Design of Phase II Non-inferiority Trials.

TitleDesign of Phase II Non-inferiority Trials.
Publication TypeJournal Article
Year of Publication2017
AuthorsJung, Sin-Ho
JournalContemp Clin Trials Commun
Volume7
Pagination23-27
Date Published2017 Sep
ISSN2451-8654
Abstract

With the development of inexpensive treatment regimens and less invasive surgical procedures, we are confronted with non-inferiority study objectives. A non-inferiority phase III trial requires a roughly four times larger sample size than that of a similar standard superiority trial. Because of the large required sample size, we often face feasibility issues to open a non-inferiority trial. Furthermore, due to lack of phase II non-inferiority trial design methods, we do not have an opportunity to investigate the efficacy of the experimental therapy through a phase II trial. As a result, we often fail to open a non-inferiority phase III trial and a large number of non-inferiority clinical questions still remain unanswered. In this paper, we want to develop some designs for non-inferiority randomized phase II trials with feasible sample sizes. At first, we review a design method for non-inferiority phase III trials. Subsequently, we propose three different designs for non-inferiority phase II trials that can be used under different settings. Each method is demonstrated with examples. Each of the proposed design methods is shown to require a reasonable sample size for non-inferiority phase II trials. The three different non-inferiority phase II trial designs are used under different settings, but require similar sample sizes that are typical for phase II trials.

DOI10.1016/j.conctc.2017.04.008
Alternate JournalContemp Clin Trials Commun
Original PublicationDesign of phase II non-inferiority trials.
PubMed ID28798964
PubMed Central IDPMC5546322
Grant ListP01 CA142538 / CA / NCI NIH HHS / United States