Related Experiment Video
Updated: Apr 16, 2026

A Clinical Trial Assessing the Safety, Efficacy, and Delivery of Olive-Oil-Based Three-Chamber Bags for Parenteral Nutrition
Published on: September 20, 2019
Statistical issues for design and analysis of single-arm multi-stage phase II cancer clinical trials
1Biostatistics and Clinical Epidemiology Center, Samsung Medical Center, Department of Biostatistics and Bioinformatics, Duke University, USA.
Background:
Phase II trials have been very widely conducted and published every year for cancer clinical research. In spite of the fast progress in design and analysis methods, single-arm two-stage design is still the most popular for phase II cancer clinical trials. Because of their small sample sizes, statistical methods based on large sample approximation are not appropriate for design and analysis of phase II trials.
Methods:
As a prospective clinical research, the analysis method of a phase II trial is predetermined at the design stage and it is analyzed during and at the end of the trial as planned by the design. The analysis method of a trial should be matched with the design method. For two-stage single arm phase II trials, Simon's method has been the standards for choosing an optimal design, but the resulting data have been analyzed and published ignoring the two-stage design aspect with small sample sizes.
Conclusions:
In this article, we review analysis methods that exactly get along with the exact two-stage design method. We also discuss some statistical methods to improve the existing design and analysis methods for single-arm two-stage phase II trials.
Related Concept Videos
Clinical Trials
There are four phases in a clinical trial. A phase one...
Clinical Trials: Overview
Comparing the Survival Analysis of Two or More Groups
Types of Biopharmaceutical Studies: Controlled and Non-Controlled Approaches
Non-controlled studies, commonly employed for initial exploration, lack a control group, rendering them susceptible to biases and external influences. In contrast,...
Cancer Survival Analysis
Bioavailability Study Design: Single Versus Multiple Dose Studies
