杭州甲磺酸贝舒地尔片III期临床试验-一项评估贝舒地尔片剂治疗慢性移植肺功能丧失在成人受试者中有效性的研究

登记号	CTR20242991	试验状态	进行中
申请人联系人	赛诺菲	首次公示信息日期	2024-08-08
申请人名称	Kadmon Pharmaceuticals, LLC/ 赛诺菲（中国）投资有限公司/ UPM Pharmaceuticals, Inc.

登记号	CTR20242991
相关登记号	暂无
药物名称	甲磺酸贝舒地尔片
药物类型	化学药物
临床申请受理号	企业选择不公示
适应症	慢性移植肺功能丧失
试验专业题目	一项在双肺移植后慢性移植肺功能丧失（CLAD）成人受试者中评价口服贝舒地尔的有效性的随机、双盲、安慰剂对照、平行分组、Ⅲ 期研究，继以开放性延长研究
试验通俗题目	一项评估贝舒地尔片剂治疗慢性移植肺功能丧失在成人受试者中有效性的研究
试验方案编号	EFC17801	方案最新版本号	修订版临床试验方案05
版本日期:	2023-12-18	方案是否为联合用药	否

申请人名称	[["Kadmon Pharmaceuticals, LLC"],["赛诺菲（中国）投资有限公司"],["UPM Pharmaceuticals, Inc."]]
联系人姓名	赛诺菲	联系人座机	65634716	联系人手机号
联系人Email	Contact-US.CN@sanofi.com	联系人邮政地址	北京市-北京市-朝阳区建国路112号	联系人邮编	100022

主要目的：证明在双肺移植后患有 1 期或 2 期 CLAD 的受试者中，贝舒地尔与安慰剂相比，通过 1 秒用力呼气容积（FEV1）测量的针对肺功能的有效性 次要目的：证明在双肺移植后患有 CLAD 的受试者中，贝舒地尔与安慰剂相比，通过 FEV1 测量的针对肺功能的有效性；通过测量用力肺活量（FVC），评估贝舒地尔对肺功能的有效性；通过测量肺总量（TLC），评估贝舒地尔对肺功能的有效性；评估贝舒地尔对 CLAD 进展的影响；通过 6 分钟步行试验评估贝舒地尔对运动能力的有效性；通过 6 分钟步行试验评估贝舒地尔对氧合状态的有效性；评估贝舒地尔对患者报告结局（PRO）的影响；评估贝舒地尔在双肺移植后患有 CLAD 的受试者中的安全性。

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	[["受试者签署知情同意书时的年龄必须不低于 18 岁"],["发生 1 期或 2 期 CLAD 的受试者：在筛选和随机化时，FEV1 为 PTBL 的＞50% 至 80%"],["在 CLAD 诊断后和随机化前接受过至少 8 周阿奇霉素（≥250 mg/天，每周至少 3 次）治疗的受试者"]]
排除标准	[["FEV1≤移植后基线值的 50%（CLAD 3 期和 4期）"],["肺功能下降可由非 CLAD 原因解释，包括但不限于急性移植肺排斥反应（＞A1）、筛选前 4 周内抗体介导的排斥反应、气道狭窄或气管支气管软化症"]]

试验药	[["中文通用名:贝舒地尔 英文通用名:Belumosudil 商品名称:易来克","剂型:片剂 规格:200mg 用法用量:受试者将接受200mg贝舒地尔,每日一次,口服给药 用药时程:双盲治疗期每日一次至26周，后转至开放标签组直到52周，后续再进入长期开放标签组直到批准上市为止"]] 序号 名称 用法
对照药	[["中文通用名:安慰剂 英文通用名:Placebo 商品名称:NA","剂型:片剂 规格:0mg/片 用法用量:受试者将接受安慰剂，每日一次,口服给药 用药时程:双盲治疗期每日一次至26周"]] 序号 名称 用法

