Dynamic development paths for expanding a proof-of-concept study to explore dose range.
In Phase II clinical development of a new drug, the two most important deliverables are proof of concept (PoC) and dose ranging. Traditionally, a PoC study is designed as the first Phase II clinical trial. In this PoC, there are two treatment groups-a high dose of the study medication, against the placebo control. After the concept is proven, the next Phase II study is a dose-ranging design with many test doses. This paper proposes a two-stage design with the first stage attempting to generate an early signal of efficacy. If successful, the second stage will adopt a "Go Fast" plan to expand the current study and add lower study doses of the test drug to explore the efficacy dose range. Otherwise, a "Go Slow" strategy is triggered, and the study will stop at a reduced sample size with high dose and placebo only.
['Deng|Qiqi|Q|', 'Bai|Xiaofei|X|0000-0003-2011-5756', 'Ting|Naitee|N|']
D017322:Clinical Trials, Phase II as Topic D006801:Humans D015233:Models, Statistical D004364:Pharmaceutical Preparations D000075082:Proof of Concept Study D012107:Research Design D018401:Sample Size
proof-of-concept dose ranging clinical trial two-stage design “Go/NoGo” decision