天津Mezagitamab注射剂III期临床试验-一项在慢性原发性免疫性血小板减少症受试者中评价Mezagitamab皮下注射的有效性和安全性的III期、随机、双盲、安慰剂对照研究

登记号	CTR20250206	试验状态	进行中
申请人联系人	魏雅静	首次公示信息日期	2025-01-21
申请人名称	Takeda Development Center Americas, Inc./ 武田亚太生物医药研发有限公司/ Spera Pharma, Inc.

登记号	CTR20250206
相关登记号	暂无
药物名称	Mezagitamab注射剂
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	慢性原发性免疫性血小板减少症
试验专业题目	一项在慢性原发性免疫性血小板减少症受试者中评价Mezagitamab皮下注射的有效性和安全性的III期、随机、双盲、安慰剂对照研究
试验通俗题目	一项在慢性原发性免疫性血小板减少症受试者中评价Mezagitamab皮下注射的有效性和安全性的III期、随机、双盲、安慰剂对照研究
试验方案编号	TAK-079-3002	方案最新版本号	方案修正案2
版本日期:	2024-11-26	方案是否为联合用药	否

申请人名称	[["Takeda Development Center Americas, Inc."],["武田亚太生物医药研发有限公司"],["Spera Pharma, Inc."]]
联系人姓名	魏雅静	联系人座机	010-84687062	联系人手机号	18513359725
联系人Email	Yajing.Wei@takeda.com	联系人邮政地址	北京市-北京市-北京朝阳区新源南路8号，启皓北京西塔2层	联系人邮编	100027

本研究由Takeda Development Center Americas, Inc.和武田亚太生物医药研发有限公司申办的一项在慢性原发性免疫性血小板减少症受试者中评价 Mezagitamab 皮下注射的有效性和安全性的 III 期、随机、 双盲、安慰剂对照研究。研究的主要目的为：评估 mezagitamab 相比安慰剂在年龄≥18 岁的慢性 ITP 受试者实现持久血小板应答的有效性。研究预计在中国筛选50例受试者，入组30例受试者。

