A non-parametric U-statistic testing approach for multi-arm clinical trials with multivariate longitudinal data

Dhrubajyoti Ghosh and Sheng Luo

Journal of Multivariate Analysis, 2025, vol. 209, issue C

Abstract: Randomized clinical trials (RCTs) often involve multiple longitudinal primary outcomes to comprehensively assess treatment efficacy. The Longitudinal Rank-Sum Test (LRST) Xu et al. (2025), a robust U-statistics-based, non-parametric, rank-based method, effectively controls Type I error and enhances statistical power by leveraging the temporal structure of the data without relying on distributional assumptions. However, the LRST is limited to two-arm comparisons. To address the need for comparing multiple doses against a control group in many RCTs, we extend the LRST to a multi-arm setting. This novel multi-arm LRST provides a flexible and powerful approach for evaluating treatment efficacy across multiple arms and outcomes, with a strong capability for detecting the most effective dose in multi-arm trials. Extensive simulations demonstrate that this method maintains excellent Type I error control while providing greater power compared to the two-arm LRST with multiplicity adjustments. Application to the Bapineuzumab (Bapi) 301 trial further validates the multi-arm LRST’s practical utility and robustness, confirming its efficacy in complex clinical trial analyses.

Keywords: Longitudinal data; Rank-Sum Test; U-statistics (search for similar items in EconPapers)
Date: 2025
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DOI: 10.1016/j.jmva.2025.105447

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