Efficacy, safety and tolerability of rozanolixizumab in patients with chronic inflammatory demyelinating polyradiculoneuropathy : A randomised, subject-blind, investigator-blind, placebo-controlled, phase 2a trial and open-label extension study
Querol, Luis (Institut de Recerca Sant Pau)
De Sèze, J. (University Hospital of Strasbourg)
Dysgaard, T. (University of Copenhagen)
Levine, T. (Bob Bové Neuroscience Institute)
Rao, T.H. (The Neurological Institute. PA)
Rivner, M. (Augusta University)
Hartung, H.P. (Palacký University)
Kiessling, P. (UCB Pharma)
Shimizu, S. (UCB Pharma)
Marmol, D. (UCB Pharma)
Bozorg, A. (UCB Pharma)
Colson, A.O. (UCB Pharma)
Massow, U. (UCB Pharma)
Eftimov, F. (Amsterdam University Medical Center (UMC))
Universitat Autònoma de Barcelona

Date:	2024
Abstract:	Background Chronic inflammatory demyelinating polyradiculoneuropathy (CIDP) is a peripheral nerve disorder characterised by weakness and sensory loss. We assessed the neonatal Fc receptor inhibitor rozanolixizumab for CIDP management. Methods CIDP01 (NCT03861481) was a randomised, subject-blind, investigator-blind, placebo-controlled, phase 2a study. Adults with definite or probable CIDP receiving subcutaneous or intravenous immunoglobulin maintenance therapy were randomised 1:1 to 12 once-weekly subcutaneous infusions of rozanolixizumab 10 mg/kg or placebo, stratified according to previous immunoglobulin administration route. Investigators administering treatment and assessing efficacy, and patients, were blinded. The primary outcome was a change from baseline (CFB) to day 85 in inflammatory Rasch-built Overall Disability Scale (iRODS) score. Eligible patients who completed CIDP01 entered the open-label extension CIDP04 (NCT04051944). Results In CIDP01, between 26 March 2019 and 31 March 2021, 34 patients were randomised to rozanolixizumab or placebo (17 (50%) each). No significant difference in CFB to day 85 in iRODS centile score was observed between rozanolixizumab (least squares mean 2. 0 (SE 3. 2)) and placebo (3. 4 (2. 6); difference -1. 5 (90% CI -7. 5 to 4. 5)). Overall, 14 (82%) patients receiving rozanolixizumab and 13 (76%) receiving placebo experienced a treatment-emergent adverse event during the treatment period. Across CIDP01 and CIDP04, rozanolixizumab was well tolerated over up to 614 days; no clinically meaningful efficacy results were seen. No deaths occurred. Conclusions Rozanolixizumab did not show efficacy in patients with CIDP in this study, although this could be due to a relatively high placebo stability rate. Rozanolixizumab was well tolerated over medium-to-long-term weekly use, with an acceptable safety profile.
Rights:	Aquest document està subjecte a una llicència d'ús Creative Commons. Es permet la reproducció total o parcial, la distribució, la comunicació pública de l'obra i la creació d'obres derivades, sempre que no sigui amb finalitats comercials, i sempre que es reconegui l'autoria de l'obra original.
Language:	Anglès
Document:	Article ; recerca ; Versió publicada
Subject:	FC RECEPTOR ; MOVEMENT DISORDERS ; MYASTHENIA ; NEUROMUSCULAR ; RANDOMISED TRIALS
Published in:	Journal of Neurology, Neurosurgery, and Psychiatry, Vol. 95 Núm. 9 (16 2024) , p. 845-854, ISSN 1468-330X

DOI: 10.1136/jnnp-2023-333112
PMID: 38729747

10 p, 435.7 KB

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