Primary Biliary CholangitisHEROES-PBCNCT05293938
A 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, 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… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Primary Biliary Cholangitis
- What is being tested?
- Obeticholic Acid 5 MG and Obeticholic Acid 10 MG
- Type of treatment
- Pill or oral medicine
- How many people
- 0 participants
- Where
- 1 siteUnited States
- Study dates
- 28 Mar 2022to Jul 2023
- Phase
- Observational
- Are there results?
- None posted in the registry
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 UK PBC registry between 01 Jun 2015 and 31 Dec 2021 and who meet the eligibility criteria will be considered for the study.
Who may be able to join
- 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)
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country.
United States1 site
Intercept Pharmaceuticals, Inc
San Diego, California, 92129
About this study
Official title: 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.
- Conditions
- Primary Biliary Cholangitis
- Being tested
- Obeticholic Acid 5 MG, Obeticholic Acid 10 MG, Standard of Care: UDCA
- 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.
Obeticholic Acid 5 MGDrug
Also called Ocaliva
once daily, oral administration
Obeticholic Acid 10 MGDrug
Also called Ocaliva
once daily, oral administration
Standard of Care: UDCADrug
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.
| Study group | Role | What they receive |
|---|---|---|
| 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) Receives: 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) Receives: Drug: Standard of Care: UDCA |
What is being measured
The primary outcome is the main question the study is designed to answer.
Time to the first occurrence of the composite endpoint of all-cause death, liver transplant, or hepatic decompensation.
Measured: Time from index date to first occurrence of the composite endpoint events, assessed up to 62 months.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Mar 2022
- Study start28 Mar 2022
- Primary completionJun 2023 (estimated)
- Study completionJul 2023 (estimated)
Why it stopped: A fundamental protocol deviation on data capture left the database no longer fit for purpose.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- 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
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Intercept Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Global PBC Study Group, Target RWE, Syneos Health, UK PBC Study Group
- Responsible party
- Sponsor
- Investigators
- Lynda Szczech, MD, Study director, Intercept Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05293938
- Sponsor's protocol ID
- 747-404
- Acronym
- HEROES-PBC
- Status last verified
- Oct 2023
- First submitted
- 4 Mar 2022
- Last updated
- 10 Oct 2023
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 10 Oct 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Study Evaluating the Effectiveness of Obeticholic Acid on Hepatic Real-World Outcomes in Patients With Primary Biliary Cholangitis
- ClinicalTrials.gov ID
- NCT05293938
- Acronym / sponsor ID
- HEROES-PBC / 747-404
- Conditions
- Primary Biliary Cholangitis
- Keywords
- Primary Biliary Cholangitis, Primary Biliary Cirrhosis, PBC, Hepatic Impairment, Liver
- Study type
- Observational
- Recruitment status
- WithdrawnReason stopped: A fundamental protocol deviation on data capture left the database no longer fit for purpose.
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Intercept PharmaceuticalsIndustry (pharma, biotech, devices)
- Collaborators
- Global PBC Study Group, Target RWE, Syneos Health, UK PBC Study Group
- Study start
- 28 Mar 2022 (actual)
- Primary completion
- Jun 2023 (estimated)
- Study completion
- Jul 2023 (estimated)
- First posted
- 24 Mar 2022 (actual)
- Results
- Not posted in the source record
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, 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 design
- Observational model
- Cohort
- Time perspective
- Retrospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| OCA Treatment Group | – | Drug: Obeticholic Acid 5 MG, Drug: Obeticholic Acid 10 MGPBC patients with a history of UDCA failure who initiated OCA in the study window (01 Jun 2015 to 31 Dec 2021) |
| Control Group | – | Drug: Standard of Care: UDCAPBC 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) |
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 UK PBC registry between 01 Jun 2015 and 31 Dec 2021 and who meet the eligibility criteria will be considered for the study.
Key inclusion criteria (4 listed)
- 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)
Key exclusion criteria (6 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Obeticholic Acid 5 MGAlso: Ocalivaonce daily, oral administration | Drug | OCA Treatment Group |
| Obeticholic Acid 10 MGAlso: Ocalivaonce daily, oral administration | Drug | OCA Treatment Group |
| Standard of Care: UDCANo 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. | Drug | Control Group |
Primary outcome
Time to the first occurrence of the composite endpoint of all-cause death, liver transplant, or hepatic decompensation.
Time frame: Time from index date to first occurrence of the composite endpoint events, assessed up to 62 months.
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Oct 2023.
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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 6 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: withdrawn, as of the sponsor's last update on 10 Oct 2023. 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?
The study was withdrawn before enrolling participants, so there are no results.
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?
Time to the first occurrence of the composite endpoint of all-cause death, liver transplant, or hepatic decompensation. (time frame: Time from index date to first occurrence of the composite endpoint events, assessed up to 62 months.). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 10 Oct 2023.