A Bayesian adaptive design for multi-dose, randomized, placebo-controlled phase I/II trials

Contemporary Clinical Trials

Volumn 33, Issue 4, 2012, Pages 739-748

  Xie, Fang ^a   Ji, Yuan ^b   Tremmel, Lothar ^c  

^a CEPHALON INC   (United States)

^b NORTHSHORE UNIVERSITY HEALTHSYSTEM   (United States)

^c Drug Metabolism and Pharmacokinetics and Clinical Pharmacology   (United States)

Author keywords

Adaptive design; Bayesian; Cohort expansion; Dose escalation; Dose finding; Simulation

Indexed keywords

ARTICLE; BAYES THEOREM; COMPUTER PROGRAM; CONTROLLED STUDY; DRUG DOSE REGIMEN; DRUG TOLERABILITY; ERROR; FALSE POSITIVE RESULT; HUMAN; ISCHIALGIA; PHASE 1 CLINICAL TRIAL (TOPIC); PHASE 2 CLINICAL TRIAL (TOPIC); RANDOMIZED CONTROLLED TRIAL; RANDOMIZED CONTROLLED TRIAL (TOPIC); SAFETY; SAMPLE SIZE; SIMULATION; STUDY DESIGN;

BAYES THEOREM; CLINICAL TRIALS, PHASE I AS TOPIC; CLINICAL TRIALS, PHASE II AS TOPIC; COMPUTER SIMULATION; DECISION SUPPORT TECHNIQUES; DOSE-RESPONSE RELATIONSHIP, DRUG; DRUG ADMINISTRATION SCHEDULE; HUMANS; MAXIMUM TOLERATED DOSE; MODELS, STATISTICAL; NO-OBSERVED-ADVERSE-EFFECT LEVEL; RANDOMIZED CONTROLLED TRIALS AS TOPIC; RESEARCH DESIGN;

EID: 84861529528     PISSN: 15517144     EISSN: 15592030     Source Type: Journal    
DOI: 10.1016/j.cct.2012.03.001     Document Type: Article

References (18)

1. Berry S.M., Carlin B.P., Lee J.J., Muller P. Bayesian adaptive methods for clinical trials 2010, CRC press.
2. Thall P.F., Simon R.M. Practical Bayesian guidelines for phase IIB clinical trials. Biometrics 1994, 50:337-349.
3. O'Quigley J., Pepe M., Fisher L. Continual reassessment method: a practical design for phase 1 clinical trials in cancer. Biometrics 1990, 46:33-48.
4. Ji Y., Li Y., Bekele B.N. Dose-finding in phase I oncology clinical trials based on toxicity probability intervals. Clin Trials 2007, 4:235-244.
5. Ji Y., Liu P., Li Y., Nebiyou B.N. A modified toxicity probability interval method for dose-finding trials. Clin Trials 2010, 7:653-663.
6. Hu B, Bekele BN, Ji Y. Adaptive Dose Insertion in Early Phase Clinical Trials. Clin Trials in press; Sep. 6, Epub.
7. Ji Y., Feng L., Shpall E.J., Kebriaei P., Champlin R., Berry D., et al. Bayesian Continual Reassessment Method for Dose-Finding Trials Infusing T Cells with Limited Sample Size. J Biopharm Stat 2010.
8. Ji Y., Bekele B.N. Adaptive randomization for multi-arm comparative clinical trials based on joint efficacy/toxicity outcomes. Biometrics 2009, 65(3):876-884.
9. Thall P.F., Simon R., Estey E.H. Bayesian sequential monitoring designs for single-arm clinical trials with multiple outcomes. Stat Med 1995, 14:357-379.
10. Thall P.F., Sung H.G. Some extensions and applications of Bayesian strategy for monitoring multiple outcomes in clinical trials. Stat Med 1998, 17:1563-1580.
11. Thall P.F., Cook J.D. Adaptive dose selection using efficacy-toxicity trade-offs: illustrations and practical considerations. J Biopharm Stat 2006, 16:623-638.
12. Cook J.D. Multc lean statistical tutorial 2005.
13. A Randomized, Double-Blind, Placebo-Controlled, Ascending-Dose Study to Evaluate the Safety and Efficacy of CEP-37247 Administered by the Transforaminal Epidural Route for the Treatment of Patients With Lumbosacral Radicular Pain Associated With Disk Herniation. www.Clinicaltrials.gov identifier: NCT01240876. Sponsored by Cephalon, Inc.
14. Carlin B.P., Louis T.A. Bayesian methods for data analysis 2009, CRC Press. 3rd Edition.
15. Nadarajah S. Reliability for some bivariate distributions. Math Probl Eng 2005, 101-111.
16. Cook J.D., Nadarajah S. Stochastic inequality probabilities for adaptively randomized clinical trials. Biom J 2006, 48(3):356-365.
17. Wathen J.K., Nguyen H.Q., Cook J.D. Inequality calculator http://biostatistics.mdanderson.org/softwaredownload.
18. McClung M.R., Lewiecki E.M., Cohen S.B., et al. Denosumab in postmenopausal women with low bone mineral density. N Engl J Med 2006, 354(8):821-831.