Seamless phase IIa/IIb adaptive design with the same primary endpoint for proof of concept and dose finding

Abstract

This paper considers combining a proof of concept (POC) study and a dose finding (DF) study where the POC and the DF share the same primary endpoint. An example based on real study conditions shows that compared to a conventional design the proposed adaptive design tests more active doses, with a smaller sample size and a shorter overall duration leading to a budget saving of 30% in study operations. Keywords: Adaptive design, Dose finding, Proof of concept, Seamless design, Budget saving