Can I read Zilucoplan in Immune-mediated Necrotising Myopathy: a Phase 2, Randomised, Double-blind, Placebo-controlled, Multicentre Trial on EtoBox?

Zilucoplan in Immune-mediated Necrotising Myopathy: a Phase 2, Randomised, Double-blind, Placebo-controlled, Multicentre Trial by Andrew L Mammen; Anthony A Amato; Mazen M Dimachkie; Hector Chinoy; Yessar Hussain; James B Lilleker; Iago Pinal-Fernandez; Yves Allenbach; Babak Boroojerdi; Mark Vanderkelen; Eumorphia Maria Delicha; Harold Koendgen; Ramin Farzaneh-Far; Petra W Duda; Camil Sayegh; Olivier Benveniste; Anthony A. Amato; Olivier Benveniste; Suur Biliciler; Hector Chinoy; Mazen M. Dimachkie; Christyn Edmundson; Miriam Freimer; Anthony Geraci; Yessar Hussain; Pedro Machado; Andrew L. Mammen; Tahseen Mozaffar; Payam Soltanzadeh; Niraja Suresh; Anneke van der Kooi; Yves Allenbach; Matthew Appleby; Richard J Barohn; Nicolas Champtiaux; Christopher Doughty; Jerrica Farias; Constantine Farmakidis; Ali A. Habib; Chafic Karam; James Lilleker; Samantha Lorusso; Mamatha Pasnoor; Iago Pinal-Fernandez; Giorgia Querin; Joost Raaphorst; George Ransley; Sami Saba; Kazim Sheikh; Andrew Snedden; Jeffrey Statland; Tuan Vu is a Medicine article available to read on EtoBox.

What is Zilucoplan in Immune-mediated Necrotising Myopathy: a Phase 2, Randomised, Double-blind, Placebo-controlled, Multicentre Trial about?

Background Immune-mediated necrotising myopathy is an autoimmune myopathy characterised by proximal muscle weakness, high creatine kinase concentrations, and autoantibodies recognising 3-hydroxy-3-methylglutaryl-coenzyme A reductase (HMGCR) or the signal recognition particle (SRP). No approved therapies exist for people with immunemediated necrotising myopathy. Previous studies have suggested that complement activation might be pathogenic in immune-mediated necrotising myopathy; therefore, zilucoplan, a complement C5 (C5) inhibitor, could be a potential therapy. We aimed to evaluate the efficacy, safety, and tolerability of zilucoplan in adult participants with anti-HMGCR or anti-SRP autoantibody-positive immune-mediated necrotising myopathy. ## Methods IMNM-01 was a phase 2, multicentre, randomised, double-blind, placebo-controlled study done at 15 hospital sites across the USA, the UK, France, and the Netherlands. Participants aged 18-74 years were eligible for inclusion if they had a clinically confirmed diagnosis of immune-mediated necrotising myopathy, positive serology for anti-HMGCR or anti-SRP autoantibodies, clinical evidence of weakness, serum total creatine kinase concen

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Author

Andrew L Mammen; Anthony A Amato; Mazen M Dimachkie; Hector Chinoy; Yessar Hussain; James B Lilleker; Iago Pinal-Fernandez; Yves Allenbach; Babak Boroojerdi; Mark Vanderkelen; Eumorphia Maria Delicha; Harold Koendgen; Ramin Farzaneh-Far; Petra W Duda; Camil Sayegh; Olivier Benveniste; Anthony A. Amato; Olivier Benveniste; Suur Biliciler; Hector Chinoy; Mazen M. Dimachkie; Christyn Edmundson; Miriam Freimer; Anthony Geraci; Yessar Hussain; Pedro Machado; Andrew L. Mammen; Tahseen Mozaffar; Payam Soltanzadeh; Niraja Suresh; Anneke van der Kooi; Yves Allenbach; Matthew Appleby; Richard J Barohn; Nicolas Champtiaux; Christopher Doughty; Jerrica Farias; Constantine Farmakidis; Ali A. Habib; Chafic Karam; James Lilleker; Samantha Lorusso; Mamatha Pasnoor; Iago Pinal-Fernandez; Giorgia Querin; Joost Raaphorst; George Ransley; Sami Saba; Kazim Sheikh; Andrew Snedden; Jeffrey Statland; Tuan Vu

Publisher

Elsevier BV

Published

2023

Language

EN

Field

Medicine (Health Sciences)