上海艾加莫德α注射液（皮下注射）III期临床试验-评价Efgartigimod PH20 SC给药治疗眼肌型重症肌无力成人受试者的有效性和安全性

登记号	CTR20244895	试验状态	进行中
申请人联系人	樊晨娇	首次公示信息日期	2024-12-26
申请人名称	argenx BV/ 再鼎医药（上海）有限公司/ Vetter Pharma-Fertigung GmbH & Co. KG

登记号	CTR20244895
相关登记号	暂无
药物名称	艾加莫德α注射液（皮下注射）
药物类型	生物制品
临床申请受理号	企业选择不公示
适应症	眼肌型重症肌无力
试验专业题目	一项在眼肌型重症肌无力成人受试者中评价通过预充式注射器进行Efgartigimod PH20 SC给药的有效性和安全性的随机、双盲、安慰剂对照、III期、平行组设计研究
试验通俗题目	评价Efgartigimod PH20 SC给药治疗眼肌型重症肌无力成人受试者的有效性和安全性
试验方案编号	ARGX-113-2315	方案最新版本号	V1.0
版本日期:	2024-06-18	方案是否为联合用药	否

申请人名称	[["argenx BV"],["再鼎医药（上海）有限公司"],["Vetter Pharma-Fertigung GmbH & Co. KG"]]
联系人姓名	樊晨娇	联系人座机	021-61632588	联系人手机号	13918653491
联系人Email	tina.fan@zailaboratory.com	联系人邮政地址	上海市-上海市-上海市浦东新区金科路4560号1号楼南楼4层	联系人邮编	200010

主要目的是在oMG受试者中评价Efgartigimod PH20 SC与安慰剂相比的有效性和安全性。其他目的是评估Efgartigimod PH20 SC的长期有效性、安全性和耐受性。

试验分类	安全性和有效性	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	[["签署ICF时年龄至少为18岁且为临床研究的当地法定知情同意年龄"],["能够提供已签署的知情同意书，并遵守研究方案要求"],["WOCBP必须同意按照方案描述基于当地法规采取避孕措施"],["筛选时WOCBP的血清妊娠试验结果必须为阴性，基线时接受IMP给药前的尿妊娠试验结果必须为阴性"],["已诊断为MG，具有该疾病一致的临床特征并经记录证实，并得到以下条件的支持： na.AChR-Ab血清阳性，或者 nb.SFEMG或RNS证实的神经肌肉传递异常，和，依酚氯铵试验阳性病史或经口服AChE抑制剂、PLEX、IVIg或皮质类固醇等治疗后MG体征改善"],["MGFA I型"],["筛选和基线MGII（PRO）眼部评分≥6，至少2个眼部项目评分≥2"],["筛选前正在接受稳定剂量的MG治疗，包括AChE抑制剂、类固醇或NSIST（联合用药或单药治疗均可）（更多信息见试验方案）"],["<3年内出现症状，除非有MRI证据表明眼外肌无脂肪替代或在过去一年内对治疗（IVIg、PLEX、溴吡斯的明和/或类固醇）有反应"],["无瞳孔异常，由既往局部疾病或手术导致的瞳孔异常除外"]]
排除标准	[["除研究适应症以外，已知患有可能干扰oMG/MG临床症状准确评估或使受试者处于过度风险的自身免疫性疾病或任何医学病症"],["2. 有恶性肿瘤病史，除非认为经充分治疗治愈，且在IMP首次给药≥3年内无复发证据。经充分治疗的以下癌症受试者可随时入选： n1).基底细胞或鳞状细胞皮肤癌n2).宫颈原位癌 n3).乳腺原位癌n4).经组织学确诊的前列腺偶发癌（TNM T1a或T1b期）"],["研究者认为在基线前未充分痊愈且具有临床意义的活动性感染"],["筛选时以下任何活动性感染的血清检测结果呈阳性： na.t提示急性或慢性感染的HBV，除非HBV DNA检测结果呈阴性 nb.t基于HCV抗体检测存在HCV感染，除非RNA检测结果呈阴性 nc.tHIV感染，基于CD4计数检测结果<200个细胞/mm3或存在AIDS标志性病变 nd.tHIV感染，基于CD4计数检测结果≥200个细胞/mm3，但未进行充分的抗病毒治疗"],["目前正在参与另一项干预性临床研究"],["已知对IMP或其任何辅料有超敏反应"],["根据研究者评估，当前存在或既往（筛选前12个月内）有酒精、毒品或药物滥用史"],["处于妊娠期或哺乳期，或计划在研究期间怀孕"],["既往参加过Efgartigimod临床研究且接受过至少1剂IMP给药"],["筛选前4周内接种了活疫苗或减毒活疫苗"],["有记录显示对PLEX无临床应答"],["筛选前3个月内进行过胸腺切除术或计划在研究期间进行胸腺切除术"],["使用某些既往或合并治疗（更多信息见试验方案）"],["完整的排除标准详见研究方案"]]

