Phase 3 Study of Obeticholic Acid in Patients With Primary Biliary Cirrhosis
(POISE Trial)

Recruiting in Palo Alto (17 mi)

+58 other locations

Age: 18+

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 3

Waitlist Available

Sponsor: Intercept Pharmaceuticals

Pivotal Trial (Near Approval)

Prior Safety Data

Trial Summary

What is the purpose of this trial?

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).

Eligibility Criteria

Inclusion Criteria

History of elevated alkaline phosphatase (ALP) levels for at least 6 months

Positive antimitochondrial antibodies (AMA) titer or if AMA negative or in low titer (<1:80) PBC specific antibodies (anti-GP210 and/or anti-SP100 and/or antibodies against the major M2 components (pyruvate dehydrogenase complex-E2 [PDC-E2], 2-oxo-glutaric acid dehydrogenase complex)

Liver biopsy consistent with PBC

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Treatment Details

Interventions

Obeticholic Acid (OCA) (Farnesoid X receptor (FXR) agonist)
Placebo (Drug)

Participant Groups

4Treatment groups

Experimental Treatment

Placebo Group

Group I: LTSE OCAExperimental Treatment1 Intervention

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.

Group II: DB OCA 5-10 mgExperimental Treatment1 Intervention

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.

Group III: DB OCA 10 mgExperimental Treatment1 Intervention

OCA 10 mg for 12 months during the DB phase.

Group IV: DB PlaceboPlacebo Group1 Intervention

Matching placebo for 12 months during the DB phase.