文摘
客观的评估的有效性和安全性rozanolixizumab富亮氨酸脑胶质瘤的治疗灭活1 (LGI1)自身免疫性脑炎(AIE)。
背景LGI1 AIE临床是一个齐次综合征由自身抗体,主要IgG4子类,以癫痫、认知障碍和神经精神症状。没有批准的治疗选项可用,目前的治疗模式是变量。Rozanolixizumab是全人源化单克隆抗体,可以交付皮下注射和抑制免疫球蛋白结合地区的新生儿Fc受体(FcRn),减少循环的抗体免疫球蛋白的浓度,包括致病IgG-autoantibodies。
设计/方法这个多中心、随机、双盲、安慰剂对照LEGIONE研究(NCT04875975)是第一第二阶段研究来评估疗效和安全性FcRn抑制的治疗LGI1 AIE。这项研究招募成人血清LGI1-autoantibodies,考虑静脉注射甲基强的松龙治疗,与最近诊断为疾病(经历几个月前研究条目)。之前诊断癫痫患者在筛选,无关LGI1 AIE,免疫球蛋白水平= 5.5 g / L,临床相关的感染或肿瘤疾病的历史将被排除在外。与一个典型的类固醇锥度,∼68名患者将随机1:1皮下注入rozanolixizumab 24周或安慰剂,分层时间从疾病发生和认知功能(可重复的测量电池的神经心理学评估状态)在随机化。主要终点是衡量没收自由(连续28天的癫痫保持到最后的治疗周期)。二次端点认知功能的变化,利用救援药物的时间发作的癫痫自由和rozanolixizumab的安全性和耐受性。探索性的药代动力学/药效学和biomarker-based端点的预期。
结果研究背景、理论基础和设计将呈现。
结论LEGIONE研究是第一个随机、双盲、安慰剂对照研究第二阶段评估FcRn抑制剂的疗效和安全性,rozanolixizumab,富亮氨酸glioma-inactivated患者1自身免疫性脑炎和招收患者。
脚注
披露:Dubey博士的机构已收到个人薪酬在500 - 4999美元的范围作为顾问的联合。Dubey博士的机构已收到个人薪酬在5000 - 9999美元的范围作为顾问阿斯特拉。Dubey博士已经收到个人补偿的范围为0 - 499美元在愁云密布的演讲人。Dubey博士已经收到个人补偿的范围为0 - 499美元在神经学的进步的演讲人。首页Dubey博士已经收到个人补偿的范围0 - 499美元为服务Moffit所癌症中心的演讲人。Dubey博士已经收到了来自国防部的研究支持。Dubey博士已经收到知识产权利益从发现或技术有关卫生保健。Dubey博士已经收到知识产权利益从发现或技术有关卫生保健。Dubey博士已经收到知识产权利益从发现或技术有关卫生保健。Titulaer博士的机构已经收到了来自荷兰癫痫基础研究支持(NEF 14 - 18和19-08)。 The institution of Dr. Titulaer has received research support from CSL Behring. The institution of Dr. Titulaer has received research support from UCB. The institution of Dr. Titulaer has received research support from Netherlands Organisation for Scientific Research (ZonMW, Memorabel initiative and E-RARE UltraAIE). The institution of Dr. Titulaer has received research support from Horizon Therapeutics. The institution of Dr. Titulaer has received research support from Dioraphte (charity). The institution of Dr. Titulaer has received research support from Guidepoint Global LLC. Dr. Koul has stock in UCB. An immediate family member of Dr. Koul has received intellectual property interests from a discovery or technology relating to health care. An immediate family member of Dr. Koul has received personal compensation in the range of $0-$499 for serving as a Scientific advisory board member with CSIR. Dr. Yates has received personal compensation for serving as an employee of UCB Biosciences, Inc. Dr. Yates has stock in UCB Biosciences, Inc. Dr. Irani has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Immunovant. Dr. Irani has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for UCB. Dr. Irani has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for UCB. Dr. Irani has received personal compensation in the range of $500-$4,999 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for Brain. Dr. Irani has received personal compensation in the range of $10,000-$49,999 for serving as an Expert Witness for kinmi. The institution of Dr. Irani has received research support from UCB / CSL. Dr. Irani has received intellectual property interests from a discovery or technology relating to health care.
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