Safety, tolerability, and efficacy of subcutaneous efgartigimod in patients with chronic inflammatory demyelinating polyradiculoneuropathy (ADHERE): a multicentre, randomised-withdrawal, double-blind, placebo-controlled, phase 2 trial
| Title: | Safety, tolerability, and efficacy of subcutaneous efgartigimod in patients with chronic inflammatory demyelinating polyradiculoneuropathy (ADHERE): a multicentre, randomised-withdrawal, double-blind, placebo-controlled, phase 2 trial |
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| Authors: | Allen, Jeffrey A; Lin, Jie; Basta, Ivana; Dysgaard, Tina; Eggers, Christian; Guptill, Jeffrey T; Gwathmey, Kelly G; Hewamadduma, Channa; Hofman, Erik; Hussain, Yessar M; Kuwabara, Satoshi; Le Masson, Gwendal; Leypoldt, Frank; Chang, Ting; Lipowska, Marta; Lowe, Murray; Lauria, Giuseppe; Querol, Luis; Simu, Mihaela-Adriana; Suresh, Niraja; Tse, Anissa; Ulrichts, Peter; Van Hoorick, Benjamin; Yamasaki, Ryo; Lewis, Richard A; van Doorn, Pieter A; Collaborators: Susanne Grinzinger; Julia Wanschitz; Thomas Seifert-Held; Kristl G Claeys; Jonathan Baets; Gauthier Remiche; Véronique Bissay; Nicolas Dubuisson; Stephanie Delstanche; Ivaylo Tarnev; Krasimir Genov; Plamen Tsvetanov; Ivan Milanov; Chongbo Zhao; Bitao Bu; Xuefan Yu; Wei Li; Haishan Jiang; Yuwei Da; Zuneng Lu; Hui Liang; Fuqiang Guo; Zunbo Li; Zhangyu Zou; Daojun Hong; Huan Yang; Junhong Guo; Jianquan Shi; Jianglong Tu; Dian He; Yiqi Wang; Jing Ding; Yali Zhang; Yuanqi Zhao; Renxi Xu; Yunhua Yue; Aihong Guo; Yuzhong Wang; Radomir Talab; Thomas Harbo; Soeren Sindrup; Jérôme De Seze; Sabrina Sacconi; Yann Péréon; Andoni Echaniz-Laguna; Laurent Magy; Guillaume Nicolas; Frédéric Taithe; Julien Cassereau; Rabab Debs; Roman Shakarishvili; Alexander Tsiskaridze; Maka Mania; Marina Janelidze; Tamar Janelidze; Michael Schroeter; Thomas Skripuletz; De-Hyung Lee; Juliane Klehmet; Benjamin Hotter; Olaf Hoffmann; Petra Baum; Jana Zschuentzsch; Kalliopi Pitarokoili; Mark Stettner; Dániel Bereczki; Gyula Pánczél; Alon Abraham; Amir Dori; Yair Lampl; Fiore Manganelli; Stefania Morino; Alessandro Padovani; Gabriele Siciliano; Angelo Schenone; Francesca Magri; Anna Mazzeo; Fabio Giannini; Sandro Sorbi; Adriano Chiò; Motoi Kuwahara; Tatsusada Okuno; Tomoko Okamoto; Norito Kokubun; Kazutoshi Nishiyama; Kenichi Kaida; Kota Bokuda; Masahisa Katsuno; Ichiro Yabe; Etsuji Saji; Takanori Yokota; Yuki Hatanaka; Jin Nakahara; Takamichi Sugimoto; Fumiaki Tanaka; Satoshi Tomita; Yoshihisa Yamano; Tomohiro Hayashi; Hiroki Yamazaki; Takashi Tokashiki; Kazuhiro Horiuchi; Guntis Karelis; Filip Eftimov; Marta Banach; Urszula Chyrchel-Paszkiewicz; Jan Kochanowicz; Krzysztof Selmaj; Tomasz Zielinski; Krzysztof Banaszkiewicz; Dan Mitrea; Ana-Maria Scutaru-Kadar; Any Axelerad; Fatima Stuchevskaya; Alexey Boyko; Farit Khabirov; Tatiana Trushnikova; Zoya Goncharova; Natalia Suponeva; Nikolay Dorogov; Eduard Yakupov; Ranko Raicevic; Svetlana Miletic Drakulic; Macarena Cabrera Serrano; Jose Luis Muñoz Blanco; Antonio Guerrero Sola; Eduardo Aguera Morales; Carmen Diaz Marin; Raúl Juntas Morales; Jiann-Horng Yeh; Jia-Ying Sung; Han-Wei Huang; Nai-Wen Tsai; Yuh-Cherng Guo; Chi-Chao Chao; Long-Sun Ro; Ihsan Sengun; Murat Terzi; Sezin Alpaydin Baslo; Filiz Koç; Hamdi Necdet Karli; Olga Shulga; Dmytro Smolko; Oleksandr Doroshenko; Nataliya Tomakh; Alla Kyrychenko; Olga Seliuk; Oleksandr Kalbus; Iryna Skrypchenko; Olha Novakovska; Francisco Javier Carod-Artal; Simon Rinaldi; Kathryn Brennan; Simon Ellis; Aisling Carr; Emma Matthews; Rebecca Traub; Tahseen Mozaffar; Matthew Elliott; Ratna Bhavaraju-Sanka; Christopher Nance; Todd Levine; Robert Lisak; Mamatha Pasnoor; Michael Pulley; Bhaskar Roy; Raghav Govindarajan; Gregory Sahagian; Sami Khella; Daniel Jacob; Hani Kushlaf; Kumaraswamy Sivakumar; Isaac Melamed; Khema Sharma; Adam Quick; Eroboghene Ubogu; David Lacomis; Arnaldo Isa; Thomas Brannagan; Shan Chen; Jonathan Katz; Marc Feinberg; Pantelis Pavlakis; Dale Lange; Mark Gudesblatt; Rup Tandan; Karissa Gable; Michael Rivner; Benjamin Barnes; Donna Luke; Zabeen Mahuwala; Samir Macwan; Patrick Kwon; James Scott; Sadiq Altamimi; Priyanka Sabharwal |
