上海Empasiprubart注射用浓溶液III期临床试验-A study to assess the efficacy and safety of Empasiprubart versus IVIg in adults with Multifocal Motor Neuropathy （empassion）一项在多灶性运动神经病成人患者中评价 Empasiprubart 对比 IVIg 的疗效和安全性的研究（empassion）

登记号	CTR20252964	试验状态	进行中
申请人联系人	王岚	首次公示信息日期	2025-07-29
申请人名称	不适用/ 百时益医药研发（北京）有限公司

登记号	CTR20252964
相关登记号	暂无
药物名称	Empasiprubart注射用浓溶液
药物类型	生物制品
临床申请受理号	JXSL2500047
适应症	多灶性运动神经病
试验专业题目	一项在多灶性运动神经病成人患者中评价Empasiprubart对比静脉注射免疫球蛋白的疗效和安全性的随机、双盲、双模拟 III 期研究
试验通俗题目	A study to assess the efficacy and safety of Empasiprubart versus IVIg in adults with Multifocal Motor Neuropathy （empassion）一项在多灶性运动神经病成人患者中评价 Empasiprubart 对比 IVIg 的疗效和安全性的研究（empassion）
试验方案编号	ARGX-117-2302	方案最新版本号	2.0（修正案1）
版本日期:	2025-01-28	方案是否为联合用药	否

申请人名称	[["不适用"],["百时益医药研发（北京）有限公司"]]
联系人姓名	王岚	联系人座机	010-61846088	联系人手机号	18691065462
联系人Email	Lan.Wang2@thermofisher.com	联系人邮政地址	北京市-北京市-东城区东直门南大街1号第23层	联系人邮编	100007

A部分主要目的：证明empasiprubart与IVIg 相比在改善功能能力方面的疗效；A部分关键次要目的：证明empasiprubart与IVIg相比对肌力和运动功能的疗效；证明empasiprubart与IVIg相比在手灵巧度方面的疗效；A部分其他次要目的：评价empasiprubart与 IVIg 相比的安全性和耐受性；评价empasiprubart的PK、PD和免疫原性；评估empasiprubart与IVIg相比在肌力、运动功能、手灵巧度、健康相关生活质量和患者报告结局方面的疗效；评价empasiprubart与 IVIg 相比对健康相关生活质量的影响。B部分次要目的：评估empasiprubart在改善功能能力、肌力、运动功能、手灵巧度、生活质量和患者报告结局方面的长期疗效；评价empasiprubart的长期安全性和耐受性；评价empasiprubart的PK、PD和免疫原性；评价empasiprubart对长期健康相关生活质量的影响。

试验分类	安全性	试验分期	III期	设计类型	平行分组
随机化	随机化	盲法	双盲	试验范围	国际多中心试验

年龄	18岁(最小年龄)至 无上限 (最大年龄)
性别	男+女
健康受试者	无
入选标准	[["至少18岁且达到当地同意参与临床研究的法定年龄"],["根据EFNS/PNS2010年指南，在筛选时确诊为明确的或很可能的 MMN"],["在过去5年内对IVIg有应答。"],["每 2、3、4或5周接受一次IVIg治疗，剂量为每个周期0.4至2.0g/kg 体重"],["在筛选前接受最佳 IVIg 维持治疗方案（由研究者评估，频率无变化，剂量变化不得 > 10%）至少8周（或对于每5周接受一次 IVIg 的受试者，至少为10周）"],["最小换算后每周 IVIg 剂量为 ≥ 0.125g/kg"],["筛选前5年内有预防荚膜细菌病原体（脑膜炎奈瑟菌和肺炎链球菌）的免疫接种记录或愿意在研究药物首次给药前至少14天接受免疫接种"]]
排除标准	[["除研究的适应症外，还已知患有会混淆研究结果或使受试者面临不必要风险的自身免疫性疾病（例如，SLE）或任何其他医学状况"],["提示MMN以外的神经病的临床体征或症状，如运动神经元病（例如，延髓征、亢进反射）或其他炎性神经病（例如，感觉神经病）"]]

