Enrollment, Enrichment, and Monitoring Strategies to Accelerate Phase 3 Clinical Trials in NASH

Date: 28 February 2020

Time: 12 pm EST | 9 am PST | 6 pm CET

Non-alcoholic steatohepatitis (NASH) is widely recognized as a challenging disease for drug development. Phase 3 clinical trials require a substantial investment of time and money, and failure can cause costly delays or trial cancellation, wasting resources. Lack of cost-effective patient enrollment methods and the inability to demonstrate efficacy have been cited as the primary obstacles to conducting phase 3 clinical trials. Getting patients enrolled in NASH clinical trials has been challenging as endpoints still require an invasive biopsy and patients are reluctant to have multiple biopsies. As the race to bring the first NASH treatment to market heats up, sponsors will need to rely on cost-effective, non-invasive solutions not just to accelerate patient recruitment, but also to determine their response to treatment.

Key Topics Covered:

  • Non-invasive imaging and wet biomarkers
    • What are the most successful non-invasive biomarkers that are currently available in NASH clinical trials?
    • Which biomarkers have the most clinical utility to assess metabolic, inflammatory or fibrotic changes as a result of NASH treatment?
  • Enrollment and enrichment strategies
    • How can non-invasive biomarkers be applied to pre-screen patients for enrollment in NASH trials and reduce screen fails?
      • Differentiate patients with simple fatty liver and those with NASH 
      • Differentiate low- and high-risk NASH (e.g. NAS>3, F>1) patients 
      • Differentiate cirrhotic and non-cirrhotic NASH patients
  • Monitoring treatment efficacy
    • How to incorporate non-invasive biomarkers for early efficacy signaling?
    • How can non-invasive biomarkers be leveraged to monitor trial participants on a long-term basis?