Trial design for evaluation of novel targeted therapies

Gynecologic Oncology

Volumn 116, Issue 2, 2010, Pages 173-176

  Farley, John ^a   Rose, Peter G ^b  

^a UNIFORMED SERVICES UNIVERSITY OF THE HEALTH SCIENCES   (United States)

^b CASE WESTERN RESERVE UNIVERSITY   (United States)

Author keywords

Clinical; Design; Trial

Indexed keywords

BIOLOGICAL MARKER; CYTOTOXIC AGENT; EPIDERMAL GROWTH FACTOR RECEPTOR 2; FLUORODEOXYGLUCOSE;

CANCER SURVIVAL; COMPUTER ASSISTED TOMOGRAPHY; DRUG EFFICACY; HUMAN; IMAGING; IN VIVO STUDY; ONCOLOGY; POSITRON EMISSION TOMOGRAPHY; PRIORITY JOURNAL; PROTEIN EXPRESSION; REVIEW; TREATMENT RESPONSE;

CLINICAL TRIALS, PHASE II AS TOPIC; ENDPOINT DETERMINATION; FEMALE; GENITAL NEOPLASMS, FEMALE; HUMANS; MEDICAL ONCOLOGY; RANDOMIZED CONTROLLED TRIALS AS TOPIC; TUMOR MARKERS, BIOLOGICAL;

EID: 73949161270     PISSN: 00908258     EISSN: 10956859     Source Type: Journal    
DOI: 10.1016/j.ygyno.2009.09.046     Document Type: Review

References (34)

