THE CLINICAL TRIAL
Primary biliary cholangitis (PBC) is a chronic liver disease caused by the destruction of the bile ducts in the liver. Bile is produced in the liver and travels through the bile ducts to the small intestine. When these ducts are damaged, bile builds in the liver and leads to inflammation, fibrosis, and cirrhosis. There is no cure for PBC. There are treatments available to slow the progression of liver damage, but they are not effective in all patients. This creates an unmet need for PBC patients. The TRANSFORM Trial is evaluating a secondary treatment for PBC and liver stiffness that, unlike current medications, is meant to specifically target the causes of inflammation and fibrosis.
With such a small study population who were often dealing with severe symptoms, study teams were facing some unique challenges with recruitment. PBC is a rare disease, affecting only up to 1 in 3,000 people. Of that small population, the clinical trial was only recruiting people with moderate-to-severe disease who had already undergone first or second line treatment. Our approach was focused on understanding the patient profile of PBC to develop a compassionate, educational study toolkit for both potential participants and site staff.