北京Zorevunersen注射液III期临床试验-一项评估 Zorevunersen 在Dravet综合征患者中有效性、安全性和耐受性的双盲临床研究

登记号	CTR20262328	试验状态	进行中
申请人联系人	蔡丽娟	首次公示信息日期	2026-06-12
申请人名称	Stoke Therapeutics, Inc./ 艾昆纬医药科技（上海）有限公司/ Sharp Sterile Manufacturing

登记号	CTR20262328
相关登记号	暂无
药物名称	Zorevunersen注射液
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	Dravet综合征
试验专业题目	EMPEROR：一项在Dravet综合征患者中评价zorevunersen（STK-001）的有效性、安全性和耐受性的多中心、随机、双盲、假对照、平行组、III期研究
试验通俗题目	一项评估 Zorevunersen 在Dravet综合征患者中有效性、安全性和耐受性的双盲临床研究
试验方案编号	STK-001-DS-301	方案最新版本号	2.0
版本日期:	2025-09-15	方案是否为联合用药	否

申请人名称	[["Stoke Therapeutics, Inc."],["艾昆纬医药科技（上海）有限公司"],["Sharp Sterile Manufacturing"]]
联系人姓名	蔡丽娟	联系人座机	010-89197555	联系人手机号
联系人Email	lijuan.cai@iqvia.com	联系人邮政地址	广东省-广州市-越秀区越华路112号珠江国际大厦4401	联系人邮编	510030

本研究旨在评估 Zorevunersen 在Dravet综合征患者中的有效性、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	2岁(最小年龄)至 17岁(最大年龄)
性别	男+女
健康受试者	无
入选标准	[["签署知情同意书/儿童知情同意书时，患者必须≥2岁且<18岁。"],["患者必须经Epilepsy Study Consortium, Inc.（ESCI）证实临床诊断为DS，定义为：n-在12月龄前出现复发性局灶性发作伴运动体征、半侧阵挛性发作或全面性强直-阵挛发作。n-无其他已知引起DS临床表现的病因。"],["患者必须经证实携带电压门控钠离子通道1型α亚单位（SCN1A）基因的致病性、可能致病性变异或意义不明确的变异。SCN1A检测结果为阴性（未发现变异）的患者不能参与随机化。"],["在6周观察期间，患者必须出现规定次数的运动性癫痫大发作。纳入计数的癫痫发作类型包括半侧阵挛性发作、局灶性发作伴运动体征、局灶进展为双侧强直-阵挛发作、全面性强直-阵挛发作、强直发作、强直/失张力发作（伴有跌倒或跌倒风险的跌倒发作）和双侧阵挛发作。"],["患者必须既往接受过至少2种癫痫发作的干预治疗。这可能包括抗癫痫发作药物（ASM）、生酮饮食和/或迷走神经刺激术（VNS），但存在以下任一情况：癫痫发作控制不充分或因AE而终止治疗。上述干预可以是正在进行的治疗。"],["患者必须接受至少一次ASM治疗。针对任何适应症而常规使用（即，非按需用药[PRN]）的苯二氮卓类药物或ASM均视为ASM治疗。"],["基线期间，所有维持ASM和癫痫发作的干预治疗（即生酮饮食或VNS）必须保持稳定（根据体重调整除外）。"]]
排除标准	[["患者经证实携带SCN1A基因的功能获得变异。"],["鉴于zorevunersen的作用机制，患者目前正在接受主要作为钠通道阻滞剂的ASM维持治疗，包括但不限于苯妥英、卡马西平、奥卡西平、拉莫三嗪、拉考沙胺、卢非酰胺或西诺氨酯。"],["患者目前正在接受神经调控技术治疗（例如，反应性神经电刺激、脑深部电刺激或经颅磁刺激），VNS除外。"],["患者在基线期间出现新的癫痫发作类型或既往癫痫发作类型（筛选访视A前最后一次发作超过12个月的癫痫发作类型）复发，或在基线期间因癫痫发作住院超过1次。"]]

试验药	[["中文通用名:Zorevunersen注射液 英文通用名:ZorevunersenInjection 商品名称:NA","剂型:注射剂 规格:7.0mg/mL,10ml/瓶 用法用量:TP1-Zorevunersen组将在第1,57,169,281天通过鞘内(IT)给予研究用药。第1,57天的给予70mg，第169,281天的给予45mg。TP2-在TP1中被分配至zorevunersen组的将于第393,477,589天接受45mg的Zorevunersen。 用药时程:每例患者参与研究的时间预计约为108周，包括基线期、治疗期1和治疗期2。"]] 序号 名称 用法
对照药	[["中文通用名:假对照 英文通用名:Sham 商品名称:NA","剂型:NA 规格:NA 用法用量:治疗期1：假对照组不给予研究药物。假对照组将接受一项旨在模拟药物给药的操作。治疗期2：在治疗期1中分配至假对照组的受试者，将在第393,477天分别接受70mg的zorevunersen给药，并在第589天接受45mg的zorevunersen给药。 用药时程:每例患者参与研究的时间预计约为108周，包括基线期、治疗期1和治疗期2。"]] 序号 名称 用法

