Phase II Clinical Trials

Statistical Advances in the Biomedical Sciences: Clinical Trials, Epidemiology, Survival Analysis, and Bioinformatics

Volumn , Issue , 2007, Pages 15-31

  Stallard, Nigel ^a  

^a UNIVERSITY OF WARWICK   (United Kingdom)

Author keywords

Clinical assessment program; Single arm phase II trial; Treatment effectiveness hypothesis test

Indexed keywords

CLINICAL ASSESSMENTS; CLINICAL TRIAL; HYPOTHESIS TESTS; PHASE II;

SOFTWARE TESTING;

EID: 84954594013     PISSN: None     EISSN: None     Source Type: Book    
DOI: 10.1002/9780470181218.ch2     Document Type: Chapter

References (61)

1. Anscombe, F. J., Sequential medical trials, J. Am. Statist. Assoc. 58, 365-383 (1963).
2. Barnard, G. A., Sequential tests in industrial statistics, J. Roy. Statist. Soc. 8(Suppl.) 1-26 (1946).
3. Bauer, P. and Kieser, M., Combining different phases in the development of medical treatments within a single trial, Statist. Med. 18, 1833-1848 (1999).
4. Bauer, P. and Köhne, K., Evaluation of experiments with adaptive interim analyses, Biometrics 50, 1029-1041 (1994).
5. Bellisant, E., Benichou, J., and Chastang, C., Application of the triangular test to phase II cancer clinical trials, Statist. Med. 9, 907-917 (1990).
6. Bland, M., An Introduction to Medical Statistics. Oxford University Press, Oxford, 2000.
7. Bretz, F., Schmidli, H., König, F., Racine, A., and Maurer, W., Confirmatory seamless phase II/III clinical trials with hypothesis selection at interim: General concepts, Biometr. J. (in press).
8. Brunier, H. C. and Whitehead, J., Sample sizes for phase II clinical trials derived from Bayesian decision theory, Statist. Med. 13, 2493-2502 (1994).
9. Bryant, J. and Day, R., Incorporating toxicity considerations into the design of two-stage phase II clinical trials, Biometrics 51, 1372-1383 (1995).
10. Carlin, B. P., Kadane, J. B., and Gelfand, A. E., Approaches for optimal sequential decision analysis in clinical trials, Biometrics 54, 964-975 (1998).
11. Chang, M. N., Therneau, T. M., Weiand, H. S., and Cha, S. S., Designs for group sequential phase II clinical trials, Biometrics 51, 1372-1383 (1987).
12. Chen, T. T., Optimal three-stage designs for phase II cancer clinical trials, Statist. Med. 16, 2701-2711 (1997).
13. Colton, T., A model for selecting one of two medical treatments, J. Am. Statist. Assoc. 58, 388-400 (1963).
14. Conaway, M. R. and Petroni, G. R., Bivariate sequential designs for phase II trials, Biometrics 51, 656-664 (1995).
15. Conaway, M. R. and Petroni, G. R., Designs for phase II trials allowing for a trade-off between response and toxicity, Biometrics 52, 1375-1386 (1996).
16. Dunnett, C. W., Selection of the best treatment in comparison to a control with an application to a medical trial, in Design of Experiments: Ranking and Selection, Santer, T. J. and Tamhane, A. C., eds., Marcel Dekker, New York, 1984.
17. Ensign, L. G., Gehan, E. A., Kamen, D. S., and Thall, P. F., An optimal three-stage design for phase II clinical trials, Statist. Med. 13, 1727-1736 (1994).
18. Evans, C. H. and Ildstad, S. T., eds., Small Clinical Trials: Issues and Challenges, National Academy Press, Washington, DC, 2001.
19. Fan, S. H. and Wang, Y.-G., Decision-theoretic designs for dose-finding clinical trials with multiple outcomes, Statist. Med. (in press).
20. Fleming, T. R., One-sample multiple testing procedure for phase II clinical trials, Biometrics 38, 143-151 (1982).
21. Hilden, J., Reader reaction: Corrected loss calculation for phase II trials, Biometrics 46, 535-538 (1990).
22. Inoue, L. Y. T., Thall, P. F., and Berry, D. A., Seamlessly expanding a randomised phase II trial to phase III, Biometrics 58, 823-831 (2002).
23. Jennison, C. and Turnbull, B.W., Group Sequential Methods with Applications to Clinical Trials, Chapman & Hall/CRC, Boca Raton, FL, 2000.
24. Julious, S. A. and Swank, D. J., Moving statistics beyond the individual clinical trial: Applying decision science to optimize a clinical development plan, Pharm. Statist. 4, 37-46 (2005).
25. Kelly, P. J., Stallard, N., and Todd, S., An adaptive group sequential design for phase II/III clinical trials that select a single treatment from several, J. Biopharm. Statist. 15, 641-658 (2005).
26. Korn, E. L., Arbuck, S. G., Pluda, J. M., Simon, R., Kaplan, R. S., and Christian, M. C., Clinical trial designs for cytostatic agents: Are new approaches needed?, J. Clin. Oncol. 19, 265-272 (2001).
27. Lan, K. K. G. and DeMets, D. L., Discrete sequential boundaries for clinical trials, Biometrika 70, 659-663 (1983).
28. Lin, Y. and Shih, W. J., Adaptive two-stage designs for single-arm phase IIA cancer clinical trial, Biometrics 60, 482-490 (2004).
