Designing an exploratory phase 2b platform trial in NASH with correlated, co-primary binary endpoints
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Non-alcoholic steatohepatitis (NASH), an inflammatory and more progressive form of nonalcoholic fatty liver disease (NAFLD), is a disease area with high unmet medical need. Platform trials provide great benefits for sponsors and trial participants in terms of accelerating drug development programs. In this article, we describe some of the activities of the EU-PEARL consortium (EU Patient-cEntric clinicAl tRial pLatforms) regarding the use of platform trials in NASH, in particular the proposed trial design, decision rules and simulation results. For a set of assumptions, we present the results of a simulation study recently discussed with the FDA in a critical path innovation meeting (CPIM) and the learnings from this meeting from a trial design perspective. Since the proposed design uses co-primary binary endpoints, we furthermore discuss the different options and practical considerations for simulating correlated binary endpoints.
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