试验药	[["中文通用名:艾加莫德α注射液（皮下注射） 英文通用名:EfgartigimodPH20SC 商品名称:NA","剂型:注射剂 规格:Efgartigimod200mg/mLrHuPH202000U/mL 用法用量:皮下一次注射 用药时程:每个治疗期时每周一次，连续3周共4次注射"]] 序号 名称 用法
对照药	[["中文通用名:安慰剂PH20SC 英文通用名:PlaceboPH20SC 商品名称:NA","剂型:注射剂 规格:rHuPH202000U/mL 用法用量:皮下一次注射 用药时程:每周一次，连续3周共4次注射"]] 序号 名称 用法

主要终点指标及评价时间	[["A部分中MGII（PRO）眼部评分从基线至第29天的变化","研究第29天","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	[["1. A部分中MGII（PRO+PE）眼部评分从基线至第29天的变化 n2. A部分中MG-ADL眼部领域评分从基线至第29天的变化 n3. A部分中MGII总评分从基线至第29天的变化","研究第29天","有效性指标"]] 序号 指标 评价时间 终点指标选择

1	姓名	赵重波	学位	博士学位	职称	主任医师
	电话	13701822316	Email	zhao_chongbo@fudan.edu.cn	邮政地址	上海市-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","奚剑英","中国","上海市","上海市"],["山东大学齐鲁医院","王勤周","中国","山东省","济南市"],["武汉大学人民医院","卢祖能","中国","湖北省","武汉市"],["四川省人民医院","许飞","中国","四川省","成都市"],["福建医科大学附属协和医院","邹漳钰","中国","福建省","福州市"],["南昌大学第一附属医院","洪道俊","中国","江西省","南昌市"],["中南大学湘雅医院","杨欢","中国","湖南省","长沙市"],["广东省中医院","招远祺","中国","广东省","广州市"],["广州中医药大学第一附属医院","江其龙","中国","广东省","广州市"],["重庆医科大学附属第一医院","肖飞","中国","重庆市","重庆市"],["山东大学齐鲁医院（青岛）","赵翠萍","中国","山东省","青岛市"],["华中科技大学同济医学院附属同济医院","卜碧涛","中国","湖北省","武汉市"],["石家庄市人民医院","乞国艳","中国","河北省","石家庄市"],["Gold Coast University Hospital","Sabet, Arman","澳大利亚","Queensland","Southport"],["The Alfred Hospital","van der Walt, Anneke","澳大利亚","Victoria","Melbourne"],["Box Hill Hospital-8 Arnold St","Buzzard, Katherine","澳大利亚","Victoria","Box Hill"],["Chiba University Hospital","Uzawa, Akiyuki","日本","Tokyo","Chuo-Ku"],["Kindai University Hospital","Samukawa, Makoto","日本","Saitama","Sayama-Shi"],["General Hanamaki Hospital","Utsugisawa, Kimiaki","日本","Iwate","Hanamaki-shi"],["Osaka University Hospital","Takahashi, Masanori","日本","saka","Suita-Shi"],["Tokyo Medical University Hospital","Masuda, Masayuki","日本","Tokyo","Shinjuku-Ku"],["Saitama Medical Center","Kaida, Kenichi","日本","Saitama","Kawagoe-Shi"],["Kyushu University Hospital","Watanabe, Mitsuru","日本","Fukuoka","Fukuoka"],["National Hospital Organization Hokkaido Medical Center","Minami, Naoya","日本","Hokkaid","Sapporo-Shi"],["Hiroshima University Hospital","Sugimoto, Takamichi","日本","Hirosima","Hiroshima"],["National Hospital Organization Utano National Hospital","Tomita, Satoshi","日本","Kyoto","Kyoto-Shi"],["IUHW Narita Hospital","Murai, Hiroyuki","日本","Tiba","Narita"],["Kyungpook National University Chilgok hospital","Park, Jin-Sung","韩国","Daegu","Daegu"],["Severance Hospital Yonsei University Health System","Shin, Ha Young","韩国","Seou","Seou"],["Korea University Anam Hospital","Kim, Byung-Jo","韩国","Seoul Teugbyeolsi","Seongbuk-Gu"],["Universit？