Clinical trial NCT05293938: A Real-World Data Study to Evaluate the Effectiveness of OCA on Hepatic Outcomes in PBC Patients
Official title: Study Evaluating the Effectiveness of Obeticholic Acid on Hepatic Real-World Outcomes in Patients With Primary Biliary Cholangitis
Study type: OBSERVATIONAL
Overall status: WITHDRAWN
Conditions: Primary Biliary Cholangitis
Keywords: Primary Biliary Cholangitis
Primary Biliary Cirrhosis
PBC
Hepatic Impairment
Liver
Lead sponsor: Intercept Pharmaceuticals
Sponsor class: INDUSTRY
Collaborators: name: Global PBC Study Group
name: Target RWE
name: Syneos Health
name: UK PBC Study Group
First posted: 2022-03-24
Enrollment: 0
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: RETROSPECTIVE
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.

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.


Eligibility criteria: 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

Primary outcomes:
measure: Time to the first occurrence of the composite endpoint of all-cause death, liver transplant, or hepatic decompensation.
timeFrame: Time from index date to first occurrence of the composite endpoint events, assessed up to 62 months.
Secondary outcomes:
Other outcomes:
measure: Time to the first occurrence of all-cause death
timeFrame: Time from index date to first occurrence of all-cause death, assessed up to 62 months.
measure: Time to the first occurrence of liver transplant
timeFrame: Time from index date to first occurrence of liver transplant, assessed up to 62 months.
measure: Time to first occurrence of hepatic decompensation
timeFrame: Time from index date to first occurrence of hospitalization for hepatic decompensation, assessed up to 62 months.
Locations:
city: San Diego
country: United States
facility: Intercept Pharmaceuticals, Inc
state: California
Study officials:
affiliation: Intercept Pharmaceuticals
name: Lynda Szczech, MD
role: STUDY_DIRECTOR
References: