Randomized clinical trials with biomarkers: Design issues

Journal of the National Cancer Institute

Volumn 102, Issue 3, 2010, Pages 152-160

  Freidlin, Boris ^a   McShane, Lisa M ^a   Korn, Edward L ^a  

^a NATIONAL CANCER INSTITUTE   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

BIOLOGICAL MARKER; CISPLATIN; DOCETAXEL; EPIDERMAL GROWTH FACTOR RECEPTOR; ERLOTINIB; EXCISION REPAIR CROSS COMPLEMENTING PROTEIN 1; FLT3 LIGAND; GEFITINIB; GEMCITABINE; MIDOSTAURIN; PEMETREXED;

ACUTE GRANULOCYTIC LEUKEMIA; CANCER DIAGNOSIS; CANCER PATIENT; CANCER THERAPY; CHEMOSENSITIVITY; CLINICAL ASSESSMENT; CLINICAL EVALUATION; CLINICAL PRACTICE; CLINICAL TRIAL; CONTROLLED CLINICAL TRIAL; DRUG EFFICACY; DRUG SENSITIVITY; DRUG TARGETING; EXON; FLUORESCENCE IN SITU HYBRIDIZATION; GENE EXPRESSION; GENE MUTATION; HUMAN; LUNG NON SMALL CELL CANCER; MEDICAL DECISION MAKING; METHODOLOGY; NOTE; PRIORITY JOURNAL; PROGNOSIS; RANDOMIZED CONTROLLED TRIAL;

ANTINEOPLASTIC AGENTS; CARCINOMA, NON-SMALL-CELL LUNG; CLINICAL TRIALS, PHASE III AS TOPIC; DNA-BINDING PROTEINS; ENDONUCLEASES; ENZYME ACTIVATION; FMS-LIKE TYROSINE KINASE 3; GENE EXPRESSION REGULATION, ENZYMOLOGIC; GENE EXPRESSION REGULATION, NEOPLASTIC; GLUTAMATES; GUANINE; HUMANS; LEUKEMIA, MYELOID, ACUTE; LUNG NEOPLASMS; MUTATION; PROTEIN KINASE INHIBITORS; QUINAZOLINES; RANDOMIZED CONTROLLED TRIALS AS TOPIC; RECEPTOR, EPIDERMAL GROWTH FACTOR; RESEARCH DESIGN; STAUROSPORINE; TUMOR MARKERS, BIOLOGICAL;

EID: 76349100026     PISSN: 00278874     EISSN: 14602105     Source Type: Journal    
DOI: 10.1093/jnci/djp477     Document Type: Note

References (26)

1. Karapetis CS, Khambata-Ford S, Jonker DJ, et al. K-ras mutations and beneft from cetuximab in advanced colorectal cancer. N Engl J Med. 2004;359(17):1757-1765.
2. Mandrekar SJ, Grothey A, Goetz MP, Sargent DJ. Clinical trial designs for prospective validation of biomarkers. Am J Pharmacogenomics. 2005; 5(5):317-325.
3. Pusztai L, Hess KR. Clinical trial design for microarray predictive marker discovery and assessment. Ann Oncol. 2004;15(12):1731-1737.
4. Sargent DJ, Conley BA, Allegra C, Collette L. Clinical trial designs for predictive marker validation in cancer treatment trials. J Clin Oncol. 2005; 23(9):2020-2027.
5. Therasse P, Carbonnelle S, Bogaerts J. Clinical trials design and treatment tailoring: general principles applied to breast cancer research. Crit Rev Oncol Hematol. 2006;59(2):98-105.
6. Buyse M. Towards validation of statistically reliable biomarkers. Eur J Cancer Suppl. 2007;5(5):89-95.
7. Simon R. The use of genomics in clinical trial design. Clin Cancer Res. 2008;14(19):5984-5993.
8. Mandrekar SJ, Sargent DJ. Clinical trial designs for predictive biomarker validation: theoretical considerations and practical challenges. J Clin Oncol. 2009;27(24):4027-4034.
9. Ransohoff DF. The process to discover and develop biomarkers for cancer: a work in progress. J Natl Cancer Inst. 2008;100(20):1419-1420.
10. Wakelee H, Kernstine K, Vokes E, et al. Cooperative group research efforts in lung cancer 2008: focus on advanced-stage non-small-cell lung cancer. Clin Lung Cancer. 2008;9(6):346-351.
11. Potti A, Mukherjee S, Petersen R, et al. A genomic strategy to refne prognosis in early-stage non-small-cell lung cancer. N Engl J Med. 2006; 355(6):570-580. (Pubitemid 44200650)
12. Kim ES, Hirsh V, Mok T, et al. Geftinib versus docetaxel in previously treated non-small-cell lung cancer (INTEREST): a randomised phase III trial. Lancet. 2008;372(9652):1809-1818.
13. Gilliland DG, Griffn JD. The roles of FLT3 in hematopoiesis and leukemia. Blood. 2002;100(5):1532-1542.
14. Cobo M, Isla D, Massuti B, et al. Customizing cisplatin based on quantitative excision repair cross-complementing 1 mRNA expression: a phase III trial in non-small-cell lung cancer. J Clin Oncol. 2007;25(19): 2747-2754.
15. Cree IA, Kurbacher CM, Lamont A, et al. A prospective randomized controlled trial of tumour chemosensitivity assay directed chemotherapy versus physician's choice in patients with recurrent platinum-resistant ovarian cancer. Anticancer Drugs. 2007;18(9):1093-1101.
16. Rosell R, Vergnenegre A, Fournel P, et al. Pharmacogenetics in lung cancer for the lay doctor. Targeted Oncol. 2008;3(3):161-171.
17. Rosell R, Taron M, Sanchez JJ, et al. Setting the benchmark for tailoring treatment with EGFR tyrosine kinase inhibitors. Future Oncol. 2007;3(3): 277-283.
18. Sparano JA. TAILORx: trial assigning individualized options for treatment (Rx). Clin Breast Cancer. 2006;7(4):347-350.
19. Ransohoff DF. Bias as a threat to the validity of cancer molecular-marker research. Nat Rev Cancer. 2005;5(2):142-149.
20. Simon R, Maitournam A. Evaluating the effciency of targeted designs for randomized clinical trials. Clin Cancer Res. 2004;10(20): 6759-6763.
21. Piantadosi S. Clinical Trials: A Methodologic Perspective. New York, NY: Wiley; 1997.
22. Grignon DJ, Caplan R, Sarkar FH, et al. p53 status and prognosis of locally advanced prostatic adenocarcinoma: a study based on RTOG 8610. J Natl Cancer Inst. 1997;89(2):158-165.
23. Tsao AS, Tang XM, Sabloff B, et al. Clinicopathologic characteristics of the EGFR gene mutation in non-small cell lung cancer. J Thorac Oncol. 2006;1(3):231-239.
24. Jennison C, Turnbull BW. Group Sequential Methods With Applications To Clinical Trials. Boca Raton, FL: Chapman & Hall/CRC; 2000.
25. O'Brien PC, Fleming TR. A multiple testing procedure for clinical trials. Biometrics. 1979;35(3):549-556.
26. Freidlin B, Korn EL. Monitoring for lack of beneft: a critical component of a randomized clinical trial. J Clin Oncol. 2009;27(4):629-633.