INT-747-202 Trial in Primary Biliary Cirrhosis
A Study of INT 747 (6-ECDCA) in Combination with Ursodeoxycholic Acid (URSO®, UDCA) in Patients with Primary Biliary Cirrhosis
Intercept Pharmaceuticals (Dr David Shapiro)
Primary Biliary Cirrhosis (PBC) is a chronic liver disease (not associated with alcohol abuse) that arises in patients who generate an autoimmune response that damages their liver biliary cells. This damage leads to bile duct obstruction, destruction of the bile ducts, inflammmation and thickening of the liver and, eventually, liver failure. Patients with liver failure will die, unless they receive a liver transplant. This study intends to examine the effect of a new drug INT-747 in patients with (PBC), used in combination with the current best therapy Urso ((the bile acid ursodeoxycholic acid). INT-747 is a new drug, a bile acid derivative, that it is thought will improve bile flow in patients with PBC. The primary research questions in this study are: What is the effect of INT-747, at three dose levels (10 mg, 25 mg and 50 mg), when used in combination with Urso in patients with proven or likely PBC, on levels of alkaline phosphatase (an indicator, obtained from a blood sample, of the level of liver inflammation)? How safe is INT-747, at three dose levels (10 mg, 25 mg and 50 mg), when used in combination with Urso in patients with proven or likely PBC? What side effects do the people taking this drug experience, and how serious are those side effects? What is the effect of INT-747, at three dose levels (10 mg, 25 mg and 50 mg), when used in combination with Urso in patients with proven or likely PBC, on: patients?? liver function and on damage to liver cells; on inflammation and thickening of the liver, as measured through indicators appearing in the patients?? blood; on on patients?? symptoms of PBC and on general and liver-specific health-related quality of life? Patients with proven or likely PBC will be allocated at random to one of three doses of INT-747, or to a placebo (a dummy treatment), administered once daily in combination with Urso for 12 weeks. The efficacy and safety will be measured to answer the questions listed above.
North East - Newcastle & North Tyneside 1 Research Ethics Committee
Date of REC Opinion
13 Oct 2008
Further Information Favourable Opinion