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，受试者年龄≥18岁。"],["受试者在开始任何试验程序之前已提供知情同意书（即通过签署书面ICF并注明日期进行记录）和任何要求的隐私授权。"],["受试者确诊为原发性ITP，并持续至少12个月。诊断符合美国血液病学会2019年ITP指南（Neunert et al. 2019）或原发性ITP研究和管理的更新国际共识报告（Provan et al. 2019），如当地适用。"],["受试者的ITP诊断得到既往ITP治疗（不包括TPO-RA）应答（定义为达到血小板计数≥50,000/μL）的支持。"],["受试者对至少1种现有ITP一线治疗（例如皮质类固醇）和至少1种现有ITP二线治疗（例如TPO-RA、利妥昔单抗、fostamatinib、麦考酚酯）应答不足或不耐受。对既往治疗应答不足定义为在既往接受适当疗程的ITP治疗后血小板计数未能持续维持在至少50,000/μL或较基线血小板计数翻倍。不耐受定义为有副作用导致治疗终止的记录。"],["自签署ICF至第1天给药之间至少2次连续测量（至少间隔5天）受试者的平均血小板计数<30,000/μL（且单次测量值≤35,000/μL，包括至少1次测量在试验用药品（IMP）首次给药（第1天）前14天内）。"],["如果受试者在筛选期间正在接受允许使用的标准ITP治疗，并打算继续使用，那么如果在IMP首次给药（即第1天）前已按照稳定剂量和频率给药至少4周，且预期在整个试验期间保持稳定，则可在试验期间继续治疗。允许使用的合并治疗可能包括以下3个类别中的1种药物： n？t一种血小板生成素受体激动剂（例如罗普司亭、艾曲泊帕、阿伐曲泊帕海曲泊帕）和/或n？t一种口服皮质类固醇（不超过泼尼松20 mg/天或等效剂量），每日一次或每2日一次，和/或n？tFostamatinib。n如果受试者未计划在试验期间继续使用这些药物，则必须进行药物洗脱。其他信息参见第6.8.2节。"],["如果受试者为有生育能力的个体（参见第13.1.1.1节），则需要根据筛选期间和试验干预首次给药前的人绒毛膜促性腺激素阴性结果证实其未妊娠。"],["如果受试者入组日本的研究中心，则ITP诊断时或诊断后的幽门螺杆菌检测结果应呈阴性，或者在第1天前至少12周提供幽门螺杆菌成功根除治疗的记录。"]]
排除标准	[["研究者认为受试者不愿和/或无法理解并完全遵守试验程序和要求（包括数字工具和应用程序）。"],["该受试者患有继发性ITP。"],["受试者在签署ICF前12个月内发生过任何血栓或栓塞事件。"],["受试者在签署ICF前3个月内接受过脾切除术。n注：脾切除受试者感染风险增加，应及时接种疫苗并接受适当的预防治疗，如第6.9.4节所述。"],["受试者发生乙型肝炎病毒、丙型肝炎病毒或人类免疫缺陷病毒（HIV）活动性感染。"],["受试者在规定时间范围内发生过以下任一类型的感染：na)t签署ICF前2周内发生过活动性细菌、病毒、真菌感染（普通感冒和甲真菌病除外）或任何其他严重感染。感染的任何抗感染疗程必须在第1天前至少2周完成。nb)t签署ICF前4周内发生过SARS-CoV-2感染。nc)t签署ICF前≤12周内发生过机会感染或接受过机会感染治疗。"],["签署ICF前5年内有恶性肿瘤史（包括骨髓增生异常综合征），但经治的非黑色素瘤皮肤癌或宫颈原位癌除外。"],["受试者（1）确诊或疑似患有慢性阻塞性肺疾病（COPD）或哮喘，（2）筛选时，使用支气管扩张剂前的1秒用力呼气量<预测正常值的50%。"],["研究者认为受试者目前存在可能干扰受试者参加试验，对受试者构成额外风险或可能混淆试验结果评估的任何医学状况（例如严重的眼部、心血管、肺、血液学、胃肠道、内分泌、肝脏、肾脏、神经系统、恶性肿瘤、感染性疾病、免疫缺陷或酒精和药物滥用）。"],["研究者认为受试者患有可能使其无法按照本方案完成治疗的严重医学或精神疾病。"],["受试者在筛选前12个月内接受过抗CD20治疗，且符合以下任一条件：na)t筛选前6个月内接受末次给药。nb)t筛选前6-12个月内接受末次给药，且受试者的CD19+计数低于正常值下限。"],["受试者在第1天前6个月内使用过任何用于免疫调节的单克隆或多克隆抗体。"],["受试者既往在第1天前4周内或5个半衰期内（以较长者为准）暴露于mezagitamab，或暴露于另一种试验用药物。"],["受试者在试验治疗首次给药前3周内使用过抗凝剂（例如维生素K拮抗剂、直接口服抗凝剂）。"],["受试者在试验治疗首次给药前4周内接种过活疫苗或减毒活疫苗，或计划在试验期间接种任何活疫苗或减毒活疫苗。"],["受试者在试验治疗首次给药前使用过以下规定的免疫抑制剂：8周内使用过烷化剂类药物（例如环磷酰胺），4周内使用过长春花生物碱（例如长春新碱），3周内使用过砜类药物（例如氨苯砜），2周内使用过抗增殖药物（例如吗替麦考酚酯和硫唑嘌呤），2周内使用过钙调磷酸酶抑制剂（例如环孢素）。"],["受试者在签署ICF前4周内使用过静脉注射免疫球蛋白（IVIg）、SC免疫球蛋白、重组人血小板生成素、抗D免疫球蛋白治疗或艾加莫德，或计划在筛选至第1天期间使用除受试者正在接受的标准ITP治疗以外的任何其他血小板减少症治疗（例如补救治疗、血液制品）。"],["受试者对重组蛋白或mezagitamab/安慰剂制剂中使用的辅料有重度过敏反应或速发严重过敏反应史。"],["受试者在筛选时存在以下任何有临床意义的实验室检查结果异常：nc)t丙氨酸氨基转移酶或天门冬氨酸氨基转移酶（AST）>3倍正常值上限（ULN）。nd)t总胆红素>1.5×ULN。（注：不应根据该标准排除确诊Gilbert综合征的受试者。）ne)t中性粒细胞绝对计数<1000/mm3。nf)t血红蛋白<9 g/dL。ng)tIgG<500 mg/dL。nh)t淋巴细胞计数<500/mm3。ni)t估计肾小球滤过率<30 mL/min/1.73 m2。"],["受试者处于哺乳期，但不同意自IMP首次给药至IMP末次给药后30天放弃哺乳。"],["受试者为有生育能力的个体，但不同意在IMP末次给药后至少150天内与异性性交时使用至少1种高效避孕法和1种屏障避孕法（首选男用避孕套）。"],["受试者为性生活活跃且能产生精子的未绝育个体，但不同意在IMP末次给药后至少150天内与任何有生育能力的异性伴侣性交时使用1种屏障避孕法（首选男用避孕套）和至少1种高效避孕法。"]]

