Primary Biliary CholangitisRESCATNCT05919433
Detection Program for Patients With Primary Biliary Cholangitis Lost in the System
An observational study of Primary Biliary Cholangitis, run by Hospital Mutua de Terrassa at 1 site in Spain.
Trial summary
Primary biliary cholangitis (PBC) has been considered a rare disease and its management has been limited by the lack of therapeutic alternatives. PBC is a slowly progressing chronic liver disease characterized by an immune-mediated destruction of the intrahepatic bile ducts, which leads to cholestasis, portal… 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
- 500 participants
- Where
- 1 siteSpain
- Study dates
- 1 May 2023to 31 Dec 2028
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
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: Patients identified in the database of the participating hospitals
Who may be able to join
- Patients with positive AMA and/or with positive ANA anti-gp210 or anti-sp100 identified in the computer databases of each of the participating centers.
- Adults (≥18 years)
Who cannot join
- Patients with Overlap Syndrome (PBC overlap with Autoimmune Hepatitis (AIH))
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
Diana Horta, MD, PhD contact
Spain1 site, 1 recruiting
- Recruiting
Hospital Universitari MútuaTerrassa
Terrassa, Barcelona, 08221
Diana Horta, MD, PhD, diana.horta.s@gmail.com
About this study
Primary biliary cholangitis (PBC) has been considered a rare disease and its management has been limited by the lack of therapeutic alternatives. PBC is a slowly progressing chronic liver disease characterized by an immune-mediated destruction of the intrahepatic bile ducts, which leads to cholestasis, portal inflammation, and ultimately liver cirrhosis and its associated complications (ascites, portal hypertension, etc), if not treated effectively. Thus, early diagnosis and close management of these patients with PBC is essential. First-line treatment with ursodeoxycholic acid (UDCA) improves liver biochemical parameters, delays histological progression, and increases liver transplant-free survival and overall survival. However, up to 40% of patients are non-responders to UDCA. Obeticholic acid (OCA) is recommended as second-line therapy in combination with UDCA for patients with an inadequate response to UDCA or as monotherapy in cases of UDCA intolerance.
According to current clinical guidelines, the diagnosis of PBC includes a combination of elevated alkaline phosphatase (ALP) levels and the presence of anti-mitochondrial antibodies (AMA) (titer >1:40) and/or anti-nuclear antibodies (ANA) anti-gp210 or anti-sp100. AMA are highly sensitive and specific for PBC and are detected in nearly 95% of PBC patients. A liver biopsy is not necessary unless there is an elevation of ALP without the presence of specific AMA and/or anti-gp210 or anti-sp100 ANA or if coexistence with other liver diseases is suspected (autoimmune hepatitis, hepatic steatosis).
Read more
The incidence of PBC has increased in recent years due to an increase in the diagnosis of cases in the initial phases, better awareness in the medical community and the development of more sensitive diagnostic tests. However, up to 31% of patients with PBC are lost without follow-up. The correct identification of patients with PBC is essential so that they can benefit from an adequate treatment and modify disease progression. To date, two studies (one Spanish and one Portuguese) showed that 27% and 45.5% of the patients lost with PBC presented advanced fibrosis, respectively.
The objective of this study is to identify, through computerized data, patients with PBC who may be lost in the system and evaluate their clinical, analytical and demographic characteristics, and in a second phase, provide access to follow-up in specialized consultations.
- Conditions
- Primary Biliary Cholangitis
- 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 |
|---|---|---|
| Patients with positive AMA and/or ANA anti-gp210/sp100 identified in the hospital database | Not described |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percentage of patients correctly diagnosed with PBC and monitored in specialized health care compared to lost to follow-up PBC patients
Measured: at inclusion
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Jun 2023
- Study start1 May 2023
- Primary completion31 Dec 2023 (estimated)
- Study completion31 Dec 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (8)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
- Case only
- Time perspective
- Other
- Enrolment
- 500 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Hospital Mutua de Terrassa
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05919433
- Sponsor's protocol ID
- RESCAT
- Acronym
- RESCAT
- Status last verified
- Jun 2023
- First submitted
- 29 May 2023
- Last updated
- 26 Jun 2023
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Publications
Carey EJ, Ali AH, Lindor KD. Primary biliary cirrhosis. Lancet. 2015 Oct 17;386(10003):1565-75. doi: 10.1016/S0140-6736(15)00154-3. Epub 2015 Sep 11.
