论文标题
在NASH中设计探索性的2B平台试验，并具有相关的共同主要二进制端点
Designing an exploratory phase 2b platform trial in NASH with correlated, co-primary binary endpoints
论文作者
论文摘要
非酒精性脂肪性肝炎（NASH）是非酒精性脂肪肝疾病（NAFLD）的进行性形式，是一种高未满足医疗需求的疾病。平台试验在加速药物开发计划方面为赞助商和试用参与者提供了巨大的好处。在本文中，我们介绍了欧盟雷素财团（以欧盟为中心的临床试验平台）的一些活动，涉及在纳什使用平台试验，尤其是拟议的试验设计，决策规则和仿真结果。对于一组假设，我们介绍了最近与两个卫生当局讨论的仿真研究的结果，以及从试验设计的角度来看这些会议的学习。由于拟议的设计使用了共有二进制端点，因此我们此外讨论了模拟相关二进制端点的不同选项和实际考虑。
Non-alcoholic steatohepatitis (NASH) is the progressive form of nonalcoholic fatty liver disease (NAFLD) and a disease with high unmet medical need. Platform trials provide great benefits for sponsors and trial participants in terms of accelerating drug development programs. In this article, we describe some of the activities of the EU-PEARL consortium (EU Patient-cEntric clinicAl tRial pLatforms) regarding the use of platform trials in NASH, in particular the proposed trial design, decision rules and simulation results. For a set of assumptions, we present the results of a simulation study recently discussed with two health authorities and the learnings from these meetings from a trial design perspective. Since the proposed design uses co-primary binary endpoints, we furthermore discuss the different options and practical considerations for simulating correlated binary endpoints.
