Clinical Trials of Novel and Targeted Therapies: Endpoints, Trial Design, and Analysis

REFERENCES

• Hanahan D., Weinberg R. The hallmarks of cancer. Cell 2000; 100(1)57–70
   PubMed Web of Science ®Google Scholar
• Geller N. Design of phase I and phase II clinical trials in cancer: a statistician's view. Cancer Invest 1984; 2: 483–491
   PubMed Web of Science ®Google Scholar
• Storer B. E. Design and analysis of phase I clinical trials. Biometrics 1989; 45(3)925–937
   PubMed Web of Science ®Google Scholar
• Korn E. L., Midthune D., Chen T. T., Rubinstein L.V., Simon R. M. A comparison of two phase I trial designs. Stat Med 1994; 13(18)1799–1806
   PubMed Web of Science ®Google Scholar
• Simon R., Freidlin B., Rubinstein L., Arbuck S. G., Collin J., Christian M. C. Accelerated titration designs for phase I clinical trials in oncology. JNCI 1997; 89(15)1138–1147
   PubMed Web of Science ®Google Scholar
• Ivanova A., Montazer-Haghighi A., Mohanty S. G., Durham S. D. Improved up-and-down designs for phase I trials. Stat Med 2003; 22: 69–82
   PubMed Web of Science ®Google Scholar
• Gezmu M. The Geometric Up-and-Down Design for Allocating Dosage Levels. 1996, Dissertation, American University
   Google Scholar
• Narayana T. V. Sequential Procedures in the Probit Analysis. 1953, Dissertation, University of North Carolina, Chapel Hill
   Google Scholar
• O'Quigley J., Pepe M., Fisher L. Continual reassessment method: a practical design for phase I clinical trials in cancer. Biometrics 1990; 45: 33–48
   Google Scholar
• Chevret S. The continual reassessment method in cancer phase I clinical trials: a simu lation study. Stat Med 1993; 12: 1093–1108
   PubMed Web of Science ®Google Scholar
• Goodman S. N., Zahurak M. L., Piantadosi S. Some practical improvements in the continual reassessment method for phase I studies. Stat Med 1995; 14: 1149–1161
   PubMed Web of Science ®Google Scholar
• Moller S. An extension of the continual reassessment methods using a preliminary up-and-down design in a dose-finding study in cancer patients, in order to investigate a greater range of doses. Stat Med 1995; 14: 911–922
   PubMed Web of Science ®Google Scholar
• Korn E. L., Arbuck S. G., Pluda J. M., Simon R., Kaplan R. S., Christian M. C. Clinical trial designs for cytostatic agents: are new approaches needed?. J Clin Onc 2001; 19(1)265–272
   PubMed Web of Science ®Google Scholar
• Parulekar W. R., Eisenhauer E.A. Phase I trial design for solid tumor studies of targeted, non-cytotoxic agents: theory and practice. JNCI 2004; 96(13)990–997
   PubMed Web of Science ®Google Scholar
• Ranson M., Jayson G. Targeted antitumor therapy-future perspective. Brit J Cancer 2005; 92(suppl 1)S28–S31
   PubMedGoogle Scholar
• Ma B. B., Britten C. D., Siu L.L. Clinical trial designs for targeted agents. Hematol. Oncol Clin North Am 2002; 16(5)1287–1305
   PubMed Web of Science ®Google Scholar
• Rowinsky E. The pursuit of optimal outcomes in cancer therapy in a new age of ration ally designed target-based anti-cancer agents. Drugs 2000; 60(suppl 1)1–14
   PubMedGoogle Scholar
• Gelmon K. A., Eisenhauer E. A., Harris A. L., Ratain M., Workman P. Anticancer agents target ing signaling molecules and cancer cell development: challenges for drug development?. JNCI 1999; 91(15)1281–1287
   PubMed Web of Science ®Google Scholar
• Hunsberger S., Rubinstein L. V., Dancey J., Korn E.L. Dose escalation trial designs based on molecularly targeted endpoint. Stat Med 2005; 24: 2171–2181
   PubMed Web of Science ®Google Scholar
• Thall P. F., Estey E., Sung H. A new statistical method for dose-finding based on efficacy and toxicity in early phase clinical trial. Invest New Drugs 1999; 17: 155–167
   PubMed Web of Science ®Google Scholar
• Thall P. F., Cook J. D. Dose-finding based on efficacy-toxicity trade-offs. Biometrics 2004; 60(3)684–693
   PubMed Web of Science ®Google Scholar
• Zhang W., Sargent DJ., Mandrekar S. An adaptive dose-finding design incorporating both toxicity and efficacy. Stat Med 2005; 25(14)2365–2383
   Web of Science ®Google Scholar
• 7Simon R. Optimal two-stage designs for phase II clinical trials. Control Clin Trials 1989; 10: 1–10
   PubMedGoogle Scholar
• Fleming T. R. One-sample multiple testing procedure for phase II clinical trials. Biometrics 1982; 38(1)143–151
   PubMed Web of Science ®Google Scholar
• Dent S., Zee B., Dancey J., Hanauske A., Wanders J., Eisenhauer E. Application of a new multinomial phase II stopping rule using response and early progression. J Clin Oncol 2001; 19(3)785–791
   PubMed Web of Science ®Google Scholar
• Zee B., Melnychuk D., Dancey J., Eisenhauer E. Multinomial phase II cancer trials incor porating response and early progression. J Biopharm Stat 1999; 9(2)351–363
   PubMedGoogle Scholar
• Von Hoff D.D. There are no bad anticancer agents, only bad clinical trial designs—twenty-first Richard and Hinda Rosenthal Foundation Award Lecture. Clin Cancer Res 1998; 4(5)1079–1086