主要终点指标及评价时间

[["FEV1 从基线至第 26 周的百分比变化","从基线至第 26 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 26 周时的应答率，定义为第 26 周时 FEV1 较基线下降≤10% 的受试者比例（关键次要终点）","第 26 周","有效性指标"],["FEV1 从基线至第 26 周的绝对变化","从基线至第 26 周","有效性指标"],["FEV1 占预计值百分比从基线至第 26 周的绝对变化","从基线至第 26 周","有效性指标"],["FVC 从基线至第 26 周的百分比变化","从基线至第 26 周","有效性指标"],["FVC 从基线至第 26 周的绝对变化","从基线至第 26 周","有效性指标"],["FVC 占预计值百分比从基线至第 26 周的绝对变化","从基线至第 26 周","有效性指标"],["TLC 从基线至第 26 周的百分比变化","从基线至第 26 周","有效性指标"],["双盲治疗期间至 CLAD 进展的时间，定义为首次发生以下事件：FEV1 较基线下降＞10%，或 CLAD 分期变化，或重新移植，或死亡","从基线至第 26 周","有效性指标"],["至重新移植或死亡的时间","贝舒地尔最后一次用药后最多至第7天","有效性指标"],["6 分钟步行距离从基线至第 26 周的百分比变化","从基线至第 26 周","有效性指标"],["6 分钟步行距离从基线至第 26 周的绝对变化","从基线至第 26 周","有效性指标"],["6 分钟步行最低血氧饱和度从基线至第 26 周的绝对变化","从基线至第 26 周","有效性指标"],["圣乔治呼吸问卷（SGRQ）从基线至第 26 周的变化（总分和各维度评分）","从基线至第 26 周","有效性指标"],["EQ-5D-5L（视觉模拟量表和个体维度）从基线至第 26 周的变化","从基线至第 26 周","有效性指标"],["患者报告结局测量信息系统-概况 29 第 2.1 版（PROMIS-概况 29 V2.1）（维度评分）从基线至第 26 周的变化","从基线至第 26 周","有效性指标"],["在双盲治疗期以及开放性延长研究中发生的治疗期间出现的不良事件（TEAE）、严重不良事件（SAE）、特别关注的不良事件（AESI）以及实验室检查异常","贝舒地尔最后一次用药后最多至第7天","安全性指标"]] 序号 指标 评价时间 终点指标选择

1	姓名	陈静瑜	学位	医学博士	职称	主任医师
	电话	13003329213	Email	chenjy@wuxiph.com	邮政地址	浙江省-杭州市-解放路88号
	邮编	310052	单位名称	浙江大学医学院附属第二医院

