An evaluation of composite endpoints using data from the EXCITE trial

使用 EXCITE 试验的数据评估复合终点

• 批准号: 10645995
• 负责人: Mark R Conaway
• 金额: $ 8.08万
• 依托单位: UNIVERSITY OF VIRGINIA
• 依托单位国家: 美国
• 财政年份: 2023
• 资助国家: 美国
• 起止时间: 2023-04-10 至 2025-03-31
• 项目状态: 未结题
• 来源: https://reporter.nih.gov/project-details/10645995
• 关键词: Affect; Agreement; Authorization documentation; Blinded; Characteristics; Clinical Trials; Consensus; Contracts; Data; Data Analyses; Data Set; Evaluation; Formulation; Funding; Future; Grant; Heterogeneity; Individual; Intervention; Investigation; Ischemia; Measures; Methods; National Institute of Child Health and Human Development; Outcome; Paper; Participant; Patients; Performance; Property; Role; Specimen; Statistical Methods; Structure; Testing; Therapeutic Intervention; Universities; Upper Extremity; Virginia; Work; arm; base; clinically relevant; data access; data hub; data resource; improved; insight; interest; intervention effect; longitudinal design; primary endpoint; primary outcome; randomized, clinical trials; repository; response; secondary analysis; secondary endpoint; trial design

In many clinical trials that assess the effect of an intervention, a single outcome does not adequately capture the effect of the intervention. In these cases, a composite outcome that combines information from several individual outcomes may be preferable. Although there have been a number of statistical tests proposed for composite outcomes, these methods tend to focus on the technical issues of maintain the type I error rate and the effects on statistical power. These are important considerations for designing trials, but do not address other key aspects, such as the characteristics of individual outcomes that would make up a clinical relevant composite measure. This proposal is in response to a Notice of Special Interest for using NICHD data resources in secondary data analyses. The proposal will do secondary analyses of the data from the EXCITE trial, available on the NICHD Data and Specimen Hub (DASH), to investigate the proposed statistical methods with actual clinical trial data, and to gain insight into the characteristics of individual outcomes that combine into a relevant composite outcome.

在许多评估干预效果的临床试验中， 没有充分体现干预的效果。在这些情况下， 一个结合了几个单独结果的信息的结果可能更可取。 虽然已经有一些统计测试提出了复合 结果，这些方法往往侧重于维持I型的技术问题， 错误率和对统计功效的影响。这些都是重要的考虑因素， 设计试验，但没有解决其他关键方面，如 构成临床相关综合指标的个体结局。这 该提案是对使用NICHD数据资源的特别关注通知的回应， 二次数据分析。该提案将对来自 EXCITE试验，可在NICHD数据和标本中心（DASH）上获得，以研究 建议的统计方法与实际的临床试验数据，并深入了解 联合收割机成相关复合结果的单个结果的特征。