Adaptive designs for dose-finding in non-cancer phase II trials: Influence of early unexpected outcomes

Clinical Trials

Volumn 5, Issue 6, 2008, Pages 595-606

  Resche Rigon, Matthieu ^a,b   Zohar, Sarah ^a   Chevret, Sylvie ^a  

^a INSERM   (France)

^b SAINT LOUIS HOSPITAL   (France)

Author keywords

[No Author keywords available]

Indexed keywords

ANDROSTANE DERIVATIVE; NEUROMUSCULAR BLOCKING AGENT; ROCURONIUM;

ARTICLE; BAYES THEOREM; DIAGNOSIS RELATED GROUP; DOSE RESPONSE; ENDOTRACHEAL INTUBATION; EPIDEMIOLOGY; HUMAN; INFANT; METHODOLOGY; NEWBORN; PHASE 2 CLINICAL TRIAL; REPRODUCIBILITY; STATISTICAL MODEL; TREATMENT FAILURE;

ANDROSTANOLS; BAYES THEOREM; BIAS (EPIDEMIOLOGY); CLINICAL TRIALS, PHASE II AS TOPIC; DOSE-RESPONSE RELATIONSHIP, DRUG; HUMANS; INFANT; INFANT, NEWBORN; INTUBATION, INTRATRACHEAL; LIKELIHOOD FUNCTIONS; LOGISTIC MODELS; NEUROMUSCULAR NONDEPOLARIZING AGENTS; OUTLIERS, DRG; REPRODUCIBILITY OF RESULTS; RESEARCH DESIGN; TREATMENT FAILURE;

EID: 61449307103     PISSN: 17407745     EISSN: 17407753     Source Type: Journal    
DOI: 10.1177/1740774508098788     Document Type: Article

References (43)

