Phase 3 Study of Obeticholic Acid in Patients With Primary Biliary Cirrhosis

NCT01473524 · POISE Phase 3Completed

Lead sponsor: Intercept Pharmaceuticals (Industry)
Enrollment: 217 (actual)
Start date: 1 Jan 2012
Primary completion: Dec 2013 (actual)
Study completion: 17 Dec 2018
Last update posted: 6 May 2021

Design

Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel
Masking: Quadruple
Arms: 4

Interventions

Obeticholic Acid (OCA) Drug
Placebo Drug

Conditions and primary outcome

Primary Biliary Cirrhosis

Primary outcome

DB Phase: Composite Endpoint Alkaline Phosphatase (ALP) And Total Bilirubin, 10 mg OCA Versus Placebo (DB Month 12); LTSE Phase: Composite Endpoint ALP And Total Bilirubin (Baseline (DB Month 12), LTSE Months 24, 36, 48, and 60)

Posted results on ClinicalTrials.gov

Source: ClinicalTrials.gov results for NCT01473524 (opens in a new tab)· Licence: public-domain

First posted 13 Feb 2017 · Last update posted 6 May 2021 · Imported 26 Oct 2026 from ClinicalTrials.gov download 2026-10-25

Primary outcomes with a statistical analysis

DB Phase: Composite Endpoint Alkaline Phosphatase (ALP) And Total Bilirubin, 10 mg OCA Versus Placebo
Time frame
DB Month 12
Measure
Number
Unit
percentage of participants
- DB OCA 10 mg vs DB PlaceboSuperiority or other
  H0: The response rates are equal between placebo and 10 mg OCA. H1: The response rates are different between placebo and 10 mg OCA.
  Estimate
  Not given
  CI, two-sided
  Not given
  p-value
  <0.0001
  Method
  Cochran-Mantel-Haenszel

Serious adverse events

Time frame: DB Phase: Baseline up to 12 months (1 year). LTSE phase: Baseline (DB Month 12) up to 60 months (5 years).

Participants with serious adverse events per group for POISE, as posted
Group	Affected	At risk
DB 5-10 mg	11	70
DB OCA 10 mg	8	73
DB Placebo	3	73
LTSE OCA (DB OCA 5-10 mg)	30	63
LTSE OCA (DB 10 mg)	19	64
LTSE OCA (DB Placebo)	20	66
Overall LTSE OCA	69	193

Posted results as ClinicalTrials.gov prints them: primary outcome analyses and serious adverse event totals only. Registry facts, not reviewed by HealthTrade and not investment advice; filing-derived results are listed separately.

Source: ClinicalTrials.gov, U.S. National Library of Medicine. Listing a study does not mean NLM has evaluated it.

Results from company and journal sources

No editor-confirmed results are on record for this study.

Publications

6 on PubMed

Long-Term Safety and Efficacy of Obeticholic Acid in Patients With Primary Biliary Cholangitis: Final Results of the POISE Long-Term Safety Extension.
Alimentary pharmacology & therapeutics · 2026 Oct
PubMed 42449190 (opens in a new tab)DOI 10.1111/apt.70832 (opens in a new tab)
Obeticholic acid is associated with improvements in AST-to-platelet ratio index and GLOBE score in patients with primary biliary cholangitis.
JHEP reports : innovation in hepatology · 2021 Feb
PubMed 33319187 (opens in a new tab)DOI 10.1016/j.jhepr.2020.100191 (opens in a new tab)
Long-Term Obeticholic Acid Therapy Improves Histological Endpoints in Patients With Primary Biliary Cholangitis.
Clinical gastroenterology and hepatology : the official clinical practice journal of the American Gastroenterological Association · 2020 May
PubMed 31606455 (opens in a new tab)DOI 10.1016/j.cgh.2019.09.050 (opens in a new tab)
Long-term efficacy and safety of obeticholic acid for patients with primary biliary cholangitis: 3-year results of an international open-label extension study.
The lancet. Gastroenterology & hepatology · 2019 Jun
PubMed 30922873 (opens in a new tab)DOI 10.1016/S2468-1253(19)30094-9 (opens in a new tab)
A Placebo-Controlled Trial of Obeticholic Acid in Primary Biliary Cholangitis.
The New England journal of medicine · 2016 Aug 18
PubMed 27532829 (opens in a new tab)DOI 10.1056/NEJMoa1509840 (opens in a new tab)
Advances in pharmacotherapy for primary biliary cirrhosis.
Expert opinion on pharmacotherapy · 2015 Apr
PubMed 25543678 (opens in a new tab)DOI 10.1517/14656566.2015.998650 (opens in a new tab)

Citations from PubMed, U.S. National Library of Medicine; abstracts are not stored. A publication lists this study’s NCT number and may be a review that cites many studies.

CT-VCA trial quality

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Data integrity

Compare what the registry, company releases, FDA documents and publications say about this study.

Drugs on HealthTrade

Obeticholic acid

Source

NCT01473524 on ClinicalTrials.gov (opens in a new tab) Licence: public-domain. Read 25 Oct 2026

Source: ClinicalTrials.gov, U.S. National Library of Medicine. Listing a study does not mean NLM has evaluated it.

Nothing on this page is medical or investment advice.