tsklinikum AKH Wien","Zimprich, Fritz","澳大利亚","Wien","Wien"],["UZ Leuven - PPDS","Claeys, Kristl","比利时","Leuven","Leuven"],["H？pital de la Citadelle","Poleur, Margaux","比利时","Liège","Liège"],["Cyprus Institute of Neurology and Genetics","Kleopa, Kleopas","塞浦路斯","Lefkosia","Egkomi"],["Fakultni nemocnice Brno","Vohanka, Stanislav","捷克共和国","Brno","Brno"],["Nemocnice Pardubickeho kraje, a.s., Pardubicka nemocnice","Ehler, Edvard","捷克共和国","Pardubice","Pardubice"],["Neurologie a rehabilitace Skopalikova","Minks, Eduard","捷克共和国","Brno","Brno"],["Rigshospitalet","Preisler, Nicolai Rasmus","丹麦","Copenhagen","Copenhagen"],["Aarhus Universitetshospital","Andersen, Henning","丹麦","Central Jutland","Aarhus N"],["Turun Yliopistollinen Keskussairaala","Jokela, Manu","芬兰","Turku","Turku"],["Hospital Pasteur (CHU Nice)","Bresch, Saskia","法国","Nice","Nice"],["Hospices Civils de Lyon - Hospital Pierre Wertheimer","Froment, Caroline","法国","Bron","Bron"],["Hospital Fondation Adolphe de Rothschild - PPDS","Gueguen, Antoine","法国","Paris","Paris"],["CHU Dijon Bourgogne","Blanc-Labarre, Christelle","法国","Dijon","Dijon"],["Centre Hospitalier National D'ophtalmologie Des Quinze Vingts","Aboab, Jennifer","法国","Paris","Paris"],["New Hospitals","Margania, Temur","格鲁吉亚","Tbilisi","Tbilisi"],["Petre Sarajishvili Institute of Neurology","Shakarishvili, Roman","格鲁吉亚","Tbilisi","Tbilisi"],["Pineo Medical Ecosystem","Tsiskaridze, Alexander","格鲁吉亚","Tbilisi","Tbilisi"],["LLC Caucasus Medical Centre","Sekhniashvili, Madona","格鲁吉亚","Tbilisi","Tbilisi"],["Charité - University tsmedizin Berlin","Meisel, Andreas","德国","Berlin","Berlin"],["University Georg August","Zschuentzsch, Jana","德国","Gottingen","Gottingen"],["University General Hospital ''ATTIKON''","Giannopoulos, Sotirios","希腊","Attiki","Chaidari"],["Eginitio Hospital","Zouvelou, Vasiliki","希腊","Attiki","Athens"],["University General Hospital of Patras","Chroni, Elisabeth","希腊","Achaia","Patras"],["Fondazione IRCCS Istituto Neurologico Carlo Besta","Antozzi, Carlo","意大利","Milano","Milano"],["ASST Papa Giovanni XXIII - Ospedale Papa Giovanni XXIII","Sgarzi, Manlio","意大利","Bergamo","Bergamo"],["Azienda Ospedaliero-Universitaria Sant'Andrea","Morino, Stefania","意大利","Roma","Roma"],["Ospedale San Raffaele S.r.l. - PPDS","Previtali, Stefano","意大利","Lombardia","Milano"],["AORN Antonio Cardarelli","Habetswallner, Francesco","意大利","Napoli","Napoli"],["IRCCS Istituto dell Scienze Neurologiche di Bologna","Giannoccaro, Maria Pia","意大利","Bologna","Bologna"],["Leids Universitair Medisch Centrum","Verschuuren, J.J.G.M.","荷兰","Leiden","Leiden"],["Krakowska Akademia Neurologii Sp. z