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COVID-19 对正在进行的临床试验的潜在影响:基于 CANNA-TICS 研究的神经耶鲁全球 Tic 严重程度量表的模拟研究。

Potential impact of COVID-19 on ongoing clinical trials: a simulation study with the neurological Yale Global Tic Severity Scale based on the CANNA-TICS study.

机构信息

Hannover Medical School, Hannover, Germany.

European Medicines Agency, Amsterdam, Netherlands.

出版信息

Pharm Stat. 2021 May;20(3):675-691. doi: 10.1002/pst.2100. Epub 2021 Feb 16.

Abstract

The COVID-19 pandemic has manifold impacts on clinical trials. In response, drug regulatory agencies and public health bodies have issued guidance on how to assess potential impacts on ongoing clinical trials and stress the importance of a risk-assessment as a pre-requisite for modifications to the clinical trial conduct. This article presents a simulation study to assess the impact on the power of an ongoing clinical trial without the need to unblind trial data and compromise trial integrity. In the context of the CANNA-TICS trial, investigating the effect of nabiximols on reducing the total tic score of the Yale Global Tic Severity Scale (YGTSS-TTS) in patients with chronic tic disorders and Tourette syndrome, the impact of the two COVID-19 related intercurrent events handled by a treatment policy strategy is investigated using a multiplicative and additive data generating model. The empirical power is examined for the analysis of the YGTSS-TTS as a continuous and dichotomized endpoint using analysis techniques adjusted and unadjusted for the occurrence of the intercurrent event. In the investigated scenarios, the simulation studies showed that substantial power losses are possible, potentially making sample size increases necessary to retain sufficient power. However, we were also able to identify scenarios with only limited loss of power. By adjusting for the occurrence of the intercurrent event, the power loss could be diminished to different degrees in most scenarios. In summary, the presented risk assessment approach may support decisions on trial modifications like sample size increases, while maintaining trial integrity.

摘要

新冠疫情对临床试验产生了多方面的影响。为此,药品监管机构和公共卫生机构发布了指导意见,说明如何评估对正在进行的临床试验的潜在影响,并强调在对临床试验进行修改之前进行风险评估的重要性。本文提出了一项模拟研究,旨在评估在无需揭开试验数据并损害试验完整性的情况下,对正在进行的临床试验的功效的影响。在 CANNA-TICS 试验的背景下,该试验研究了纳比西莫司对减少慢性抽动障碍和妥瑞氏综合征患者耶鲁全球抽动严重程度量表(YGTSS-TTS)总抽动评分的影响,使用乘法和加法数据生成模型,研究了一种治疗策略处理的两种与 COVID-19 相关的并发事件对试验的影响。使用调整和未调整并发事件发生情况的分析技术,对 YGTSS-TTS 作为连续和二分类终点的分析,检验了经验功效。在研究的情景中,模拟研究表明,可能会出现大幅度的功效损失,可能需要增加样本量以保持足够的功效。但是,我们也能够确定只有有限的功效损失的情况。通过调整并发事件的发生,在大多数情况下,可以在不同程度上减少功效损失。总之,所提出的风险评估方法可以支持对临床试验修改(如增加样本量)的决策,同时保持试验的完整性。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/ecd4/8014297/b3a824f0c826/PST-20-675-g001.jpg

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