试验药	[["中文通用名:Empasiprubart静脉输注用浓缩液 英文通用名:EmpasiprubartIV 商品名称:NA","剂型:静脉输注用浓缩液 规格:165mg/mL 用法用量:静脉（IV）输液：初始两剂和每4周一次的维持剂量 用药时程:120周"],["中文通用名:氯化钠注射液 英文通用名:SodiumChiorideInjection 商品名称:NA","剂型:注射剂 规格:100ml:0.9g 用法用量:不适用 用药时程:24周"]] 序号 名称 用法
对照药	[["中文通用名:ARGX-117安慰剂、输注用浓缩液 英文通用名:ARGX-117placebo,concentrateforsolutionforinfusion 商品名称:NA","剂型:注射剂 规格:不适用 用法用量:不适用 用药时程:24周"],["中文通用名:静注人免疫球蛋白（pH4） 英文通用名:HumanImmunoglobulin（pH4）forIntravenousInjection 商品名称:静注人免疫球蛋白（pH4）","剂型:注射剂 规格:5g/瓶 用法用量:IV，每个周期0.4至2.0g/kg体重，最小换算后每周剂量≥0.125g/kg，采用受试者的维持剂量和给药方案b 用药时程:24周"]] 序号 名称 用法

主要终点指标及评价时间	[["第24周时基于Rasch模型的25项 MMN 整体残疾量表(MMN- RODS)百分位数评分较基线的变化","第24周","有效性指标"]] 序号 指标 评价时间 终点指标选择
次要终点指标及评价时间	[["第24周时受累最严重的手部握力（3天移动平均值）较基线的变化","第24周","有效性指标"],["第24周时改良版英国医学研究委员会肌力检查-14项总评分较基线的变化","第24周","有效性指标"],["第24周时惯用手完成九-孔柱测试的时间较基线的变化百分比","第24周","有效性指标"]] 序号 指标 评价时间 终点指标选择

1	姓名	林洁	学位	博士	职称	副主任医师
	电话	13916940621	Email	Linjie15@fudan.edu.com	邮政地址	上海市-上海市-静安区乌鲁木齐中路12号
	邮编	200040	单位名称	复旦大学附属华山医院

