Designing an exploratory phase 2b platform trial in NASH with correlated, co-primary binary endpoints.
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 desc...
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| Format: | Article |
| Language: | English |
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Public Library of Science (PLoS)
2023-01-01
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| Series: | PLoS ONE |
| Online Access: | https://journals.plos.org/plosone/article/file?id=10.1371/journal.pone.0281674&type=printable |
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| author | Elias Laurin Meyer Peter Mesenbrink Nicholas A Di Prospero Juan M Pericàs Ekkehard Glimm Vlad Ratziu Elena Sena Franz König EU-PEARL NASH Investigators |
| author_facet | Elias Laurin Meyer Peter Mesenbrink Nicholas A Di Prospero Juan M Pericàs Ekkehard Glimm Vlad Ratziu Elena Sena Franz König EU-PEARL NASH Investigators |
| author_sort | Elias Laurin Meyer |
| collection | DOAJ |
| description | 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. |
| format | Article |
| id | doaj-art-459636e7038a492385f00e8dbcee2ab5 |
| institution | Kabale University |
| issn | 1932-6203 |
| language | English |
| publishDate | 2023-01-01 |
| publisher | Public Library of Science (PLoS) |
| record_format | Article |
| series | PLoS ONE |
| spelling | doaj-art-459636e7038a492385f00e8dbcee2ab52025-02-05T05:32:43ZengPublic Library of Science (PLoS)PLoS ONE1932-62032023-01-01183e028167410.1371/journal.pone.0281674Designing an exploratory phase 2b platform trial in NASH with correlated, co-primary binary endpoints.Elias Laurin MeyerPeter MesenbrinkNicholas A Di ProsperoJuan M PericàsEkkehard GlimmVlad RatziuElena SenaFranz KönigEU-PEARL NASH InvestigatorsNon-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.https://journals.plos.org/plosone/article/file?id=10.1371/journal.pone.0281674&type=printable |
| spellingShingle | Elias Laurin Meyer Peter Mesenbrink Nicholas A Di Prospero Juan M Pericàs Ekkehard Glimm Vlad Ratziu Elena Sena Franz König EU-PEARL NASH Investigators Designing an exploratory phase 2b platform trial in NASH with correlated, co-primary binary endpoints. PLoS ONE |
| title | Designing an exploratory phase 2b platform trial in NASH with correlated, co-primary binary endpoints. |
| title_full | Designing an exploratory phase 2b platform trial in NASH with correlated, co-primary binary endpoints. |
| title_fullStr | Designing an exploratory phase 2b platform trial in NASH with correlated, co-primary binary endpoints. |
| title_full_unstemmed | Designing an exploratory phase 2b platform trial in NASH with correlated, co-primary binary endpoints. |
| title_short | Designing an exploratory phase 2b platform trial in NASH with correlated, co-primary binary endpoints. |
| title_sort | designing an exploratory phase 2b platform trial in nash with correlated co primary binary endpoints |
| url | https://journals.plos.org/plosone/article/file?id=10.1371/journal.pone.0281674&type=printable |
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