The randomized discontinuation trial: A phase II design to assess growth-inhibitory agents

Molecular Cancer Therapeutics

Volumn 6, Issue 4, 2007, Pages 1180-1185

  Stadler, Walter M ^a  

^a UNIVERSITY OF CHICAGO   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

5 AMINO 4 IMIDAZOLECARBOXAMIDE DERIVATIVE; ANTINEOPLASTIC AGENT; GEFITINIB; PLACEBO; RAF PROTEIN; SORAFENIB; TRASTUZUMAB;

ACCURACY; BREAST CANCER; CANCER INHIBITION; CANCER SURVIVAL; CLINICAL TRIAL; COLON CANCER; CONTROLLED CLINICAL TRIAL; DISEASE COURSE; DOUBLE BLIND PROCEDURE; DRUG TARGETING; DRUG WITHDRAWAL; HUMAN; HYPOTHESIS; KIDNEY CANCER; MALIGNANT NEOPLASTIC DISEASE; MEDICAL DECISION MAKING; METHODOLOGY; OUTCOME ASSESSMENT; PATIENT SELECTION; PHASE 2 CLINICAL TRIAL; PREDICTION; PRIORITY JOURNAL; RANDOMIZED CONTROLLED TRIAL; RESEARCH ETHICS; RESEARCH SUBJECT; REVIEW; STATISTICAL ANALYSIS; TREATMENT OUTCOME; TUMOR VOLUME; UNSPECIFIED SIDE EFFECT;

ANTINEOPLASTIC AGENTS; CLINICAL TRIALS, PHASE II; HUMANS; RANDOMIZED CONTROLLED TRIALS;

EID: 34248193851     PISSN: 15357163     EISSN: None     Source Type: Journal    
DOI: 10.1158/1535-7163.MCT-06-0249     Document Type: Review

References (35)

