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Record W4408589746 · doi:10.1007/s00415-025-12958-9

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

2025· article· en· W4408589746 on OpenAlexaff
Vera Bril, Artur Drużdż, Julian Großkreutz, Ali A. Habib, Henry J. Kaminski, Renato Mantegazza, Sabrina Sacconi, Kimiaki Utsugisawa, Tuan H. 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 R. Beydoun, Franz Blaes, Silvia Bonarino, Anna Boss Soevang, Nazibrola Botchorishvili, Stéphan A. Botez, Ivo Božović, Paulina Budzinska, Pietro Businaro, Lucia Campetella, Alejandro Oncina-Cánovas, Carlos Casasnovas, Hou‐Chang Chiu, Adam Comer, Elena Cortés Vicente, Roberto D’Angelo, Lubna Daniyal, Annie Dionne, Péter Diószeghy, Laura Fionda, Denis Flemm, Rita Frangiamore, Manuela Gambella, Matteo Garibaldi, Matteo Gastaldi, Christian Geis, Hannah George, Stefan Gingele, María-Jesús Martín, Yuh‐Cherng Guo, Gerardo Gutiérrez‐Gutiérrez, Francesco Habetswallner, Lina Hassoun, Sonja Holm‐Yildiz, Faraz Hussain, Francisca Iniesta, Viktoriya Irodenko, Marina Janelidze, Min Su Kang, Chafic Karam, D. S. Korobko, Sergey Kotov, Michal Kretkowski, N. Kvirkvelia, Antonio Lauletta, Yi‐Chung Lee, Luca Leonardi, Kore Liow, Arnau Llauradó Gayete, Sara Llufriú, Catherine Lomen‐Hoerth, Jan D. Lünemann, Lorenzo Maggi, Esmeralda Hernandez, 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, Е. S. Novikova, Izabella Obál, Anita Palsgård, Claudia Papi, Lorena Pérez, Stojan Perić, Nicolai Preisler, Giorgia Querin, Konrad Rejdak, Kourosh Rezania, Elena Rinaldi, Rita Rinaldi, Michael H. Rivner, Annekathrin Roediger, Laura Rosow, Símone Rossi, Elena Rossini, Stephen J. Ryan, Lotte Levison, Albert Saiz, María Salvadó, Daniel Sánchez‐Tejerina, Margret Schwarz, María Sepúlveda, Khema R. Sharma, Sheetal Shroff, Ольга Григорьевна Сидорова, Guilhem Solé, Javier Sotoca, Mads Stemmerik, Aleksandar Stojanov, Tanya Stojkovic, Kai Su, Sebastian Szklener, Alexander Tsiskaridze, Laura Tufano, Michaela Týblová, Eiko Uenaka, Astrid Unterlauft, Fiammetta Vanoli, Tamar Vashadze, Nuria Vidal Fernández, Marie‐Hélène Violleau, Nicolas Weiss, Nanna Witting, Jiann‐Horng Yeh, Leila Zaidi, Leonid Zaslavskiy, Jana Zschüntzsch

Bibliographic record

VenueJournal of Neurology · 2025
Typearticle
Languageen
FieldMedicine
TopicMyasthenia Gravis and Thymoma
Canadian institutionsToronto General HospitalUniversity of Toronto
FundersUCB
KeywordsOpen labelMyasthenia gravisMedicineNeuroradiologyNeurologyRandomized controlled trialSurgeryInternal medicinePsychiatry

Abstract

fetched live from OpenAlex

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).

Fetched live from OpenAlex and de-inverted. Abstracts are not stored in this database: the inverted indexes are 8.6 GB of the frame’s 9.3 GB of text, and the host has 13 GB free.

How this classification was reachedexpand

Full frame distilled prediction

Teacher imitation

Not calibrated prevalence, not ground truth. Human validation pending. Learned from the 10,348 direct Codex labels and 10,348 direct Gemma labels. Candidate is the union of thresholded teacher heads; consensus is their intersection. These outputs are machine_predicted_unvalidated and are not human labels or direct frontier model labels.

metaresearch head score (Codex)0.002
metaresearch head score (Gemma)0.001
Version: codex-gemma-dda1882f352aValidation status: machine_predicted_unvalidated
Candidate categoriesnone
Consensus categoriesnone
DomainCandidate signal: none · Consensus signal: none
Study designCandidate signal: Randomized trial · Consensus signal: Randomized trial
GenreCandidate signal: Empirical · Consensus signal: Empirical
Teacher disagreement score0.160
Threshold uncertainty score0.445

Codex and Gemma teacher scores by category

CategoryCodexGemma
Metaresearch0.0020.001
Meta-epidemiology (narrow)0.0000.000
Meta-epidemiology (broad)0.0020.000
Bibliometrics0.0000.000
Science and technology studies0.0000.000
Scholarly communication0.0000.000
Open science0.0000.000
Research integrity0.0000.001
Insufficient payload (model declined to judge)0.0000.000

Machine scores (provisional)

The two teacher heads of the student model, read on this work. A score orders the frame for review; it never asserts a category, and the validation status ships verbatim with every row.

Baseline scores from an immature model (maturity gate not passed, 7 training rounds). Scores rank; they never assert a category.

Opus teacher head0.021
GPT teacher head0.323
Teacher spread0.302 · how far apart the two teachers sit on this one work
Validation statusscore_only:v0-immature-baseline · verbatim from the scoring run: score_only means the number may rank works, and no category label ships from it

Classification

machine, unvalidated

Machine predicted; a candidate call from one teacher head, not a consensus.

The models applied no category: nothing in the taxonomy fit this work.
Study designRandomized trial
Domainnot available
GenreEmpirical

How this classification was reached, model by model and score by score, is at the end of the page under "How this classification was reached".

Quick stats

Citations6
Published2025
Admission routes1
Has abstractyes

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