| Contributors: | Jeffrey A, Allen; Jie, Lin; Ivana, Basta; Tina, Dysgaard; Christian, Egger; Jeffrey T, Guptill; Kelly G, Gwathmey; Channa, Hewamadduma; Erik, Hofman; Yessar M, Hussain; Satoshi, Kuwabara; Gwendal, Le Masson; Frank, Leypoldt; Ting, Chang; Marta, Lipowska; Murray, Lowe; Giuseppe, Lauria; Luis, Querol; Mihaela-Adriana, Simu; Niraja, Suresh; Anissa, Tse; Peter, Ulricht; Benjamin, Van Hoorick; Ryo, Yamasaki; Richard A, Lewi; Pieter A, van Doorn; Susanne Grinzinger, Collaborators; Wanschitz, Julia; Seifert-Held, Thoma; G Claeys, Kristl; Baets, Jonathan; Remiche, Gauthier; Bissay, Véronique; Dubuisson, Nicola; Delstanche, Stephanie; Tarnev, Ivaylo; Genov, Krasimir; Tsvetanov, Plamen; Milanov, Ivan; Zhao, Chongbo; Bu, Bitao; Yu, Xuefan; Li, Wei; Jiang, Haishan; Da, Yuwei; Lu, Zuneng; Liang, Hui; Guo, Fuqiang; Li, Zunbo; Zou, Zhangyu; Hong, Daojun; Yang, Huan; Guo, Junhong; Shi, Jianquan; Tu, Jianglong; He, Dian; Wang, Yiqi; Ding, Jing; Zhang, Yali; Zhao, Yuanqi; Xu, Renxi; Yue, Yunhua; Guo, Aihong; Wang, Yuzhong; Talab, Radomir; Harbo, Thoma; Sindrup, Soeren; De Seze, Jérôme; Sacconi, Sabrina; Péréon, Yann; Echaniz-Laguna, Andoni; Magy, Laurent; Nicolas, Guillaume; Taithe, Frédéric; Cassereau, Julien; Debs, Rabab; Shakarishvili, Roman; Tsiskaridze, Alexander; Mania, Maka; Janelidze, Marina; Janelidze, Tamar; Schroeter, Michael; Skripuletz, Thoma; Lee, De-Hyung; Klehmet, Juliane; Hotter, Benjamin; Hoffmann, Olaf; Baum, Petra; Zschuentzsch, Jana; Pitarokoili, Kalliopi; Stettner, Mark; Bereczki, Dániel; Pánczél, Gyula; Abraham, Alon; Dori, Amir; Lampl, Yair; Manganelli, Fiore; Morino, Stefania; Padovani, Alessandro; Siciliano, Gabriele |
| Publication Year: | 2024 |
| Collection: | IRIS Università degli Studi di Napoli Federico II |
| Description: | Background: Chronic inflammatory demyelinating polyradiculoneuropathy (CIDP) is an autoimmune disease of the peripheral nervous system that can lead to severe disability from muscle weakness and sensory disturbances. Around a third of patients do not respond to currently available treatments, and many patients with a partial response have residual neurological impairment, highlighting the need for effective alternatives. Efgartigimod alfa, a human IgG1 antibody Fc fragment, has demonstrated efficacy and safety in patients with generalised myasthenia gravis. We evaluated the safety, tolerability, and efficacy of subcutaneous efgartigimod PH20 in adults with CIDP. Methods: ADHERE, a multistage, double-blind, placebo-controlled trial, enrolled participants with CIDP from 146 clinical sites from Asia-Pacific, Europe, and North America. Participants with evidence of clinically meaningful deterioration entered an open-label phase of weekly 1000 mg subcutaneous efgartigimod PH20 for no longer than 12 weeks (stage A). Those with confirmed evidence of clinical improvement (ECI; treatment responders) entered a randomised-withdrawal phase of 1000 mg subcutaneous efgartigimod PH20 weekly treatment versus placebo for a maximum of 48 weeks (stage B). Participants were randomised (1:1) through interactive response technology and stratified by their adjusted Inflammatory Neuropathy Cause and Treatment (aINCAT) score change during stage A and their most recent CIDP medication within 6 months before screening. Investigators, the clinical research organisation, and participants were masked to the treatment. The primary endpoint in stage A, evaluated in the stage A safety population, was confirmed ECI (≥1 points aINCAT decrease, ≥4 points [centile metric] Inflammatory Rasch-built Overall Disability Scale increase, or ≥8 kPa grip strength increase after four injections and two consecutive visits). The primary endpoint in stage B, evaluated in the modified intention-to-treat population, was the risk of relapse (time to first aINCAT ... |
| Document Type: | article in journal/newspaper |
| Language: | English |
| Relation: | info:eu-repo/semantics/altIdentifier/wos/WOS:001319433700001; volume:23; issue:10; journal:LANCET NEUROLOGY; https://hdl.handle.net/11588/992269 |
| DOI: | 10.1016/S1474-4422(24)00309-0 |
| Availability: | https://hdl.handle.net/11588/992269; https://doi.org/10.1016/S1474-4422(24)00309-0 |
| Rights: | info:eu-repo/semantics/openAccess ; license:Copyright dell'editore ; license uri:publisher |
| Accession Number: | edsbas.A49C5EEE |
| Database: | BASE |