Methodology and application of adaptive and sequential approaches in contemporary clinical trials

Journal of Probability and Statistics

Volumn , Issue , 2012, Pages

  Chen, Zhengjia ^a   Zhao, Yichuan ^b   Cui, Ye ^b   Kowalski, Jeanne ^a  

^a EMORY UNIVERSITY   (United States)

^b GEORGIA STATE UNIVERSITY   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

EID: 84871391750     PISSN: 1687952X     EISSN: 16879538     Source Type: Journal    
DOI: 10.1155/2012/527351     Document Type: Review

References (61)

1. Chow S. C., Chang M., Adaptive design methods in clinical trials-a review. Orphanet Journal of Rare Diseases 2008 3 1, article 11 2-s2.0-44949171503 10.1186/1750-1172-3-11
2. Gallo P., Chuang-Stein C., Dragalin V., Gaydos B., Krams M., Pinheiro J., Adaptive designs in clinical drug development-an executive summary of the PhRMA working group. Journal of Biopharmaceutical Statistics 2006 16 3 275 283 10.1080/10543400600614742 2227370
3. Potter D. M., Phase I studies of chemotherapeutic agents in cancer patients: a review of the designs. Journal of Biopharmaceutical Statistics 2006 16 5 579 604 10.1080/10543400600860295 2252309 (Pubitemid 44844014)
4. Lin Y., Shih W. J., Statistical properties of the traditional algorithm-based designs for phase I cancer clinical trials. Biostatistics 2001 2 2 203 215 10.1093/biostatistics/2.2.203 (Pubitemid 33278607)
5. Leung D. H. Y., Wang Y. G., Isotonic designs for phase I trials. Controlled Clinical Trials 2001 22 2 126 138 2-s2.0-0035075179 10.1016/S0197-2456(00)00132-X (Pubitemid 32289449)
6. Simon R., Freidlin B., Rubinstein L., Arbuck S. G., Collins J., Christian M. C., Accelerated titration designs for phase I clinical trials in oncology. Journal of the National Cancer Institute 1997 89 15 1138 1147 2-s2.0-0030749549 (Pubitemid 27337928)
7. Storer B. E., Design and analysis of phase I clinical trials. Biometrics 1989 45 3 925 937 10.2307/2531693 1029610 ZBL0715.62241 (Pubitemid 19249331)
8. O'Quigley J., Pepe M., Fisher L., Continual reassessment method: a practical design for phase 1 clinical trials in cancer. Biometrics 1990 46 1 33 48 10.2307/2531628 1059105 ZBL0715.62242 (Pubitemid 20245604)
9. Babb J., Rogatko A., Zacks S., Cancer phase I clinical trials: efficient dose escalation with overdose control. Statistics in Medicine 1998 17 10 1103 1120 (Pubitemid 28221028)
10. Korn E. L., Midthune D., Timothy Chen T., A comparison of two phase I trial designs. Statistics in Medicine 1994 13 18 1799 1806 10.1002/sim. 4780131802
11. Faries D., Practical modifications of the continual reassessment method for phase I cancer clinical trials. Journal of Biopharmaceutical Statistics 1994 4 2 147 164 2-s2.0-0028060511 (Pubitemid 24248939)
12. Zacks S., Rogatko A., Babb J., Optimal Bayesian-feasible dose escalation for cancer phase I trials. Statistics & Probability Letters 1998 38 3 215 220 10.1016/S0167-7152(98)00016-9 1629891 ZBL0903.62064 (Pubitemid 128181982)
13. Mugno R., Zhus W., Rosenberger W. F., Adaptive urn designs for estimating several percentiles of a dose-response curve. Statistics in Medicine 2004 23 13 2137 2150 2-s2.0-3242786541 10.1002/sim.1808 (Pubitemid 38968034)
14. Chen Z., Krailo M. D., Sun J., Azen S. P., Range and trend of expected toxicity level (ETL) in standard A + B designs: a report from the children's oncology group. Contemporary Clinical Trials 2009 30 2 123 128 2-s2.0-60249085300 10.1016/j.cct.2008.10.006
15. Chen Z., Krailo M. D., Azen S. P., Tighiouart M., A novel toxicity scoring system treating toxicity response as a quasi-continuous variable in phase I clinical trials. Contemporary Clinical Trials 2010 31 5 473 482 2-s2.0-77957754119 10.1016/j.cct.2010.05.010
16. Chen Z., Tighiouart M., Kowalski J., Dose escalation with overdose control using a quasi-continuous toxicity score in cancer phase I clinical trials. Contemporary Clinical Trials 2012 33 5 949 958 10.1016/j.cct.2012.04.007
17. Tighiouart M., Rogatko A., Dose finding with escalation with overdose control (EWOC) in cancer clinical trials. Statistical Science 2010 25 2 217 226 10.1214/10-STS333 2789991
18. Rosenberger W. F., Haines L. M., Competing designs for phase I clinical trials: a review. Statistics in Medicine 2002 21 18 2757 2770 2-s2.0-0037201011 10.1002/sim.1229
