用于生物标志物引导治疗的癌症II期临床试验。
Phase II cancer clinical trials for biomarker-guided treatments.
作者信息
Jung Sin-Ho
机构信息
a Department of Biostatistics and Bioinformatics , Duke University , Durham , NC , USA.
出版信息
J Biopharm Stat. 2018;28(2):256-263. doi: 10.1080/10543406.2017.1372777. Epub 2017 Nov 27.
Abstract
The design and analysis of cancer clinical trials with biomarker depend on various factors, such as the phase of trials, the type of biomarker, whether the used biomarker is validated or not, and the study objectives. In this article, we demonstrate the design and analysis of two Phase II cancer clinical trials, one with a predictive biomarker and the other with an imaging prognostic biomarker. Statistical testing methods and their sample size calculation methods are presented for each trial. We assume that the primary endpoint of these trials is a time to event variable, but this concept can be used for any type of endpoint.
摘要
使用生物标志物的癌症临床试验的设计与分析取决于多种因素，如试验阶段、生物标志物类型、所使用的生物标志物是否经过验证以及研究目标。在本文中，我们展示了两项II期癌症临床试验的设计与分析，一项使用预测性生物标志物，另一项使用成像预后生物标志物。针对每项试验给出了统计检验方法及其样本量计算方法。我们假定这些试验的主要终点是一个事件发生时间变量，但这一概念可用于任何类型的终点。
相似文献
1
Phase II cancer clinical trials for biomarker-guided treatments.
J Biopharm Stat. 2018;28(2):256-263. doi: 10.1080/10543406.2017.1372777. Epub 2017 Nov 27.
2
Performance of adaptive designs for single-armed phase II oncology trials.
J Biopharm Stat. 2015;25(3):602-15. doi: 10.1080/10543406.2014.920863.
3
An analytical approach to assess the predictive value of biomarkers in Phase II decision making.
J Biopharm Stat. 2013;23(5):1106-23. doi: 10.1080/10543406.2013.814377.
4
Early average change in tumor size in a phase 2 trial: efficient endpoint or false promise?
J Natl Cancer Inst. 2007 Oct 3;99(19):1422-3. doi: 10.1093/jnci/djm167. Epub 2007 Sep 25.
5
Biomarker enrichment considerations in oncology early development single-arm studies.
J Biopharm Stat. 2018;28(2):282-291. doi: 10.1080/10543406.2017.1379533. Epub 2017 Oct 30.
6
Current issues in oncology drug development, with a focus on Phase II trials.
J Biopharm Stat. 2009;19(3):556-62. doi: 10.1080/10543400902802474.
7
Design of phase II cancer trials using a continuous endpoint of change in tumor size: application to a study of sorafenib and erlotinib in non small-cell lung cancer.
J Natl Cancer Inst. 2007 Oct 3;99(19):1455-61. doi: 10.1093/jnci/djm158. Epub 2007 Sep 25.
8
Nine-year change in statistical design, profile, and success rates of Phase II oncology trials.
J Biopharm Stat. 2016;26(1):141-9. doi: 10.1080/10543406.2015.1092030.
9
The design of phase II clinical trials testing cancer therapeutics: consensus recommendations from the clinical trial design task force of the national cancer institute investigational drug steering committee.
Clin Cancer Res. 2010 Mar 15;16(6):1764-9. doi: 10.1158/1078-0432.CCR-09-3287. Epub 2010 Mar 9.
10
Methods for proper handling of overrunning and underrunning in phase II designs for oncology trials.
Stat Med. 2015 Jun 15;34(13):2128-37. doi: 10.1002/sim.6479. Epub 2015 Mar 17.
引用本文的文献
1
Diversity in Oncology Clinical Trials: Current Landscape for Industry-Sponsored Clinical Trials in Asia.
Oncol Ther. 2024 Mar;12(1):115-129. doi: 10.1007/s40487-023-00254-3. Epub 2023 Dec 8.
2
Design and Analysis of Cancer Clinical Trials for Personalized Medicine.
J Pers Med. 2021 May 4;11(5):376. doi: 10.3390/jpm11050376.
本文引用的文献
1
Pemetrexed Plus Cisplatin Versus Gemcitabine Plus Cisplatin According to Thymidylate Synthase Expression in Nonsquamous Non-Small-Cell Lung Cancer: A Biomarker-Stratified Randomized Phase II Trial.
J Clin Oncol. 2015 Aug 1;33(22):2450-6. doi: 10.1200/JCO.2014.59.9324. Epub 2015 Jun 29.
2
Randomized phase II trial designs with biomarkers.
J Clin Oncol. 2012 Sep 10;30(26):3304-9. doi: 10.1200/JCO.2012.43.3946. Epub 2012 Aug 6.
3
On sample size calculation for comparing survival curves under general hypothesis testing.
J Biopharm Stat. 2012;22(3):485-95. doi: 10.1080/10543406.2010.550701.
4
Significance of thymidylate synthase and thyroid transcription factor 1 expression in patients with nonsquamous non-small cell lung cancer treated with pemetrexed-based chemotherapy.
J Thorac Oncol. 2011 Aug;6(8):1392-9. doi: 10.1097/JTO.0b013e3182208ea8.
5
Randomized clinical trials with biomarkers: design issues.
J Natl Cancer Inst. 2010 Feb 3;102(3):152-60. doi: 10.1093/jnci/djp477. Epub 2010 Jan 14.
6
Significance of thymidylate synthase for resistance to pemetrexed in lung cancer.
Cancer Sci. 2010 Jan;101(1):161-6. doi: 10.1111/j.1349-7006.2009.01358.x. Epub 2009 Sep 10.
7
Clinical trial designs for predictive biomarker validation: theoretical considerations and practical challenges.
J Clin Oncol. 2009 Aug 20;27(24):4027-34. doi: 10.1200/JCO.2009.22.3701. Epub 2009 Jul 13.
8
Sample size computation for two-sample noninferiority log-rank test.
J Biopharm Stat. 2005;15(6):969-79. doi: 10.1080/10543400500265736.
9
On the efficiency of targeted clinical trials.
Stat Med. 2005 Feb 15;24(3):329-39. doi: 10.1002/sim.1975.
10
Evaluating the efficiency of targeted designs for randomized clinical trials.
Clin Cancer Res. 2004 Oct 15;10(20):6759-63. doi: 10.1158/1078-0432.CCR-04-0496.
Zotero 插件