The Most Often Utilized Clinical Endpoints for Assessing Treatment Efficacy in Nonalcoholic Fatty Liver or Nonalcoholic Steatohepatitis

OBJECTIVES: The aim of this study was to determine the most often utilized clinical endpoints for assessing treatment efficacy in nonalcoholic fatty liver (NAFL) or nonalcoholic steatohepatitis (NASH).

METHODS: The data from ClinicalTrials.gov was used. Phase 2 trials with more than 50 people were considered, excluding trials assessing device testing, food restriction, operations, and diagnostics. Trials were tagged and classified according to outcome measures, and the most frequent primary, secondary, and additional outcomes across all trials were determined. These endpoints were afterwards compared with outcomes considered in economic models recently developed in NASH.

RESULTS: The initial search returned 287 trials for pre-screening. After filtering by the specified criteria, the trial number was reduced to 179. Following the review process, a total of 83 primary, 291 secondary, and 52 other endpoints were identified. The majority of the highlighted endpoints were related to evaluating the subject's fat content or liver condition. The primary endpoints most frequently observed were “liver fat” (44 trials), “MRI-PDFF” - magnetic resonance imaging-derived proton density fat fraction (30 trials), and “adverse events” (28 trials). Among secondary endpoints, “ALT” - alanine transaminase level appeared most commonly (67 trials), followed by “fibrosis” (55 trials) and “AST” - aspartate aminotransferase level (48 trials).

CONCLUSIONS: Numerous clinical trials are currently underway to explore different treatments for NASH, each with its own set of outcomes under consideration. Recognizing this variety and understanding the most prevalent endpoints will facilitate more effective comparisons between clinical studies.