19. Le Tourneau C., Lee J. J., Siu L. L., Dose escalation methods in phase I cancer clinical trials. Journal of the National Cancer Institute 2009 101 10 708 720 2-s2.0-66849118694 10.1093/jnci/djp079
20. Gehan E. A., The determination of the number of patients required in a preliminary and a follow-up trial of a new chemotherapeutic agent. Journal of Chronic Diseases 1961 13 4 346 353 2-s2.0-50549174560 10.1016/0021-9681(61) 90060-1
21. Simon R., Optimal two-stage designs for phase II clinical trials. Controlled Clinical Trials 1989 10 1 1 10 2-s2.0-0024536437 (Pubitemid 19099520)
22. Fleming T. R., One-sample multiple testing procedure for phase II clinical trials. Biometrics 1982 38 1 143 151 2-s2.0-0020108590
23. Chang M. N., Therneau T. M., Wieand H. S., Cha S. S., Design for group sequential phase II clinical trials. Biometrics 1987 43 4 865 874 2-s2.0-0023522405 (Pubitemid 18016772)
24. Thall P. F., Simon R., A Bayesian approach to establishing sample size and monitoring criteria for phase II clinical trials. Controlled Clinical Trials 1994 15 6 463 481 2-s2.0-0028033082 10.1016/0197-2456(94)90004-3
25. Lee J. J., Liu D. D., A predictive probability design for phase II cancer clinical trials. Clinical Trials 2008 5 2 93 106 2-s2.0-43649085358 10.1177/1740774508089279 (Pubitemid 351682519)
26. Yin G., Chen N., Lee J. J., Phase II trial design with Bayesian adaptive randomization and predictive probability. Journal of the Royal Statistical Society C 2012 61 2 219 235 10.1111/j.1467-9876.2011.01006.x 2905060
27. Rubinstein L., Crowley J., Ivy P., Leblanc M., Sargent D., Randomized phase II designs. Clinical Cancer Research 2009 15 6 1883 1890 2-s2.0-63449114216 10.1158/1078-0432.CCR-08-2031
28. Thall P. F., A review of phase 2-3 clinical trial designs. Lifetime Data Analysis 2008 14 1 37 53 10.1007/s10985-007-9049-x 2412716 ZBL1169.62392
29. Inoue L. Y. T., Thall P. F., Berry D. A., Seamlessly expanding a randomized phase II trial to phase III. Biometrics 2002 58 4 823 831 10.1111/j.0006-341X.2002.00823.x 1945019 ZBL1210.62168 (Pubitemid 36133760)
30. Lavin P. T., An alternative model for the evaluation of antitumor activity. Cancer Clinical Trials 1981 4 4 451 457 2-s2.0-0019760378
31. Wang Y., Sung C., Dartois C., Ramchandani R., Booth B. P., Rock E., Gobburu J., Elucidation of relationship between tumor size and survival in non-small-cell lung cancer patients can aid early decision making in clinical drug development. Clinical Pharmacology and Therapeutics 2009 86 2 167 174 2-s2.0-67651171223 10.1038/clpt.2009.64
32. Karrison T. G., Maitland M. L., Stadler W. M., Design of phase II cancer trials using a continuous endpoint of change in tumor size: application to a study of sorafenib and erlotinib in non-small-cell lung cancer. Journal of the National Cancer Institute 2007 99 19 1455 1461 10.1093/jnci/djm158
33. An M. W., Mandrekar S. J., Branda M. E., Comparison of continuous versus categorical tumor measurement-based metrics to predict overall survival in cancer treatment trials. Clinical Cancer Research 2011 17 20 6592 6599 10.1158/1078-0432.CCR-11-0822
34. Yothers G., Toward progression-free survival as a primary end point in advanced colorectal cancer. Journal of Clinical Oncology 2007 25 33 5153 5154 2-s2.0-36849069358 10.1200/JCO.2007.13.6796 (Pubitemid 350232244)
35. Buyse M., Thirion P., Carlson R. W., Burzykowski T., Molenberghs G., Piedbois P., Relation between tumour response to first-line chemotherapy and survival in advanced colorectal cancer: a meta-analysis. Meta-Analysis Group in Cancer. The Lancet 2000 356 9227 373 378 2-s2.0-0034729931 (Pubitemid 30487493)
36. Schulz K. F., Grimes D. A., Generation of allocation sequences in randomised trials: chance, not choice. The Lancet 2002 359 9305 515 519 2-s2.0-0037045587 10.1016/S0140-6736(02)07683-3 (Pubitemid 34164304)
37. Lachin J. M., Matts J. P., Wei L. J., Randomization in clinical trials: conclusions and recommendations. Controlled Clinical Trials 1988 9 4 365 374 2-s2.0-0024244278 10.1016/0197-2456(88)90049-9 (Pubitemid 19012639)
38. Taves D. R., Minimization: a new method of assigning patients to treatment and control groups. Clinical Pharmacology and Therapeutics 1974 15 5 443 453 2-s2.0-0016211783
