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Designing an exploratory phase 2b platform trial in NASH with correlated, co-primary binary endpoints

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Listed:
  • Elias Laurin Meyer
  • Peter Mesenbrink
  • Nicholas A Di Prospero
  • Juan M Pericàs
  • Ekkehard Glimm
  • Vlad Ratziu
  • Elena Sena
  • Franz König
  • on behalf of the EU-PEARL NASH Investigators

Abstract

Non-alcoholic steatohepatitis (NASH) is the progressive form of nonalcoholic fatty liver disease (NAFLD) and a disease 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 two health authorities and the learnings from these meetings 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.

Suggested Citation

  • Elias Laurin Meyer & Peter Mesenbrink & Nicholas A Di Prospero & Juan M Pericàs & Ekkehard Glimm & Vlad Ratziu & Elena Sena & Franz König & on behalf of the EU-PEARL NASH Investigators, 2023. "Designing an exploratory phase 2b platform trial in NASH with correlated, co-primary binary endpoints," PLOS ONE, Public Library of Science, vol. 18(3), pages 1-27, March.
    • Handle: RePEc:plo:pone00:0281674
    DOI: 10.1371/journal.pone.0281674
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    References listed on IDEAS

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    1. Wei Jiang & Shuang Song & Lin Hou & Hongyu Zhao, 2021. "A Set of Efficient Methods to Generate High-Dimensional Binary Data With Specified Correlation Structures," The American Statistician, Taylor & Francis Journals, vol. 75(3), pages 310-322, July.
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