Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
Review
. 2024 Nov;21(11):809-823.
doi: 10.1038/s41575-024-00955-8. Epub 2024 Jul 17.

A roadmap for clinical trials in MASH-related compensated cirrhosis

Juan M Pericàs  1   2 , Quentin M Anstee  3   4 , Salvador Augustin  5 , Ramón Bataller  6 , Annalisa Berzigotti  7 , Andreea Ciudin  8   9 , Sven Francque  10 , Juan G Abraldes  11 , Virginia Hernández-Gea  12   6 , Mònica Pons  13 , Thomas Reiberger  14 , Ian A Rowe  15 , Peter Rydqvist  16 , Elmer Schabel  17 , Frank Tacke  18 , Emmanuel A Tsochatzis  19 , Joan Genescà  13   12
Affiliations
Review

A roadmap for clinical trials in MASH-related compensated cirrhosis

Juan M Pericàs et al. Nat Rev Gastroenterol Hepatol. 2024 Nov.

Erratum in

  • Author Correction: A roadmap for clinical trials in MASH-related compensated cirrhosis.
    Pericàs JM, Anstee QM, Augustin S, Bataller R, Berzigotti A, Ciudin A, Francque S, Abraldes JG, Hernández-Gea V, Pons M, Reiberger T, Rowe IA, Rydqvist P, Schabel E, Tacke F, Tsochatzis EA, Genescà J. Pericàs JM, et al. Nat Rev Gastroenterol Hepatol. 2024 Nov;21(11):824. doi: 10.1038/s41575-024-00976-3. Nat Rev Gastroenterol Hepatol. 2024. PMID: 39103628 No abstract available.

Abstract

Although metabolic dysfunction-associated steatohepatitis (MASH) is rapidly becoming a leading cause of cirrhosis worldwide, therapeutic options are limited and the number of clinical trials in MASH-related compensated cirrhosis is low as compared to those conducted in earlier disease stages. Moreover, designing clinical trials in MASH cirrhosis presents a series of challenges regarding the understanding and conceptualization of the natural history, regulatory considerations, inclusion criteria, recruitment, end points and trial duration, among others. The first international workshop on the state of the art and future direction of clinical trials in MASH-related compensated cirrhosis was held in April 2023 at Vall d'Hebron University Hospital in Barcelona (Spain) and was attended by a group of international experts on clinical trials from academia, regulatory agencies and industry, encompassing expertise in MASH, cirrhosis, portal hypertension, and regulatory affairs. The presented Roadmap summarizes important content of the workshop on current status, regulatory requirements and end points in MASH-related compensated cirrhosis clinical trials, exploring alternative study designs and highlighting the challenges that should be considered for upcoming studies on MASH cirrhosis.

PubMed Disclaimer

References

    1. Rinella, M. E. et al. A multi-society Delphi consensus statement on new fatty liver disease nomenclature. J. Hepatol. 79, 1542–1556 (2023).
    1. Younossi, Z. et al. Global burden of NAFLD and NASH: trends, predictions, risk factors and prevention. Nat. Rev. Gastroenterol. Hepatol. 15, 11–20 (2018). - PubMed
    1. Brown, G. T. & Kleiner, D. E. Histopathology of nonalcoholic fatty liver disease and nonalcoholic steatohepatitis. Metabolism 65, 1080–1086 (2016). - PubMed
    1. Anstee, Q. M., Reeves, H. L., Kotsiliti, E., Govaere, O. & Heikenwalder, M. From NASH to HCC: current concepts and future challenges. Nat. Rev. Gastroenterol. Hepatol. 16, 411–428 (2019). - PubMed
    1. Estes, C., Razavi, H., Loomba, R., Younossi, Z. & Sanyal, A. J. Modeling the epidemic of nonalcoholic fatty liver disease demonstrates an exponential increase in burden of disease. Hepatology 67, 123–133 (2018). - PubMed

LinkOut - more resources