Optimal two-stage randomized multinomial designs for Phase II oncology trials
Related Experiment Video
Updated: Jun 23, 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
Linda Z Sun1, Cong Chen, Kamlesh Patel
1Biostatistics and Research Decision Sciences, Merck Research Laboratories, Upper Gwynedd, PA 19454, USA. linda_sun@merck.com
Abstract:
A new two-stage design is proposed that is suitable for early detection of the anticancer activity of experimental therapies in Phase II oncology trials. The endpoints of interest are response rate and early progression rate. The anticancer activity is defined by a positive signal in one endpoint and a non-negative signal in the other endpoint. The two endpoints are modeled by the multinomial distribution. The design is optimal in that it minimizes the patient exposure when the experimental therapies are inactive. The design parameters are found by a grid searching algorithm under type I and type II error rate constraints. Examples of the design are also presented in this paper.
Related Concept Videos
Bioequivalence Experimental Study Designs: Repeated Measures, Cross-Over, Carry-Over, and Latin Square Designs
Bioequivalence Experimental Study Designs: Completely Randomized and Randomized Block Designs
Clinical Trials: Overview
Clinical Trials
There are four phases in a clinical trial. A phase one...
Crossover Experiments
Crossover designs are performed even with smaller sample sizes since the samples can act as their controls. These are better than simple randomized trials since patients are exposed to all the treatments.
Randomized Experiments
Simple randomization
Simple...