[["复旦大学附属华山医院","林洁","中国","上海市","上海市"],["福建医科大学附属协和医院","邹漳钰","中国","福建省","福州市"],["北京大学第三医院","樊东升","中国","北京市","北京市"],["西安市人民医院（西安市第四医院）","康娟","中国","陕西省","西安市"],["University of North Carolina at Chapel Hill-101 Manning Dr","Rebecca Traub","美国","North Carolina","Chapel Hill"],["UC Irvine - MDA ALS and Neuromuscular Center","Jeffrey Mullen","美国","California","Orange"],["Indiana University Health Neuroscience Center","Alexandru Barboi","美国","Indiana","Indianapolis"],["Oregon Health and Science University","Nizar Chahin","美国","Oregon","Portland"],["The Cleveland Clinic Foundation","Yuebing Li","美国","Ohio","Cleveland"],["Stanford Neuroscience Health Center","Srikanth Muppidi","美国","California","Palo Alto"],["University of Colorado- Anschutz Medical Campus - PPDS","Varun Sreenivasan","美国","Colorado","Aurora"],["Yale University School of Medicine -15 York St","Bhaskar Roy","美国","Connecticut","New Haven"],["University of Cincinnati","Hani Kushlaf","美国","Ohio","Cincinnati"],["University of Minnesota-420 Delaware Str SE","Georgios Manousakis","美国","Minnesota","Minneapolis"],["University of Miami Miller School of Medicine","Khema Sharma","美国","Florida","Miami"],["National Neuromuscular Research Institute","Yessar Hussain","美国","Texas","Austin"],["Columbia University Medical Center","Thomas Brannagan","美国","New York","New York"],["Robert Wood Johnson Medical School","Shan Chen","美国","New Jersey","New Brunswick"],["Dent Neurologic Institute - Amherst","Luisa Rojas Estupinan","美国","New York","Amherst"],["Penn Medicine University City","Sami Khella","美国","Pennsylvania","Philadelphia"],["Healthcare Innovations Institute, LLC","Sonia Kalirao","美国","Florida","Coral Springs"],["Samir Macwan, M.D., Inc.","Samir Macwan","美国","California","Rancho Mirage"],["University of California San Francisco","Madina Tugizova","美国","California","San Francisco"],["The Neurology Group","Sadiq Altamimi","美国","California","Pomona"],["Medstar Health Research Institute","Nicholas Samuel Streicher","美国","District of Columbia","Washington"],["Northwestern University-Regenstein Center for Neurological Care","Arjun Seth","美国","Illinois","Chicago"],["Perelman Center for Advanced Medicine","Chafic Karam","美国","Pennsylvania","Philadelphia"],["University of Kansas Medical Center","Mamatha Pasnoor","美国","Kansas","Kansas City"],["University of South Florida -13330 USF Laurel Dr","Kathleen Murray","美国","Florida","Tampa"],["NeuroCarePlus","Nicolas Nammour","美国","Texas","Houston"],["EZR Research LLC - Boca Raton -801 Meadows Rd","Patricio Espinosa Prado","美国","Florida","Boca Raton"],["Toronto General Hospital","Hans Katzberg","加拿大","Ontario","Toronto"],["Montreal Neurological Institute and Hospital","Rami Massie","加拿大","Quebec","Montreal"],["CHU de Quebec","Annie Dionne","加拿大","Quebec","Quebec"],["London Health Sciences Centre","Anita Florendo-Cumbermack","加拿大","Ontario","London"],["Neuro-Outaouais Clinic","Francois Jacques","加拿大","Quebec","Gatineau"],["St. Paul's Neurology Associates","Katherine Beadon","加拿大","British Columbia","Vancouver"],["University General Hospital ''ATTIKON'' - General Hospital of West Attica H AGIA VARVARA","Georgios Tsivgoulis","希腊","Attiki","Chaidari"],["AHEPA University General Hospital of Thessaloniki","Vasilios Kimiskidis","希腊","Thessaloniki","Thessaloniki"],["UZ Leuven - PPDS","Kristl Claeys","比利时","Vlaams Brabant","Leuven"],["UZ Antwerpen","Jonathan Baets","比利时","Antwerpen","Edegem"],["CHU Limoges-Dupuytren 1","Laurent Magy","法国","Haute-Vienne","Limoges"],["Hopital Roger Salengro","Céline Tard","法国","Nord","Lille"],["Hospital Universitario de Bellvitge","Carlos Casasnovas Pons","西班牙","Barcelona","L'hospitalet de Llobregat"],["Hospital Clinico San Carlos","Lucia Galan Davila","西班牙","Madrid","Madrid"],["Hospital de La Santa Creu i Sant Pau","Luis Querol","西班牙","Barcelona","Barcelona"],["Hospital Universitari i Politecnic La Fe de Valencia","Teresa Sevilla Mantecón","西班牙","Valencia","Valencia"],["Hospital Universitario de Basurto","Solange Kapetanovic","西班牙","Vizcaya","Bilbo"],["Hospital Universitario de Donostia","Roberto Fernandez