Model-based phase I designs incorporating toxicity and efficacy for single and dual agent drug combinations: Methods and challenges

Statistics in Medicine

Volumn 29, Issue 10, 2010, Pages 1077-1083

  Mandrekar, Sumithra J ^a   Qin, Rui ^a   Sargent, Daniel J ^a  

^a MAYO CLINIC   (United States)

Author keywords

Continual reassessment method; Dose finding; Efficacy; Model based designs; Toxicity; Trinary outcome

Indexed keywords

ARTICLE; COMBINATION CHEMOTHERAPY; DRUG EFFICACY; DRUG TOXICITY; MONOTHERAPY; OPTIMAL DRUG DOSE; SIMULATION; STATISTICAL MODEL;

BAYES THEOREM; CLINICAL TRIALS, PHASE I AS TOPIC; COMPUTER SIMULATION; DOSE-RESPONSE RELATIONSHIP, DRUG; DRUG COMBINATIONS; DRUG TOXICITY; HUMANS; MODELS, STATISTICAL;

EID: 77951528602     PISSN: 02776715     EISSN: 10970258     Source Type: Journal    
DOI: 10.1002/sim.3706     Document Type: Article

References (26)

1. Storer BE. Choosing a phase I design. In Handbook of Statistics in Clinical Oncology, Crowley J, Ankerst DP (eds). Chapman & Hall/CRC: Boca Raton, FL, 2006; 59-75.
2. Storer BE. Design and analysis of phase I clinical trials. Biometrics 1989; 45(3):925-937. (Pubitemid 19249331)
3. 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. (Pubitemid 20245604)
4. Linsley PS. New look at an old costimulator [comment]. Nature Immunology 2005; 6(3):231-232. (Pubitemid 41724757)
5. 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)
6. Goodman SN, Zahurak ML, Piantadosi S. Some practical improvements in the continual reassessment method for phase I studies. Statistics in Medicine 1995; 14(11):1149-1161.
7. Shen LZ, O'Quigley J. Consistency of continual reassessment method under model misspecification. Biometrika 1996; 83(2):395-405.
8. Braun T. The bivariate continual reassessment method: extending the CRM to phase I trials of two competing outcomes. Controlled Clinical Trials 2002; 23(3):240-256. (Pubitemid 34621206)
9. Thall PF, Cook JD. Dose-finding based on efficacy-toxicity trade-offs. Biometrics 2004; 60(3):684-693.
10. Yin G, Li Y, Ji Y. Bayesian dose-finding in phase I/ II clinical trials using toxicity and efficacy odds ratios. Biometrics 2006; 62(3):777-784. (Pubitemid 44410635)
11. Bekele BN, Shen Y. A Bayesian approach to jointly modeling toxicity and biomarker expression in a phase I/ II dose-finding trial. Biometrics 2005; 61(2):343-354. (Pubitemid 40853408)
12. Dragalin V, Fedorov VV. Adaptive designs for dose-finding based on efficacy-toxicity response. Journal of Statistical Planning and Inference 2006; 136(6):1800-1823. (Pubitemid 43193550)
13. O'Quigley J, Hughes MD, Fenton T. Dose-finding designs for HIV studies. Biometrics 2001; 57(4):1018-1029.
14. Ivanova A. A new dose-finding design for bivariate outcomes. Biometrics 2003; 59(4):1001-1007. (Pubitemid 38021229)
15. Thall PF, Russell KE. A strategy for dose-finding and safety monitoring based on efficacy and adverse outcomes in phase I/ II clinical trials. Biometrics 1998; 54(1):251-264.
16. Mandrekar SJ, Cui Y, Sargent DJ. An adaptive phase I design for identifying a biologically optimal dose for dual agent drug combinations. Statistics in Medicine 2007; 26(11):2317-2330. (Pubitemid 46730251)
17. Zhang W, Sargent DJ, Mandrekar SJ. An adaptive dose-finding design incorporating both toxicity and efficacy. Statistics in Medicine 2006; 25(14):2365-2383. (Pubitemid 44057044)
18. Agresti A. Categorical Data Analysis (2nd edn). Wiley: Hoboken, NJ, 2002.
19. Zohar S, Chevret S. Recent developments in adaptive designs for Phase I/ II dose-finding studies. Journal of Biopharmaceutical Statistics 2007; 17(6):1071-1083. (Pubitemid 350135835)
20. Rogatko A, Schoeneck D, Jonas W, Tighiouart M, Khuri FR, Porter A. Translation of innovative designs into phase I trials. Journal of Clinical Oncology 2007; 25(31):4982-4986. (Pubitemid 350220431)
21. Conaway M, Dunbar S, Peddada S. Designs for single or multiple-agent phase I trials. Biometrics 2004; 60(3):661-669. (Pubitemid 39181110)
22. Harris M. Monoclonal antibodies as therapeutic agents for cancer. Lancet Oncology 2004; 5(5):292-302. (Pubitemid 38541581)
23. Hunsberger S, Rubinstein LV, Dancey J, Korn EL. Dose escalation trial designs based on a molecularly targeted endpoint. Statistics in Medicine 2005; 24(14):2171-2181. (Pubitemid 40960214)
24. Rubinstein L, Simon R. Phase I clinical trial design. In Handbook of Anticancer Drug Development, Budman D, Calvert A, Rowinsky E (eds). Elsevier: Amsterdam, 2003.
25. Wang K, Ivanova A. Two-dimensional dose finding in discrete dose space. Biometrics 2005; 61(1):217-222. (Pubitemid 40380982)
26. Fan SK, Wang YG. Decision-theoretic designs for dose-finding clinical trials with multiple outcomes. Statistics in Medicine 2006; 25(10):1699-1714. (Pubitemid 43757806)