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AASLD-FDA Workshop Discussion of NASH 

AASLD-FDA Workshop
Trial Designs and Endpoints for Liver Disease Secondary to Nonalcoholic Fatty Liver Disease (NAFLD)
Held: September 5 - 6

Course Syllabus [PDF]

This workshop is now available via LiverLearning®.

Course Directors
Arun J. Sanyal, MD; Arthur J. McCullough, MD; Scott L. Friedman, MD; and Lara Dimick-Santos, MD, FACS

Course Summary
This workshop will allow participants to increase the knowledge of the science of clinical trial design for fatty liver diseases and allow drug development to proceed.

Nonalcoholic Steatohepatitis (NASH) is rapidly becoming the leading cause for transplantation in the developed world. However, the FDA has not approved treatment for NAFLD/NASH or liver fibrosis and cirrhosis secondary to NASH. In addition, the lack of established guidelines for clinical trial design, population selection, and benefit endpoints for these diseases is stalling progress in drug development. Participants will discuss how to structure clinical trials to obtain evidence and answer questions about access to care and clinical management.

Learning Objectives:

  • Ability to design clinical trials for drugs that meet federal requirements and expectations
  • Identify appropriate populations for trials
  • Design trials with appropriate endpoints
  • Design trials that answer clinical management questions
  • Provide information for the FDA to use to develop policies regarding trial design and endpoints in drug and diagnostics development for NASH and fibrosis

 ‭(Hidden)‬ Custom List View Classes

 Thursday, September 5

Talk TitleFilter
Session 1: FDA Perspectives on Clinical Trial Designs and Natural History in NAFLD
Moderators: Arun J. Sanyal, MD and Scott L. Friedman, MD
8:00 am - 8:05 am
Introduction and Overview of Meeting
8:05 am - 8:25 am
FDA Perspectives: Key Needs in the Field to Guide Development in NASH and Fibrosis
Lara Dimick-Santos, MD, FACS
8:25 am - 8:50 am
Defining Clinical Outcomes in NAFLD for Adults and Children
Arthur J. McCullough, MD
8:50 am - 9:20 am
Rates of Fibrosis Progression/Regression in Varying Phenotypes of NAFLD and Confounding Variables that Affect It
David E. Kleiner, MD, PhD
9:20 am - 9:45 am
Open Forum and Panel Discussion
9:45 am - 10:00 am
Session 2: Treatment Trials in NASH (What Are the Options for Surrogate and Clinical Benefit Endpoints for NAFLD/NASH Trials?)
Moderators: Brent A. Tetri, MD and Lara Dimick-Santos, MD, FACS
10:00 am - 10:25 am
Defining Drug Efficacy for NASH: What are the Best Endpoints for Early Phase and Late Phase Studies in Adults - A Clinician's Perspective  

Arun J. Sanyal, MD
10:25 am - 10:45 am
Safety Related Outcomes in Treatment Trials for NAFLD
Naga P. Chalasani, MD
10:45 am - 11:10 am
Trial Design Considerations and Endpoints for Development of Diagnostics for Pediatric NAFLD
Joel E. Lavine, MD, PhD
11:10 am - 12:00 pm
Panel and Open Forum Discussion
12:00 pm - 1:00 pm
Session 3: Diagnostics in NASH (How Are Surrogate and Clinical Benefits Endpoints Measured?)
Moderators: Joel E. Lavine, MD, PhD, and Arthur J. McCullough, MD
1:00 pm - 1:25 pm
What are the Key Clinical Questions that Require Development of Diagnostic Tools?
Rohit Loomba, MD, MHSc
1:25 pm - 1:40 pm
Key Issues in Trial Design for Diagnostics for NASH
Brent A. Tetri, MD
1:40 pm - 2:00 pm
Evaluation of the State of Imaging Tests for Liver Fibrosis and Fat
Claude B. Sirlin, MD
2:00 pm - 2:20 pm
Safety Related Outcomes and Diagnostics in Treatment Trials for Pediatric NAFLD
Miriam B. Vos, MD, MSPH
2:20 pm - 3:00 pm
Open Forum and Panel Discussion
3:00 pm - 3:15 pm
Session 4: Clinical Trials for Advanced Fibrosis
Moderators: Arun J. Sanyal, MD and Don C. Rockey, MD
3:15 pm - 3:40 pm
Relating HVPG and Changes in HVPG to Clinical Outcomes in Cirrhosis -- Quality Metrics for Liver Histology and HVPG Measurement in Studies of Fibrosis
Guadalupe Garcia-Tsao, MD
3:40 pm - 4:00 pm
Stratification of Subjects for Anti-fibrotic Trials: Use of Genetic Markers, HVPG, Quantitative Liver Function Tests etc.
Scott L. Friedman, MD
4:00 pm - 4:20 pm
What is the Best Intermediate-Term Surrogate: (1-2 years) Endpoint for Treatment Trials with Fibrosis as a Therapeutic Target?
Arun J. Sanyal, MD
4:20 pm - 5:00 pm
Open Forum for Discussion
Adjourn Day One

 Friday, September 6

Talk TitleFilter
Session 1: Diagnostics in Fibrosis
Naga P. Chalasani, MD and Don C. Rockey, MD
8:00 am - 8:20 am
Elastography as a Way to Assess Changes in Fibrosis
Marc Ghany, MD, MHSc
8:20 am - 8:40 am
Quantitative Liver Function Tests and Their Utility to Assess Liver Related Outcomes
Gregory T. Everson, MD
8:40 am - 9:00 am
Strengths and Weaknesses of Measures of Fibrosis: Defining A Gold Standard For Diagnostics Development
Scott L. Friedman, MD
9:00 am - 9:30 am
Open Forum and Panel Discussion
9:30 am - 10:00 am
Session 2: Wrap - Up Discussions
10:00 am - 11:00 am
Trial Design, Population Prioritization and Endpoints for Diagnostics and Therapeutics for NASH and Advanced Fibrosis and Cirrhosis
Arthur J. McCullough, MD
11:00 am - 12:00 pm
Trial Design, Population Prioritization and Endpoints for Diagnostics and Therapeutics for Advanced Fibrosis and Cirrhosis
Don C. Rockey, MD
12:00 pm - 1:00 pm
1:00 pm - 2:00 pm
Safety Related Endpoints in NASH and Fibrosis/Cirrhosis Trials
Naga P. Chalasani, MD
2:00 pm - 2:30 pm
Wrap Up
Arun J. Sanyal, MD

This program is supported in part by educational grants from GENFIT Corporation and Gilead.