论文标题
无缝的I/II平台设计，具有潜在Covid-19疗法的活动时间效率终点
A Seamless Phase I/II Platform Design with a Time-To-Event Efficacy Endpoint for Potential COVID-19 Therapies
论文作者
论文摘要
在寻找有效治疗的Covid-19时，最初的重点一直在重新使用治疗上。为了最大程度地找到成功治疗的机会，需要针对该疾病开发的新型治疗方法。在本手稿中，我们描述和评估了敏捷平台的统计设计，敏捷平台是一种自适应随机无缝的I/II期试验平台，旨在快速建立安全剂量的安全范围，并使用贝叶斯顺序试验设计对潜在功效进行研究。都考虑了单剂和组合处理。我们发现该设计可以识别出可靠的潜在治疗方法，这些治疗方法可靠地可靠地识别出小到中等的样本量。
In the search for effective treatments for COVID-19, initial emphasis has been on re-purposed treatments. To maximise the chances of finding successful treatments, novel treatments that have been developed for this disease in particular, are needed. In this manuscript we describe and evaluate the statistical design of the AGILE platform, an adaptive randomized seamless Phase I/II trial platform that seeks to quickly establish a safe range of doses and investigates treatments for potential efficacy using a Bayesian sequential trial design. Both single agent and combination treatments are considered. We find that the design can identify potential treatments that are safe and efficacious reliably with small to moderate sample sizes.