   PubMed Web of Science ®Google Scholar
• Chan S., Scheulen M. E., Johnston S., et al. Phase II study of temsirolimus (CCI-779), a novel inhibitor of mTOR, in heavily pretreated patients with locally advanced or metastatic breast cancer. J Clin Oncol 2005; 23(23)5314–5322
   PubMed Web of Science ®Google Scholar
• Perez E. A., Geeraerts L., Suman V. J., et al. A randomized phase II study of sequential docetaxel and doxorubicin/cyclophosphamide in patients with metastatic breast cancer. Ann Oncol 2002; 13(8)1225–1235
   PubMed Web of Science ®Google Scholar
• Herson J., Carter S. K. Calibrated phase II clinical trials in oncology. Stat Med 1986; 5(5)441–447
   PubMed Web of Science ®Google Scholar
• Rubinstein L. V., Korn E. L., Freidlin B., Hunsberger S., Ivy SP., Smith M. A. Design issues of randomized phase II trials and a proposal for phase II screening trials. J Clin Oncol 2005; 23(28)7199–7206
   PubMed Web of Science ®Google Scholar
• Simon R., Wittes R. E., Ellenberg S. S. Randomized phase II clinical trials. Cancer Treat Rep 1985; 69(12)1375–1381
   PubMedGoogle Scholar
• Sargent D. J., Goldberg R. M. A flexible design for multiple armed screening trials. Stat Med 2001; 20(7)1051–1060
   PubMed Web of Science ®Google Scholar
• Thall P. F., Simon R., Ellenberg S. S. A two-stage design for choosing among several exper imental treatments and a control in clinical trials. Biometrics 1989; 45(2)537–547
   PubMed Web of Science ®Google Scholar
• Strauss N., Simon R. Investigating a sequence of randomized phase II trials to discover promising treatments. Stat Med 1995; 14: 1479–1489
   PubMed Web of Science ®Google Scholar
• 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 2005; 23(16)3726–3732
   PubMed Web of Science ®Google Scholar
• Freidlin B., Simon R. Evaluation of randomized discontinuation design. J Clin Oncol 2005; 23(22)5094–5098
   PubMed Web of Science ®Google Scholar
• Wieand H. S. Randomized phase II trials: what does randomization gain?. J Clin Oncol 2005; 23(9)1794–1795
   PubMed Web of Science ®Google Scholar
• Simon R., Maitournam A. Evaluating the efficiency of targeted designs for randomized clinical trials. Clin Cancer Res 2004; 10(20)6759–6763
   PubMed Web of Science ®Google Scholar
• Maitournam A., Simon R. On the efficiency of targeted clinical trials. Stat Med 2005; 24(3)329–339
   PubMed Web of Science ®Google Scholar
• Loi S., Buyse M., Sotiriou C., Cardoso F. Challenges in breast cancer clinical trial design in the postgenomic era. Curr Opin Oncol 2004; 16: 536–541
   PubMed Web of Science ®Google Scholar
• Slamon D. J., Leyland-Jones B., Shak S., et al. Use of chemotherapy plus a monoclonal anti body against HER2 for metastatic breast cancer that overexpresses HER2. N Engl J Med 2001; 344(ll)783–792
   PubMedGoogle Scholar
• Romond E. H., Perez E. A., Bryant J., et al. Trastuzumab plus adjuvant chemotherapy for operable HER2-positive breast cancer. N Engl J Med 2005; 353(16)1673–1684
   PubMed Web of Science ®Google Scholar
• Herbst R. S., Giaccone G., Schiller J. H., 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(5)785–794
   PubMed Web of Science ®Google Scholar
• Giaccone G., Herbst R. S., 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(5)777–784
   PubMed Web of Science ®Google Scholar
• Lynch T. J., Bell D. W., Sordella R., et al. Activating mutations in the epidermal growth factor receptor underlying responsiveness of non-small-cell lung cancer to gefitinib. N Engl J Med 2004; 350(21)2129–2139
   PubMed Web of Science ®Google Scholar
• Cunningham D., Humblet Y., Siena S., et al. Cetuximab monotherapy and cetuximab plus irinotecan in irinotecan-refractory metastatic colorectal cancer. N Engl J Med 2004; 351(4)337–345
   PubMed Web of Science ®Google Scholar
• Chung K. Y., Shia J., Kemeny N. E., et al. Cetuximab shows activity in colorectal cancer patients with tumors that do not express the epidermal growth factor receptor by immu- nohistochemistry. J Clin Oncol 2005; 23(9)1803–1810
   PubMed Web of Science ®Google Scholar
• Saltz L. B. Can the addition of cetuximab to irinotecan improve outcome in colorectal cancer?. Nat Clin Pract Oncol 2005; 2(1)20–21
   PubMedGoogle Scholar
• Sargent D. J., Conley B. A., Allegra C., Collette L. Clinical trial designs for predictive marker validation in cancer treatment trials. J Clin Oncol 2005; 23(9)2020–2027
   PubMed Web of Science ®Google Scholar
• Hecht J. R., Trarbach T., Jaeger E., et al. A randomized, double-blind, placebo-controlled, phase III study in patients (pts) with metastatic adenocarcinoma of the colon or rectum receiving first-line chemotherapy with oxaliplatin/5-fluorouracil/leucovorin and PTK787/ZK 222584 or placebo (CONFIRM-1). J Clin Oncol 2005 ASCO Annual Meeting Proceedings 2005; 23(16S)3
   Google Scholar