Elevidys ENVISION 3期临床试验
研究概览
- 年龄
- 8岁及以上
- 阶段
- III 期
- 申办方
- Sarepta Therapeutics
- 治疗方式
- 基因治疗
- 变异要求
- 基于记录临床发现和既往基因检测的确诊DMD诊断。
- 适用性别
- 男性
- 行走能力
- 可行走与不可行走
- 研究开始（实际）
- 2023-05-31
- 主要完成（预计）
- 2027-05-31
- 研究完成（预计）
- 2028-06-30
- 入组人数（预计）
- 148
- 国家
- 美国澳大利亚比利时加拿大德国中国香港特别行政区以色列意大利日本韩国西班牙瑞典台湾英国
研究要求与标准
类固醇使用
筛选前至少12周口服皮质类固醇稳定每日剂量，预期整个研究期间保持不变（因体重变化调整除外）。
纳入标准
- 基于记录临床发现和既往基因检测的确诊DMD诊断。
- 仅队列1：按方案规定标准不能行走。
- 仅队列2：按方案规定标准可行走，筛选时≥8至<18岁。
- 能够配合运动评估测试。
- 筛选前至少12周口服皮质类固醇稳定每日剂量，预期整个研究期间保持不变（因体重变化调整除外）。
- 重组腺相关病毒血清型rh74（rAAVrh74）抗体滴度按方案要求未升高。
- DMD基因致病性移码突变或提前终止密码子，外显子8和/或9缺失突变除外。
排除标准
- 在方案规定时限内暴露于基因治疗、研究性药物或任何旨在增加抗肌萎缩蛋白表达的治疗。
- 方案规定的诊断评估或实验室检测异常。
- 研究者认为存在对基因转移造成不必要风险的其他具有临床意义的疾病、医学状况或慢性药物治疗需求。
联系人与地点
联系人与地点
联系人
尚未提供
地点
本研究共有 46 个地点
United States
Arkansas 地点
Little Rock, Arkansas, United States, 72202
Arkansas Children's Hospital
California 地点
Palo Alto, California, United States, 94304
Lucile Packard Children's Hospital Stanford
Sacramento, California, United States, 95817
University of California at Davis Medical Center
San Diego, California, United States, 92123
Rady Children's Hospital-San Diego
Florida 地点
Gainesville, Florida, United States, 32608
University of Florida, UF Health Center for Pediatric Neuromuscular and Rare Diseases
Illinois 地点
Chicago, Illinois, United States, 60611
Ann & Robert H. Lurie Children's Hospital of Chicago
Maryland 地点
Baltimore, Maryland, United States, 21287
The Johns Hopkins Hospital, Charlotte R. Bloomberg Children's Center, Pediatric Clinical Research Unit
Massachusetts 地点
Boston, Massachusetts, United States, 02115
Boston Children's Hospital
Missouri 地点
St Louis, Missouri, United States, 63110
Washington University of St. Louis, St. Louis Children's Hospital
New York 地点
Rochester, New York, United States, 14642
University of Rochester, Department of Neurology
North Carolina 地点
Durham, North Carolina, United States, 27705
Lenox Baker Children's Hospital (Duke University)
Ohio 地点
Columbus, Ohio, United States, 43205
Nationwide Children's Hospital
Pennsylvania 地点
Philadelphia, Pennsylvania, United States, 19104
Children's Hospital of Philadelphia
Virginia 地点
Norfolk, Virginia, United States, 23510
Children's Hospital of the King's Daughters
Australia
New South Wales 地点
Westmead, New South Wales, Australia, 2145
The Children's Hospital at Westmead
Victoria 地点
Parkville, Victoria, Australia, 3052
The Royal Children's Hospital
Belgium
Oost-Vlaanderen 地点
Ghent, Oost-Vlaanderen, Belgium, 9000
Universitair Ziekenhuis Gent
Canada
Ontario 地点
Ottawa, Ontario, Canada, K1H8L1
The Children's Hospital of Eastern Ontario
Quebec 地点
Montreal, Quebec, Canada, H4A3J1
Research Institute McGill University Health Centre
Québec, Canada, G1V 4G2
Centre Hospitalier Universitaire de Québec - Université Laval (pavillon Centre Hospitalier Universitaire Laval)
Germany
Bavaria 地点
München, Bavaria, Germany
LMU- Klinikum der Universitat Munchen, Kinderklinik und Kinderpoliklinik im Dr. von Haunerschen Kinderspital, Abeteilung Neuropadiatrie, Campus Innenstadt
North Rhine-Westphalia 地点
Essen, North Rhine-Westphalia, Germany
Universitatsklinikum Essen, Klinik fur Kinderheilkunde I, Abteilung Neuropadiatrie Essen
Hamburg, Germany
Universitatsklinikum Hamburg Eppendorf
Hong Kong
Hong Kong, Hong Kong
Hong Kong Children's Hospital
Israel
Petah Tikva, Israel
Institute of Neruology, Schneider Children's Medical Center of Israel
Tel Aviv, Israel, 6423906
Tel Aviv Sourasky Medical Center
Italy
Genova, Italy, 16147
U.O.S.D Centro Traslazionale di Miologia e Patologie Neurodegenerative, Istituto G. Gaslini, Istituto Pediatrico di Ricovero e Cura a Carattere Scientifico
Milan, Italy, 20122
UOC Neurologia, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico
Milan, Italy, 20133
IRCCS Istituto Neurologico Carlo Besta Neurepsichiatria Infantile 2 - Epilettologia e Neurologia dello Sviluppo
Roma, Italy, 00168
UOC Neuropsichiatria Infantile, Area Salute del Bambino, Fondazione Policlinico Universitario A. Gemelli IRCCS
Japan
Osaka 地点
Toyonaka-shi, Osaka, Japan, 560-8552
National Hospital Organization Osaka Toneyama Medical Center
Tokyo 地点
Kodaira, Tokyo, Japan, 187-8551
National Center of Neurology and Psychiatry
Shinjuku-ku, Tokyo, Japan, 162-866
Tokyo Women's Medical University Hospital
South Korea
Gyeongsangnam-do 地点
Yangsan, Gyeongsangnam-do, South Korea, 50612
Pusan National University Yangsan Hospital
NAP 地点
Seoul, NAP, South Korea, 03080
Seoul National University Hospital
Daegu, South Korea, 41944
Kyungpook National University Hospital
Seoul, South Korea, 06273
Gangnam Severance Hospital, Yonsei University Health System
Spain
Barcelona 地点
Esplugues de Llobregat, Barcelona, Spain, 08950
Hospital Sant Joan de Deu
Valencia, Spain, 46026
Hospital Universitari Politecnic La Fe
Sweden
Gothenburg, Sweden, 416 85
Sahlgrenska Universitetssjukhuset
Solna, Sweden, 171 76
Karolinska Universitetssjukhuset/Astrid Lindgrens Barnsjukhus, Barnneurologen
Taiwan
Kaohsiung City, Taiwan
Kaohsiung Medical University Chung-Ho Memorial Hospital
Taipei, Taiwan
National Taiwan University Hospital
United Kingdom
Greater London 地点
London, Greater London, United Kingdom, WC1N 3JH
Great Ormond Street Hospital for Children Foundation Trust
Oxfordshire 地点
Oxford, Oxfordshire, United Kingdom
Oxford University Hospitals NHS Foundation Trust
Newcastle upon Tyne, United Kingdom, NE13BZ
Institute of Translational and Clinical Research
临床试验注册
NCT 编号
NCT05881408本信息仅供教育参考。做出医疗决定前请务必咨询研究负责人。