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Clinical Trial
. 2025 Oct 11;272(10):686.
doi: 10.1007/s00415-025-13420-6.

Self-administration of rozanolixizumab via manual push and infusion pump methods in patients with generalised myasthenia gravis: a randomised, phase 3, open-label, crossover study

Vera Bril  1 , Carlo Antozzi  2 , Tomasz Berkowicz  3 , Artur Drużdż  4 , Rachana K Gandhi Mehta  5 , Zabeen K Mahuwala  6 , Jana Zschüntzsch  7 , Marion Boehnlein  8 , Virginie Kerbusch  9 , Andreea Lavrov  8 , Mark Morris  10 , Puneet Singh  10 , M Isabel Leite  11
Affiliations
Clinical Trial

Self-administration of rozanolixizumab via manual push and infusion pump methods in patients with generalised myasthenia gravis: a randomised, phase 3, open-label, crossover study

Vera Bril et al. J Neurol. .

Abstract

Background: The phase 3, open-label, randomised, crossover MG0020 study investigated rozanolixizumab self-administration, efficacy, and safety in patients with generalised myasthenia gravis (gMG) using infusion pump and manual push methods.

Methods: Adults with gMG received once-weekly rozanolixizumab for 18 weeks, comprising a 6-week Training Period and two 6-week Self-Administration Periods where patients were randomised 1:1 to Sequence 1 (infusion pump then manual push) or Sequence 2 (manual push then infusion pump). The primary endpoint was successful rozanolixizumab self-administration (choosing correct infusion site, administering subcutaneously, delivering intended dose), evaluated by a healthcare professional (HCP) at weeks 12 and 18. Secondary endpoints included treatment-emergent adverse events (TEAEs). Additional endpoints included change from baseline in total immunoglobulin G (IgG) and Myasthenia Gravis Activities of Daily Living (MG-ADL) score, and patients' administration method preference.

Results: Sixty-two patients received treatment; 55 were randomised (Sequence 1: n = 28; Sequence 2: n = 27). The self-administration success rate was 100% with both methods. Decreases from baseline in IgG and MG-ADL score were maintained during self-administration with both methods. TEAEs occurred in 47/62 (75.8%) patients; most events (161/165 [97.6%]) were mild or moderate. Incidence was comparable for both methods. Most patients (35/55 [63.6%]) preferred self-administration to HCP administration (5/55 [9.1%]); more preferred manual push (25/55 [45.5%]) to infusion pump (17/55 [30.9%]).

Conclusions: All patients successfully self-administered rozanolixizumab; more patients preferred manual push. Efficacy and safety were consistent with the known HCP-administered profile. These results support rozanolixizumab self-administration and manual push administration in patients with gMG.

Trial registration: NCT05681715 (registered 4 January 2023).

Keywords: Generalised myasthenia gravis; Infusion pump; Manual push; Patient preference; Rozanolixizumab; Self-administration.

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Conflict of interest statement

