Primary Biliary CholangitisHEROES PBCNCT05292872
Real-World Data Study to Evaluate the Effectiveness of OCA on Hepatic Outcomes in PBC Patients
An observational study of Primary Biliary Cholangitis, run by Intercept Pharmaceuticals at 1 site in United States.
Trial summary
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). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Primary Biliary Cholangitis
- How many people
- 4,577 participants
- Where
- 1 siteUnited States
- Study dates
- 1 Jun 2015to 31 Dec 2021
- Phase
- Observational
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
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.
Who may be able to join
- 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)
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country.
United States1 site
Intercept Pharmaceuticals, Inc
San Diego, California, 92121
About this study
Official title: Replicate Studies Evaluating the Effectiveness of Obeticholic Acid on Hepatic Real-World Outcomes in Patients With Primary Biliary Cholangitis
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.
- Conditions
- Primary Biliary Cholangitis
- Keywords
- Primary Biliary Cholangitis, Primary Biliary Cirrhosis, PBC, Hepatic Impairment, Liver
- Medical subject headings
- Liver Cirrhosis, Biliary
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| 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. |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: Up to 67 months
Secondary outcomes (3)
Risk of Death
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).
Measured: Up to 67 months
Risk of Liver Transplantation
The primary source for liver transplant was the Organ Transplant Network (OPTN) transplant registry. Risk is presented using the number of liver transplantation.
Measured: Up to 67 months
Risk of Hospitalization for Hepatic Decompensation
The primary source for hospitalization for hepatic decompensation were Komodo Health claims. Risk is presented using the number of hospitalization for hepatic decompensation.
Measured: Up to 67 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Mar 2022
- Study start1 Jun 2015
- Primary completion31 Dec 2021
- Study completion31 Dec 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Primary Biliary CholangitisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Retrospective
- Enrolment
- 4,577 participants (actual)
Sponsor and investigators
- Lead sponsor
- Intercept Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Target RWE, Syneos Health, Komodo Health, Inc.
- Responsible party
- Sponsor
- Investigators
- Lynda Szczech, MD, Study director, Intercept Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05292872
- Sponsor's protocol ID
- 747-405
- Acronym
- HEROES PBC
- Status last verified
- Dec 2024
- First submitted
- 4 Mar 2022
- Last updated
- 24 Jan 2025
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Publications
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.
PubMed 39630028
Study at a glance
From the registry record, last updated by the sponsor on 24 Jan 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Replicate Studies Evaluating the Effectiveness of Obeticholic Acid on Hepatic Real-World Outcomes in Patients With Primary Biliary Cholangitis
- ClinicalTrials.gov ID
- NCT05292872
- Acronym / sponsor ID
- HEROES PBC / 747-405
- Conditions
- Primary Biliary Cholangitis
- Keywords
- Primary Biliary Cholangitis, Primary Biliary Cirrhosis, PBC, Hepatic Impairment, Liver
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 4,577 participants (actual)
- Lead sponsor
- Intercept PharmaceuticalsIndustry (pharma, biotech, devices)
- Collaborators
- Target RWE, Syneos Health, Komodo Health, Inc.
- Study start
- 1 Jun 2015 (actual)
- Primary completion
- 31 Dec 2021 (actual)
- Study completion
- 31 Dec 2021 (actual)
- First posted
- 23 Mar 2022 (actual)
- Results
- Posted 24 Jan 2025
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Cohort
- Time perspective
- Retrospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| 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. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- 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.
Key inclusion criteria (4 listed)
- 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 (8 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
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.
3 secondary outcomes
Risk of Death
Time frame: Up to 67 months
Risk of Liver Transplantation
Time frame: Up to 67 months
Risk of Hospitalization for Hepatic Decompensation
Time frame: Up to 67 months
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Jan 2025.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 39630028Brookhart 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Lynda Szczech, MD study director
Intercept Pharmaceuticals
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Primary Biliary Cholangitis, run by Intercept Pharmaceuticals at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 24 Jan 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Yes. Results are posted in the registry (first posted 24 Jan 2025). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Risk of the First Event of the Composite Events (time frame: Up to 67 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT05292872, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 24 Jan 2025.