TITE(e)-U-Keyboard:a Two-stage Phase Ⅰ/Ⅱ Design
10.11783/j.issn.1002-3674.2023.06.003
- VernacularTitle:TITE(e)-U-Keyboard两阶段Ⅰ/Ⅱ期试验设计研究
- Author:
Juan HE
1
;
Wenyun YANG
;
Yucheng ZHANG
Author Information
1. 中国药科大学生物统计与计算药学研究中心(210009)
- Keywords:
Late-onset efficacy;
Phase Ⅰ/Ⅱ clinical trial;
Two-stage design
- From:
Chinese Journal of Health Statistics
2023;40(6):811-816
- CountryChina
- Language:Chinese
-
Abstract:
Objective Aiming at the problem of delayed therapeutic effect of tumor targeting agents and cellular immunotherapy,an innovative TITE(e)-U-Keyboard design is established based on the framework of phaseⅠ/Ⅱclinical trial design.Methods Approximated binomial likelihood method was used to incorporate the unobserved efficacy outcome information and construct approximated distribution to assist decision making.TITE(e)-U-Keyboard was compared with theⅠ/Ⅱ trial design method(U-BOIN、BOIN12)from multiple perspectives.Results Simulation results show that the accuracy of OBD selection and the number of patients allocated at optimal dose of the three methods were similar,but TITE(e)-U-Keyboard requires the shortest trial duration.Sensitivity analysis shows that the effectiveness evaluation window has an effect on the TITE(e)-U-Keyboard duration,which increases with the increase of effectiveness evaluation window.Conclusion TITE(e)-U-Keyboard significantly reduces the trial duration while maintaining good simulation performance and it can address the problem of delayed treatment effects in phase Ⅰ/Ⅱ clinical trials,thus accelerating the clinical trial process.
