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Published on: September 20, 2019
Optimal two-stage designs for single-arm phase II oncology trials with two binary endpoints
1Institute of Medical Biometry and Informatics, University of Heidelberg, Im Neuenheimer Feld 305, 69120 Heidelberg, Germany. kunz@imbi.uni-heidelberg.de
Methods of Information in Medicine
|November 9, 2010
Summary
This study introduces a new two-stage clinical trial design for oncology that evaluates two endpoints, offering a more thorough assessment of anticancer drug efficacy than single-endpoint methods.
Area of Science:
- Oncology
- Clinical Trial Design
- Biostatistics
Background:
- Standard phase II oncology trials use single-arm, two-stage designs with one endpoint.
- This approach may miss promising drugs that show disease stabilization despite a lower tumor response rate.
Purpose of the Study:
- To develop and present an analytical solution for a two-stage clinical trial design incorporating two endpoints.
- To provide optimal and minimax solutions for calculating type I and type II error rates.
- To address inference for two endpoints after rejecting the global null hypothesis.
Main Methods:
- An analytical solution for calculating error rates in a two-stage, two-endpoint design.
- Derivation of a multiple test procedure to control experimentwise type I error rate.
- Tabulation of the new design for various parameter values.
Main Results:
- The proposed method allows for a more comprehensive assessment of anti-tumor efficacy.
- Optimal and minimax designs with two endpoints can differ significantly in sample size characteristics.
- Admissible designs offer a valuable compromise between design characteristics.
Conclusions:
- The new procedure extends Simon's two-stage design to accommodate two endpoints.
- This enables a more holistic evaluation of a new treatment's anti-tumor efficacy.
- The approach provides a more nuanced understanding of drug performance in clinical trials.
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