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Endpoints and patient stratification in clinical trials for alcoholic hepatitis

Philippe Mathurin1, Mark Thursz2

  • 1Service des Maladies de l'Appareil Digestif, PU-PH, Hôpital Claude Huriez, 1er étage- Aile Est, Rue Michel Polonovski, 59037 Lille Cedex, France.

Journal of Hepatology
|January 20, 2019
PubMed

Insights

Developing new treatments for alcoholic hepatitis faces challenges. Using liver function tests as endpoints in early trials is more practical than mortality, aiding drug development for this unmet need.

Area of Science:

  • Hepatology
  • Clinical Trial Design
  • Pharmacology

Background:

  • Clinical development pathways are established in many medical fields.
  • Novel therapeutics for alcoholic hepatitis development faces significant unanswered questions.
  • Current trial designs may hinder pharmaceutical investment in this area.

Purpose of the Study:

  • To discuss alternative endpoints for alcoholic hepatitis clinical trials.
  • To propose pragmatic solutions for early-phase study designs.
  • To facilitate the development of new therapeutics for alcoholic hepatitis.

Main Methods:

  • Literature review and expert discussion on clinical trial endpoints.
  • Analysis of the practicality of mortality versus functional endpoints in Phase II trials.
  • Recommendations for trial stratification based on disease severity.

Main Results:

  • Mortality as an endpoint in Phase II trials is impractical due to patient numbers.
  • Dynamic assessment of liver function is a pragmatic alternative endpoint for early studies.
  • Stratification by disease severity is crucial for balanced patient distribution.

Conclusions:

  • Alternative endpoints, specifically dynamic liver function assessment, are recommended for early-phase alcoholic hepatitis trials.
  • Stratification by disease severity is essential for robust trial outcomes.
  • Consensus on early-phase trial design is needed to advance therapeutic development for alcoholic hepatitis.
Keywords:
ABICGAHSInfectionMELDSurrogate endpoints

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