Group Sequential Design and Monitoring of Clustered Data in Randomized Eye Trials [0.03%]
随机化眼病临床试验的分组序贯设计和疗效评价方法学研究
Guoqing Diao,Qiang Zhang,Toshimitsu Hamasaki et al.
Guoqing Diao et al.
Group sequential designs are the most commonly used methods for randomized clinical trials with interim monitoring plans, which control the type I error rate and protect statistical power. However, to the best of our knowledge, group sequen...
An Adaptive Biomarker-based Umbrella Trial Design Using Bayesian Latent Class Model [0.03%]
一种基于自适应生物标志物的伞式试验设计，使用贝叶斯潜在类别模型
Jiaying Guo,Mengyi Lu,Yan Han et al.
Jiaying Guo et al.
A Bayesian adaptive umbrella trial design is proposed to identify effective treatments for biomarker-defined subgroups. This design employs a mixture regression model to jointly model short-term binary and long-term time-to-event outcomes a...
A Bayesian Adaptive Marker-Stratified Design for Phase II Clinical Trials Using Calibrated Spike-and-Slab priors [0.03%]
基于校准的_spike-and-slab_先验的贝叶斯自适应分层标志物Ⅱ期临床试验设计
Mu Shan,Mengyi Lu,Leng Han et al.
Mu Shan et al.
The marker-stratified design (MSD) is useful for assessing subgroup-specific treatment effects of molecularly targeted agents (MTA). In MSD, patients are first classified into the marker-positive and marker-negative subgroups, and then rand...
On the Two-Step Hybrid Design for Augmenting Randomized Trials Using Real-World Data [0.03%]
基于真实世界数据增强随机临床试验的两阶段混合设计方法研究
Jiapeng Xu,Ruben P A van Eijk,Alicia Ellis et al.
Jiapeng Xu et al.
Hybrid clinical trials, which borrow real-world data (RWD) from patient registries, claims databases, or electronic health records (EHRs) to augment randomized clinical trials, are of increasing interest. Hybrid clinical trials are especial...
Two-stage Adaptive Enrichment Designs with Survival Outcomes and Adjustment for Misclassification in Predictive Biomarkers [0.03%]
基于生存结局的两阶段自适应富集设计及预测生物标志物误分类调整方法
Yanping Chen,Yong Lin,Shou-En Lu et al.
Yanping Chen et al.
Biomarker enrichment clinical trial designs are versatile tools to assess the treatment effect and increase the efficiency of clinical trials. In this paper, we propose a two-stage enrichment clinical trial design with survival outcomes, an...
A novel longitudinal rank-sum test for multiple primary endpoints in clinical trials: Applications to neurodegenerative disorders [0.03%]
一种新的纵向秩和检验：用于临床试验多个主要终点的新型纵向秩和检验：神经退行性疾病的應用
Xiaoming Xu,Dhrubajyoti Ghosh,Sheng Luo
Xiaoming Xu
Neurodegenerative disorders such as Alzheimer's disease (AD) present a significant global health challenge, characterized by cognitive decline, functional impairment, and other debilitating effects. Current AD clinical trials often assess m...
Isotonic Phase I cancer clinical trial design utilizing patient-reported outcomes [0.03%]
基于患者报告结果的同效性I期抗癌临床试验设计
Nolan A Wages,Ruitao Lin
Nolan A Wages
This article considers the concept of designing Phase I clinical trials using both clinician- and patient-reported outcomes to adaptively allocate study participants to tolerable doses and determine the maximum tolerated dose (MTD) at the s...
Assessment of treatment effect heterogeneity for multiregional randomized clinical trials [0.03%]
多区域随机临床试验的治疗效应异质性评估
Haotian Zhuang,Xiaofei Wang,Stephen L George
Haotian Zhuang
Multiregional clinical trials (MRCTs) have become increasingly common in recent years. Detecting underlying regional heterogeneity is a critical issue for these trials. Existing methods for assessing treatment effect heterogeneity across re...
Unsupervised Imputation of Non-ignorably Missing Data Using Importance-Weighted Autoencoders [0.03%]
利用加权自动编码器对非可忽略性缺失数据进行无监督填充
David K Lim,Naim U Rashid,Junier B Oliva et al.
David K Lim et al.
Deep Learning (DL) methods have dramatically increased in popularity in recent years. While its initial success was demonstrated in the classification and manipulation of image data, there has been significant growth in the application of D...
Thomas R Fleming,Lisa V Hampson,Bharani Bharani-Dharan et al.
Thomas R Fleming et al.
Indolent cancers are characterized by lengthy overall survival (OS) times. Therefore, powering a clinical trial to provide definitive assessment of the effects of an experimental intervention on OS in a reasonable timeframe is generally inf...