Endpoints and clinical trial design for nonalcoholic steatohepatitis

Hepatology. 2011 Jul;54(1):344-53. doi: 10.1002/hep.24376.

Abstract

Nonalcoholic fatty liver disease is a common cause of chronic liver disease in the general population. Nonalcoholic steatohepatitis (NASH), the aggressive form of nonalcoholic fatty liver disease, is associated with an increased risk of liver-related mortality and cardiovascular disease. At present, a liver biopsy is the only generally acceptable method for the diagnosis of NASH and assessment of its progression toward cirrhosis. Although several treatments have shown evidence of efficacy in clinical trials of varying design, there are no approved treatments for NASH, and published trials are often too divergent to allow meaningful comparisons. There is thus a lack of established noninvasive, point-of-care diagnostics and approved treatment on one hand and a substantial population burden of disease on the other. These provide the rationale for developing consensus on key endpoints and clinical trial design for NASH.

Conclusion: This article summarizes the consensus arrived at a meeting of the American Association for the Study of Liver Diseases on the key endpoints and specific trial design issues that are germane for development of diagnostic biomarkers and treatment trials for NASH.

Publication types

  • Consensus Development Conference
  • Research Support, N.I.H., Intramural
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Biomarkers / metabolism
  • Biopsy
  • Clinical Trials as Topic*
  • Disease Progression
  • Endpoint Determination*
  • Fatty Liver / diagnosis*
  • Fatty Liver / metabolism
  • Fatty Liver / therapy*
  • Humans
  • Liver / pathology
  • Non-alcoholic Fatty Liver Disease
  • Societies, Medical
  • United States

Substances

  • Biomarkers