A Novel Model of the Continual Reassessment Method in Phase I Trial

Overview

Journal Sci Rep

Specialty Science

Date 2023 Mar 28

PMID 36977709

Authors

Weijia Zhang

Wanni Lei

Xiaojun Zhu

Affiliations

Soon will be listed here.

Abstract

For the model-based designs, the continual reassessment method (CRM) is widely used to identify the maximum tolerated dose (MTD) in phase I clinical trials. To improve the performance of classic CRM models, we propose a new CRM and its dose-toxicity probability function based on the Cox model whatever the treatment response is immediately observed or delayed. In the process of dose-finding trial, we can use our model in situations when either the response is delayed or not and can derive the likelihood function and posterior mean toxicity probabilities to find the MTD. Simulation is carried out to evaluate the performance of the proposed model with the classic CRM models. We also evaluate the operating characteristics of the proposed model by the Efficiency, Accuracy, Reliability, and Safety (EARS) criteria.

References

Goodman S, Zahurak M, Piantadosi S . Some practical improvements in the continual reassessment method for phase I studies. Stat Med. 1995; 14(11):1149-61. DOI: 10.1002/sim.4780141102. View

Ji Y, Liu P, Li Y, Bekele B . A modified toxicity probability interval method for dose-finding trials. Clin Trials. 2010; 7(6):653-63. PMC: 5038924. DOI: 10.1177/1740774510382799. View

Simon R, Freidlin B, Rubinstein L, Arbuck S, Collins J, Christian M . Accelerated titration designs for phase I clinical trials in oncology. J Natl Cancer Inst. 1997; 89(15):1138-47. DOI: 10.1093/jnci/89.15.1138. View

Le Tourneau C, Lee J, Siu L . Dose escalation methods in phase I cancer clinical trials. J Natl Cancer Inst. 2009; 101(10):708-20. PMC: 2684552. DOI: 10.1093/jnci/djp079. View

Yan F, Mandrekar S, Yuan Y . Keyboard: A Novel Bayesian Toxicity Probability Interval Design for Phase I Clinical Trials. Clin Cancer Res. 2017; 23(15):3994-4003. PMC: 5554436. DOI: 10.1158/1078-0432.CCR-17-0220. View

Lee S, Cheung Y . Model calibration in the continual reassessment method. Clin Trials. 2009; 6(3):227-38. PMC: 2884971. DOI: 10.1177/1740774509105076. View

Stylianou M, Follmann D . The accelerated biased coin up-and-down design in phase I trials. J Biopharm Stat. 2004; 14(1):249-60. DOI: 10.1081/bip-120028518. View

Babb J, Rogatko A, Zacks S . Cancer phase I clinical trials: efficient dose escalation with overdose control. Stat Med. 1998; 17(10):1103-20. DOI: 10.1002/(sici)1097-0258(19980530)17:10<1103::aid-sim793>3.0.co;2-9. View

Ishizuka N, Ohashi Y . The continual reassessment method and its applications: a Bayesian methodology for phase I cancer clinical trials. Stat Med. 2001; 20(17-18):2661-81. DOI: 10.1002/sim.735. View

10.

Wages N, Petroni G . A web tool for designing and conducting phase I trials using the continual reassessment method. BMC Cancer. 2018; 18(1):133. PMC: 5799912. DOI: 10.1186/s12885-018-4038-x. View

11.

Wheeler G, Mander A, Bedding A, Brock K, Cornelius V, Grieve A . How to design a dose-finding study using the continual reassessment method. BMC Med Res Methodol. 2019; 19(1):18. PMC: 6339349. DOI: 10.1186/s12874-018-0638-z. View

12.

Storer B . Design and analysis of phase I clinical trials. Biometrics. 1989; 45(3):925-37. View

13.

OQuigley J, Pepe M, Fisher L . Continual reassessment method: a practical design for phase 1 clinical trials in cancer. Biometrics. 1990; 46(1):33-48. View

14.

Pan H, Yuan Y . A default method to specify skeletons for Bayesian model averaging continual reassessment method for phase I clinical trials. Stat Med. 2016; 36(2):266-279. PMC: 5026535. DOI: 10.1002/sim.6941. View

15.

Neuenschwander B, Branson M, Gsponer T . Critical aspects of the Bayesian approach to phase I cancer trials. Stat Med. 2008; 27(13):2420-39. DOI: 10.1002/sim.3230. View

16.

North B, Kocher H, Sasieni P . A new pragmatic design for dose escalation in phase 1 clinical trials using an adaptive continual reassessment method. BMC Cancer. 2019; 19(1):632. PMC: 6595589. DOI: 10.1186/s12885-019-5801-3. View

17.

Angell M . The ethics of clinical research in the Third World. N Engl J Med. 1997; 337(12):847-9. DOI: 10.1056/NEJM199709183371209. View

18.

Faries D . Practical modifications of the continual reassessment method for phase I cancer clinical trials. J Biopharm Stat. 1994; 4(2):147-64. DOI: 10.1080/10543409408835079. View

19.

Yuan Z, Chappell R, Bailey H . The continual reassessment method for multiple toxicity grades: a Bayesian quasi-likelihood approach. Biometrics. 2007; 63(1):173-9. DOI: 10.1111/j.1541-0420.2006.00666.x. View

20.

Liu S, Pan H, Xia J, Huang Q, Yuan Y . Bridging continual reassessment method for phase I clinical trials in different ethnic populations. Stat Med. 2015; 34(10):1681-94. DOI: 10.1002/sim.6442. View