Design and Analysis of Clinical Trials with Multiple Co-primary Endpoints

具有多个共同主要终点的临床试验的设计和分析

• 批准号: 22700290
• 负责人: SOZU Takashi
• 金额: $ 1.08万
• 依托单位: Kyoto University
• 依托单位国家: 日本
• 项目类别: Grant-in-Aid for Young Scientists (B)
• 财政年份: 2010
• 资助国家: 日本
• 起止时间: 2010 至 2011
• 项目状态: 已结题
• 来源: https://kaken.nii.ac.jp/grant/KAKENHI-PROJECT-22700290/
• 关键词: 医薬品開発; 臨床試験; 標本サイズ; 生物統計学; 多重性; 優越性; 非劣性; 症例数設計

Statistical methodologies for design and analysis of clinical trials with multiple co-primary endpoints were developed, where the study objective of the trials is to demonstrate statistical significance for all of the endpoints.(1) Power and sample size determinations for each scale of endpoint were provided. The derived convenient sample size formula for continuous normally distributed endpoints provided a clear relationship between the required sample size and the correlations among the endpoints.(2) A further investigation was necessary to improve the testing method combining superiority and non-inferiority of a new treatment compared to a control treatment.(3) The required sample sizes of two methods to alleviate conservative approach of testing co-primary endpoints were practically comparable in typical clinical trial settings.

开发了用于设计和分析具有多个协同主要终点的临床试验的统计学方法，其中试验的研究目的是证明所有终点的统计学显著性。(1)提供了每个终点量表的把握度和样本量测定。推导出的连续正态分布终点的方便样本量公式提供了所需样本量与终点之间相关性之间的明确关系。(2)有必要进行进一步研究，以改进新治疗与对照治疗相比的优效性和非劣效性的检测方法。(3)在典型的临床试验环境中，两种方法所需的样本量几乎相当，以减轻检验协同主要终点的保守方法。

项目成果

Sample Size Determination in Superiority Clinical Trials with Multiple Co-Primary Correlated Endpoints

• DOI: 10.1080/10543406.2011.551329
• 发表时间: 2011-01-01
• 期刊: JOURNAL OF BIOPHARMACEUTICAL STATISTICS
• 影响因子: 1.1
• 作者: Sozu, Takashi;Sugimoto, Tomoyuki;Hamasaki, Toshimitsu
• 通讯作者: Hamasaki, Toshimitsu

Adjustment methods for testing co-primary endpoints in clinical trials

临床试验中测试共同主要终点的调整方法

• 发表时间: 2012
• 作者: Sozu T;Hamasaki T
• 通讯作者: Hamasaki T

Sample size determination in clinical trials with multiple co-primary endpoints including mixed continuous and binary variables

• DOI: 10.1002/bimj.201100221
• 发表时间: 2012-09-01
• 期刊: BIOMETRICAL JOURNAL
• 影响因子: 1.7
• 作者: Sozu, Takashi;Sugimoto, Tomoyuki;Hamasaki, Toshimitsu
• 通讯作者: Hamasaki, Toshimitsu

Sample size determination in clinical trials with multiple co-primary binary endpoints

• DOI: 10.1002/sim.3972
• 发表时间: 2010-09-20
• 期刊: STATISTICS IN MEDICINE
• 影响因子: 2
• 作者: Sozu, Takashi;Sugimoto, Tomoyuki;Hamasaki, Toshimitsu
• 通讯作者: Hamasaki, Toshimitsu

Sample size determination in clinical trials with multiple co-primary endpoints

具有多个共同主要终点的临床试验中样本量的确定

• 发表时间: 2011
• 作者: Sozu T;Sugimoto T;Hamasaki T
• 通讯作者: Hamasaki T