The Chronic Progressive Repeated Measures (CPRM) Model for Clinical Trials Comparing Change Over Time in Quantitative Trait Outcomes

Author:

Zhao Yu,Edland Steven D.

Abstract

Repeated measures analysis is a common analysis plan for clinical trials comparing change over time in quantitative trait outcomes in treatment versus control. Mixed model for repeated measures (MMRM) assuming an unstructured covariance of repeated measures is the default statistical analysis plan, with alternative covariance structures specified in the event that the MMRM model with unstructured covariance does not converge. We here describe a parsimonious covariance structure for repeated measures analysis that is specifically appropriate for longitudinal repeated measures of chronic progressive conditions. This model has the parsimonious features of the mixed effects model with random slopes and intercepts, but without restricting the repeated measure means to be linear with time. We demonstrate with data from completed trials that this pattern of longitudinal trajectories spreading apart over time is typical of Alzheimer’s disease. We further demonstrate that alternative covariance structures typically specified in statistical analysis plans using MMRM perform poorly for chronic progressive conditions, with the compound symmetry model being anticonservative, and the autoregressive model being poorly powered. Finally, we derive power calculation formulas for the chronic progressive repeated measures model that have the advantage of being independent of the design of the pilot studies informing the power calculations. When data follow the pattern of a chronic progressive condition. These power formulas are also appropriate for sizing clinical trials using MMRM analysis with unstructured covariance of repeated measures.

Publisher

Lifescience Global

同舟云学术

1.学者识别学者识别

2.学术分析学术分析

3.人才评估人才评估

"同舟云学术"是以全球学者为主线,采集、加工和组织学术论文而形成的新型学术文献查询和分析系统,可以对全球学者进行文献检索和人才价值评估。用户可以通过关注某些学科领域的顶尖人物而持续追踪该领域的学科进展和研究前沿。经过近期的数据扩容,当前同舟云学术共收录了国内外主流学术期刊6万余种,收集的期刊论文及会议论文总量共计约1.5亿篇,并以每天添加12000余篇中外论文的速度递增。我们也可以为用户提供个性化、定制化的学者数据。欢迎来电咨询!咨询电话:010-8811{复制后删除}0370

www.globalauthorid.com

TOP

Copyright © 2019-2024 北京同舟云网络信息技术有限公司
京公网安备11010802033243号  京ICP备18003416号-3