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
Date Published2017 Sep

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.

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