试验药	[["中文通用名:Mezagitamab注射剂 英文通用名:MezagitamabInjection 商品名称:NA","剂型:300mg/2ml 规格:300mg包装规格：2瓶/盒 用法用量:皮下注射600mg/4ml 用药时程:0.5h"]] 序号 名称 用法
对照药	[["中文通用名:NA 英文通用名:NA 商品名称:NA","剂型:0mg/2ml 规格:0mg包装规格：2瓶/盒 用法用量:皮下注射0mg/4ml 用药时程:0.5h"]] 序号 名称 用法

主要终点指标及评价时间	[["第24周实现持久血小板应答","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	[["1.截至第24周血小板计数≥50000/μL的累积周数n2.至首次实现血小板计数≥50000/μL的时间n3.截至第24周血小板计数≥30000/μL且较基线至少翻倍的累积周数n4.实现血小板完全应答","24周","有效性指标"],["第24周时ITP-PAQ症状量表评分较基线的变化。","24周","有效性指标"],["接受的补救疗。n至首次接受补救治疗的时间。","24周","有效性指标"],["出血事件的发生率","24周","有效性指标"],["干预期间和干预后的PK浓度。","24周","有效性指标+安全性指标"],["ADA发生率和ADA滴度变化。n中和ADA发生率。","24周","有效性指标+安全性指标"]] 序号 指标 评价时间 终点指标选择

1	姓名	杨仁池	学位	医学博士	职称	主任医师
	电话	13512078851	Email	rcyang@ihcams.ac.cn	邮政地址	天津市-天津市-和平区南京路288号
	邮编	300020	单位名称	中国医学科学院血液学研究所

