drugset / Trial / NCT01473524
Phase 3 Study of Obeticholic Acid in Patients With Primary Biliary Cirrhosis
RandomizedParallel-groupQuadruple-blindTreatment
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).
Timeline
- Start
- 2012-01
- Primary completion
- 2013-12
- Completion
- 2018-12-17
Outcome
Met primary endpoint
paper The primary end point occurred in more patients in the 5-10-mg group (46%) and the 10-mg group (47%) PMID 27532829 ↗
paper 10-mg group (47%) than in the placebo group (10%; P<0.001 for both comparisons). PMID 27532829 ↗
registry DB OCA 10 mg vs DB Placebo; p <0.0001; Cochran-Mantel-Haenszel NCT01473524 ↗
Publications
- Background Lindor KD, Gershwin ME, Poupon R, Kaplan M, Bergasa NV, Heathcote EJ; American Association for Study of Liver Diseases. Primary biliary cirrhosis. Hepatology. 2009 Jul;50(1):291-308. doi: 10.1002/hep.22906. No abstract available.
- Results Nevens F, Andreone P, Mazzella G, Strasser SI, Bowlus C, Invernizzi P, Drenth JP, Pockros PJ, Regula J, Beuers U, Trauner M, Jones DE, Floreani A, Hohenester S, Luketic V, Shiffman M, van Erpecum KJ, Vargas V, Vincent C, Hirschfield GM, Shah H, Hansen B, Lindor KD, Marschall HU, Kowdley KV, Hooshmand-Rad R, Marmon T, Sheeron S, Pencek R, MacConell L, Pruzanski M, Shapiro D; POISE Study Group. A Placebo-Controlled Trial of Obeticholic Acid in Primary Biliary Cholangitis. N Engl J Med. 2016 Aug 18;375(7):631-43. doi: 10.1056/NEJMoa1509840.
- Results Trauner M, Nevens F, Shiffman ML, Drenth JPH, Bowlus CL, Vargas V, Andreone P, Hirschfield GM, Pencek R, Malecha ES, MacConell L, Shapiro D. Long-term efficacy and safety of obeticholic acid for patients with primary biliary cholangitis: 3-year results of an international open-label extension study. Lancet Gastroenterol Hepatol. 2019 Jun;4(6):445-453. doi: 10.1016/S2468-1253(19)30094-9. Epub 2019 Mar 26.
- Bowlus CL, Nevens F, Kowdley KV, Bonder A, Capozza T, Li J, Nair R, Trauner M, Jones DE. Long-Term Safety and Efficacy of Obeticholic Acid in Patients With Primary Biliary Cholangitis: Final Results of the POISE Long-Term Safety Extension. Aliment Pharmacol Ther. 2026 Jul 14. doi: 10.1111/apt.70832. Online ahead of print.
- Harms MH, Hirschfield GM, Floreani A, Mayo MJ, Pares A, Liberman A, Malecha ES, Pencek R, MacConell L, Hansen BE. Obeticholic acid is associated with improvements in AST-to-platelet ratio index and GLOBE score in patients with primary biliary cholangitis. JHEP Rep. 2020 Sep 29;3(1):100191. doi: 10.1016/j.jhepr.2020.100191. eCollection 2021 Feb.
- Bowlus CL, Pockros PJ, Kremer AE, Pares A, Forman LM, Drenth JPH, Ryder SD, Terracciano L, Jin Y, Liberman A, Pencek R, Iloeje U, MacConell L, Bedossa P. Long-Term Obeticholic Acid Therapy Improves Histological Endpoints in Patients With Primary Biliary Cholangitis. Clin Gastroenterol Hepatol. 2020 May;18(5):1170-1178.e6. doi: 10.1016/j.cgh.2019.09.050. Epub 2019 Oct 10.
- Mousa HS, Lleo A, Invernizzi P, Bowlus CL, Gershwin ME. Advances in pharmacotherapy for primary biliary cirrhosis. Expert Opin Pharmacother. 2015 Apr;16(5):633-43. doi: 10.1517/14656566.2015.998650. Epub 2014 Dec 29.
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Obeticholic Acid | Small molecule | 5 mg | Oral |
| Subject | Obeticholic Acid | Small molecule | 10 mg | Oral |