Torron","西班牙","Guipúzcoa","Donostia-San Sebastian"],["Hospital Universitario Germans Trias i Pujol","Giuseppe Lucente","西班牙","Barcelona","Badalona"],["Hospital Quironsalud Malaga","Jesus Romero Imbroda","西班牙","Málaga","Malaga"],["Ospedale Policlinico San Martino IRCCS","Luana Benedetti","意大利","Liguria","Genova"],["Az Ospedaliera Universitaria Policlinico G Martino","Luca Gentile","意大利","Sicilia","Messina"],["AUSL di Bologna - Ospedale Bellaria","Vincenzo Donadio","意大利","Emilia-Romagna","Bologna"],["Fondazione Serena Onlus - Centro Clinico NeMO Brescia","Massimiliano Filosto","意大利","Brescia","Gussago"],["Azienda Ospedaliera Universitaria Policlinico Paolo Giaccone","Filippo Brighina","意大利","Sicilia","Palermo"],["Inselspital - Universitatsspital Bern","Helly Hammer","瑞士","Bern （de）","Bern"],["Klinikum Wels-Grieskirchen GmbH - Standort Wels","Raffi Topakian","奥地利","Oberosterreich","Wels"],["Royal Hallamshire Hospital - PPDS","Channa Hewamadduma","英国","South Yorkshire","Sheffield"],["Salford Royal Hospital - PPDS","Ryan （Yann Shern） Keh","英国","Lancashire","Salford"],["Sahlgrenska Universitetssjukhuset","Lenka Novakova Nyren","瑞典","Vastra Gotalands lan","Goteborg"],["CLINIREM Sp z o.o. - Lublin","Urszula Chyrchel-Paszkiewicz","波兰","Lubelskie","Lublin"],["SP ZOZ Szpital Uniwersytecki w Krakowie-Jakubowskiego 2","Agnieszka Slowik","波兰","Malopolskie","Kraków"],["MICS Centrum Medyczne Bydgoszcz","Lukasz Rzepinski","波兰","Kujawsko-pomorskie","Bydgoszcz"],["CLINIREM Sp z o.o. - Warszawa","Anna Domzal-Stryga","波兰","Mazowieckie","Warszawa"],["Universitatsklinikum Munster","Gerd Meyer zu Hoerste","德国","Nordrhein-Westfalen","Munster"],["Universitatsklinikum Tubingen","Alexander Grimm","德国","Baden-Wurttemberg","Tubingen"],["Hospital Universitário da Universidade Estadual de Campinas - UNICAMP","Marcondes Franca Junior","巴西","Sao Paulo","Campinas"],["PSEG Centro de Pesquisa Clínica","Paulo Sgobbi de Souza","巴西","Sao Paulo","Sao Paulo"],["Hospital Das Clinicas da Faculdade de Medicina de Ribeirao Preto - USP - PPDS","Wilson Marques Junior","巴西","Sao Paulo","Ribeirao Preto"],["Unidade de pesquisa clínica e inovacao - UPCI - IDOMED","Osvaldo Nascimento","巴西","Rio de Janeiro","Rio de Janeiro"],["Hospital de Clinicas de Porto Alegre （HCPA） - PPDS","Pablo Brea Winckler","巴西","Rio Grande do Sul","Porto Alegre"],["Escola Bahiana de Medicina e Saude Publica","Marcela Costa","巴西","Bahia","Salvador"],["Brain and Mind Center","Judith Spies","澳大利亚","New South Wales","Camperdown"],["Princess Alexandra Hospital","Stefan Blum","澳大利亚","Queensland","Woolloongabba"],["Royal Melbourne Hospital","Belinda Cruse","澳大利亚","Victoria","Parkville"],["Royal Brisbane & Women's Hospital","Robert Henderson","澳大利亚","Queensland","Herston"],["Chiba University Hospital","Kazumoto Shibuya","日本","Tiba","Chuo-Ku"],["National Center of Neurology and Psychiatry","Tomoko Okamoto","日本","Tokyo","Kodaira-Shi"],["Nagoya University Hospital","Yuki Fukami","日本","Akita","Nagoya"],["Teikyo University Hospital","Yuki Hatanaka","日本","Tokyo","Itabashi-Ku"],["Yamaguchi University Hospital","Masayuki Nakamori","日本","Yamaguti","Ube-Shi"],["Southern TOHOKU Medical Clinic","Chikako Kaneko","日本","Hukusima","Koriyama-Shi"],["Institute of Science Tokyo Hospital","Sonoko Misawa","日本","Tokyo","Bunkyo-ku"],["Juntendo University Hospital","Yuji Tomizawa","日本","Tokyo","Bunkyo-ku"],["ULS de Santo António, EPE - Hospital de Santo António","Ernestina Santos","葡萄牙","Porto","Porto"],["Hospital of Lithuanian University of Health Sciences Kauno klinikos","Kestutis Petrikonis","立陶宛","Kauno Apskritis","Kaunas"],["Astra Kliinik","Katrin Gross-Paju","爱沙尼亚","Harjumaa","Tallinn"],["Fakultni nemocnice Hradec Kralove","Zbysek Pavelek","捷克","Královéhradecky kraj","Hradec Kralove"]]

序号	机构名称	主要研究者	国家	省（州）	城市

[["复旦大学附属华山医院伦理审查委员会","同意","2025-07-11"]]

序号	名称	审查结论	批准日期/备案日期

进行中（尚未招募）

目标入组人数	国内: 15 ； 国际: 100 ；
已入组人数	国内: 登记人暂未填写该信息； 国际: 1 ；
实际入组总人数	国内: 登记人暂未填写该信息； 国际: 1 ；

第一例受试者签署知情同意书日期	国内：登记人暂未填写该信息；     国际：2025-02-27；
第一例受试者入组日期	国内：登记人暂未填写该信息；     国际：2025-05-22；
试验完成日期	国内：登记人暂未填写该信息；     国际：登记人暂未填写该信息；