o.o.","Szczudlik, Andrzej","波兰","Kraków","Kraków"],["CLINIREM Sp z o.o. - Lublin","Chyrchel-Paszkiewicz, Urszula","波兰","Lublin","Lublin"],["SP ZOZ Szpital Uniwersytecki w Krakowie-ul Botaniczna 3","Slowik, Agnieszka","波兰","Kraków","Kraków"],["MICS Centrum Medyczne Bydgoszcz","Rzepinski, Lukasz","波兰","Bydgoszcz","Bydgoszcz"],["Centrum Medyczne Neurologia Slaska","Smilowski, Marek","波兰","Katowice","Katowice"],["ULS de Santa Maria, EPE - Hospital de Santa Maria - PPDS","de Carvalho, Mamede","葡萄牙","Lisbon","Lisbon"],["ULS de Santo António, EPE - Hospital de Santo António","Santos, Ernestina","葡萄牙","Porto","Porto"],["King Faisal Specialist Hospital & Research Center - Riyadh","Alshehri, Ali","沙特阿拉伯","Ar Riya","Riyadh"],["University Clinical Center of Serbia - Dr Subotica 6 - PPDS","Peric, Stojan","塞尔维亚","Beograd","Beograd"],["University Clinical Center Nis","Djordjevic, Gordana","塞尔维亚","Nis","Nis"],["Hospital Clinico San Carlos","Galan Davila, Lucia","西班牙","Madrid","Madrid"],["Hospital Universitario Vall d'Hebron - PPDS","Juntas Morales, Raúl","西班牙","Barcelona","Barcelona"],["Hospital Universitario Ramon y Cajal","Alvarez Velasco, Rodrigo","西班牙","Madrid","Madrid"],["Hospital Universitario de Donostia","Lopez de Munain Arregui, Adolfo","西班牙","Guipúzcoa- Donostia","San Sebastian"],["Hospital del Mar","Rubio Perez, Miguel","西班牙","Barcelona","Barcelona"],["Hospital Clinico Universitario de Valencia","Pareja, Ana","西班牙","Valencia","Valencia"],["Sahlgrenska Universitetssjukhuset","Novakova Nyren, Lenka","瑞典","Goteborg","Goteborg"],["Mediclinic Parkview","Al Zahmi, Fatima","阿拉伯联合酋长国","Dubai","Dubai"],["Sheikh Shakbout Medical City","Shatila, Ahmed","阿拉伯联合酋长国","Abu Dhabi","Abu Dhabi"],["Southampton General Hospital","Pinto, Ashwin","英国","Southampton","Southampton"],["Dementech Neurosciences Clinical Academic Centre","Papachatzaki, Maria-Martha","英国","London","London"],["Panthera Biopartners - Glasgow - PPDS","Goodfellow, John","英国","Glasgow","Glasgow"],["Royal Sussex County Hospital","Barritt, Andrew","英国","Brighton","Brighton"],["Leeds General Infirmary","Taleti, Esteban","英国","Leeds","Leeds"],["Toronto General Hospital","Barnett Tapia, Carolina","加拿大","Ontario","Toronto"],["McGill University Health Centre - 1033 Pine Avenue West","Blanchard, Oliver","加拿大","Quebec","Montreal"],["University of Alberta","Siddiqi, Zaeem","加拿大","Alberta","Edmonton"],["London Health Sciences Centre","Nicolle, Michael","加拿大","Ontario","London"],["Hopital de L'enfant Jesus","Dionne, Annie","加拿大","Quebec","Quebec City"],["Ottawa Hospital Research Institute","Breiner, Ariel","加拿大","Ontario","Ottawa"],["Gordon