Trials Sponsors Conditions Drugs Pricing

Conditions

Primary Biliary Cholangitis

Keywords

Primary Biliary CholangitisPrimary Biliary CirrhosisPBCHepatic ImpairmentLiver

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

Age18 Years+

Sexall

Healthy VolunteersNo

Age GroupsAdult (18-64), Older Adult (65+)

Sampling MethodNon-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

Sponsors & Collaborators

Komodo Health, Inc.
Industrycollaborator
Target RWE
Industrycollaborator
Intercept Pharmaceuticals

Study Sites (1)

Intercept Pharmaceuticals, Inc

San Diego, California, 92121, United States

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: 39630028DERIVED

MeSH Terms

Conditions

Liver Cirrhosis, Biliary

Condition Hierarchy (Ancestors)

Cholestasis, IntrahepaticCholestasisBile Duct DiseasesBiliary Tract DiseasesDigestive System DiseasesLiver DiseasesLiver CirrhosisFibrosisPathologic ProcessesPathological 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.

Study Officials

Lynda Szczech, MD
Intercept Pharmaceuticals
STUDY DIRECTOR

Publication Agreements

PI is Sponsor Employee: No
Restrictive Agreement: No

Enrollment

4,577 participants (actual enrollment reported by the registry).

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

Actual (reported)

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

Brief Summary

Registry Overview

Trial Relationships

Study Timeline

Study Start

Primary Completion

Study Completion

First Submitted

First Posted

Results Posted

Conditions

Keywords

Outcome Measures

Primary Outcomes (1)

Secondary Outcomes (3)

Study Arms (2)

OCA-treated

Non-OCA Treated

Eligibility Criteria

You may qualify if:

Sponsors & Collaborators

Study Sites (1)

Related Publications (1)

MeSH Terms

Conditions

Condition Hierarchy (Ancestors)

Limitations and Caveats

Results Point of Contact

Study Officials

Publication Agreements

Enrollment

Study Design

Study Record Dates

You may not qualify if:

Source record

Documents

Locations

Brief Summary

Registry Overview

Trial Relationships

Related Scientific Literature

Study Timeline

Study Start

Primary Completion

Study Completion

First Submitted

First Posted

Results Posted

Conditions

Keywords

Outcome Measures

Primary Outcomes (1)

Secondary Outcomes (3)

Study Arms (2)

OCA-treated

Non-OCA Treated

Eligibility Criteria

You may qualify if:

Sponsors & Collaborators

Study Sites (1)

Related Publications (1)

MeSH Terms

Conditions

Condition Hierarchy (Ancestors)

Limitations and Caveats

Results Point of Contact

Study Officials

Publication Agreements

Enrollment

Study Design

Study Record Dates

You may not qualify if:

Source record

Documents

Locations