Statistical Designs for Early Phases of Cancer Clinical Trials

REFERENCES

• Babb , J. , Rogatko , A. , Zacks , S. ( 1998 ). Cancer Phase I clinical trials: Efficient dose escalation with overdose control . Statistics in Medicine 17 : 1103 – 1120 .
   PubMed Web of Science ®Google Scholar
• Berry , D. A. , Eick , S. G. ( 1995 ). Adaptive assignment versus balanced randomization in clinical trials: A decision analysis . Statistics in Medicine 14 : 231 – 246 .
   PubMed Web of Science ®Google Scholar
• Braun , T. M. ( 2006 ). Generalizing the TITE-CRM to adapt for early-and late-onset toxicities . Statistics in Medicine 25 : 2071 – 2083 .
   PubMed Web of Science ®Google Scholar
• Burzykowski , T. , Buyse , M. , et al. ( 2008 ). Evaluation of tumor response, disease control, progression-free survival, and time to progression as potential surrogate end points in metastatic breast cancer . Journal of Clinical Oncology 26 : 1987 – 1992 .
   PubMed Web of Science ®Google Scholar
• Carter , S. K. ( 1987 ). The Phase I study . In: Hellmann , K. K. , Carter , S. K. , eds. Fundamentals of Cancer Chemotherapy . New York : McGraw-Hill , pp. 285 – 300 .
   Google Scholar
• Chevret , S. ( 2006 ). Statistical Methods for Dose-finding Experiments . Chichester , UK : Wiley .
   Google Scholar
• Cheung , Y. K. , Chappell , R. ( 2000 ). Sequential designs for Phase I clinical trials with late-onset toxicities . Biometrics 56 : 1177 – 1182 .
   PubMed Web of Science ®Google Scholar
• Collins , J. M. , Zaharko , D. S. , Dedrick , R. L. , Chabner , B. A. ( 1986 ). Potential roles for preclinical pharmacology in Phase I clinical trials . Cancer Treat Reports 70 : 73 – 80 .
   PubMedGoogle Scholar
• Dent , S. , Zee , B. , Dancey , J. , Hanauske , A. , Wanders , J. , Eisenhauer , E. ( 2001 ). Application of a new multinomial Phase II stopping rule using response and early progression . Journal of Clinical Oncology 19 : 785 – 791 .
   PubMed Web of Science ®Google Scholar
• Dhani , N. , Tu , D. , Sargent , D. J. , Seymour , L. , Moore , M. J. (2009). Alternate endpoints for screening Phase II studies. Clinical Cancer Research 15:1873–1882.
   PubMed Web of Science ®Google Scholar
• Douillard , J. Y. , Cunningham , D. , Roth , A. D. , Navarro , M. , James , R. D. , Karasek , P. , Jandik , P. , Iveson , T. , Carmichael , J. , Alakl , M. , Gruia , G. , Awad , L. , Rougier , P. ( 2000 ). Irinotecan combined with fluorouracil compared with fluorouracil alone as first-line treatment for metastatic colorectal cancer: a multicentre randomised trial . Lancet 355 : 1041 – 47 .
   PubMed Web of Science ®Google Scholar
• Ensign , L. G. , Gehan , E. A. , Kamen , D. S. , Thall , P. F. ( 2006 ). An optimal three-stage design for Phase II clinical trials . Statistics in Medicine 13 : 1727 – 1736 .
   Web of Science ®Google Scholar
• Faries , D. Practical modifications of the continual reassessment method for Phase I cancer clinical trials . Journal of Biopharmaceutical Statistics 4 : 147 – 164 .
   PubMedGoogle Scholar
• Fleming , T. R. , Rothmann , M. D. , Lu , H. L. ( 2009 ). Issues in using progression-free survival when evaluating oncology products . Journal of Clinical Oncology 27 : 2874 – 2880 .
   PubMed Web of Science ®Google Scholar
• Foo , K. F. , Tan , E. H. , Leong , S. S. , Wee , J. T. S. , Tan , T. , Fong , K. W. , Koh , L. , Tai , B. C. , Lian , L. G. , Machin , D. ( 2002 ). Gemcitabine in metastatic nasopharyngeal carcinoma of the undifferentiated type . Annals of Oncology 13 : 150 – 156 .
