. Rinaldi | M. Boehnlein | C. Tranchant | M. Týblová | I. Bozovic | D. Lavrnić | L. Klotz | H. Kaminski | K. Sekiguchi | H. Chiu | F. Bergh | J. Caress | Marie-Hélène Violleau | A. Genge | R. Morales | S. Beydoun | G. Solé | F. Woltering | T. Mozaffar | K. Utsugisawa | Y. Nagane | T. Vu | Masanori Takahashi | N. Chihara | F. Blaes | Christian Geis | Sara J. Byerly | A. Tsiskaridze | G. Querin | S. Kotov | G. Djordjević | S. Botez | R. Massie | G. Matte | J. Yeh | F. Habetswallner | B. Greve | A. Bozorg | C. Casasnovas | P. Diószeghy | K. Rezania | T. Kubota | J. M. Lallana | L. Magy | L. Fionda | G. Antonini | J. Chanson | K. Ghorab | C. Bodkin | R. Pascuzzi | Jason R. Thonhoff | Y. Oji | Yuh-Cherng Guo | Rikizo Matsumoto | D. Korobko | R. Iorio | S. Mathis | A. Habib | Aleksandar Stojanov | Aleksa Palibrk | Rachana Mehta | Michael H. Rivner | Shigeki Ohashi | K. Bender | N. Malkova | N. Nakamura | Y. Morgado | J. Zschüntzsch | E. Vicente | D. Blackmore | Mei-Fen Lee | M. Gastaldi | A. Dionne | Brandy M Quarles | M. Takaki | M. Figueras | S. Yoshimura | A. Brown | M. Gambella | R. Aly | E. Naddaf | L. Zaslavskiy | A. Totzeck | F. Tuccillo | T. Berkowicz | Ya Hui Chen | Zabeen K. Mahuwala | K. Tsukita | Yasushi Suzuki | M. Cavalli | M. Alcantara | M. Masingue | E. Ng | S. Glaubitz | M. Masuda | C. Lomen‐Hoerth | M. Hałas | Y. Tomizawa | Tomoaki Shima | Teiichiro Miyazaki | Takuro Hirayama | C. Franklin | E. Novikova | S. Shroff | T. Ueda | Pietro Businaro | M. Yasuda | V. Nedkova-Hristova | Y. Okubo | G. Gutiérrez | Rachel Zeng | M. Jakubíková | Kei Ishizuchi | I. Obál | Sara Klein | A. Drużdż | G. Watanabe | M. Stemmerik | Kazumasa Yokoyama | Ritesh Raman | N. Ido | Makiko Naito | Pranali Ravikumar | P. Pavlakis | S. Perić | Y. Ozawa | Jennifer M. Martinez-Thompson | A. Nagaoka | Lucy Lam | Anna Lammerskitten | Annekathrin Roediger | Shara Holzberg | Naoki Yamamoto | O. (Sidorova) | Eiko Uenaka | D. Lange | S. Akhter | Christine Bindler | J. Junkerová | Tomoko Tsuda | Angela Micheels | A. Kalb | K. Higashi | M. Bérubé | Yosuke Onishi | K. Kummer | F. Mazzacane | Stefanie Meyer | N. Okamoto | Alisha Thomas | Viktoriya S. Irodenko | Simone Rossi | S. Holm-Yildiz | P. Isabekova | L. Ashworth | M. Nakayama | Kazuki Sato | Faraz S. Hussain | L. Guennec | T. Alekseeva | Y. Fujisawa | Yuri Fukushige | Hiroyuki Naitō | H. Andersen | Jens Schmidt | S. Akita | Lubna Daniyal | Kai-Hua Su | S. Mannan | Temur Margania | Kanako Minemoto | Yuka Takematsu | I. Hernandez | Paulina Budzińska | Hiromi Yamada | D. Padgett | Evelyn Sarpong | Seiko Yoshida | Luis Querol Gutiérrez | Paqui Iniesta | H. George | Renee P Wagner | G. Miller | V. Goldobin | L. Rosow | Akira Tsujino | Julian Grosskreutz | M. Gayfieva | Sihame Messai | Arnaud Verloes | Leila Zaidi | Nicolas Weiss | I. Rukhadze | Eike Hänsel | Bianca Meilick | Mandy Reuschel | Lars-Malte Teußer | A. Unterlauft | C. Goedel | Vasilios Tsoutsikas | Margret Schwarz | Haruna Umehara | Makoto Kinoshita | Emi Yoneda | Saeka Fujita | Masami Ono | Sana Yamamoto | Kazuki Oikoshi | Miki Sanko | Rune Tsuji | Yumi Hyodo | Akiko Minohara | Nana Haraguchi | Mari Kirimura | Emi Sunagawa | Ayaka Suzuki | Aozora Wada | Mitsuo Yata | Emiko Kawasaki | Hirohiko Ono | Kameda Ayumi | Kumiko Kuwada | Monika Kitowska | Kandyda Derkacz | E. Skornyakova | N. Fernández | Anna Canovas Segura | Daniel Tejerina | Sara Llufriú Durán | María Sepúlveda Gázquez | Eugenia María Martínez Hernández | Ashwathy Goutham | K. Bond | Joanne Buckner | Jessyca Clemons | Asha Farmer | Summer Harris | M. Hiatt | Lynn Smith | Rose-Ann Smith | Brian Strittmatter | K. Moulton | Carol Denny | Bart Hanson | Eric Sorenson | Jane L Sultze | Delena Weis | Gelasio Baras | Stephen Ryan | K. Liow | Pantelis P. Pavlakis | M. Violleau | Isela Hernández | Naoko Okamoto | J. Grosskreutz | M. Rivner | Catherine lomen-Hoerth | Takehiro Ueda | Maryam Gayfieva | Denis Korobko | Velina Nedkova-Hristova | Jane L. Sultze