主要终点指标及评价时间	[["根据每日癫痫发作日记评估癫痫发作日记的变化","第28周时","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	[["根据每日癫痫发作日记评估癫痫发作日记的变化","第52周时","有效性指标"],["文兰适应行为量表第三版（Vineland-3）多维结局评分。n多维结局评分通过较基线的变化进行评估。评分采用非参数方法，基于治疗组与假对照组之间的排序比较。","第52周时","有效性指标"],["文兰适应行为量表第三版（Vineland-3）子领域评分。n采用对各子领域的评分较基线的变化进行评估。评分为基于分值的量表，具体评分范围因子领域而异。","第52周时","有效性指标"]] 序号 指标 评价时间 终点指标选择

1	姓名	姜玉武	学位	医学博士	职称	主任医师
	电话	010-83572211	Email	jiangyuwu@bjmu.edu.cn	邮政地址	北京市-北京市-大兴区乐园路5号北京大学第一医院儿童医学中心5楼
	邮编	102627	单位名称	北京大学第一医院

[["北京大学第一医院","姜玉武","中国","北京市","北京市"],["复旦大学附属儿科医院","王艺","中国","上海市","上海市"],["武汉儿童医院","孙丹","中国","湖北省","武汉市"],["广州医科大学附属第二医院","刘晓蓉","中国","广东省","广州市"],["Hopital de la Timone","Mathieu Milh","France","Bouches-du-Rhone","Marseille"],["Hopital Robert Debre- Paris","Stephane Auvin","France","Paris","Paris"],["Ospedale Pediatrico Bambino Gesu","Nicola Specchio","Italy","Roma","Roma"],["Hopital Necker - Enfants Malades","Rima Nabbout","France","Paris","Paris cedex 15"],["Azienda Ospedaliero Universitaria Ospedale Pediatrico Meyer","Renzo Guerrini","Italy","Firenze","Firenze"],["Fondazione Policlinico Universitario Agostino Gemelli IRCCS","Domenica Battaglia","Italy","Roma","Roma"],["Great Ormond Street Hospital for Children","Helen Cross","United Kingdom","Greater London","London"],["Sheffield Children's Hospital","Archana Desurkar","United Kingdom","South Yorkshire","Sheffield"],["Royal Hospital for Children","Andreas Brunklaus","United Kingdom","Strathclyde","Glasgow"],["National Center of Neurology and Psychiatry","Takashi Saito","Japan","Tokyo-To","Kodaira-shi"],["Hokkaido University Hospital","Kiyoshi Egawa","Japan","Hokkaido","Sapporo-shi"],["Okayama University Hospital","Toshiki Takenouchi","Japan","Okayama-Ken","Okayama-shi"],["NHO Nishiniigata Chuo Hospital","Yu Kobayashi","Japan","Niigata-Ken","Niigata-shi"],["Hospital Ruber Internacional","Antonio Gil-Nagel Rein","Spain","Madrid","Madrid"],["Clinica Universidad de Navarra","Rocio Sanchez-Carpintero Abad","Spain","Navarra","Pamplona"],["Ludwig Maximilian University Hospital Munich Division of Pediatric Neurology","Ingo Borggraefe","Germany","Bayern","Munich"],["Universitaetsklinikum Freiburg","Jan Schoenberger","Germany","Baden Wuerttemberg","Freiburg"],["Universitaetsklinikum Frankfurt Goethe-Universitaet","Susanne Schubert-Bast","Germany","Hessen","Frankfurt"],["Universitaetsklinikum Bonn AoeR","Walid Fazeli","Germany","Nordrhein Westfalen","Bonn"],["Ann & Robert H Lurie Children's Hospital of Chicago","Linda Laux","United States","Illinois","Chicago"],["Beaumont Health System- Colon & Rectal Specialists","Donald Phillips","United States","Michigan","Rochester Hills"],["Cedars Sinai Medical Center","Deborah Holder","United States","California","Los Angeles"],["Children's National Medical Center","John Schreiber","United States","District of Columbia","Washington"],["Cook Children's Medical Center","Michael Perry","United States","Texas","Fort Worth"],["Massachusetts General Hospital","Elizabeth Thiele","United States","Massachusetts","Boston"],["Children's Hospital Colorado","Kelly Knupp","United States","Colorado","Aurora"],["AdventHealth Orlando","Ammar Hussain","United States","Florida","Orlando"],["Nicklaus Children's Hospital","Matt Lallas","United States","Florida","Miami"],["Oregon Health & Science University (OHSU) - PARENT","Colin Roberts","United States","Oregon","Portland"],["University of North Carolina at Chapel Hill","Yael Shiloh-Malawsky","United States","North Carolina","Chapel Hill"],["Duke University