   PubMed Web of Science ®Google Scholar
• Gasparini , M. , Eisele , J. ( 2000 ). A curve-free method for Phase I clinical trials . Biometrics 56 : 609 – 615
   PubMed Web of Science ®Google Scholar
• Gehan , E. A. ( 1961 ). The determination of the number of patients required in a preliminary and a follow-up of a new chemotherapeutic agent . Journal of Chronic Diseases 13 : 346 – 353 .
   PubMedGoogle Scholar
• Gerke , O. , Siedentop , H. ( 2007 ). Optimal Phase I dose-escalation trial designs in oncology—A simulation study . Statistics in Medicine 27 : 5329 – 5344 .
   Web of Science ®Google Scholar
• Goffin , J. , Baral , S. , Tu , D. , Nomikos , D. , Seymour , L. ( 2005 ) Objective responses in patients with malignant melanoma or renal cell cancer in early clinical studies do not predict regulatory approval . Clinical Cancer Research 11 : 5928 – 5934 .
   PubMed Web of Science ®Google Scholar
• Green , S. J. , Dahlberg , S. ( 1992 ). Planned versus attained design in Phase II clinical trials . Statistics in Medicine 10 : 1 – 10 .
   Google Scholar
• Goodman , S. N. , Zahurak , M. L. , Piantadosi , S. ( 1995 ). Some practical improvements in the continual reassessment method for Phase I studies . Statistics in Medicine 14 : 1149 – 1161 .
   PubMed Web of Science ®Google Scholar
• Heitjan , D. F. ( 1997 ). Bayesian interim analysis of Phase II cancer clinical trials . Statistics in Medicine 16 : 1791 – 1802 .
   PubMed Web of Science ®Google Scholar
• Herson , J. ( 1979 ). Predictive probability early termination for Phase II clinical trials . Biometrics 35 : 775 – 783 .
   PubMed Web of Science ®Google Scholar
• Herndon , H. J. ( 1998 ). A design alternative for two-stage, Phase II, multicenter cancer clinical trials . Controlled Clinical Trials 19 : 440 – 450 .
   PubMedGoogle Scholar
• Ji , Y. , Li , Y. , Bekele , B. ( 2007 ). Dose-finding in Phase I clinical trials based on toxicity probability intervals . Clinical Trials 4 : 235 – 244 .
   PubMed Web of Science ®Google Scholar
• Johns , D. , Andersen , J. S. ( 1999 ). Use of predictive probabilities in Phase II and Phase III clinical trials . Journal of Biopharmaceutical Statistics 9 : 67 – 69 .
   PubMedGoogle Scholar
• Kolata , G. ( 2009 ). Lack of study volunteers hobbles cancer fight. New York Times, August 3, 2009, p.A1 .
   Google Scholar
• Kola , I. , Landis , J. ( 2004 ). Can the pharmaceutical industry reduce attrition rates? Nature Reviews Drug Discovery 3 : 711 – 716 .
   PubMed Web of Science ®Google Scholar
• Korn , E. L. , Midthune , D. , Chen , T. T. , Rubinstein , L. V. , Christian , M. C. , Simon , R. M. ( 1994 ). A comparison of two Phase I trial designs . Statistics in Medicine 13 : 1799 – 1806 .
   PubMed Web of Science ®Google Scholar
• Kramar , A. , Lebecq , A. , Candalh , E. ( 1999 ). Continual reassessment methods in Phase I trials of the combination of two drugs in oncology . Statistics in Medicine 18 : 1849 – 1864 .
   PubMed Web of Science ®Google Scholar
• Lee , J. J. , Feng , L. ( 2005 ). Randomized Phase II designs in cancer clinical trials: Current status and future directions . Journal of Clinical Oncology 23 : 4450 – 4457 .
