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Completed

NCT Number: NCT05292872

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

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.

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Key information

Conditions

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

Age range

18 year and older

Sex eligibility

All sexes

Study type

Observational

Primary location

Intercept Pharmaceuticals, Inc

San Diego, California, 92121, United States

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Key Inclusion Criteria:

  • 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)

Key Exclusion Criteria:

  • 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

Treatment and study plan

Primary outcomes

  1. Risk of the First Event of the Composite Events

    Time frame: Up to 67 months

    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.

Secondary outcomes

  1. Risk of Death

    Time frame: Up to 67 months

    The primary source for death was the Social Security Death Index (SSDI) along with obituary search, which was compared to Komodo Health claims and LabCorp/Quest laboratory data. The secondary objectives were to estimate the effect of OCA treatment versus non-OCA treatment on each component of the composite endpoint, with the same censoring rule applied as in the primary composite endpoint. Risk is presented using the the number of all-cause death within the risk period (prior to censoring).

  2. Risk of Liver Transplantation

    Time frame: Up to 67 months

    The primary source for liver transplant was the Organ Transplant Network (OPTN) transplant registry. Risk is presented using the number of liver transplantation.

  3. Risk of Hospitalization for Hepatic Decompensation

    Time frame: Up to 67 months

    The primary source for hospitalization for hepatic decompensation were Komodo Health claims. Risk is presented using the number of hospitalization for hepatic decompensation.

Sponsors and collaborators

Lead sponsor

Intercept Pharmaceuticals

Industry

Collaborators

  • Komodo Health, Inc.
  • Syneos Health
  • Target RWE

Registry information

Official study title

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

Acronym: HEROES PBC

Important dates

Study start
2015
Primary completion
2021
Study completion
2021
First posted
Mar 23, 2022
Registry last updated
Jan 24, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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