Hospital","Muhammad Zafar","United States","North Carolina","Durham"],["Mayo Clinic","Elaine Wirrell","United States","Minnesota","Rochester"],["Northeast Regional Epilepsy Group","Eric Segal","United States","New Jersey","Hackensack"],["UCSF Medical Center","Nilika Singhal","United States","California","San Francisco"],["LeBonheur Children's Hospital","James Wheless","United States","Tennessee","Memphis"],["Phoenix Children's Hospital","Angus Wilfong","United States","Arizona","Phoenix"],["New York University Medical Center","Orrin Devinsky","United States","New York","New York"],["Primary Children's Hospital","Matthew Sweney","United States","Utah","Salt Lake City"],["Universitaetsklinikum Heidelberg","Steffen Syrbe","Germany","Baden Wuerttemberg","Heidelberg"],["CS Mott's Children's Hospital","Laurel Reed","United States","Michigan","Ann Arbor"],["Istituto Giannina Gaslini-Ospedale Pediatrico IRCCS","Pasquale Striano","Italy","Genova","Genova"],["Minnesota Epilepsy Group, P.A.","Douglas Smith","United States","Minnesota","St. Paul"],["Charite - Campus Virchow-Klinikum","Angela Kaindl","Germany","Berlin","Berlin"],["Arkansas Children's Hospital","Debopam Samanta","United States","Arkansas","Little Rock"],["Boston Children's Hospital","Christelle Moufawad El Achkar","United States","Massachusetts","Boston"],["Kyoto University Hospital","Takeshi Yoshida","Japan","Kyoto-Fu","Kyoto-shi"],["Fukuoka Children's Hospital","Ryutaro Kira","Japan","Fukuoka-Ken","Fukuoka-shi"],["Osaka City General Hospital","Shin Okazaki","Japan","Osaka-Fu","Osaka-shi"],["Rhode Island Hospital","John Gaitanis","United States","Rhode Island","Providence"],["Children's Hospital of Orange County","Maija-Riikka Steenari","United States","California","Orange"],["St Marianna University Hospital","Yusaku Miyamoto","Japan","Kanagawa-Ken","Kawasaki-shi"],["Baylor College of Medicine","Anuranjita Nayak","United States","Texas","Houston"],["University of Michigan","Renee Shellhaas","United States","Michigan","Ann Arbor"],["Yokohama City University Medical Center","Yoshihiro Watanabe","Japan","Kanagawa-Ken","Yokohama-shi"],["Weill Cornell Medicine","Asim Shahid","United States","New York","New York"],["University of Rochester Medical Center","Laurie Seltzer","United States","New York","Rochester"],["UVA Health System","Erika Axeen","United States","Virginia","Charlottesville"],["Nemours Children's Health","Fernando Galan","United States","Florida","Jacksonville"],["Kobe University Hospital","Hiroaki Nagase","Japan","Hyogo-Ken","Kobe-shi"],["Nagoya University Hospital","Jun Natsume","Japan","Aichi-Ken","Nagoya-shi"],["Nationwide Children's Hospital","Anup Patel","United States","Ohio","Columbus"],["Cleveland Clinic","Ajay Gupta","United States","Ohio","Cleveland"],["Jichi Medical University Hospital","Kazuhiro Muramatsu","Japan","Tochigi-Ken","Shimotsuke-shi"],["Miyagi Children's Hospital","Kazuhiro Haginoya","Japan","Miyagi-Ken","Sendai-shi"],["NHO Shizuoka Institute of Epilepsy and Neurological Disorders","Tokito Yamaguchi","Japan","Shizuoka-Ken","Shizuoka-shi"],["Hospital Blua Sanitas Valdebebas","Angel Aledo Serrano","Spain","Madrid","Hortaleza"]]

序号	机构名称	主要研究者	国家	省（州）	城市

[["北京大学第一医院生物医学研究伦理委员会","同意","2026-05-11"]]

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: 2 ； 国际: 170 ；
已入组人数	国内: 登记人暂未填写该信息； 国际: 146 ；
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：2025-06-04；
第一例受试者入组日期	国内：登记人暂未填写该信息；     国际：2025-08-04；
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；