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...
A Two-Stage Covariate-Adjusted Response-Adaptive Enrichment Design [0.03%]
一种两阶段的调整协变量的以疗效适应性的富集设计方法
Li Yang,Guoqing Diao,William F Rosenberger
Li Yang
In the precision medicine paradigm, it is of interest to identify subgroups that benefit most from the treatment. However, the subgroup often cannot be identified until after a large-scale clinical trial. Clinical trials are often designed ...
Comb-BOIN12: A Utility-Based Bayesian Optimal Interval Design for Dose Optimization in Cancer Drug-Combination Trials [0.03%]
基于效用的贝叶斯最优区间设计在癌症药物联合试验中的剂量优化 Comb-BOIN12
Mengyi Lu,Jingyi Zhang,Ying Yuan et al.
Mengyi Lu et al.
Drug combinations are increasingly utilized in cancer treatment to enhance drug effectiveness through synergistic therapeutic effects. However, determining the optimal biological dose combination (OBDC) in small-scale drug combination trial...
Monitoring Ongoing Clinical Trials under Fractional Brownian Motion with Drift [0.03%]
漂移带分数布朗运动的临床试验监控方法研究
Peng Zhang,Weichung Joe Shih,Yong Lin et al.
Peng Zhang et al.
The standard Brownian motion (Bm) with a linear drift is a convenient statistical structure for monitoring ongoing clinical trials in practice for more than four decades (Lan and DeMets, 1983). Under this model, the most current one-point s...