1. Markman M. Antineoplastic agents in the management of ovarian cancer: current status and emerging therapeutic strategies. Trends Pharmacol. Sci. 29 (2008) 515-519
2. Jemal A., Siegel R., Ward E., et al. Cancer statistics, 2008. CA Cancer J. Clin. 58 (2008) 71-96
3. Adjei A.A., Christian M., and Ivy P. Novel designs and end points for phase II clinical trials. Clin. Cancer Res. 15 (2009) 1866-1872
4. Kola I., and Landis J. Can the pharmaceutical industry reduce attrition rates?. Nat. Rev., Drug Discov. 3 (2004) 711-715
5. Leighl N.B., Paz-Ares L., Douillard J.Y., 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. 23 (2005) 2831-2839
6. Moore M.J., Hamm J., Dancey J., et al. Comparison of gemcitabine versus the matrix metalloproteinase inhibitor BAY 12-9566 in patients with advanced or metastatic adenocarcinoma of the pancreas: a phase III trial of the National Cancer Institute of Canada Clinical Trials Group. J. Clin. Oncol. 21 (2003) 3296-3302
7. Shepherd F.A., Giaccone G., Seymour L., et al. Prospective, randomized, double-blind, placebo-controlled trial of marimastat after response to first-line chemotherapy in patients with small-cell lung cancer: a trial of the National Cancer Institute of Canada-Clinical Trials Group and the European Organization for Research and Treatment of Cancer. J. Clin. Oncol. 20 (2002) 4434-4439
8. Dhani N., Tu D., Sargent D.J., Seymour L., and Moore M.J. Alternate endpoints for screening phase II studies. Clin. Cancer Res. 15 (2009) 1873-1882
9. Goldberg R.M., Sargent D.J., Morton R.F., et al. A randomized controlled trial of fluorouracil plus leucovorin, irinotecan, and oxaliplatin combinations in patients with previously untreated metastatic colorectal cancer. J. Clin. Oncol. 22 (2004) 23-30
10. Grothey A., Hedrick E.E., Mass R.D., et al. Response-independent survival benefit in metastatic colorectal cancer: a comparative analysis of N9741 and AVF2107. J. Clin. Oncol. 26 (2008) 183-189
11. Escudier B., Eisen T., Stadler W.M., et al. Sorafenib in advanced clear-cell renal-cell carcinoma. N. Engl. J. Med. 356 (2007) 125-134
12. Ratain M.J., Eisen T., Stadler W.M., et al. Phase II placebo-controlled randomized discontinuation trial of sorafenib in patients with metastatic renal cell carcinoma. J. Clin. Oncol. 24 (2006) 2505-2512
13. Abou-Alfa G.K., Schwartz L., Ricci S., et al. Phase II study of sorafenib in patients with advanced hepatocellular carcinoma. J. Clin. Oncol. 24 (2006) 4293-4300
14. Llovet J.M., Ricci S., Mazzaferro V., et al. Sorafenib in advanced hepatocellular carcinoma. N. Engl. J. Med. 359 (2008) 378-390
15. Shankar L.K., Van den Abbeele A., Yap J., Benjamin R., Scheutze S., and Fitzgerald T.J. Considerations for the use of imaging tools for phase II treatment trials in oncology. Clin. Cancer Res. 15 (2009) 1891-1897
16. Therasse P., Arbuck S.G., Eisenhauer E.A., 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. J. Natl. Cancer Inst. 92 (2000) 205-216
17. Suzuki C., Jacobsson H., Hatschek T., et al. Radiologic measurements of tumor response to treatment: practical approaches and limitations. Radiographics 28 (2008) 329-344
18. Ratain M.J., and Sargent D.J. Optimising the design of phase II oncology trials: the importance of randomisation. Eur. J. Cancer 45 (2009) 275-280
19. Cannistra S.A. Phase II trials in Journal of Clinical Oncology. J. Clin. Oncol. 27 (2009) 3073-3076
20. Rubinstein L., Crowley J., Ivy P., Leblanc M., and Sargent D. Randomized phase II designs. Clin. Cancer Res. 15 (2009) 1883-1890
21. Stadler W.M., 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. 23 (2005) 3726-3732
22. Rosner G.L., Stadler W., and Ratain M.J. Randomized discontinuation design: application to cytostatic antineoplastic agents. J. Clin. Oncol. 20 (2002) 4478-4484
23. Burzykowski T., Buyse M., Piccart-Gebhart M.J., et al. Evaluation of tumor response, disease control, progression-free survival, and time to progression as potential surrogate end points in metastatic breast cancer. J. Clin. Oncol. 26 (2008) 1987-1992
24. Buyse M., Thirion P., Carlson R.W., Burzykowski T., Molenberghs G., and Piedbois P. Relation between tumour response to first-line chemotherapy and survival in advanced colorectal cancer: a meta-analysis. Meta-Analysis Group in Cancer. Lancet 356 (2000) 373-378
25. Goffin J., Baral S., Tu D., Nomikos D., and Seymour L. Objective responses in patients with malignant melanoma or renal cell cancer in early clinical studies do not predict regulatory approval. Clin. Cancer Res. 11 (2005) 5928-5934
26. McShane L.M., Hunsberger S., and Adjei A.A. Effective incorporation of biomarkers into phase II trials. Clin. Cancer Res. 15 (2009) 1898-1905
27. Park J.W., Kerbel R.S., Kelloff G.J., et al. Rationale for biomarkers and surrogate end points in mechanism-driven oncology drug development. Clin. Cancer Res. 10 (2004) 3885-3896
28. Smith I.E. Efficacy and safety of Herceptin in women with metastatic breast cancer: results from pivotal clinical studies. Anticancer Drugs 12 Suppl. 4 (2001) S3-S10
29. Pusztai L., Anderson K., and Hess K.R. Pharmacogenomic predictor discovery in phase II clinical trials for breast cancer. Clin. Cancer Res. 13 (2007) 6080-6086
30. Wahl R.L., Jacene H., Kasamon Y., and Lodge M.A. From RECIST to PERCIST: evolving considerations for PET response criteria in solid tumors. J. Nucl. Med. 50 Suppl. 1 (2009) 122S-150S
31. Demetri G.D., von Mehren M., Blanke C.D., et al. Efficacy and safety of imatinib mesylate in advanced gastrointestinal stromal tumors. N. Engl. J. Med. 347 (2002) 472-480
32. Herbst R.S., Davies A.M., Natale R.B., et al. Efficacy and safety of single-agent pertuzumab, a human epidermal receptor dimerization inhibitor, in patients with non small cell lung cancer. Clin. Cancer Res. 13 (2007) 6175-6181
33. Van den Abbeele A.D. The lessons of GIST-PET and PET/CT: a new paradigm for imaging. Oncologist 13 Suppl. 2 (2008) 8-13
34. Shankar L.K., Hoffman J.M., Bacharach S., et al. Consensus recommendations for the use of 18F-FDG PET as an indicator of therapeutic response in patients in National Cancer Institute Trials. J. Nucl. Med. 47 (2006) 1059-1066