[["中国医学科学院血液学研究所","杨仁池","中国","天津市","天津市"],["复旦大学附属金山医院（上海市金山区核化伤害应急救治中心、上海市金山区眼病防治所）","程韵枫","中国","上海市","上海市"],["广西医科大学第一附属医院","程鹏","中国","广西壮族自治区","南宁市"],["四川大学华西医院（四川省国际医院）","龚玉萍","中国","四川省","成都市"],["山东大学齐鲁医院","侯明","中国","山东省","济南市"],["南昌大学第一附属医院","黄瑞滨","中国","江西省","南昌市"],["华中科技大学同济医学院附属协和医院","胡豫","中国","湖北省","武汉市"],["中国医科大学附属盛京医院（中国医科大学附属第二医院）","廖爱军","中国","辽宁省","沈阳市"],["河北医科大学第一医院","刘清池","中国","河北省","石家庄市"],["广东省人民医院","罗成伟","中国","广东省","广州市"],["陕西省人民医院（陕西省临床医学研究院）","王一","中国","陕西省","西安市"],["浙江省中医院、浙江中医药大学附属第一医院（浙江省东方医院）","叶宝东","中国","浙江省","杭州市"],["苏州大学附属第一医院","殷杰","中国","江苏省","苏州市"],["河南省肿瘤医院","周虎","中国","河南省","郑州市"],["昆明医科大学第二附属医院（云南省泌尿专科医院、云南省肝胆胰外科医院）","周泽平","中国","云南省","昆明市"],["中国医学科学院北京协和医院","朱铁楠","中国","北京市","北京市"],["The Perth Blood Institute Limited","Ross Baker","澳大利亚","Perth","Perth"],["The Canberra Hospital","Philip Choi","澳大利亚","Canberra","Canberra"],["Westmead Hospital - Crown Princess Mary Cancer Centre (Westmead Cancer Care Centre)","Jennifer Curnow","澳大利亚","Westmead","Westmead"],["Monash University - Australian Centre for Blood Diseases (ACBD)","Zane Kaplan","澳大利亚","Monash","Monash"],["The Alfred Hospital","Huyen Tran","澳大利亚","Sydney","Sydney"],["South Eastern Sydney Local Health District","Silvia Zheng","澳大利亚","Sydney","Sydney"],["University of New South Wales (UNSW) - Liverpool Hospital - Liverpool Cancer Therapy Centre","Danny Hsu","澳大利亚","New South Wales","New South Wales"],["Concord Repatriation General Hospital Dept of Haematology, Haematology Clinical Research Unit","Vivien Chen","澳大利亚","Sydney","Sydney"],["Peter MacCallum Cancer Centre","Isaac Goncalves","澳大利亚","Melbourne","Melbourne"],["UMHAT Sv. Ivan Rilski","Atanas Radinoff","保加利亚","Sofia","Sofia"],["Hematology Clinic","Tzvetan Alaykov","保加利亚","Sofia","Sofia"],["Military Medical Academy Multiprofile Hospital for Active Treatment - Sofia","Viktoria Barbukova","保加利亚","Sofia","Sofia"],["Hospital Henri Mondor","Marc Michel","法国","Creteil","Creteil"],["Hopital Haut-Leveque, Medecine interne et maladies infectieuses","Jean-Francois Viallard","法国","Pessac Cedex","Pessac Cedex"],["Chu Dijon - Bourgogne","Bernard Bonnotte","法国","Dijon","Dijon"],["MVZ Gerinnungszentrum Hochtaunus","Rosa Alesci","德国","Bad Homburg","Bad Homburg"],["Universit？tsklinikum Jena","Thomas Stauch","德国","Jena","Jena"],["University Hospital of Duisburg-Essen","Alexander Roth","德国","Essen","Essen"],["General Hospital of Thessaloniki George Papanikolaou","Antonia Syrigou","希腊","Thessaloniki","Thessaloniki"],["Olympion General Clinic & Rehabilitation Center","Argiris Symeonidis","希腊","Patras","Patras"],["Queen Mary Hospital, Department of Medicine","Yok Lam Kwong","中国香港","Hong Kong","Hong Kong"],["A.O.U. Maggiore della Carità","Andrea Patriarca","意大利","Novara","Novara"],["Azienda Ospedaliera Di Rilievo Nazionale E Di Alta Specializzazione Garibaldi","Ugo Consoli","意大利","Catania","Catania"],["Azienda ULSS 8 Berica - Ospedale San Bortolo","Giuseppe Carli","意大利","Vicenza","Vicenza"],["AOU Citta della Salute e della Scienza di Torino","Alessandra Borchiellini","意大利","Torino","Torino"],["Azienda