[["浙江大学医学院附属第二医院","陈静瑜","中国","浙江省","杭州市"],["浙江大学医学院附属第二医院","黄曼","中国","浙江省","杭州市"],["四川省人民医院","郭璐","中国","四川省","成都市"],["无锡市人民医院","许红阳","中国","江苏省","无锡市"],["中日友好医院","梁朝阳","中国","北京市","北京市"],["安徽省立医院","柯立","中国","安徽省","合肥市"],["郑州大学第一附属医院","李向楠","中国","河南省","郑州市"],["广州医科大学附属第一医院","巨春蓉","中国","广东省","广州市"],["福建医科大学附属协和医院","叶冰","中国","福建省","福州市"],["University of Alberta - Edmonton","Jason Weatherald","CANADA","Edmonton","Edmonton"],["University Health Network","Tereza Martinu","CANADA","Toronto","Toronto"],["Vancouver General Hospital - The Lung Centre","Celine Bergeron","CANADA","Vancouver","Vancouver"],["Emory University Hospital","Purav Shah","UNITED STATES","Atlanta","Atlanta"],["Johns Hopkins University School of Medicine","Christian Merlo","UNITED STATES","Baltimore","Baltimore"],["Advent Health Transplant Institute","Martin Zamora","UNITED STATES","Orlando","Orlando"],["Baylor University Medical Center - Dallas","Todd Grazia","UNITED STATES","Dallas","Dallas"],["Baylor University Medical Center - Houston","Puneet Garcha","UNITED STATES","Houston","Houston"],["Brigham & Women's Hospital/VA Boston Lung Transplant Program","Nirmal Sharma","UNITED STATES","Boston","Boston"],["Cleveland Clinic Foundation","Marie Budev","UNITED STATES","Cleveland","Cleveland"],["Corewell Health","Reda Girgis","UNITED STATES","Grand Rapids","Grand Rapids"],["David Geffen School of Medicine at UCLA","S. Samuel Weigt","UNITED STATES","Los Angeles","Los Angeles"],["Duke Lung Transplant Program","Jamie Todd","UNITED STATES","Durham","Durham"],["Houston Methodist Research Institute","Jihad Youssef","UNITED STATES","Houston","Houston"],["Icahn School of Medicine at Mount Sinai","Harish Seethamraju","UNITED STATES","New York","New York"],["Inova Fairfax Medical Campus - IHVI ALD and Lung Transplant Services","Shambhu Aryal","UNITED STATES","Falls Church","Falls Church"],["Loyola University Medical Center","Daniel Dilling","UNITED STATES","Maywood","Maywood"],["Mayo Clinic","Remzi Bag","UNITED STATES","Jacksonville","Jacksonville"],["Montefiore Medical Center","Ali Mansour","UNITED STATES","Bronx","Bronx"],["New York Presbyterian/Columbia University Medical Center","Selim Arcasoy","UNITED STATES","New York","New York"],["Northwestern University","Ambalavanan Arunachalam","UNITED STATES","Chicago","Chicago"],["NYU Langone Medical Center","Darya Rudym","UNITED STATES","New York","New York"],["OSU Wexner Medical Center","Bronwyn Small","UNITED STATES","Columbus","Columbus"],["St. Joseph's Hospital & Medical Center - Norton Thoracic Research Department","Rajat Walia","UNITED STATES","Phoenix","Phoenix"],["Stanford Health Care - Center for Advance Lung Disease","Amit Banga","UNITED STATES","Stanford","Stanford"],["Tampa General Hospital","Kapilkumar Patel","UNITED STATES","Tampa","Tampa"],["Temple University Hospital","Gerard Criner","UNITED STATES","Philadelphia","Philadelphia"],["UAB Lung Health Center","Keith Wille","UNITED STATES","Birmingham","Birmingham"],["UC San Diego Health","Gordon Yung","UNITED STATES","La Jolla","La Jolla"],["University of Iowa Hospitals and Clinics","Tahuanty Pena","UNITED STATES","Iowa City","Iowa City"],["University of Kentucky - College of Nursing","Sravanthi Nandavaram","UNITED STATES","Lexington","Lexington"],["University of Maryland School of Medicine","Eberlein Michael","UNITED STATES","Baltimore","Baltimore"],["University of Miami - Jackson Memorial Hospital","Andres pelaez","UNITED STATES","Miami","Miami"],["University of Michigan Hospital - 1500 E Medical Center Dr","Dennis Lyu","UNITED STATES","Ann Arbor","Ann Arbor"],["University of Pennsylvania - Harron Lung Center","Andrew Courtwright","UNITED STATES","Philadelphia","Philadelphia"],["UT Health San Antonio - University Hospital","Holly Keyt","UNITED STATES","San Antonio","San Antonio"],["UT Southwestern Medical Center","Vaidehi Kaza","UNITED STATES","Dallas","Dallas"],["Vanderbilt University Medical Center","Anil Trindade","UNITED STATES","Nashville","Nashville"],["Washington University School of Medicine - Division of Pulmonary & Critical Care Medicine","Ramsey Hachem","UNITED STATES","St. Louis","St. Louis"],["Fiona Stanley Hospital","michael musk","Australia","Murdoch","Murdoch"],["Prince Charles Hospital","Peter Hopkins","Australia","Chermside","Chermside"],["St Vincents Hospital","David Darley","Australia","Darlinghurst","Darlinghurst"],["Semmelweis Egyetem","Veronika Muller","Hungry","Budapest","Budapest"],["APHM - CHU Marseille - Hopital Nord","Martine Reynaud","France","Marseille","Marseille"],["APHP - Hopital Bichat","VINCENT BUNEL","France","Paris","Paris"],["Centre Hospitalier Universitaire De Bordeaux","Xavier Demant","France","PESSAC","PESSAC"],["CHU Nantes - Hopital Nord Laennec","Morgane Penhouet","FRANCE","Saint Herblain","Saint Herblain"],["Groupe Hospitalier Paris-Saint Joseph - Hopital Marie Lannelongue","Jerome Le Pavec","FRANCE","La Plessis Robinson","La Plessis Robinson"],["Hopital Foch","Olivier BRUGIERE","FRANCE","Suresnes","Suresnes"],["LMU Klinikum - Grohadern","Juergen Barton","GERMANY","München","München"],["Complexo Hospitalario Universitario A Corura.","MERCEDES DE LA TORRE BRAVOS","SPAIN","La Corura","La Corura"],["Hospital Universitari i Politècnic La Fe","Amparo Sole","SPAIN","Valencia","Valencia"],["Hospital Universitari Vall d'Hebron.","Berta Saez Gimenez","SPAIN","Barcelona","Barcelona"],["Hospital Universitario Marques De Valdecilla.","Victor Mora Cuesta","SPAIN","Santander","Santander"],["Hospital Universitario Puerta de Hierro Majadahonda.","Myriam Aguilar Perez","SPAIN","Majadahonda","Majadahonda"],["Cliniques Universitaires UCL","Francois Carlier","BELGIUM","Godinne","Godinne"],["UZ Leuven","Robin Vos","BELGIUM","Leuven","Leuven"],["Rigshospitalet","Michael Perch","DENMARK","Copenhagen","Copenhagen"],["HUS Helsinki University Hospital","Jussi Tikkanen","FINLAND","Helsinki","Helsinki"],["Universitair Medisch Centrum Groningen","Erik Verschuuren","NETHERLANDS","Groningen","Groningen"],["Azienda Ospedaliera Universitaria Senese","David Bennett","ITALY","Siena","Siena"],["Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico","Francesco B. A. Blasi","ITALY","Milano","Milano"],["Fondazione IRCCS Policlinico San Matteo","Domenica Federica Briganti","ITALY","Pavia","Pavia"],["Istituto Mediterraneo Per I Trapianti E Terapie Ad Alta Specializzazione S.r.l.","lavinia martino","ITALY","Palermo","Palermo"]]

序号	机构名称	主要研究者	国家	省（州）	城市

[["浙江大学医学院附属第二医院人体研究伦理委员会","同意","2024-06-28"]]

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: 10 ； 国际: 180 ；
已入组人数	国内: 登记人暂未填写该信息； 国际: 6 ；
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：2023-10-10；
第一例受试者入组日期	国内：登记人暂未填写该信息；     国际：2024-06-07；
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；