and Leslie Diamond Health Care Centre","Mezei, Michelle","加拿大","British Columbia","Vancouver"],["Maritime Neurology","Leckey, Richard","加拿大","Nova Scotia","Halifax"],["Centre de recherche du Centre Hospitalier de l'Universite de Montreal","Botez, Stephan","加拿大","Quebec","Montreal"],["University of North Carolina at Chapel Hill-170 Manning Dr","Howard, James","美国","North Carolina","Chapel Hill"],["University of California Irvine-200 S Manchester Ave","Habib, Ali","美国","Maryland","Columbia"],["University of South Florida - 13330 USF Laurel Drtn","Vu, Tua","美国","Florida","Tampa"],["University of Kansas Medical Center Research Institute, Inc.","Pasnoor, Mamatha","美国","Kansas","Fairway"],["Cleveland Clinic Main Campus","Andrapalliyal, Nirmal","美国","Ohio","Cleveland"],["Houston Methodist Neurological Institute","Greene (Simpson), Ericka","美国","Maryland","Columbia"],["Penn Medicine University City","Khella, Sami","美国","Pennsylvania","Philadelphia"],["National Neuromuscular Research Institute","Hussain, Yessar","美国","Texas","Austin"],["Neurology Associates PA - 331 N Maitland Ave","Isa, Arnaldo","美国","Florida","Maitland"],["Robert Wood Johnson Medical School","Chen, Shan","美国","New Jersey","New Brunswick"],["SFM Clinical Research, LLC","Feinberg, Marc","美国","Maryland","Columbia"],["Duke Neurological Disorders Clinic-40 Duke Medicine Cir","Juel, Vern","美国","North Carolina","Durham"],["Neurology Offices of South Florida","Costell, Brian","美国","Maryland","Columbia"],["Massachusetts General Hospital","Guidon, Amanda","美国","Massachusetts","Boston"],["Carolinas HealthCare System Neurosciences Institute-Neurology","Desai, Urvi","美国","North Carolina","Charlotte"],["Northwestern Memorial Hospital - 259 E Erie St.","Seth, Arjun","美国","Illinois","Chicago"],["HonorHealth Neurology - Bob Bové Neuroscience Institute - PPDS","Muley, Suraj","美国","Arizona","Scottsdale"],["Martha Morehouse Medical plaza","Freimer, Miriam","美国","Ohio","Columbus"],["Henry Ford Health System","Grover, Kavita","美国","Michigan","Detroit"],["Saint Louis University - 1438 S Grand Blvd","Hayat, Ghazala","美国","Missouri","St. Louis"],["Loma Linda University","Rosenfeld, Jeffrey","美国","California","Loma Linda"],["USC Roski Eye Institute - Los Angeles","Gluckstein, Jeffrey","美国","California","Los Angeles"]]

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","2024-10-29"]]

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: 24 ； 国际: 124 ；
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：2024-11-12；
第一例受试者入组日期	国内：登记人暂未填写该信息；     国际：2024-12-16；
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；