Ospedaliera Universitaria Federico II","Fabrizio Pane","意大利","Napoli","Napoli"],["Azienda Ospedaliera Universitaria Policlinico Tor Vergata","Francesco Buccisano","意大利","Rome","Rome"],["ASST Grande Ospedale Metropolitano Niguarda - Presidio Ospedaliero Ospedale Niguarda","Monica Carpendo","意大利","Milano","Milano"],["IRCCS Azienda Ospedaliero-Universitaria di Bologna","Nicola Vianelli","意大利","Bologna","Bologna"],["Azienda sanitaria universitaria Giuliano Isontina (ASU GI) (Azienda Sanitaria Universitaria Integrata di Trieste)","Elisa Lucchini","意大利","Trieste","Trieste"],["Seoul National University Hospital","Youngil Koh","韩国","Seoul","Seoul"],["Seoul National University Bundang Hospital Division of Hematology and Medical Oncology","Soo-Mee Bang","韩国","Bundang-gu","Bundang-gu"],["Severance Hospital, Yonsei University Health System","June-Won Cheong","韩国","Seoul","Seoul"],["Kyungpook National University Hospital","Sang-Kyun Sohn","韩国","Jung-ku","Jung-ku"],["Samsung Medical Center","Jun Ho Jang","韩国","Seoul","Seoul"],["Erasmus Medical Center, Department of Hematology","Arend Jan Gerard Jansen","荷兰","Rotterdam","Rotterdam"],["HagaZiekenhuis","Tanja Netelenbos","荷兰","The Hague","The Hague"],["Sykehuset Ostfold","Waleed Ghanima","挪威","Gralum","Gralum"],["Universitetet i Bergen","Galina Tsykunova","挪威","Bergen","Bergen"],["Akershus Universitetssykehus","Hoa Tran","挪威","Lorenskog","Lorenskog"],["N. Copernicus Provincial Multispecialist Center of Oncology and Traumatology in Lodz","Jacek Trelinski","波兰","Lodz","Lodz"],["Pratia Onkologia Katowice - PRATIA - PPDS","Sebastian Grosicki","波兰","Katowice","Katowice"],["Instytut Hematologii i Transfuzjologii","Jerzy Windyga","波兰","Warszawa","Warszawa"],["IN-VIVO sp. z o.o.","Jaroslaw Czyz","波兰","Bydgoszcz","Bydgoszcz"],["Hospital Universitario 12 de Octubre","Denis Zafra Torres","西班牙","Madrid","Madrid"],["Hospital Universitario Virgen del Rocio - PPDS","Maria Eva Mingot Castellano","西班牙","Sevilla","Sevilla"],["Hospital General Universitario Gregorio Maranon","Cristina Pascual Izquierdo","西班牙","Madrid","Madrid"],["Hospital Universitario De Salaman","Jose Ramon Gonzalez-Porras","西班牙","Salamanca","Salamanca"],["Skanes University hospital, Hematology Department","Alexandros Arvanitakis","瑞典","Malmo","Malmo"],["Karolinska Universitetssjukhuset","Cecilia Karlstrom","瑞典","Huddinge","Huddinge"],["Ankara University Faculty of Medicine Cebeci Hospital, Department of Hematology","Selami Toprak","土耳其","Mamak/Ankaea","Mamak/Ankaea"],["Cerrahpasa Tip Fakultesi","Muhlis Cem Ar","土耳其","Faith","Faith"],["Hematology Services Trakya University School of Medicine","Ahmet Muzaffer Demir","土耳其","Edirne","Edirne"],["Aydin Adnan Menderes University Medical Faculty-Hematology Department","Irfan Yavasoglu","土耳其","Efeler/Aydin","Efeler/Aydin"],["University College London Hospitals, NHS Foundation Trust","Marie Ann Scully","英国","London","London"],["Royal Liverpool and Broadgreen University Hospitals NHS Trust","David Simcox","英国","Liverpool","Liverpool"],["Imperial College Healthcare NHS Trust","Nichola