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Published on: September 20, 2019
A two-stage phase II trial design utilizing both primary and secondary endpoints
Xun Lin1, Randy Allred, Glen Andrews
1Pfizer Global Research and Development, La Jolla Laboratories, 10555 Science Center Drive, San Diego, CA 92121, USA. Xun.Lin@Pfizer.com
Pharmaceutical Statistics
|January 26, 2007
Summary
This study introduces a novel two-stage clinical trial design for oncology, incorporating both primary and secondary endpoints. This approach enhances treatment activity assessment by considering multiple parameters, improving drug development efficiency.
Area of Science:
- Clinical Trials
- Oncology Drug Development
- Biostatistics
Background:
- Phase II oncology trials assess initial treatment activity.
- Traditional designs, like Simon's 2-stage, often focus on a single parameter (e.g., response rate).
- There is a need for designs that can evaluate multiple endpoints simultaneously.
Purpose of the Study:
- To present a novel two-stage clinical trial design for oncology.
- To incorporate both primary and secondary endpoints into the decision-making process.
- To evaluate the statistical properties of this new design.
Main Methods:
- Development of a two-stage clinical trial design.
- Inclusion of both primary and secondary endpoints in decision rules.
- Monte-Carlo simulations to investigate performance.
Main Results:
- The proposed design utilizes multiple endpoints for decision rules.
- Family-wise Type 1 error rate and statistical power were analyzed.
- Performance was evaluated under various simulated scenarios.
Conclusions:
- The presented two-stage design offers a method for evaluating multiple endpoints in Phase II oncology trials.
- This approach may provide a more comprehensive assessment of treatment activity.
- Further investigation into its application and statistical properties is warranted.
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