ArticlePloS one2023
Designing an exploratory phase 2b platform trial in NASH with correlated, co-primary binary endpoints.
Article in PloS one, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 4 papers.
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
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Who cites it
4 citing papers in PubMed.
- Design, simulate, refine: simulation-guided clinical trials for accelerated drug development.Nature reviews. Drug discovery · 2026Review
- Why and how should we simulate platform trials? Learnings from EU-PEARL.BMC medical research methodology · 2025Article
- Needs assessment for creation of a platform trial network in metabolic-dysfunction associated steatohepatitis.Communications medicine · 2024Article
- Design Considerations for a Phase II Platform Trial in Major Depressive Disorder.Pharmaceutical statisticsArticle
Corrections and comments
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Authors and funding
9 authors.
Funding
No grant is acknowledged in the PubMed record.
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.
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