Primary Biliary Cholangitis (PBC)

TARGET-PBC

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

In the U.S alone, studies and research has shown that an estimated 65 out of every 100,000 women and 12 out of every 100,000 men have primary biliary cholangitis. The exact cause of this disease is still unknown, according to the U.S. Department of Health and Human Services, and most individuals have no symptoms at all when they are first diagnosed. 

There has been incredible progress in the research of PBC, and the TARGET-PBC study aims to additionally contribute to this by generating  new real-world evidence and disease insights. TARGET-PBC is a 5-year longitudinal, observational study of patients with PBC designed to specifically address important clinical questions that remain incompletely answered from typical trials. TARGET offers partners unlimited access to query the TARGET-PBC database, which includes biorepository access to further enhance disease understandings and help identify appropriate treatment strategies. 

study goals:

  • 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

 

enrollment goal:

Up to 1,500

TARGET-PBC Steering Committee

Cynthia Levy, MD (Chair)
Assistant Director of Schiff Center for Liver Diseases & Program Director of the Transplant Hepatology Fellowship, Miami, FL

Christopher Bowlus, MD
Division Chief of Gastroenterology and Hepatology, Sacramento, CA

Elizabeth Carey, MD
Associate Professor of Medicine, Hepatology, Phoenix, AZ

Marlyn Mayo, MD
Associate Professor, Gastroenterology, Dallas, TX

W. Ray Kim, MD
Professor of Medicine, Gastroenterology and Hepatology, Stanford, CA