Designing an exploratory phase 2b platform trial in NASH with correlated, co-primary binary endpoints
| Autor: | Meyer, Elias Laurin, Mesenbrink, Peter, Di Prospero, Nicholas A., Pericàs, Juan M., Glimm, Ekkehard, Ratziu, Vlad, Sena, Elena, König, Franz |
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| Přispěvatelé: | Institut Català de la Salut, [Meyer EL, König F] Center for Medical Data Science, Medical University of Vienna, Vienna, Austria. [Mesenbrink P] Novartis Pharmaceuticals Corporation, One Health Plaza, East Hanover, NJ, United States of America. [Di Prospero NA] Janssen Research and Development, Raritan, NJ, United States of America. [Pericàs JM] Servei d’Hepatologia, Vall d’Hebron Hospital Universitari, Barcelona, Spain. Vall d’Hebron Institut de Recerca (VHIR), Barcelona, Spain. Centros de Investigación Biomédica en Red Enfermedades Hepáticas y Digestivas (CIBERehd), ISCIII, Madrid, Spain. [Glimm E] Novartis Pharma AG, Basel, Switzerland. Institute of Biometry and Medical Informatics, University of Magdeburg, Magdeburg, Germany. [Ratziu V] Assistance Publique-Hôpitaux de Paris, Hôpital Pitie-Salpetriere, University of Paris, Paris, France. [Sena E] Servei d’Hepatologia, Vall d’Hebron Hospital Universitari, Barcelona, Spain. Vall d’Hebron Institut de Recerca (VHIR), Barcelona, Spain, Vall d'Hebron Barcelona Hospital Campus |
| Jazyk: | angličtina |
| Rok vydání: | 2022 |
| Předmět: |
Medicaments - Desenvolupament
enfermedades del sistema digestivo::enfermedades hepáticas::hígado graso::esteatosis hepática no alcohólica [ENFERMEDADES] técnicas de investigación::métodos::diseño de la investigación [TÉCNICAS Y EQUIPOS ANALÍTICOS DIAGNÓSTICOS Y TERAPÉUTICOS] FOS: Computer and information sciences Esteatosi hepàtica - Tractament Multidisciplinary Medicina clínica - Investigació Investigative Techniques::Methods::Research Design [ANALYTICAL DIAGNOSTIC AND THERAPEUTIC TECHNIQUES AND EQUIPMENT] Digestive System Diseases::Liver Diseases::Fatty Liver::Non-alcoholic Fatty Liver Disease [DISEASES] Otros calificadores::Otros calificadores::/farmacoterapia [Otros calificadores] Applications (stat.AP) Other subheadings::Other subheadings::/drug therapy [Other subheadings] Statistics - Applications |
| Zdroj: | Scientia |
| Popis: | Fibrosis; Drug research and development; Decision making Fibrosis; Investigación y desarrollo de medicamentos; Toma de decisiones Fibrosi; Recerca i desenvolupament de medicaments; Presa de decisions 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. EU-PEARL has received funding from the Innovative Medicines Initiative 2 Joint Undertaking under grant agreement No 853966-2. This Joint Undertaking receives support from the European Union’s Horizon 2020 research and innovation programme and EFPIA and CHILDREN’S TUMOR FOUNDATION, GLOBAL ALLIANCE FOR TB DRUG DEVELOPMENT NON PROFIT ORGANISATION, SPRINGWORKS THERAPEUTICS INC. The funders had no role in study design, data collection and analysis, decision to publish, or preparation of the manuscript. |
| Databáze: | OpenAIRE |
| Externí odkaz: |
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