文献检索文档翻译深度研究
Suppr Zotero 插件Zotero 插件
邀请有礼套餐&价格历史记录

新学期,新优惠

限时优惠:9月1日-9月22日

30天高级会员仅需29元

1天体验卡首发特惠仅需5.99元

了解详情
不再提醒
插件&应用
Suppr Zotero 插件Zotero 插件浏览器插件Mac 客户端Windows 客户端微信小程序
高级版
套餐订阅购买积分包
AI 工具
文献检索文档翻译深度研究
关于我们
关于 Suppr公司介绍联系我们用户协议隐私条款
关注我们

Suppr 超能文献

核心技术专利:CN118964589B侵权必究
粤ICP备2023148730 号-1Suppr @ 2025

具有时间事件疗效终点的 COVID-19 疗法的无缝 I/II 期平台设计。

A seamless Phase I/II platform design with a time-to-event efficacy endpoint for potential COVID-19 therapies.

机构信息

Faculty for Informatics and Data Science, University Regensburg, Germany.

MRC Biostatistics Unit, University of Cambridge, UK.

出版信息

Stat Methods Med Res. 2024 Nov;33(11-12):2115-2130. doi: 10.1177/09622802241288348. Epub 2024 Oct 14.


DOI:10.1177/09622802241288348
PMID:39397762
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC11577684/
Abstract

In the search for effective treatments for COVID-19, the initial emphasis has been on re-purposed treatments. To maximize the chances of finding successful treatments, novel treatments that have been developed for this disease in particular, are needed. In this article, 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. The bespoke Bayesian design (i) utilizes randomization during dose-finding, (ii) shares control arm information across the platform, and (iii) uses a time-to-event endpoint with a formal testing structure and error control for evaluation of potential efficacy. 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.

摘要

在寻找有效的 COVID-19 治疗方法时,最初的重点是重新利用已有的治疗方法。为了最大程度地提高找到成功治疗方法的机会,需要开发专门针对这种疾病的新治疗方法。在本文中,我们描述并评估了 AGILE 平台的统计设计,AGILE 平台是一个适应性随机无缝的 I/II 期试验平台,旨在快速确定安全剂量范围,并研究潜在有效的治疗方法。该定制的贝叶斯设计 (i) 在剂量发现过程中利用随机化,(ii) 在平台内共享对照臂信息,以及 (iii) 使用具有正式测试结构和错误控制的时事件终点来评估潜在的疗效。既考虑单药治疗,也考虑联合治疗。我们发现,该设计可以在小到中等样本量下可靠地识别出安全有效的潜在治疗方法。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/6aa9/11577684/52a0c4fc47ec/10.1177_09622802241288348-fig4.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/6aa9/11577684/e1afccf0d210/10.1177_09622802241288348-fig1.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/6aa9/11577684/3917432df496/10.1177_09622802241288348-fig2.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/6aa9/11577684/3562d144dff0/10.1177_09622802241288348-fig3.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/6aa9/11577684/52a0c4fc47ec/10.1177_09622802241288348-fig4.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/6aa9/11577684/e1afccf0d210/10.1177_09622802241288348-fig1.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/6aa9/11577684/3917432df496/10.1177_09622802241288348-fig2.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/6aa9/11577684/3562d144dff0/10.1177_09622802241288348-fig3.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/6aa9/11577684/52a0c4fc47ec/10.1177_09622802241288348-fig4.jpg

相似文献

[1]
A seamless Phase I/II platform design with a time-to-event efficacy endpoint for potential COVID-19 therapies.

Stat Methods Med Res. 2024-11

[2]
AGILE-ACCORD: A Randomized, Multicentre, Seamless, Adaptive Phase I/II Platform Study to Determine the Optimal Dose, Safety and Efficacy of Multiple Candidate Agents for the Treatment of COVID-19: A structured summary of a study protocol for a randomised platform trial.

Trials. 2020-6-19

[3]
Protocol for the DisCoVeRy trial: multicentre, adaptive, randomised trial of the safety and efficacy of treatments for COVID-19 in hospitalised adults.

BMJ Open. 2020-9-21

[4]
A Trial of Favipiravir and Hydroxychloroquine combination in Adults Hospitalized with moderate and severe Covid-19: A structured summary of a study protocol for a randomised controlled trial.

Trials. 2020-10-31

[5]
[Repurposing of chlorpromazine in COVID-19 treatment: the reCoVery study].

Encephale. 2020-6

[6]
Safety and Efficacy of Imatinib for Hospitalized Adults with COVID-19: A structured summary of a study protocol for a randomised controlled trial.

Trials. 2020-10-28

[7]
A Phase 3 Open-label, Randomized, Controlled Study to Evaluate the Efficacy and Safety of Intravenously Administered Ravulizumab Compared with Best Supportive Care in Patients with COVID-19 Severe Pneumonia, Acute Lung Injury, or Acute Respiratory Distress Syndrome: A structured summary of a study protocol for a randomised controlled trial.

Trials. 2020-7-13

[8]
ACCORD: A Multicentre, Seamless, Phase 2 Adaptive Randomisation Platform Study to Assess the Efficacy and Safety of Multiple Candidate Agents for the Treatment of COVID-19 in Hospitalised Patients: A structured summary of a study protocol for a randomised controlled trial.

Trials. 2020-7-31

[9]
Norwegian Coronavirus Disease 2019 (NO COVID-19) Pragmatic Open label Study to assess early use of hydroxychloroquine sulphate in moderately severe hospitalised patients with coronavirus disease 2019: A structured summary of a study protocol for a randomised controlled trial.

Trials. 2020-6-5

[10]
Evaluating the efficacy and safety of human anti-SARS-CoV-2 convalescent plasma in severely ill adults with COVID-19: A structured summary of a study protocol for a randomized controlled trial.

Trials. 2020-6-8

本文引用的文献

[1]
Efficient Adaptive Designs for Clinical Trials of Interventions for COVID-19.

Stat Biopharm Res. 2020-7-29

[2]
Tocilizumab in patients admitted to hospital with COVID-19 (RECOVERY): a randomised, controlled, open-label, platform trial.

Lancet. 2021-5-1

[3]
Adding flexibility to clinical trial designs: an example-based guide to the practical use of adaptive designs.

BMC Med. 2020-11-19

[4]
Effect of Hydroxychloroquine in Hospitalized Patients with Covid-19.

N Engl J Med. 2020-10-8

[5]
Dexamethasone in Hospitalized Patients with Covid-19.

N Engl J Med. 2021-2-25

[6]
Endpoints for randomized controlled clinical trials for COVID-19 treatments.

Clin Trials. 2020-10

[7]
Remdesivir for the Treatment of Covid-19 - Preliminary Report. Reply.

N Engl J Med. 2020-9-3

[8]
The Evolution of Master Protocol Clinical Trial Designs: A Systematic Literature Review.

Clin Ther. 2020-7

[9]
AGILE-ACCORD: A Randomized, Multicentre, Seamless, Adaptive Phase I/II Platform Study to Determine the Optimal Dose, Safety and Efficacy of Multiple Candidate Agents for the Treatment of COVID-19: A structured summary of a study protocol for a randomised platform trial.

Trials. 2020-6-19

[10]
A minimal common outcome measure set for COVID-19 clinical research.

Lancet Infect Dis. 2020-6-12

文献AI研究员

20分钟写一篇综述,助力文献阅读效率提升50倍

立即体验

用中文搜PubMed

大模型驱动的PubMed中文搜索引擎

马上搜索

推荐工具

医学文档翻译智能文献检索