Declarations. Conflict of interest: V. Bril is a consultant for Akcea, Alexion Pharmaceuticals, Alnylam, argenx, CSL, Grifols, Immunovant, Ionis, Janssen Pharmaceuticals (now Johnson & Johnson Innovative Medicine), Momenta (now Johnson & Johnson), Novo Nordisk, Octapharma, Pfizer, Powell Mansfield, Roche, Sanofi, Takeda Pharmaceuticals and UCB. She has received research support from Akcea, Alexion Pharmaceuticals, argenx, CSL, Grifols, Immunovant, Ionis, Momenta (now Johnson & Johnson), Octapharma, Takeda Pharmaceuticals, UCB and Viela Bio (now Amgen). C. Antozzi has received funding for congress and Institutional Review Board participation from Alexion Pharmaceuticals, argenx, Biogen, Janssen Pharmaceuticals (now Johnson & Johnson Innovative Medicine), Momenta (now Johnson & Johnson) and UCB. T. Berkowicz has nothing to disclose. A. Drużdż has nothing to disclose. R.K. Gandhi Mehta has received research funding from Akcea Pharmaceuticals (now AstraZeneca), EMD Serono, Novartis and UCB. She has served on advisory boards for Amgen and UCB and has received speaker honoraria from UCB. Z.K. Mahuwala has received compensation for advisory board participation from Alexion Pharmaceuticals and Janssen Pharmaceuticals (now Johnson & Johnson Innovative Medicine) and has served as a panellist for Academic CME. She has received research funding for clinical trials paid to the University of Kentucky from Alexion Pharmaceuticals, argenx, Immunovant, Janssen Pharmaceuticals (now Johnson & Johnson Innovative Medicine), RemeGen Biosciences and UCB. J. Zschüntzsch has been awarded research grants from the German Innovationfond, German Society for Muscle Disease (DGM), and the Innovative Medicines Initiative 2 Joint Undertaking (IMI JU) of the European Commission (Grant Number: 101034427-2). She has received speaker honoraria or travel grants from Alexion, argenx, Kedrion, Roche and Sanofi. She serves on scientific or educational advisory boards for Alexion, Amicus, argenx, iThera, Kedrion, Sanofi, and UCB. She has received research support from argenx and UCB. She is a member of the European Reference Network for Rare Neuromuscular Diseases (ERN EURO-NMD) and a member of the medical advisory boards of the DGM and the German Myasthenia Gravis Society. M. Boehnlein is an employee and shareholder of UCB. V. Kerbusch is a paid Consultant for UCB. A. Lavrov is an employee and shareholder of UCB. M. Morris is an employee and shareholder of UCB. P. Singh is an employee and shareholder of UCB, and is a previous employee and shareholder of GSK. M.I. Leite is funded by the NHS (Myasthenia and Related Disorders Service and National Specialised Commissioning Group for Neuromyelitis Optica, UK) and by the University of Oxford, UK. She has been awarded research grants from UK associations for patients with myasthenia and with muscular disorders (Myaware and Muscular Dystrophy UK, respectively) and the University of Oxford. She has received speaker honoraria or travel grants from Biogen, the Guthy-Jackson Charitable Foundation, Novartis and UCB. She serves on scientific or educational advisory boards for argenx, Horizon Therapeutics (now Amgen) and UCB. Ethical approval: Study protocol, amendments, and study participant informed consent were approved by a national, regional or independent ethics committee or institutional review board. All patients provided the written informed consent. This study was conducted in accordance with local regulations, International Conference on Harmonization Good Clinical Practice requirements, and the principles of the Declaration of Helsinki.

Figures

Fig. 1
Fig. 1
Study design. aPatients completing all treatment periods, including the End of Treatment Visit, and moving on to either a post-study access programme or commercially available rozanolixizumab during the Safety Follow-Up Period underwent an earlier End of Study Visit prior to this move. HCP, healthcare professional; R, randomisation.
Fig. 2
Fig. 2
Patient study disposition. AE, adverse event; IP, infusion pump; MP, manual push; RSS, randomised safety set; SA, self-administration; SS, safety set
Fig. 3
Fig. 3
Self-administration success rate by study sequence. Full analysis set. Successful self-administration was assessed at week 12 (visit 13) for Self-Administration Period 1 and week 18 (visit 19) for Self-Administration Period 2. Percentages are based on the number of patients with non-missing data. Patients are grouped according to the self-administration method within the study period. RLZ, rozanolixizumab
Fig. 4
Fig. 4
Median percentage change from baseline in total IgG. Randomised safety set. IgG values up to and including 8 weeks after the start date of rescue therapy were excluded. Median baseline value was 9.87 for Sequence 1 (N = 28) and 9.37 for Sequence 2 (N = 27). aBaseline values were defined as the last available measurement before the first administration of study drug at week 1. CFB, change from baseline; HCP, healthcare professional; IgG, immunoglobulin G; IP, infusion pump; MP, manual push; R, randomisation
Fig. 5
Fig. 5
Mean change from baseline in MG-ADL score. Randomised safety set. Mean (SD) baseline value was 7.07 (3.89) for Sequence 1 (N = 28) and 7.52 (3.94) for Sequence 2 (N = 27). aBaseline values were defined as the last available measurement before the first administration of study drug at week 1. CFB, change from baseline; HCP, healthcare professional; IP, infusion pump; MG-ADL, Myasthenia Gravis Activities of Daily Living; MP, manual push; R, randomisation; SD, standard deviation
Fig. 6
Fig. 6
Patient preference for (a) HCP-administered versus self-administered rozanolixizumab and (b) manual push versus infusion pump self-administration. Randomised safety set. Questionnaire was administered at the last self-administration visit and answers were based on patients’ overall self-administration experience. Percentages are based on the number of patients with an assessment at week 18. Self-administration includes study drug administration by patient or caregiver. HCP, healthcare professional; IP, infusion pump, MP, manual push; RLZ, rozanolixizumab
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