PubMed 26364546Kaplan MM, Gershwin ME. Primary biliary cirrhosis. N Engl J Med. 2005 Sep 22;353(12):1261-73. doi: 10.1056/NEJMra043898. No abstract available.
PubMed 16177252European Association for the Study of the Liver. EASL Clinical Practice Guidelines: The diagnosis and management of patients with primary biliary cholangitis. J Hepatol. 2017 Jul;67(1):145-172. doi: 10.1016/j.jhep.2017.03.022. Epub 2017 Apr 18.
PubMed 28427765Mayo MJ. Mechanisms and molecules: What are the treatment targets for primary biliary cholangitis? Hepatology. 2022 Aug;76(2):518-531. doi: 10.1002/hep.32405. Epub 2022 Mar 10.
PubMed 35152430Pares A; Leading PBC Group. Practical management of primary biliary cholangitis. Rev Esp Enferm Dig. 2022 Jul;114(7):410-417. doi: 10.17235/reed.2021.8219/2021.
PubMed 34663072Leung KK, Hirschfield GM. Autoantibodies in Primary Biliary Cholangitis. Clin Liver Dis. 2022 Nov;26(4):613-627. doi: 10.1016/j.cld.2022.06.004.
PubMed 36270719Olveira-Martin A, Yebra-Carmona J, Amaral-Gonzalez C, Tejedor M, Eiras P, Hernandez-Perez M, Suarez-Cabredo C, Spigarelli-de Rabago I, Suarez-Ferrer C, Morales-Arraez D, Chico I, Diaz-Flores F, Rodriguez R, Llorente S, Molina-Perez E, Hernandez-Guerra de Aguilar MN. Retrieval and treatment of patients with primary biliary cholangitis who are lost in the health system. Rev Esp Enferm Dig. 2021 Nov;113(11):776-779. doi: 10.17235/reed.2021.8174/2021.
PubMed 34470449Garrido I, Liberal R, Cardoso MJ, Macedo G. The impact of undiagnosed primary biliary cholangitis. Eur J Gastroenterol Hepatol. 2021 Dec 1;33(1S Suppl 1):e1027-e1031. doi: 10.1097/MEG.0000000000002268.
PubMed 34402472
Study at a glance
From the registry record, last updated by the sponsor on 26 Jun 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Detection Program for Patients With Primary Biliary Cholangitis Lost in the System
- ClinicalTrials.gov ID
- NCT05919433
- Acronym / sponsor ID
- RESCAT / RESCAT
- Conditions
- Primary Biliary Cholangitis
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 500 participants (estimated)
- Lead sponsor
- Hospital Mutua de TerrassaUniversity, hospital or other
- Study start
- 1 May 2023 (actual)
- Primary completion
- 31 Dec 2023 (estimated)
- Study completion
- 31 Dec 2028 (estimated)
- First posted
- 26 Jun 2023 (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.
Primary biliary cholangitis (PBC) has been considered a rare disease and its management has been limited by the lack of therapeutic alternatives. PBC is a slowly progressing chronic liver disease characterized by an immune-mediated destruction of the intrahepatic bile ducts, which leads to cholestasis, portal inflammation, and ultimately liver cirrhosis and its associated complications (ascites, portal hypertension, etc), if not treated effectively. Thus, early diagnosis and close management of these patients with PBC is essential. First-line treatment with ursodeoxycholic acid (UDCA) improves liver biochemical parameters, delays histological progression, and increases liver transplant-free survival and overall survival. However, up to 40% of patients are non-responders to UDCA. Obeticholic acid (OCA) is recommended as second-line therapy in combination with UDCA for patients with an inadequate response to UDCA or as monotherapy in cases of UDCA intolerance.
According to current clinical guidelines, the diagnosis of PBC includes a combination of elevated alkaline phosphatase (ALP) levels and the presence of anti-mitochondrial antibodies (AMA) (titer >1:40) and/or anti-nuclear antibodies (ANA) anti-gp210 or anti-sp100. AMA are highly sensitive and specific for PBC and are detected in nearly 95% of PBC patients. A liver biopsy is not necessary unless there is an elevation of ALP without the presence of specific AMA and/or anti-gp210 or anti-sp100 ANA or if coexistence with other liver diseases is suspected (autoimmune hepatitis, hepatic steatosis).