39. Pocock S. J., Simon R., Sequential treatment assignment with balancing for prognostic factors in the controlled clinical trial. Biometrics 1975 31 1 103 115 2-s2.0-0016799829
40. Frane J. W., A method of biased coin randomization, its implementation, and its validation. Drug Information Journal 1998 32 2 423 432 2-s2.0-0031782053 (Pubitemid 28233085)
41. Kang M., Ragan B. G., Park J. H., Issues in outcomes research: an overview of randomization techniques for clinical trials. Journal of Athletic Training 2008 43 2 215 221 2-s2.0-41949141262 (Pubitemid 351508038)
42. Pocock S. J., Group sequential methods in the design and analysis of clinical trials. Biometrika 1977 64 2 191 199 2-s2.0-0017744944 10.1093/biomet/64.2.191 (Pubitemid 8145484)
43. O'Brien P. C., Fleming T. R., A multiple testing procedure for clinical trials. Biometrics 1979 35 3 549 556 2-s2.0-0018687930 (Pubitemid 10242750)
44. DeMets D. L., Lan K. K. G., Interim analysis: the alpha spending function approach. Statistics in Medicine 1994 13 13-14 1341 1352 2-s2.0-0027934302 10.1002/sim.4780131308 (Pubitemid 24236184)
45. Hwang I. K., Shih W. J., Decani J. S., Group sequential designs using a family of type I error probability spending functions. Statistics in Medicine 1990 9 12 1439 1445 2-s2.0-0025598008 10.1002/sim.4780091207
46. Jennison C., Turnbull B. W., Group Sequential Methods with Applications to Clinical Trials 2000 CRC Press 1710781
47. Berry S. M., Kadane J. B., Optimal Bayesian randomization. Journal of the Royal Statistical Society B 1997 59 4 813 819 10.1111/1467-9868.00098 1483216 ZBL0886.62026 (Pubitemid 127686982)
48. Thall P. F., Wathen J. K., Practical Bayesian adaptive randomisation in clinical trials. European Journal of Cancer 2007 43 5 859 866 10.1016/j.ejca.2007.01.006 (Pubitemid 46366693)
49. Rosner G. L., Bayesian monitoring of clinical trials with failure-time endpoints. Biometrics 2005 61 1 239 245 10.1111/j.0006-341X.2005.031037.x 2135866 (Pubitemid 40380985)
50. Gelman A., Carlin J. B., Stern H. S., Rubin D. B., Bayesian Data Analysis 1995 Boca Raton, Fla, USA Chapman & Hall 1385925
51. Berry D. A., Ho C. H., One-sided sequential stopping boundaries for clinical trials: a decision-theoretic approach. Biometrics 1988 44 1 219 227 10.2307/2531909 931636 ZBL0707.62263 (Pubitemid 18098823)
52. Eales J. D., Jennison C., An improved method for deriving optimal one-sided group sequential tests. Biometrika 1992 79 1 13 24 10.1093/biomet/79.1.13 1158514 ZBL0850.62633
53. Cressie N., Biele J., A sample-size-optimal Bayesian procedure for sequential pharmaceutical trials. Biometrics 1994 50 3 700 711 10.2307/2532784 1309313 ZBL0822.62091 (Pubitemid 24320818)
54. Barber S., Jennison C., Optimal asymmetric one-sided group sequential tests. Biometrika 2002 89 1 49 60 10.1093/biomet/89.1.49 1888345 ZBL0998.62067 (Pubitemid 41312002)
55. Krams M., Lees K. R., Hacke W., Grieve A. P., Orgogozo J. M., Ford G. A., Acute stroke therapy by inhibition of neutrophils (ASTIN): an adaptive dose-response study of UK-279,276 in acute ischemic stroke. Stroke 2003 34 11 2543 2548 2-s2.0-0242694372 10.1161/01.STR.0000092527.33910.89 (Pubitemid 37392736)
56. Luce B. R., Kramer J. M., Goodman S. N., Connor J. T., Tunis S., Whicher D., Schwartz J. S., Rethinking randomized clinical trials for comparative effectiveness research: the need for transformational change. Annals of Internal Medicine 2009 151 3 206 209 2-s2.0-68249130994
57. Posch M., Bauer P., Adaptive two stage designs and the conditional error function. Biometrical Journal 1999 41 6 689 696 2-s2.0-0033458198
58. Cui L., Hung H. M. J., Wang S. J., Modification of sample size in group sequential clinical trials. Biometrics 1999 55 3 853 857 2-s2.0-0032886394 (Pubitemid 29427088)
59. Shih W. J., Group sequential, sample size re-estimation and two-stage adaptive designs in clinical trials: a comparison. Statistics in Medicine 2006 25 6 933 941 10.1002/sim.2252 2225183 (Pubitemid 43337981)
60. Proschan M. A., Hunsberger S. A., Designed extension of studies based on conditional power. Biometrics 1995 51 4 1315 1324 2-s2.0-0029589385 10.2307/2533262 (Pubitemid 26030176)
61. Proschan M. A., Two-stage sample size re-estimation based on a nuisance parameter: a review. Journal of Biopharmaceutical Statistics 2005 15 4 559 574 10.1081/BIP-200062852 2190569 (Pubitemid 40967299)