|Title||Design of Phase II Non-inferiority Trials.|
|Publication Type||Journal Article|
|Year of Publication||2017|
|Journal||Contemp Clin Trials Commun|
|Date Published||2017 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 Journal||Contemp Clin Trials Commun|
|Original Publication||Design of phase II non-inferiority trials.|
|PubMed Central ID||PMC5546322|
|Grant List||P01 CA142538 / CA / NCI NIH HHS / United States|
Design of Phase II Non-inferiority Trials.