   PubMed Web of Science ®Google Scholar
• Leong , S. S. , Wee , J. , Tay , M. H. , Toh , C. K. , Tan , S. B. , Thng , C. H. , Foo K. F., Lim , W. T. , Tan , T. , Tan , E. H. ( 2005 ). Paclitaxel, carboplatin, and gemcitabine in metastatic nasopharyngeal carcinoma. A Phase II trial using a triplet combination . Cancer 103 : 569 – 575 .
   PubMed Web of Science ®Google Scholar
• Leung , D. H. Y , Wang , Y. G. (2001). Isotonic designs for Phase I trials. Controlled Clinical Trials 22:126–138.
   PubMedGoogle Scholar
• Lin , Y. , Shih , W. J. ( 2001 ). Statistical properties of the traditional algorithm-based designs for Phase I cancer clinical trials . Biostatistics 2 : 203 – 215 .
   PubMedGoogle Scholar
• Lin , Y. , Shih , W. J. ( 2004 ). Adaptive two-stage designs for single-arm Phase IIA cancer clinical trials . Biometrics 60 : 482 – 490 .
   PubMed Web of Science ®Google Scholar
• Lu , Y. , Jin , H. , Lamborn , R. ( 2005 ). A design of Phase II cancer trials using total and complete response endpoints . Statistics in Medicine 24 : 3155 – 3170 .
   PubMed Web of Science ®Google Scholar
• Moller , S. ( 1995 ). 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 . Statistics in Medicine 14 : 911 – 922 .
   PubMed Web of Science ®Google Scholar
• Muler , J. H. , McGinn , C. J. , Normolle , D. , Lawrence , T. , Brown , D. , Hejna , G. , Zalupski , M. M. ( 2004 ). Phase I trial using a time-to-event continual reassessment strategy for dose escalation of cisplatin combined with gemcitabine and radiation therapy in pancreatic cancer . Journal of Clinical Oncology 22 : 238 – 243 .
   PubMed Web of Science ®Google Scholar
• O'Quigley , J. ( 2006 ). Phase I and Phase I/II dose finding algorithms using continual reassessment method . In: Crowley , J , Ankerst , D. P. , eds. Handbook of Statistics in Clinical Oncology . 2nd ed. Boca Raton , FL : Chapman & Hall/CRC , pp. 31 – 58 .
   Google Scholar
• O'Quigley , J. , Hughes , M. D. , Fenton , T. ( 2001 ). Dose-finding designs for HIV studies . Biometrics 57 : 1018 – 1029 .
   PubMed Web of Science ®Google Scholar
• O'Quigley , J. , Pepe , M. , Fisher , L. ( 1990 ). Continual reassessment method: A practical design for Phase I clinical trials in cancer . Biometrics 46 : 33 – 48 .
   PubMed Web of Science ®Google Scholar
• O'Quigley , J. , Reiner , E. ( 1998 ). A stopping rule for the continual reassessment methods . Biometrika 85 : 741 – 748 .
   Web of Science ®Google Scholar
• Shu , J. , O'Quigley , J. ( 2008 ). Dose-escalation designs in oncology: ADEPT and the CRM . Statistics in Medicine 27 : 5345 – 5353 .
   PubMed Web of Science ®Google Scholar
• Panageas , K. S. , Smith , A. , Gönen , M. , Chapman , P. B. ( 2002 ). An optimal two-stage Phase II design utilizing complete and partial response information separately . Controlled Clinical Trials 23 : 367 – 379 .
   PubMedGoogle Scholar
• Rinaldi , D. A. , Burris , H. A. , Dorr , F. A. , Woodworth , J. R. , Kuhn , J. G. , Eckardt , J. R. , Rodriguez , G. , Corso , S. W. , Fields , S. M. , Langley , C. ( 1995 ). Initial Phase I evaluation of the novel thymidylate synthase inhibitor, LY231514, using the modified continual reassessment method for dose escalation . Journal of Clinical Oncology 13 : 2842 – 2850 .
   PubMed Web of Science ®Google Scholar
• Simon , R. ( 1989 ). Optimal two-stage designs for Phase II clinical trials . Control Clinical Trials 10 : 1 – 10 .
