Biomarker based clinical trial design

Chinese Clinical Oncology

Volumn 3, Issue 3, 2014, Pages

  Simon, Richard ^a  

^a NATIONAL CANCER INSTITUTE   (United States)

Author keywords

Adaptive design; Clinical trial design; Companion diagnostic; Enrichment trial; Predictive biomarker

Indexed keywords

EID: 84960450147     PISSN: 23043865     EISSN: 23043873     Source Type: Journal    
DOI: 10.3978/j.issn.2304-3865.2014.02.03     Document Type: Article

References (42)

1. Sawyers CL. The cancer biomarker problem. Nature 2008;452:548-52.
2. Simon RM. eds. Genomic clinical trials and predictive medicine. Cambridge, UK: Cambridge University Press, 2013.
3. Simon RM. An agenda for clinical trials: clinical trials in the genomic era. Clin Trials 2004;1:468-70.
4. Simon R. New challenges for 21st century clinical trials. Clin Trials 2007;4:167-9; discussion 173-7.
5. Mamounas EP, Tang G, Fisher B, et al. Association between the 21-gene recurrence score assay and risk of locoregional recurrence in node-negative, estrogen receptor-positive breast cancer: results from NSABP B-14 and NSABP B-20. J Clin Oncol 2010;28:1677-83.
6. Pusztai L, Hess KR. Clinical trial design for microarray predictive marker discovery and assessment. Ann Oncol 2004;15:1731-7.
7. Jones CL, Holmgren E. An adaptive Simon Two-Stage Design for Phase 2 studies of targeted therapies. Contemp Clin Trials 2007;28:654-61.
8. Freidlin B, McShane LM, Polley MY, et al. Randomized phase II trial designs with biomarkers. J Clin Oncol 2012;30:3304-9.
9. Kim ES, Herbst RS, Wistuba II, et al. The BATTLE trial: personalizing therapy for lung cancer. Cancer Discov 2011;1:44-53.
10. Korn EL, Freidlin B. Outcome--adaptive randomization: is it useful? J Clin Oncol 2011;29:771-6.
11. Barker AD, Sigman CC, Kelloff GJ, et al. I-SPY 2: an adaptive breast cancer trial design in the setting of neoadjuvant chemotherapy. Clin Pharmacol Ther 2009;86:97-100.
12. Stratton MR, Campbell PJ, Futreal PA. The cancer genome. Nature 2009;458:719-24.
13. Simon R, Roychowdhury S. Implementing personalized cancer genomics in clinical trials. Nat Rev Drug Discov 2013;12:358-69.
14. Simon R, Polley E. Clinical trials for precision oncology using next generation sequencing. Personalized Medicine 2013;10:485-95.
15. Shak S. Overview of the trastuzumab (Herceptin) anti-HER2 monoclonal antibody clinical program in HER2-overexpressing metastatic breast cancer. Herceptin Multinational Investigator Study Group. Semin Oncol 1999;26:71-7.
16. Chapman PB, Hauschild A, Robert C, et al. Improved survival with vemurafenib in melanoma with BRAF V600E mutation. N Engl J Med 2011;364:2507-16.
17. Shaw AT, Yeap BY, Solomon BJ, et al. Effect of crizotinib on overall survival in patients with advanced non-small-cell lung cancer harbouring ALK gene rearrangement: a retrospective analysis. Lancet Oncol 2011;12:1004-12.
18. Simon R, Maitournam A. Evaluating the efficiency of targeted designs for randomized clinical trials. Clin Cancer Res 2004;10:6759-63.
19. Simon R, Maitournam A. Evaluating the efficiency of targeted designs for randomized clinical trials: supplement and correction. Clin Cancer Res 2006;12:3229.
20. Maitournam A, Simon R. On the efficiency of targeted clinical trials. Stat Med 2005;24:329-39.
21. Hoering A, Leblanc M, Crowley JJ. Randomized phase III clinical trial designs for targeted agents. Clin Cancer Res 2008;14:4358-67.
22. Mandrekar SJ, Sargent DJ. Clinical trial designs for predictive biomarker validation: theoretical considerations and practical challenges. J Clin Oncol 2009;27:4027-34.
23. Simon R. The use of genomics in clinical trial design. Clin Cancer Res 2008;14:5984-93.
24. Freidlin B, Sun Z, Gray R, et al. Phase III clinical trials that integrate treatment and biomarker evaluation. J Clin Oncol 2013;31:3158-61.
25. Simon R, Wang SJ. Use of genomic signatures in therapeutics development in oncology and other diseases. Pharmacogenomics J 2006;6:166-73.
26. Wang SJ, O'Neill RT, Hung HM. Approaches to evaluation of treatment effect in randomized clinical trials with genomic subset. Pharm Stat 2007;6:227-44.
27. Freidlin B, McShane LM, Korn EL. Randomized clinical trials with biomarkers: design issues. J Natl Cancer Inst 2010;102:152-60.
28. Mandrekar SJ, Sargent DJ. Predictive biomarker validation in practice: lessons from real trials. Clin Trials 2010;7:567-73.
29. Karuri SW, Simon R. A two-stage Bayesian design for co-development of new drugs and companion diagnostics. Stat Med 2012;31:901-14.
30. Hong F, Simon R. Run-in phase III trial design with pharmacodynamics predictive biomarkers. J Natl Cancer Inst 2013;105:1628-33.
31. Simon RM, Paik S, Hayes DF. Use of archived specimens in evaluation of prognostic and predictive biomarkers. J Natl Cancer Inst 2009;101:1446-52.
32. Jiang W, Freidlin B, Simon R. Biomarker-adaptive threshold design: a procedure for evaluating treatment with possible biomarker-defined subset effect. J Natl Cancer Inst 2007;99:1036-43.
33. Wang SJ, Hung HM, O'Neill RT. Adaptive patient enrichment designs in therapeutic trials. Biom J 2009;51:358-74.
34. Rosenblum M, Van der Laan MJ. Optimizing randomized trial designs to distinguish which subpopulations benefit from treatment. Biometrika 2011;98:845-60.
35. Follmann D. Adaptively changing subgroup proportions in clinical trials. Statistic Sinca 1997;7:1085-102.
36. Simon N, Simon R. Adaptive enrichment designs for clinical trials. Biostatistics 2013;14:613-25.
37. Freidlin B, Simon R. Adaptive signature design: an adaptive clinical trial design for generating and prospectively testing a gene expression signature for sensitive patients. Clin Cancer Res 2005;11:7872-8.
38. Wang SJ, James Hung HM, O'Neill RT. Impacts on type I error rate with inappropriate use of learn and confirm in confirmatory adaptive design trials. Biom J 2010;52:798-810.
39. Scher HI, Nasso SF, Rubin EH, et al. Adaptive clinical trial designs for simultaneous testing of matched diagnostics and therapeutics. Clin Cancer Res 2011;17:6634-40.
40. Matsui S, Simon R, Qu P, et al. Developing and validating continuous genomic signatures in randomized clinical trials for predictive medicine. Clin Cancer Res 2012;18:6065-73.
41. Gu X, Yin G, Lee JJ. Bayesian two-step Lasso strategy for biomarker selection in personalized medicine development for time-to-event endpoints. Contemp Clin Trials 2013;36:642-50.
42. Freidlin B, Jiang W, Simon R. The cross-validated adaptive signature design. Clin Cancer Res 2010;16:691-8.