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.

尽管代谢功能障碍相关脂肪性肝炎 （MASH） 正迅速成为全球肝硬化的主要原因，但治疗选择有限，并且与疾病早期阶段进行的临床试验相比，MASH 相关代偿期肝硬化的临床试验数量较少。此外，设计 MASH 肝硬化的临床试验在自然病程的理解和概念化、监管考虑、纳入标准、招募、终点和试验持续时间等方面提出了一系列挑战。2023 年 4 月在巴塞罗那（西班牙）的 Vall d'Hebron 大学医院举行了第一届关于 MASH 相关代偿期肝硬化临床试验的最新技术和未来方向的国际研讨会，来自学术界、监管机构和行业的一组临床试验国际专家出席了研讨会，包括 MASH、肝硬化、 门静脉高压症和监管事务。提出的路线图总结了研讨会关于 MASH 相关代偿性肝硬化临床试验的现状、监管要求和终点的重要内容，探索了替代研究设计，并强调了即将到来的 MASH 肝硬化研究应考虑的挑战。