TY - JOUR
T1 - A roadmap for clinical trials in MASH-related compensated cirrhosis
AU - Pericàs, Juan M.
AU - Anstee, Quentin M.
AU - Augustin, Salvador
AU - Bataller, Ramón
AU - Berzigotti, Annalisa
AU - Ciudin, Andreea
AU - Francque, Sven
AU - Abraldes, Juan G.
AU - Hernández-Gea, Virginia
AU - Pons, Mònica
AU - Reiberger, Thomas
AU - Rowe, Ian A.
AU - Rydqvist, Peter
AU - Schabel, Elmer
AU - Tacke, Frank
AU - Tsochatzis, Emmanuel A.
AU - Genescà, Joan
N1 - Publisher Copyright:
© Springer Nature Limited 2024.
PY - 2024
Y1 - 2024
N2 - 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.
AB - 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.
UR - http://www.scopus.com/inward/record.url?scp=85198850695&partnerID=8YFLogxK
U2 - 10.1038/s41575-024-00955-8
DO - 10.1038/s41575-024-00955-8
M3 - Article
AN - SCOPUS:85198850695
SN - 1759-5045
JO - Nature Reviews Gastroenterology and Hepatology
JF - Nature Reviews Gastroenterology and Hepatology
ER -