TY -的T1 -一个阶段3成人患者的疗效和安全性研究Ravulizumab Neuromyelitis视谱系障碍:研究设计和方法JF -神经学乔-神经病学SP - S4 LP - S5做wnl.0000903072.14566 10.1212/01.。首页c2六世- 99 - 23补充2非盟-肖恩Pittock AU -艾伦Kerstin盟娅斯敏Mashhoon AU -马库斯Yountz Y1 - 2022/12/05 UR - //www.ez-admanager.com/content/99/23_Supplement_2首页/S4.abstract N2 -客观呈现的3期临床试验的设计和原理alxn - 1210 -动- 307 (NCT04201262)。背景Eculizumab补充组件5 (C5)抑制剂批准成人anti-aquaporin-4抗体阳性(AQP4 +)视neuromyelitis谱系障碍(NMOSD)。Ravulizumab绑定相同的C5抗原决定基,有更长的半衰期延长给药间隔(每8对每2周)。我们设计了一个创新的试验没有并发安慰剂暴露评估的有效性和安全性ravulizumab与AQP4 + NMOSD成年人。设计/方法NA。结果alxn1210 -动- 307是一个开放、多中心、单臂研究使用安慰剂组的预防试验作为一个比较器。恒常性和预防维护,包括类似的患者群体,审判过程和端点。敏感性分析是判断病人的不同特点。计算测量混杂,创造价值的主要终点(time-to-first裁决受审复发)。鉴于NMOSD攻击的严重影响,eculizumab批准杜绝使用并发比较器出于伦理方面的原因,因为它需要分配病人安慰剂当有效的治疗存在。non-inferiority功效试验也被认为是,但招聘非常大的样本容量充分权力这一件疾病的研究是不可行的。 Thus, a standard randomized clinical trial design was used. The trial enrolled 58 adults with EDSS score = 7 to receive an infusion of ravulizumab every 8 weeks after the loading dose. The primary treatment period will end when the last enrolled patient reaches week 50 unless a predefined number of patients have an adjudicated on-trial relapse by that time. The entire treatment period will last up to ∼4.5 years.Conclusions ALXN1210-NMO-307 is an ongoing study evaluating the efficacy and safety of ravulizumab in patients with AQP4+ NMOSD. It is designed to be consistent with PREVENT, and robust statistical methods will address the potential impact of an external comparator. ER -