Primary Biliary Cholangitis (PBC)

TARGET-PBC is currently enrolling


TARGET-PBC represents a unique opportunity in the study of rare diseases. By fostering collaborations between academic and community centers, TARGET-PBC will reveal the true natural history of the disease and the response to available therapies in various subgroups, many of which have been underrepresented in phase 3 trials. TARGET-PBC will answer crucial questions involving the safety and efficacy of approved and off-label therapies used in the real world and will identify at-risk populations in need of novel therapies.” – Cynthia Levy, MD

While there has been incredible progress in primary biliary cholangitis (PBC) research, there is still significant opportunity to better understand it in order to identify appropriate treatment strategies.  TARGET-PBC provides access to a biorepository linked to validated clinical outcomes, including patient reported outcomes, putting TARGET-PBC in a very unique place.  This offers partners a new platform to collect real world data to enhance their understanding of the natural history of PBC, providing better clinical outcomes and care for patients.

Initial Goals include: 

  • Evaluate PBC treatment regimens being used in clinical practice
  • Examine populations under-represented in phase II-III clinical trials
  • Examine biochemical response and its association with long-term outcomes
  • Estimate adverse event frequency and severity and describe management practices

Launched: October, 2016

Targeted number of patients: 1,500

TARGET-PBC Steering Committee

Cynthia Levy, MD 
Associate Professor of Medicine, Division of Gastroenterology and Hepatology, University of Miami

Chris Bowlus, MD
Division Chief of Gastroenterology and Hepatology, University of California-Davis

Elizabeth Carey, MD
Associate Professor of Medicine, Hepatology, Mayo Clinic

Marlyn Mayo, MD
Associate Professor, Gastroenterology, UT Southwestern

W.Ray Kim, MD
Professor of Medicine (Gastroenterology and Hepatology) Stanford University Medical Center