29. Lindgren, B. W., Statistical Theory, Macmillan, New York, 1968.
30. Leung, D. H.-Y. and Wang, Y.-G., Reader reaction: A Bayesian decision approach for sample size determination in phase II trials, Biometrics 57, 309-312 (2006).
31. Machin, D., Campbell, M. J., Fayers, P. M., and Pinol, A. P. Y., Sample Size Tables for Clinical Studies, Blackwell Science Ltd., Oxford, 1997.
32. Mariani, L. and Marubini, E., Design and analysis of phase II cancer trials: A review of statistical methods and guidelines for medical researchers, Int. Statist. Rev. 64, 61-88 (1996).
33. Novick, M. R. and Grizzle, J. E., A Bayesian approach to the analysis of data from a clinical trial, J. Am. Statist. Assoc. 60, 81-96 (1965).
34. Schoenfeld, D., Statistical considerations for pilot studies, Int. J. Radiat. Oncol. Bio. Phys. 6, 371-374 (1980).
35. Shuster, J., Optimal two-stage designs for single arm phase II cancer trials, J. Biopharm. Statist. 12, 39-51 (2002).
36. Simon, R., Optimal two-stage designs for phase II clinical trials, Control. Clin. Trials 10, 1-10 (1989).
37. Senn, S., Some statistical issues in project prioritization in the pharmaceutical industry, Statist. Med. 15, 2689-2702 (1996).
38. Senn, S., Further statistical issues in project prioritization in the pharmaceutical industry, Drug Inform. J. 32, 253-259 (1998).
39. Spiegelhalter, D., Freedman, L. S., and Parmar, M. K. B., Bayesian approaches to randomised trials, J. Roy. Statist. Soc. Ser. A 157, 357-416 (1994).
40. Stallard, N., Sample size determination for phase II clinical trials based on Bayesian decision theory, Biometrics 54, 279-294 (1998).
41. Stallard, N., Decision-theoretic designs for phase II clinical trials allowing for competing studies, Biometrics 59, 402-409 (2003).
42. Stallard, N., Thall, P. F., and Whitehead, J., Decision theoretic designs for phase II clinical trials with multiple outcomes, Biometrics 55, 971-977 (1999).
43. Stallard, N. and Todd, S., Exact sequential tests for single samples of discrete responses using spending functions, Statist. Med. 19, 3051-3064 (2000).
44. Stallard, N. and Todd, S., Sequential designs for phase III clinical trials incorporating treatment selection, Statist. Med. 22, 689-703 (2003).
45. Sylvester, R. J., A Bayesian approach to the design of phase II clinical trials, Biometrics 44, 823-836 (1988).
46. Sylvester, R. J. and Staquet, M. L., Design of phase II clinical trials in cancer using decision theory, Cancer Treat. Rep. 64, 519-524 (1980).
47. Thall, P. F. and Cheng, S.-C., Treatment comparisons based on two-dimensional safety and efficacy alternatives in oncology trials, Biometrics 55, 746-753 (1999).
48. Thall, P. F. and Cheng, S.-C., Optimal two-stage designs for clinical trials based on safety and efficacy, Statist. Med. 20, 1023-1032 (2001).
49. Thall, P. F. and Estey, E. H., A Bayesian strategy for screening cancer treatments prior to phase II clinical evaluation, Statist. Med. 12, 1197-1211 (1993).
50. Thall, P. F., Simon, R., Practical Bayesian guidelines for phase IIB clinical trials, Biometrics 50, 337-349 (1994).
51. Thall, P. F., Simon, R., and Ellenberg, S. S., Two-stage selection and testing designs for comparative clinical trials, Biometrika 75, 303-310 (1988).
52. Thall, P. F., Simon, R., and Ellenberg, S. S., A two-stage design for choosing among several experimental treatments and a control in clinical trials, Biometrics 45, 537-547 (1989).
53. Thall, P. F., Simon, R., and Estey, E. H., Bayesian sequential monitoring designs for single-arm clinical trials with multiple outcomes, Statist. Med. 14, 357-379 (1995).
54. Thall, P. F. and Sung, H.-G., Some extensions and applications of a Bayesian strategy for multiple outcomes on clinical trials, Statist. Med. 17, 1563-1580 (1998).
55. Thall, P. F., Sung, H.-G., and Estey, E. H., Selecting therapeutic strategies based on efficacy and death in multicourse clinical trials, J. Am. Statist. Assoc. 97, 29-39 (2002).
56. Todd, S. and Stallard, N., A new clinical trial design combining phases II and III: sequential designs with treatment selection and a change of endpoint, Drug Inform. J. 39, 109-118 (2005).
57. Wald, A., Sequential Analysis, Wiley, New York, 1947.
58. Wang, Y.-G., Leung, D. H.-Y., Li, M., and Tan, S.-B., Bayesian designs with frequentist and Bayesian error rate considerations, Statist. Meth. Med. Res. 14, 445-456 (2005).
59. Whitehead, J., Designing phase II studies in the context of a programme of clinical research, Biometrics 41, 373-383 (1985).
60. Whitehead, J., Sample sizes for phase II and phase III clinical trials, an integrated approach, Statist. Med. 5, 459-464 (1986).
61. Whitehead, J., The Design and Analysis of Sequential Clinical Trials, Wiley, Chichester, 1997.