The incidence of PBC has increased in recent years due to an increase in the diagnosis of cases in the initial phases, better awareness in the medical community and the development of more sensitive diagnostic tests. However, up to 31% of patients with PBC are lost without follow-up. The correct identification of patients with PBC is essential so that they can benefit from an adequate treatment and modify disease progression. To date, two studies (one Spanish and one Portuguese) showed that 27% and 45.5% of the patients lost with PBC presented advanced fibrosis, respectively.
The objective of this study is to identify, through computerized data, patients with PBC who may be lost in the system and evaluate their clinical, analytical and demographic characteristics, and in a second phase, provide access to follow-up in specialized consultations.
Study design
- Observational model
- Case only
- Time perspective
- Other
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Patients with positive AMA and/or ANA anti-gp210/sp100 identified in the hospital database | – | – |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Patients identified in the database of the participating hospitals
Key inclusion criteria (2 listed)
- Patients with positive AMA and/or with positive ANA anti-gp210 or anti-sp100 identified in the computer databases of each of the participating centers.
- Adults (≥18 years)
Key exclusion criteria (1 listed)
- Patients with Overlap Syndrome (PBC overlap with Autoimmune Hepatitis (AIH))
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
Percentage of patients correctly diagnosed with PBC and monitored in specialized health care compared to lost to follow-up PBC patients
Time frame: at inclusion
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 26 Jun 2023.
- Background: PubMed 26364546Carey EJ, Ali AH, Lindor KD. Primary biliary cirrhosis. Lancet. 2015 Oct 17;386(10003):1565-75. doi: 10.1016/S0140-6736(15)00154-3. Epub 2015 Sep 11.
- Background: PubMed 16177252Kaplan MM, Gershwin ME. Primary biliary cirrhosis. N Engl J Med. 2005 Sep 22;353(12):1261-73. doi: 10.1056/NEJMra043898. No abstract available.
- Background: PubMed 28427765European Association for the Study of the Liver. EASL Clinical Practice Guidelines: The diagnosis and management of patients with primary biliary cholangitis. J Hepatol. 2017 Jul;67(1):145-172. doi: 10.1016/j.jhep.2017.03.022. Epub 2017 Apr 18.
- Background: PubMed 35152430Mayo MJ. Mechanisms and molecules: What are the treatment targets for primary biliary cholangitis? Hepatology. 2022 Aug;76(2):518-531. doi: 10.1002/hep.32405. Epub 2022 Mar 10.
- Background: PubMed 34663072Pares A; Leading PBC Group. Practical management of primary biliary cholangitis. Rev Esp Enferm Dig. 2022 Jul;114(7):410-417. doi: 10.17235/reed.2021.8219/2021.
- Background: PubMed 36270719Leung KK, Hirschfield GM. Autoantibodies in Primary Biliary Cholangitis. Clin Liver Dis. 2022 Nov;26(4):613-627. doi: 10.1016/j.cld.2022.06.004.
- Background: PubMed 34470449Olveira-Martin A, Yebra-Carmona J, Amaral-Gonzalez C, Tejedor M, Eiras P, Hernandez-Perez M, Suarez-Cabredo C, Spigarelli-de Rabago I, Suarez-Ferrer C, Morales-Arraez D, Chico I, Diaz-Flores F, Rodriguez R, Llorente S, Molina-Perez E, Hernandez-Guerra de Aguilar MN. Retrieval and treatment of patients with primary biliary cholangitis who are lost in the health system. Rev Esp Enferm Dig. 2021 Nov;113(11):776-779. doi: 10.17235/reed.2021.8174/2021.
- Background: PubMed 34402472Garrido I, Liberal R, Cardoso MJ, Macedo G. The impact of undiagnosed primary biliary cholangitis. Eur J Gastroenterol Hepatol. 2021 Dec 1;33(1S Suppl 1):e1027-e1031. doi: 10.1097/MEG.0000000000002268.
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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.
Diana Horta, MD, PhD contact
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 Hospital Mutua de Terrassa at 1 site in Spain.
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 2 inclusion and 1 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: looking for participants now, as of the sponsor's last update on 26 Jun 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 Spain. 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?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Diana Horta, MD, PhD (diana.horta.s@gmail.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Percentage of patients correctly diagnosed with PBC and monitored in specialized health care compared to lost to follow-up PBC patients (time frame: at inclusion). 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 NCT05919433, which also lists 8 linked publications.
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 26 Jun 2023.