[1]  A. Regnault,et al.  Development of the Myasthenia Gravis (MG) Symptoms PRO: a case study of a patient-centred outcome measure in rare disease , 2021, Orphanet Journal of Rare Diseases.

[2]  N. Law,et al.  The Lived Experience of Myasthenia Gravis: A Patient-Led Analysis , 2021, Neurology and Therapy.

[3]  J. Statland,et al.  Safety, efficacy, and tolerability of efgartigimod in patients with generalised myasthenia gravis (ADAPT): a multicentre, randomised, placebo-controlled, phase 3 trial , 2021, The Lancet Neurology.

[4]  Shigeaki Suzuki,et al.  Health‐related quality of life and treatment strategies in myasthenia gravis , 2020, Clinical and Experimental Neuroimmunology.

[5]  C. Rodolico,et al.  MuSK-Associated Myasthenia Gravis: Clinical Features and Management , 2020, Frontiers in Neurology.

[6]  J. Murthy Myasthenic Crisis – Comorbidities, Complications, Long-Term Outcomes: The Challenges , 2019, Annals of Indian Academy of Neurology.

[7]  G. Cutter,et al.  Examining the Impact of Refractory Myasthenia Gravis on Healthcare Resource Utilization in the United States: Analysis of a Myasthenia Gravis Foundation of America Patient Registry Sample , 2019, Journal of clinical neurology.

[8]  P. van Damme,et al.  Randomized phase 2 study of FcRn antagonist efgartigimod in generalized myasthenia gravis , 2019, Neurology.

[9]  N. Gilhus,et al.  Myasthenia gravis , 2019, Nature Reviews Disease Primers.

[10]  C. Meier,et al.  Generation and characterization of a high affinity anti-human FcRn antibody, rozanolixizumab, and the effects of different molecular formats on the reduction of plasma IgG concentration , 2018, mAbs.

[11]  D. Sanders,et al.  Randomized study of adjunctive belimumab in participants with generalized myasthenia gravis , 2018, Neurology.

[12]  P. Urban,et al.  Treatment Standards and Individualized Therapy of Myasthenia Gravis , 2018, Neurology International Open.

[13]  J. Howard Myasthenia gravis: the role of complement at the neuromuscular junction , 2018, Annals of the New York Academy of Sciences.

[14]  G. L. Masson,et al.  Safety and efficacy of eculizumab in anti-acetylcholine receptor antibody-positive refractory generalised myasthenia gravis (REGAIN): a phase 3, randomised, double-blind, placebo-controlled, multicentre study. , 2017, The Lancet. Neurology.

[15]  J. Pines,et al.  Inpatient cost analysis for treatment of myasthenia gravis , 2017, Muscle & nerve.

[16]  M. Benatar,et al.  International consensus guidance for management of myasthenia gravis , 2016, Neurology.

[17]  A. Vincent,et al.  Pathogenesis of myasthenia gravis: update on disease types, models, and mechanisms , 2016, F1000Research.

[18]  Nils Erik Gilhus,et al.  Myasthenia gravis: subgroup classification and therapeutic strategies , 2015, The Lancet Neurology.

[19]  Wei Zhang,et al.  The role of MuSK in synapse formation and neuromuscular disease. , 2013, Cold Spring Harbor perspectives in biology.

[20]  J. Olesen,et al.  The economic cost of brain disorders in Europe , 2012, European journal of neurology.

[21]  M. Conaway,et al.  MG‐ADL: Still a relevant outcome measure , 2011, Muscle & nerve.

[22]  M. Conaway,et al.  The MG Composite , 2010, Neurology.

[23]  R. Hohlfeld,et al.  Review: Progress in the treatment of myasthenia gravis , 2008, Therapeutic advances in neurological disorders.

[24]  W. Lehmacher,et al.  Adaptive Sample Size Calculations in Group Sequential Trials , 1999, Biometrics.

[25]  R. Barohn,et al.  Reliability Testing of the Quantitative Myasthenia Gravis Score a , 1998, Annals of the New York Academy of Sciences.