Title

Sample size determination for clinical trials in patients with nonlinear disease progression

Authors

Authors

X. Yan;X. G. Su

Comments

Authors: contact us about adding a copy of your work at STARS@ucf.edu

Abbreviated Journal Title

J. Biopharm. Stat.

Keywords

longitudinal trial; nonlinear disease progression; sample size; study; duration; within-patient correlation; Pharmacology & Pharmacy; Statistics & Probability

Abstract

This paper provides explicit sample size determination formulas for planning a long-term trial in patients with chronic disease by using available results from existing short-term studies that may predict long-term disease progression patterns. The sample size calculation formulas are flexible to incorporate different nonlinear disease progression patterns. Various within-patient correlation structures are considered. By using the proposed formulas, sample size sensitivity can be easily explored for possible choices of study duration, assumed nonlinear disease progression patterns, randomization ratio, and expected clinical meaningful difference in the end of study. In addition, sample size calculation formulas are provided when the primary endpoint is change from baseline. Discussions on the relationship among required sample size, study duration, randomization ratio are also included.

Journal Title

Journal of Biopharmaceutical Statistics

Volume

16

Issue/Number

1

Publication Date

1-1-2006

Document Type

Article

Language

English

First Page

91

Last Page

105

WOS Identifier

WOS:000236233500007

ISSN

1054-3406

Share

COinS