   PubMedGoogle Scholar
• Simon , R. , Freidlin , B. , Rubinstein , L. , Arbuck , S. G. , Collins , J. , Christian , M. C. ( 1997 ). Accerlated titration designs for Phase I clinical trials in oncology . Journal of the National Cancer Institute 89 : 1138 – 1147 .
   PubMed Web of Science ®Google Scholar
• Simon , R. , Maitournam , A. ( 2004 ). Evaluating the efficiency of targeted designs for randomized clinical trials . Clinical Cancer Research 10 : 6759 – 6763 .
   PubMed Web of Science ®Google Scholar
• Smith , T. L. , Lee , J. J ., Kantarjian , H. M. , Legha , S. S. , Raber , M. N. Design and results of Phase I cancer clinical trials: Three-year experience at M.D. Anderson cancer Center . Journal of Clinical Oncology 14 : 287 – 295 .
   PubMed Web of Science ®Google Scholar
• Stallard , N. , Thall , P. F. , Whitehead , J. ( 1999 ). Decision theoretic designs for Phase II clinical trials with multiple outcome . Biometrics 55 : 971 – 977 .
   PubMed Web of Science ®Google Scholar
• Storer , B. E. ( 1989 ). Design and analysis of Phase I clinical trials . Biometrics 45 : 925 – 937 .
   PubMed Web of Science ®Google Scholar
• Sylvester , R. J. ( 1988 ). A Bayesian approach to the design of Phase II clinical trials . Biometrics 44 : 823 – 836 .
   PubMed Web of Science ®Google Scholar
• Tan , S. B. , Machin , D. (2002). Bayesian two-stage designs for Phase II trials. Statistics in Medicine 21:1991–2012.
   PubMed Web of Science ®Google Scholar
• Tan , S. B. , Machin , D. ( 2006 ). Letter to editor: Bayesian two-stage designs for Phase II trials . Statistics in Medicine 25 : 3407 – 3408 .
   PubMed Web of Science ®Google Scholar
• Thall , P. F. , Russel , K. T. ( 1998 ). A strategy for dose-finding and safety monitoring based on efficacy and adverse outcomes in Phase I/II clinical trials . Biometrics 54 : 251 – 264 .
   PubMed Web of Science ®Google Scholar
• Van Glabbeke , M. , Steward , W. , Armand , J. P. ( 2002 ). Nonrandomised Phase II trials of drug combinations: often meaningless, sometimes misleading. Are there alternative strategies? European Journal of Cancer 38 : 635 – 638 .
   PubMed Web of Science ®Google Scholar
• Wei , L. J. ( 1978 ). The adaptive biased coin design for sequential experiments . Annals of Statistics 6 : 92 – 100 .
   Web of Science ®Google Scholar
• Whitehead , J. , Brunier , H. ( 1995 ). Bayesian decision procedures for dose determining experiments . Statistics in Medicine 14 : 33 – 48 .
   Web of Science ®Google Scholar
• Wieand , H. S. ( 2005 ). Randomized Phase II trials: What does randomization gain? Journal of Clinical Oncology 23 : 1794 – 1795 .
   PubMed Web of Science ®Google Scholar
• Wooley , P. V. , Schein , P. S. ( 1979 ). Methods of Cancer Research . New York , NY : Academic Press .
   Google Scholar
• Zhang , W. , Sargent , D. J. , Mandrekar , S. ( 2006 ). An adaptive dose-finding design incorporating both toxicity and efficacy . Statistics in Medicine 25 : 2365 – 2383 .
   PubMed Web of Science ®Google Scholar
• Zhou , Y. , Whitehead , J. ( 2003 ). Practical implementation of Bayesian dose-escalation procedures . Drug Information Journal 37 : 45 – 59 .
   Web of Science ®Google Scholar
• Zohar , S. , Chervet , S. ( 2001 ). The continual reassessment method: Comparison of Bayesian stopping rules for dose-ranging studies . Statistics in Medicine 20 : 2827 – 2843 .
   PubMed Web of Science ®Google Scholar