Conditions: Primary Biliary Cirrhosis
Keywords: Primary Biliary Cholangitis Primary Biliary Cirrhosis PBC Cirrhosis Liver
Allocation: RANDOMIZED | Intervention model: PARALLEL
Enrollment: 217
Healthy volunteers: no

Brief summary:
The main objectives of the study were to assess the effects of Obeticholic Acid (OCA) on serum alkaline phosphatase (ALP) and total bilirubin, together as a composite endpoint and on safety in participants with primary biliary cirrhosis (PBC).

Detailed description:
The study included 2 phases: a 12-month randomized, double-blind (DB), placebo-controlled, parallel group phase, followed by a long-term safety extension (LTSE) phase up to 5 years. Participants from the 12-month DB phase, including those who received placebo, were eligible to participate in the open-label LTSE phase. The Month 12 visit from the DB phase served as the Day 1 visit of the LTSE phase. After completion of the 12-month DB phase all participants were offered the opportunity to enter an open-label LTSE for up to 5 years beginning at 5 mg OCA.

Data for the LTSE phase is reported by the randomized dose group assigned in the DB phase.

Arms:
arm 1: OCA 5 milligram (mg) for 6 months and then titrating up to 10 mg based on tolerability and response for remaining 6 months of the DB phase. arm 2: OCA 10 mg for 12 months during the DB phase. arm 3: Matching placebo for 12 months during the DB phase. arm 4: After completion of the 12-month DB phase all participants were offered the opportunity to enter an open-label LTSE for up to 5 years beginning at 5 mg OCA. Initially, participants were allowed to titrate to doses up to 25 mg, however, the maximum dose was then limited to 10 mg. Participants who were previously titrated above 10 mg OCA daily were down-titrated to ≤10 mg OCA daily.

Interventions: intervention 1: Obeticholic Acid (OCA) intervention 2: Placebo
Intervention descriptions: intervention 1: OCA was administered orally once daily and provided in tablet form in 2 strengths: 5 mg and 10 mg. intervention 2: Matching placebo tablets were administered orally once daily.

Protocol document: