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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

L. Querol, J. De Sèze, T. Dysgaard, T. Levine, TH. Rao, M. Rivner, HP. Hartung, P. Kiessling, S. Shimizu, D. Marmol, A. Bozorg, AO. Colson, U. Massow, F. Eftimov, CIDP01 Study Investigators

. 2024 ; 95 (9) : 845-854. [pub] 20240816

Jazyk angličtina Země Anglie, Velká Británie

Typ dokumentu časopisecké články, randomizované kontrolované studie, klinické zkoušky, fáze II, multicentrická studie

Perzistentní odkaz   https://www.medvik.cz/link/bmc24019325
E-zdroje Online Plný text

NLK ProQuest Central od 1944-07-01 do Před 6 měsíci
Nursing & Allied Health Database (ProQuest) od 1944-07-01 do Před 6 měsíci
Health & Medicine (ProQuest) od 1944-07-01 do Před 6 měsíci
Psychology Database (ProQuest) od 1944-07-01 do Před 6 měsíci

Odkazy

PubMed 38729747
DOI 10.1136/jnnp-2023-333112
Knihovny.cz E-zdroje

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.

Citace poskytuje Crossref.org

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