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

NCT05293938 | Withdrawn FDA Drug

• 1 other identifier: 747-404
• Study Type: observational
• Target: N/A
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Primary Biliary Cholangitis

Keywords

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

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (3)

• 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.

Study Arms (2)

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; Drug: Obeticholic Acid 10 MG

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

Interventions

Obeticholic Acid 5 MG DRUG

once daily, oral administration

Also known as: Ocaliva

OCA Treatment Group

Obeticholic Acid 10 MG DRUG

once daily, oral administration

Also known as: Ocaliva

OCA Treatment Group

Standard of Care: UDCA DRUG

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.

Control Group

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

You may qualify if:

• Definite or probable PBC diagnosis
• UDCA failure
• Age ≥18 years at the index date
• 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
• Patients with laboratory values indicative of hepatic decompensation or significant hepatobiliary injury
• History of liver transplant
• Evidence of OCA, fenofibrate, or bezafibrate use
• History or presence of hepatic decompensating events
• Participation in a clinical trial for a PBC medication

Sponsors & Collaborators

• Intercept Pharmaceuticals: lead
• Global PBC Study Group: collaborator
• Target RWE: collaborator
• Syneos Health: collaborator
• UK PBC Study Group: collaborator

Study Sites (1)

Intercept Pharmaceuticals, Inc

San Diego, California, 92129, United States

Location

MeSH Terms

Conditions

Liver Cirrhosis, Biliary

Interventions

obeticholic acid

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

Study Officials

• Lynda Szczech, MD

  Intercept Pharmaceuticals

  STUDY DIRECTOR

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 24, 2022
• Study Start: March 28, 2022
• Primary Completion: June 1, 2023
• Study Completion: July 1, 2023
• Last Updated: October 10, 2023

Record last verified: 2023-10

Locations

US (1)