Design and implementation of an adaptive confirmatory trial in Japanese patients with palmoplantar pustulosis.

ABSTRACT:

Background/aims

More and more literature describes how to overcome challenges in the implementation of adaptive designs for trials for drug approval process. Most adaptive trials were conducted in Western Europe or USA for Phase II or Phase II/III settings; however, examples of non-oncology pivotal adaptive trials used for regulatory approval in Japan are rare. This article elaborates on our experience with designing and implementing a Phase III adaptive confirmatory trial permitting unblinded sample size re-estimation and futility analysis after a single interim analysis in Japanese patients with palmoplantar pustulosis (PPP) receiving guselkumab.

Methods

We provide insights into consideration at the design stage of an adaptive study, including design options, development d

SUBMITTER: Zheng R 

PROVIDER: S-EPMC9192787 | biostudies-literature | 2022 Aug

REPOSITORIES: biostudies-literature