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
Language English Country England, Great Britain Media electronic
Document type Journal Article, Randomized Controlled Trial, Clinical Trial, Phase II, Multicenter Study
PubMed
38729747
PubMed Central
PMC11347201
DOI
10.1136/jnnp-2023-333112
PII: jnnp-2023-333112
Knihovny.cz E-resources
- Keywords
- FC RECEPTOR, MOVEMENT DISORDERS, MYASTHENIA, NEUROMUSCULAR, RANDOMISED TRIALS,
- MeSH
- Polyradiculoneuropathy, Chronic Inflammatory Demyelinating * drug therapy MeSH
- Adult MeSH
- Antibodies, Monoclonal, Humanized * therapeutic use adverse effects administration & dosage MeSH
- Immunoglobulins, Intravenous therapeutic use adverse effects MeSH
- Single-Blind Method MeSH
- Middle Aged MeSH
- Humans MeSH
- Aged MeSH
- Treatment Outcome MeSH
- Check Tag
- Adult MeSH
- Middle Aged MeSH
- Humans MeSH
- Male MeSH
- Aged MeSH
- Female MeSH
- Publication type
- Journal Article MeSH
- Clinical Trial, Phase II MeSH
- Multicenter Study MeSH
- Randomized Controlled Trial MeSH
- Names of Substances
- Antibodies, Monoclonal, Humanized * MeSH
- Immunoglobulins, Intravenous MeSH
- rozanolixizumab MeSH Browser
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.
Brain and Mind Center Medical Faculty University of Sydney Sydney New South Wales Australia
Center for Network Research in Rare Diseases CIBERER Madrid Spain
Department of Neurology Augusta University Augusta Atlanta Georgia USA
Department of Neurology Medical Faculty Heinrich Heine University Düsseldorf Germany
Department of Neurology Palacký University Olomouc Czech Republic
Honor Health Neurology Bob Bové Neuroscience Institute Scottsdale Arizona USA
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ClinicalTrials.gov
NCT04051944
