A study of OCA in Patients with Primary Biliary Cirrhosis (747-301)
Research type
Research Study
Full title
A Phase 3,Double Blind, Placebo Controlled Trial and Long Term Safety Extension of Obeticholic Acid in Patients with Primary Biliary Cirrhosis
IRAS ID
95694
Contact name
David Jones
Sponsor organisation
Intercept Pharmaceuticals, Inc.
Eudract number
2011-004728-36
Clinicaltrials.gov Identifier
Duration of Study in the UK
7 years, 0 months, 0 days
Research summary
Primary biliary cirrhosis (PBC) is a rare, kind of cirrhosis caused when the body’s own immune system damages the bile ducts in the liver. PBC permanently damages the liver and healthy tissue is replaced with scar tissue (fibrosis). As more scar tissue develops, the structure and function of the liver are affected and it can result in complications such as liver cancer, bleeding in the veins in the food pipe and liver failure. The only drug approved to treat PBC is ursodeoxycholic acid (UDCA). However, approximately 30 -40% of patients fail to see any improvement when treated with UDCA.
The new drug is obeticholic acid (OCA) and it has been studied in 2 trials in patients with PBC and has been shown to reduce the levels of important markers of PBC disease. This study aims to confirm these effects over a longer duration of treatment.
This trial will evaluate the effectiveness and safety of OCA in 180 patients with PBC. These will be either patients who did not see an improvement when treated with UDCA or patients cannot tolerate UDCA (monotherapy).
This study has 2 parts:
(1) the randomised, double blind (DB), placebo controlled study (neither patients nor doctor will know study treatment administered), testing 2 doses of OCA (5 and 10 mg) and placebo (dummy drug) administered once daily for 12 months.
(2) the long term safety extension where all patients will receive the study drug, OCA, at doses up to 25 mg for 5 years.
Patients will visit the clinic approximately every 3 months and will provide blood and urine samples and complete questionnaires about their disease. Patients may also have a scan of their liver every 6 months and a scan of their bones every 12 months.
The study will be conducted at up to 80 international trial sites who are experienced in treating patients with PBC.REC name
North East - Newcastle & North Tyneside 1 Research Ethics Committee
REC reference
12/NE/0004
Date of REC Opinion
21 Feb 2012
REC opinion
Further Information Favourable Opinion