Phase 3 Study of Bezafibrate in Combination With Ursodeoxycholic Acid in Primary Biliary Cirrhosis
Status:
Completed
Trial end date:
2016-12-31
Target enrollment:
Participant gender:
Summary
Primary biliary cirrhosis (PBC) is a chronic cholestatic liver disease that eventually leads
to end-stage liver failure and death unless liver transplantation (LT) is performed.
Ursodeoxycholic acid (UDCA) administered orally at the daily dose of 13-15 mg/kg is currently
the only drug approved for the treatment of PBC. UDCA consistently improves biochemical liver
tests, prolongs survival without LT, and delays histological progression as well as the
occurrence of portal hypertension. However, a significant proportion (40%) of patients
treated with UDCA shows an incomplete biochemical response and remains at high risk of death
or LT. The development of new treatments in combination with UDCA is therefore needed.
Several candidates exist among which is Bezafibrate. Bezafibrate belongs to the fibrates'
pharmacological class, which has been developed 4 decades ago for the treatment of mixed
hyperlipidaemia. Bezafibrate is cheap, widely available and well tolerated. There is now a
substantial body of circumstantial evidence supporting that fibrates, and Bezafibrate in
particular, are well tolerated and can improve biochemical liver tests in patients with PBC
with incomplete response to UDCA. However, despite several positive successful pilot studies,
there are still no phase 3 randomized placebo-controlled trials of fibrates for the treatment
of PBC. The purpose of this protocol is therefore to conduct such a trial in a selected
population of patients with PBC based on an incomplete biochemical response after 6 months of
UDCA therapy.