Cooper","英国","London","London"],["Glasgow Royal Infirmary","Catherine Bagot","英国","Glasgow","Glasgow"],["Leicester Royal Infirmary","Mamta Garg","英国","Leicester","Leicester"],["Leeds Teaching Hospitals NHS Trust","Quentin Hill","英国","Leeds","Leeds"],["Southampton General Hospital","Syed Kazmi","英国","Southampton","Southampton"],["Barts Health NHS Trust, Royal London Hospital","Frederick Chen","英国","London","London"],["Georgetown University Medical Center (GUMC) - Lombardi Comprehensive Cancer Center (LCCC)","Catherine Broome","美国","Washington, DC","Washington, DC"],["Penn Blood Disorder Center","Adam Cuker","美国","Philadelphia","Philadelphia"],["East Carolina University","Darla Liles","美国","Greenville","Greenville"],["Keck Medicine of USC","Howard Liebman","美国","Los Angeles","Los Angeles"],["Massachusetts General Hospital","David Kuter","美国","Boston","Boston"],["University of Washington","Sandhya Panch","美国","Seattle","Seattle"],["Duke University Medical Center","Ara Metjian","美国","Durham","Durham"],["One Baylor Plaza","Senthil Sukumar","美国","Houston","Houston"],["Rocky Mountain Clinical Research - CenExel - PPDS","Christopher Benton","美国","Englewood","Englewood"],["Lewis Katz School of Medicine at Temple University","Michael Bromberg","美国","Philadelphia","Philadelphia"],["UMass Chan Medical School Cancer Research Office (CRO)","Daniel Winokur","美国","Worcester","Worcester"],["University of Iowa","Steven Lentz","美国","Iowa City","Iowa City"],["Virginia Oncology Associates - Virginia Beach Princess Anne)","Celeste Bremer","美国","Virginia Beach","Virginia Beach"],["University Of Louisville","Kamila Cisak","美国","Louisville","Louisville"],["Versiti Wisconsin Inc, WI 53233","Lisa Kreuziger","美国","Milwaukee","Milwaukee"],["Clinical Hospital Centar Zagreb","Drazen Pulanic","克罗地亚","Zagreb","Zagreb"],["Tokyo Metropolitan Bokutoh Hospital","Nobuharu Kosugi","日本","Tokyo","Tokyo"],["Nihon University Itabashi Hospital","Shimon Otake","日本","Itabashi-ku","Itabashi-ku"],["Chiba Aoba Municipal Hospital","Akira Yokota","日本","Chiba","Chiba"],["Saitama Medical University Hospital","Yoshitaka Miyakawa","日本","Iruma-gun","Iruma-gun"],["Osaka University Hospital","Hisashi Kato","日本","Suita-shi","Suita-shi"],["Tohoku University Hospital","Noriko Fukuhara","日本","Sendai-shi","Sendai-shi"],["Yokohama City University Medical Center","Shin Fujisawa","日本","Yokohama-Shi","Yokohama-Shi"],["Yamanashi Prefectural Central Hospital","Masaki Iino","日本","Kofu-shi","Kofu-shi"],["National Hospital Organization Mito Medical Center","Chikashi Yoshida","日本","Higashi Ibaraki-gun","Higashi Ibaraki-gun"],["The University of Tokyo Hospital","Mineo Kurokawa","日本","Tokyo","Tokyo"]]

序号	机构名称	主要研究者	国家	省（州）	城市

[["中国医学科学院血液学研究所伦理审查委员会","同意","2024-12-19"]]

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: 30 ； 国际: 117 ；
已入组人数	国内: 登记人暂未填写该信息； 国际: 登记人暂未填写该信息；
实际入组总人数	国内: 登记人暂未填写该信息； 国际: 登记人暂未填写该信息；

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：登记人暂未填写该信息；
第一例受试者入组日期	国内：登记人暂未填写该信息；     国际：登记人暂未填写该信息；
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；