| Autor: |
Elena Sena, Frank Tacke, Quentin M. Anstee, Nicholas Di Prospero, Mette Skalshøi- Kjær, Sergio Muñoz-Martínez, Jesús Rivera-Esteban, Alba Jiménez-Masip, Jesús M. Bañales, María Martínez-Gómez, Franz Koenig, Joan Genescà, Vlad Ratziu, Juan M. Pericàs |
| Jazyk: |
angličtina |
| Rok vydání: |
2024 |
| Předmět: |
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| Zdroj: |
Communications Medicine, Vol 4, Iss 1, Pp 1-8 (2024) |
| Druh dokumentu: |
article |
| ISSN: |
2730-664X |
| DOI: |
10.1038/s43856-024-00560-5 |
| Popis: |
Abstract Background The EU Patient-cEntric clinicAl tRial pLatforms (EU-PEARL) project (IMI2-853966) aimed to develop tools to establish integrated research platforms (IRP) for conducting adaptive-design trials in various diseases, including metabolic-dysfunction associated steatohepatitis (MASH). One essential component of a successful MASH IRP is a robust and reliable Clinical Research Network (CRN). Herein, we outline the required elements and anticipated steps to set-up such a CRN. Methods We identified European clinical research sites that could potentially serve as the foundation for MASH IRP and a CRN. A survey was sent to sites to assess their interest in joining a CRN, their familiarity with platform trials, and their capacity to participate in a future MASH IRP. Results A total of 141 investigators were invited to participate in the survey, and 40% responded. More than half of the answers (52%) identify MASH with advanced fibrosis (F3-4) as the subpopulation with the greatest unmet need. Regarding the difficulty in identifying candidates for trials, 65% find it is moderately difficult and 30% very difficult. Most respondents (94%) believe that a platform trial could offer substantial benefits to patients. Nearly all researchers express interest in participating in a platform trial (78%), with 22% indicating their interest would be contingent on initial industry funding. Conclusion While preliminary, our findings on responding sites are encouraging for the potential establishment of a CRN for a MASH IRP. However, funding schemes and sustainability strategies to provide proof-of-platform in MASH seem key in the short-term scenario. |
| Databáze: |
Directory of Open Access Journals |
| Externí odkaz: |
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