1. Chevret S. Basic concepts in dose-finding. In: Chevret SE (ed.), Statistical Methods for Dose-finding Experiments, John Wiley & Sons, Chichester, 2006, pp. 5-18.
2. Korn EL, Arbuck SG, Pluda JM et al. Clinical trial designs for cytostatic agents: are new approaches needed? J Clin Oncol 2001; 19(1): 265-72.
3. Storer BE. Design and analysis of phase I clinical trials. Biometrics 1989; 45: 925-37.
4. O'Quigley J, Pepe M, Fisher L. Continual reassessment method: a practical design for phase I clinical trials in cancer. Biometrics 1990; 46: 33-48.
5. Gatsonis C, Greenhouse JB. Bayesian methods for phase I clinical trials. Stat Med 1992; 11: 1377-89.
6. Whitehead J, Brunier H. Bayesian decision procedures for dose determining experiments. Stat Med 1995; 14: 885-93; discussion 895-99.
7. Babb J, Rogatko A, Zacks S. Cancer phase I clinical trials: efficient dose escalation with overdose control. Stat Med 1998; 17: 1103-20.
8. 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: 251-64.
9. Gasparini M, Eisele J. A curve-free method for phase I clinical trials. Biometrics 2000; 56: 609-15.
10. Faries D. Practical modifications of the continual reassessment method for phase Icancer clinical trials. J Biopharm Stat 1994; 4: 147-64.
11. Goodman SN, Zahurak ML, Piantadosi S. Some practical improvements in the continual reassessment method for phase I studies. Stat Med 1995; 14: 1149-61.
12. Korn EL, Midthune D, Chen TT et al. A comparison of two phase I trial designs. Stat Med 1994; 13: 1799-1806.
13. 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: 911-22; discussion 923.
14. O'Quigley J, Shen LZ. Continual reassessment method: a likelihood approach. Biometrics 1996; 52: 673-84.
15. Ting N. Dose Finding in Drug development. Springer New-York, 2006.
16. Roberts R, Rodriguez W, Murphy D, Crescenzi T. Pediatric drug labeling. Improving the safety and efficacy of pediatrics therapies. JAMA 2003; 290: 905-11.
17. Giacoia GP, Mattison DR. Newborns and drug studies: the NICHD/FDA newborn drug development initiative. Clin Ther 2005; 27: 796-813.
18. Pons G, Treluyer JM, Dimet J, Merle Y. Potential benefit of Bayesian forecasting for therapeutic drug monitoring in neonates. Ther Drug Monit 2002; 24: 9-14.
19. Center for Drug Evaluation and Research. New Drug Development and Review. http://www.fda.gov/cder/handbook/: FDA; 2002.
20. Desfrere L, Zohar S, Morville P et al. Dose-finding study of ibuprofen in patent ductus arteriosus using the continual reassessment method. J Clin Pharm Ther 2005; 30: 121-32.
21. Treluyer JM, Zohar S, Rey E et al. Minimum effective dose of midazolam for sedation of mechanically ventilated neonates. J Clin Pharm Ther 2005; 30: 479-85.
22. Fabre E, Chevret S, Piechaud JF et al. An approach for dose finding of drugs in infants: sedation by midazolam studied using the continual reassessment method. Br J Clin Pharm 1998; 46: 395-401.
23. De Spirlet M, Treluyer JM, Chevret S et al. Tocolytic effects of intravenous nitroglycerin. Fundamental and Clinical Pharmacology 2004; 18: 207-13.
24. Storer BE. Choosing a phase I design. In: Crowley J (ed.), Statistics in Clinical Oncology. Marcel Dekker, New York, 2001, pp. 73-91.
25. Lefrere F, Zohar S, Bresson JL et al. A double-blind low dose-finding phase II study of granulocyte colonystimulating factor combined with chemotherapy for stem cell mobilization in patients with non-Hodgkin s lymphoma. Haematologica 2006; 91: 550-53.
26. Carvalho JC, Balki M, Kingdom J, Windrim R. Oxytocin requirements at elective cesarean delivery: a dose-finding study. Obstet Gynecol. 2004; 104: 1005-10.
27. Copas JB. Regression, prediction and shrinkage (with discussion). J Royal Statistical Society, Series B 1983; 45: 311-54.
28. Wetherill GB, Glazebrook KD. Sequential Methods in Statistics. Chapman and Hall, London, 1986.
29. Wierda JMKH, Meretoja OA, Taivainen T, Proost JH. Pharmacokinetics and phamacokinetic-dynamic modelling of rocuronium in infants and children. Br J Anaesth 1997; 78: 690-95.
30. Scheiber G, Ribeiro FC, Marichal A et al. Intubating conditions and onset of action after rocuronium in young children. Pediatr Anesth 1996; 83: 320-24.
31. Grand S, Noble S, Wood A et al. Assessment of intubating conditions in adults after induction with propofol and varying doses of remifentanil. Br J Anaesth 1998; 81: 540-43.
32. Potter DM. Phase I studies of chemotherapeutic agents in cancer patients: A review of the designs. J Biopharm Stat 2006; 16: 579-604.
33. Zohar S, Latouche A, Taconnet M, Chevret S. Software to compute and conduct sequential Bayesian phase I or II dose-ranging clinical trials with stopping rules. Comput Methods Programs Biomed 2003; 72: 117-25.
34. Cook RD, Weisberg S. Residuals and Influence in Regression. Chapman and Hall, London, 1982.
35. Hu F, Rosenberger WF. Analysis of time trends in adaptive designs with application to a neurolophysiology experiment. Stat Med 2000; 19: 2067-42.
36. Hu F. The weighted likelihood. Can J Stat 2002; 30: 347-71.
37. Park C, Basu A, Lindsay BG. The residual adjustement function and weighted likelihood: a graphical interpretation of robustness of minimum disparity estimators. Computational Statistics and Data Analysis 2002; 39: 21-33.
38. Markatou M, Basu A, Lindsay BG. Weighted likelihood estimating equations: the discret case with applications to logistic regression. Journal of Statistical Planning and Inference 1997; 57: 215-32.
39. Whitehead J, Williamson D. Bayesian decision procedures based on logistic regression models for dosefinding studies. J Biopharm Stat 1998; 8: 445-67.
40. Barnett V, Lewis T. Outliers in Statistical Data. John Wiley & Sons, Chichester, 1994.
41. Cook D. Assessment of local influence. Journal of the Royal Statistical Society, Series B 1986; 48: 133-69.
42. Pan JX, Fang KT, von Rosen D. Local influence assessment in the growth curve model with unstructured covariance. Journal of Statistical Planning and Inference 1997; 62: 263-78.
43. Ivanova A, Montazer-Haghighi A, Mohanty SG, Durham SD. Improved up-and-down designs for phase I trials. Stat Med 2003; 22: 69-82.