Real-World Data Study to Evaluate the Effectiveness of OCA on Hepatic Outcomes in PBC Patients

NCT05292872 | Completed Results

• 1 other identifier: 747-405
• Study Type: observational
• Target: 4,577
• Locations: 1 country
• Sites: 1

Brief Summary

This is an observational, retrospective cohort study of patients with primary biliary cholangitis (PBC) who failed ursodeoxycholic acid (UDCA) treatment, using a real-world data source, the Komodo Health United States (US) claims database. The study is designed to evaluate the effectiveness of obeticholic acid (OCA). All patients who meet diagnostic criteria for PBC in the database between 01 Jun 2015 and 31 Dec 2021 and who meet all eligibility criteria were considered for this study.

Conditions

Primary Biliary Cholangitis

Keywords

Primary Biliary Cholangitis; Primary Biliary Cirrhosis; PBC; Hepatic Impairment; Liver

Outcome Measures

Primary Outcomes (1)

• Risk of the First Event of the Composite Events

  The primary analysis outcome was assessed with hazard ratio (HR) comparing hazard of first event of the composite endpoint among OCA-treated participants and SMR-weighted non-OCA-treated PBC participants indexes. It included all-cause death, liver transplant, hospitalization for hepatic decompensation based on first occurrence of: variceal bleed, ascites (including hepatic hydrothorax and spontaneous bacterial peritonitis) and hepatic encephalopathy. OCA-treated indexes were censored 90 days after OCA discontinuation, or if fibrates were initiated. Control indexes were censored if a participant-initiated OCA therapy, initiated fibrate therapy, reinitiated UDCA for participants who had discontinued UDCA for \>6 months, or end of study period (31 Dec 2021), whichever came first. The 2.5th and 97.5th percentile of nonparametric bootstrap samples were used to estimate 95% CI for HR and to perform a test of hypothesis. Risk is presented using the number of composite event and components.

  Up to 67 months

Secondary Outcomes (3)

• Risk of Death

  Up to 67 months

• Risk of Liver Transplantation

  Up to 67 months

• Risk of Hospitalization for Hepatic Decompensation

  Up to 67 months

Study Arms (2)

OCA-treated

PBC participants with a history of UDCA failure (inadequate response, intolerance, or discontinuation) who initiated Obeticholic acid (OCA) in the study window.

Non-OCA Treated

PBC participants with a history of UDCA failure who were eligible but not treated with OCA (or off-label fibrates) in the study window.

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

All patients who meet diagnostic criteria in each database between 01 Jun 2015 and 31 Dec 2021 and who meet the eligibility criteria were considered for these studies.

You may qualify if:

• Definite or probable PBC diagnosis
• Inadequate response or intolerance to UDCA
• Age ≥18 years at the index date
• Continuous enrollment and evaluable data for at least 12 months before the index date (inclusive)

You may not qualify if:

• History or presence of other concomitant liver diseases
• History of non-skin malignancy or melanoma
• History of HIV
• Medical conditions that may cause non-hepatic increases in ALP
• Patients with laboratory values indicative of hepatic decompensation or significant hepatobiliary injury
• History of liver transplant
• Evidence of fenofibrate, or bezafibrate use
• History or presence of hepatic decompensating events

Sponsors & Collaborators

• Intercept Pharmaceuticals: lead
• Target RWE: collaborator
• Syneos Health: collaborator
• Komodo Health, Inc.: collaborator

Study Sites (1)

Intercept Pharmaceuticals, Inc

San Diego, California, 92121, United States

Location

Related Publications (1)

• Brookhart MA, Mayne TJ, Coombs C, Breskin A, Ness E, Bessonova L, Chu YJ, Li J, Fried MW, Hansen BE, Kowdley KV, Jones D, Mells G, Trivedi PJ, Hiu S, Kareithi DN, Wason J, Smith R, Seeger JD, Hirschfield GM. Hepatic real-world outcomes with obeticholic acid in primary biliary cholangitis (HEROES): A trial emulation study design. Hepatology. 2025 Jun 1;81(6):1647-1659. doi: 10.1097/HEP.0000000000001174. Epub 2025 Jan 3.

  PMID: 39630028 DERIVED

MeSH Terms

Conditions

Liver Cirrhosis, Biliary

Condition Hierarchy (Ancestors)

Cholestasis, Intrahepatic; Cholestasis; Bile Duct Diseases; Biliary Tract Diseases; Digestive System Diseases; Liver Diseases; Liver Cirrhosis; Fibrosis; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Limitations and Caveats

This is an observational real-world data study to evaluate the effectiveness of OCA on hepatic outcomes in participants with PBC and the study did not plan to evaluate safety endpoints.

Results Point of Contact

• Title: Medical Information
• Organization: Intercept Pharmaceuticals, Inc.

medinfo@interceptpharma.com

844-782-4278

Study Officials

• Lynda Szczech, MD

  Intercept Pharmaceuticals

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: RETROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 4, 2022
• First Posted: March 23, 2022
• Study Start: June 1, 2015
• Primary Completion: December 31, 2021
• Study Completion: December 31, 2021
• Last Updated: January 24, 2025
• Results First Posted: January 24, 2025

Record last verified: 2024-12

Locations

US (1)