1. Therasse P, Arbuck SG, Eisenhauer EA, et al. New guidelines to evaluate the response to treatment in solid tumors. European Organization for Research and Treatment of Cancer, National Cancer Institute of the United States, National Cancer Institute of Canada [see comments]. J Natl Cancer Inst 2000;92:205-16.
2. Sheiner LB. Learning versus confirming in clinical drug development. Clin Pharmacol Ther 1997;61:275-91.
3. Booth B, Glassman R, Ma P. Oncology's trials. Nat Rev Drug Discov 2003;2:609-10.
4. Gray R, Manola J, Saxman S, et al. Phase II clinical trial design: methods in translational research from the Genitourinary Committee at the Eastern Cooperative Oncology Group. Clin Cancer Res 2006;12:1966-9.
5. Inoue LY, Thall PF, Berry DA. Seamlessly expanding a randomized phase II trial to phase III. Biometrics 2002;58:823-31.
6. Ratain MJ, Eckhardt SG. Phase II studies of modern drugs directed against new targets: if you are fazed, too, then resist RECIST. J Clin Oncol 2004;22:4442-5.
7. Stadler W. New trial designs to assess antitumor and antiproliferative agents in prostate cancer. Invest New Drugs 2002;20:201-8.
8. Moertel CG, Hanley JA. The effect of measuring error on the results of therapeutic trials in advanced cancer. Cancer 1976;38:388-94.
9. Gehan EA. Update on planning of phase II clinical trials. Drugs Exp Clin Res 1986;12:43-50.
10. Simon R. Optimal two-stage designs for phase II clinical trials. Control Clin Trials 1989;10:1-10.
11. Leighl NB, Paz-Ares L, Douillard JY, et al. Randomized phase III study of matrix metalloproteinase inhibitor BMS-275291 in combination with paclitaxel and carboplatin in advanced non-small-cell lung cancer: National Cancer Institute of Canada-Clinical Trials Group Study BR. 18. J Clin Oncol 2005;23:2831-9.
12. Bissett D, O'Byrne KJ, von Pawel J, et al. Phase III study of matrix metalloproteinase inhibitor prinomastat in non-small-cell lung cancer. J Clin Oncol 2005;23:842-9.
13. Sparano JA, Bernardo P, Stephenson P, et al. Randomized phase III trial of marimastat versus placebo in patients with metastatic breast cancer who have responding or stable disease after first-line chemotherapy: Eastern Cooperative Oncology Group trial E2196. J Clin Oncol 2004; 22:4683-90.
14. Thatcher N, Chang A, Parikh P, et al. Gefitinib plus best supportive care in previously treated patients with refractory advanced non-small-cell lung cancer: results from a randomised, placebo-controlled, multicentre study (Iressa Survival Evaluation in Lung Cancer). Lancet 2005;366: 1527-37.
15. Herbst RS, Giaccone G, Schiller JH, et al. Gefitinib in combination with paclitaxel and carboplatin in advanced non-small-cell lung cancer: a phase III trial-INTACT 2. J Clin Oncol 2004;22:785-94.
16. Giaccone G, Herbst RS, Manegold C, et al. Gefitinib in combination with gemcitabine and cisplatin in advanced non-small-cell lung cancer: a phase III trial-INTACT 1. J Clin Oncol 2004;22:777-84.
17. Brenton JD, Carey LA, Ahmed AA, Caldas C. Molecular classification and molecular forecasting of breast cancer: ready for clinical application? J Clin Oncol 2005;23:7350-60.
18. Simon R, Maitournam A. Evaluating the efficiency of targeted designs for randomized clinical trials. Clin Cancer Res 2004;10:6759-63.
19. Bellacosa A, Kumar CC, Di Cristofano A, Testa JR. Activation of AKT kinases in cancer: implications for therapeutic targeting. Adv Cancer Res 2005;94:29-86.
20. Wakeling AE, Guy SP, Woodburn JR, et al. ZD1839 (Iressa): an orally active inhibitor of epidermal growth factor signaling with potential for cancer therapy. Cancer Res 2002;62:5749-54.
21. Shepherd FA, Rodrigues Pereira J, Ciuleanu T, et al. Erlotinib in previously treated non-small-cell lung cancer. N Engl J Med 2005;353: 123-32.
22. Hirsch FR, Witta S. Biomarkers for prediction of sensitivity to EGFR inhibitors in non-small cell lung cancer. Curr Opin Oncol 2005; 17:118-22.
23. Lavin PT. An alternative model for the evaluation of antitumor activity. Cancer Clin Trials 1981;4:451-7.
24. Kopec JA, Abrahamowicz M, Esdaile JM. Randomized discontinuation trials: utility and efficiency. J Clin Epidemiol 1993;46: 959-71.
25. Capra WB. Comparing the power of the discontinuation design to that of the classic randomized design on time-to-event endpoints. Control Clin Trials 2004;25:168-77.
26. Freidlin B, Simon R. Evaluation of randomized discontinuation design. J Clin Oncol 2005;23:5094-8.
27. Rosner GL, Stadler W, Ratain MJ. Randomized discontinuation design: application to cytostatic antineoplastic agents. J Clin Oncol 2002;20:4478-84.
28. Stadler WM, Rosner G, Small E, et al. Successful implementation of the randomized discontinuation trial design: an application to the study of the putative antiangiogenic agent carboxyaminoimidazole in renal cell carcinoma-CALGB 69901. J Clin Oncol 2005;23: 3726-32.
29. Ratain MJ, Eisen T, Stadler WM, et al. Phase II placebo-controlled randomized discontinuation trial of sorafenib in patients with metastatic renal cell carcinoma. J Clin Oncol 2006;24:2505-12.
30. Moore M, Hirte HW, Siu L, et al. Phase I study to determine the safety and pharmacokinetics of the novel Raf kinase and VEGFR inhibitor BAY 43-9006, administered for 28 days on/7 days off in patients with advanced, refractory solid tumors. Ann Oncol 2005;16: 1688-94.
31. Strumberg D, Richly H, Hilger RA, et al. Phase I clinical and pharmacokinetic study of the novel Raf kinase and vascular endothelial growth factor receptor inhibitor BAY 43-9006 in patients with advanced refractory solid tumors. J Clin Oncol 2005;23:965-72.
32. Wilhelm S, Chien DS. BAY 43-9006: preclinical data. Curr Pharm Des 2002;8:2255-7.
33. Wilhelm SM, Carter C, Tang L, et al. BAY 43-9006 exhibits broad spectrum oral antitumor activity and targets the RAF/MEK/ERK pathway and receptor tyrosine kinases involved in tumor progression and angiogenesis. Cancer Res 2004;64:7099-109.
34. Betensky RA, Louis DN, Cairncross JG. Influence of unrecognized molecular heterogeneity on randomized clinical trials. J Clin Oncol 2002; 20:2495-9.
35. Slamon DJ, Godolphin W, Jones LA, et al. Studies of the HER-2/neu proto-oncogene in human breast and ovarian cancer. Science 1989;244: 707-12.