Single-Arm Phase II Survival Trial Design provides a comprehensive summary to the most commonly- used methods for single-arm phase II trial design with time-to-event endpoints. Single-arm phase II trials are a key component for successfully developing advanced cancer drugs and treatments, particular for target therapy and immunotherapy in which time-to-event endpoints are often the primary endpoints. Most test statistics for single-arm phase II trial design with time-to-event endpoints are not available in commercial software.
- Covers the most frequently used methods for single-arm phase II trial design with time-to-event endpoints in a comprehensive fashion.
- Provides new material on phase II immunotherapy trial design and phase II trial design with TTP ratio endpoint.
- Illustrates trial designs by real clinical trial examples
- Includes R code for all methods proposed in the book, enabling straightforward sample size calculation.
Table of Contents
1. Introduction of Single-Arm Phase II Trial Design
2. Phase II Trial Design Under Parametric Model
3. One-Stage Design Evaluating Survival Probabilities
4. Two-Stage Design Evaluating Survival Probabilities
5. One-Stage Design Evaluating Survival Distributions
6. Two-Stage Design Evaluating Survival Distributions
7. Phase II Immunotherapy Trial Design
8. Phase II Trial Design with GMI Endpoint
9. Bayesian Single-Arm Phase II Trial Design
Appendix A. Probability of Failure Under Uniform Accrual
Appendix B. Asymptotic Distribution of Nelson-Aalen Estimate of the Cumulative Hazard
Appendix C. Derivation Asymptotic Distribution of the OSLRT
Appendix D. Derivation of equations (6.8) and (6.9)
Appendix E. Crossing point for the mixture cure model
Appendix F. Derivation asymptotic distribution of the score test Q
Appendix G. Generate random variables from GBVE model
Appendix H. Derivation censoring survival distribution of GMI
Appendix I. Proof of monotonicity of the posterior probability
Appendix J. Relationship between frequentist and Bayesian type I error rates
Appendix K. R Code
Jianrong (John) Wu is a professor in the Division of Cancer Biostatistics, Department of Internal Medicine, University of Kentucky; Biostatistics and Bioinformatics Shared Resource, Markey Cancer Center. Dr. Wu has more than 20 years’ experience of designing and conducting cancer clinical trials at St. Jude Children’s Research Hospital and Markey Cancer Center and has developed many novel statistical methods for designing and monitoring single-arm phase II and randomized phase III trial design with time-to-event endpoints.