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

Study Evaluating the Effectiveness of Obeticholic Acid on Hepatic Real-World Outcomes in Patients With Primary Biliary Cholangitis

This is an observational, retrospective cohort study, using the UK PBC registry, comparing patients with primary biliary cholangitis (PBC) who failed ursodeoxycholic acid (UDCA) treatment and were treated with obeticholic acid (OCA) to patients with PBC who failed UDCA treatment and were not treated with second-line therapy. The study is designed to evaluate the effectiveness of 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 will be considered for this study.

Study Overview

• Status: Withdrawn
• Conditions: Primary Biliary Cholangitis
• Intervention / Treatment: Drug: Obeticholic Acid 5 MG; Drug: Obeticholic Acid 10 MG; Drug: Standard of Care: UDCA

• Study Type: Observational

Contacts and Locations

Study Locations

• United States
  • California
    • San Diego, California, United States, 92129
      • Intercept Pharmaceuticals, Inc

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 16 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

All patients who meet diagnostic criteria in UK PBC registry between 01 Jun 2015 and 31 Dec 2021 and who meet the eligibility criteria will be considered for the study.

Description

Key Inclusion Criteria:

1. Definite or probable PBC diagnosis
2. UDCA failure
3. Age ≥18 years at the index date
4. Evaluable data for at least 12 months before the index date (inclusive)

Key Exclusion Criteria:

1. History or presence of other concomitant liver diseases
2. Patients with laboratory values indicative of hepatic decompensation or significant hepatobiliary injury
3. History of liver transplant
4. Evidence of OCA, fenofibrate, or bezafibrate use
5. History or presence of hepatic decompensating events
6. Participation in a clinical trial for a PBC medication

Study Plan

How is the study designed?

Design Details

• Observational Models: Cohort
• Time Perspectives: Retrospective

Number of groups / cohorts

2

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
OCA Treatment Group; PBC patients with a history of UDCA failure who initiated OCA in the study window (01 Jun 2015 to 31 Dec 2021)	Drug: Obeticholic Acid 5 MG; once daily, oral administration; Other Names: Ocaliva; Drug: Obeticholic Acid 10 MG; once daily, oral administration; Other Names: Ocaliva
Control Group; PBC patients with a history of UDCA failure who were eligible but were not treated with OCA (or off-label fibrates) in the study window (01 Jun 2015 to 31 Dec 2021)	Drug: Standard of Care: UDCA; No study medication is provided by the sponsor or by the investigators. The decision to initiate, continue or discontinue UDCA, or to modify UDCA dosing, is entirely at the discretion of the treating physician as per their standard of care and is in no way influenced by the sponsor or participating institutions. UDCA utilization is recorded and included in the study record.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Time to the first occurrence of the composite endpoint of all-cause death, liver transplant, or hepatic decompensation.	Time from index date to first occurrence of the composite endpoint events, assessed up to 62 months.

Other Outcome Measures

Outcome Measure	Time Frame
Time to the first occurrence of all-cause death	Time from index date to first occurrence of all-cause death, assessed up to 62 months.
Time to the first occurrence of liver transplant	Time from index date to first occurrence of liver transplant, assessed up to 62 months.
Time to first occurrence of hepatic decompensation	Time from index date to first occurrence of hospitalization for hepatic decompensation, assessed up to 62 months.

Collaborators and Investigators

• Sponsor: Intercept Pharmaceuticals
• Collaborators: Syneos Health; Global PBC Study Group; Target RWE; UK PBC Study Group
• Investigators: Study Director: Lynda Szczech, MD, Intercept Pharmaceuticals

Study record dates

Study Major Dates

• Study Start (Actual): March 28, 2022
• Primary Completion (Estimated): June 1, 2023
• Study Completion (Estimated): July 1, 2023

Study Registration Dates

• First Submitted: March 4, 2022
• First Submitted That Met QC Criteria: March 14, 2022
• First Posted (Actual): March 24, 2022

Study Record Updates

• Last Update Posted (Estimated): October 9, 2023
• Last Update Submitted That Met QC Criteria: October 5, 2023
• Last Verified: October 1, 2023

More Information

Terms related to this study

• Keywords: Hepatic Impairment; Liver; Primary Biliary Cholangitis; PBC; Primary Biliary Cirrhosis
• Additional Relevant MeSH Terms: Digestive System Diseases; Pathologic Processes; Liver Diseases; Fibrosis; Biliary Tract Diseases; Bile Duct Diseases; Cholestasis, Intrahepatic; Cholestasis; Liver Cirrhosis; Cholangitis; Liver Cirrhosis, Biliary; Gastrointestinal Agents; Cathartics; Chenodeoxycholic Acid
• Other Study ID Numbers: 747-404

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No