文摘
摘要目的:呈现闪亮的结果开放扩展研究(NCT02594124与later-onset SMA)参与者。
背景:几个临床试验已经证明了一个有利的好处:nusinersen风险,建立临床有意义的运动机能功效。
设计/方法:参与者从CS2/12,珍惜和拥抱可以参加发光。协议修正案后,所有的参与者转换到修改后的维护给药方案(MMDR;12毫克nusinersen每4个月)。参与者启动MMDR负荷剂量期间或结束时最后一次访问日期后120天。端点从MMDR第一天被评估。
结果:83名参与者从珍惜nusinersen和42 sham-procedure组转换发光;24从CS2/12转换。使用发光2018年10月15日临时数据,意味着(±SD)哈默史密斯功能性运动放大了(HFMSE)得分MMDR第一天与MMDR一天240年访问(参与者)为26.0(11.01)对于那些收到nusinersen珍惜/ (n = 61),和21.2(7.75)的随机在珍惜/ nusinersen sham-procedure发光(n = 36)。意味着(±SD)修订上肢模块(RULM)分数MMDR第一天为这些组是23.4 (5.54;n = 61), 21.2 (4.31;分别n = 36)。意味着(±SD) HFMSE MMDR第一天与MMDR一天240年访问(参与者)为33.2(12.26)对于那些从CS2/12转换与SMA II型(n = 9)和56.2(6.85)对于SMA类型III (n = 13)。意思是(±SD) RULM MMDR第一天是26.4 (4.81;II型;n = 8)和36.7 (0.58; Type III; n=3), respectively. Data from the 2019 SHINE data cut for these cohorts and those who transitioned from EMBRACE, as well as an updated safety profile, will be presented.
结论:持续发光分析数据将会增加信息的长期安全性/耐受性和疗效重复nusinersen剂量later-onset SMA患者。
研究支持:生原体
披露:奇博士已经收到个人赔偿咨询、担任科学顾问委员会说,或其他活动对AveXis SMA研究顾问委员会,生原体,Cytokinetics,基因泰克,Ionis制药、Inc .和罗氏公司;教育发言人费用从生原体. .奇博士从AveXis收到资助的研究支持,生原体,Ionis制药、Inc .,罗氏,国家卫生研究院和SMA基础. .博士结束之后收到个人赔偿咨询、担任科学顾问委员会说,与AveXis或其他活动,生原体,百时美施贵宝,Cytokinetics,马拉松,PTC疗法,罗氏,Santhera, Sarepta。博士结束之后已经收到了来自美国国立卫生研究院的研究支持/国家神经疾病和中风研究所,为SMA Slaney家族基金,致力于走基金,SMA的基础;CureSMA、Ionis制药公司和生原体,AveXis, Cytokinetics Fibrogen, PTC(列车自动控制系统)。法勒博士已经收到个人赔偿咨询、担任科学顾问委员会说,与顾问委员会或其他活动AveXis,生原体,罗氏。法勒收到了研究支持研究格兰特博士从生原体;首席研究员持续AveXis,罗氏公司和生原体临床试验。麦古力已收到个人赔偿咨询博士担任科学顾问委员会说,或其他活动对AveXis SMA研究顾问委员会,生原体,Ionis制药、Inc .)、诺华和罗氏公司. . Dr. Mercuri has received research support from Principal Investigator for ongoing Biogen/Ionis Pharmaceuticals, Inc. and Roche clinical trials; support from Biogen for a natural history registry; funding from Famiglie SMA Italy, Italian Telethon, and SMA Europe.. Dr. Kirschner has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Advisory boards for SMA studies for AveXis, Biogen, Ionis Pharmaceuticals, Inc., Novartis, and Roche.. Dr. Kirschner has received research support from Research grant from Avexis, Biogen, PTC, Roche, Santhera, and Sarepta.. Dr. Kuntz has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Advisory boards for Argenyx, Audentes, AveXis, Biogen, Cytokinetics, PTC, Roche, and Sarepta.. Dr. Kuntz has received research support from Clinical trial research contracts with Audentes, AveXis, Biogen, Pfizer, Roche, and Sarepta.. Dr. Acsadi has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with AveXis, Biogen, Genentech, and Sarepta.Dr. Tulinius has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Advisory boards for AveXis, Biogen, and PTC.. Dr. Tulinius has received research support from Principal investigator for Ionis Pharmaceuticals Inc./Biogen trials in Sweden.. Dr. Montes has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Advisory boards for Biogen, Cytokinetics, Roche, Scholar Rock, and SMA Foundation; consultant for Biogen and Ionis Pharmaceuticals, Inc.; research support from Eunice Kennedy Shriver National Institute for Child Health and Human Development and Muscular D. Dr. Montes has received research support from Research support from Eunice Kennedy Shriver National Institute for Child Health and Human Development and Muscular Dystrophy Association. Dr. Gambino has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Biogen. Dr. Gambino holds stock and/or stock options in Biogen. Dr. Foster has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities as an employee of Biogen. Dr. Foster holds stock and/or stock options in Holds stock/stock options in Biogen. Dr. Bhan has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Employee of Biogen. Dr. Bhan holds stock and/or stock options in Holds stock/stock options in Biogen. Dr. Wong has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Employee of Biogen. Dr. Kandinov has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Employee of Biogen. Dr. Kandinov holds stock and/or stock options in Holds stock/stock options in Biogen. Dr. Farwell has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Biogen. Dr. Farwell holds stock and/or stock options in Biogen.
