拉沙热疗法II/III期平台试验的适应性设计

IF 7.2 2区医学 Q1 IMMUNOLOGY

Emerging Infectious Diseases Pub Date : 2025-01-21 DOI:10.3201/eid3102.240251

Josephine Bourner, Michel Vaillant, Alex Paddy Abdel Salam, Marie Jaspard, Camille Fritzell, Shevin T. Jacob, Tom E. Fletcher, Michael Ramharter, Nnennaya Ajayi, Sylvanus Okogbenin, Cyril Erameh, Donald Grant, Robert Samuels, Oladele Oluwafemi Ayodeji, Armand Sprecher, Bronner P. Gonçalves, Tansy Edwards, Piero Olliaro

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引用次数: 0

摘要

目前用利巴韦林治疗拉沙热的建议只有微弱的证据支持。鉴于利巴韦林在具有地方性传播和流行潜力的地区的持续作用，迫切需要重新评估利巴韦林并研究其他潜在的治疗候选药物；然而，尚未建立适合拉沙热流行病学的强有力的临床试验方法。我们提出了一项自适应II/III期多中心随机对照平台试验，该试验使用具有平等分配比例的优势框架，并考虑到选择主要终点和通过使用中期分析估计目标样本量的挑战。

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Adaptive Design for Phase II/III Platform Trial of Lassa Fever Therapeutics

The current recommendation for treating Lassa fever with ribavirin is supported only by weak evidence. Given the persistent effects in areas with endemic transmission and epidemic potential, there is an urgent need to reassess ribavirin and investigate other potential therapeutic candidates; however, a robust clinical trial method adapted to Lassa fever epidemiology has not yet been established. We propose an adaptive phase II/III multicenter randomized controlled platform trial that uses a superiority framework with an equal allocation ratio and accounts for challenges selecting the primary end point and estimating the target sample size by using an interim analysis.

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来源期刊

Emerging Infectious Diseases 医学-传染病学

CiteScore

17.30

自引率

1.70%

发文量

505

审稿时长

1 months

期刊介绍： Emerging Infectious Diseases is a monthly open access journal published by the Centers for Disease Control and Prevention. The primary goal of this peer-reviewed journal is to advance the global recognition of both new and reemerging infectious diseases, while also enhancing our understanding of the underlying factors that contribute to disease emergence, prevention, and elimination. Targeted towards professionals in the field of infectious diseases and related sciences, the journal encourages diverse contributions from experts in academic research, industry, clinical practice, public health, as well as specialists in economics, social sciences, and other relevant disciplines. By fostering a collaborative approach, Emerging Infectious Diseases aims to facilitate interdisciplinary dialogue and address the multifaceted challenges posed by infectious diseases.