National Database on Primary Biliary Cholangitis (PBC322)

July 24, 2025 updated by: University of Milano Bicocca

Multicenter, Nationwide, Observational, Retrospective and Prospective Study Based on the Development of a Patients Database Linked to a Biological Sample Storage

Primary biliary cholangitis (PBC) is a rare, autoimmune, cholestatic liver disease. No data about the disease epidemiology exist in Italy. Therefore this study aims to develop a national PBC patient database linked to a biological sample storage.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Detailed Description

Primary biliary cholangitis (PBC) is an immune-mediated liver disease characterized by chronic inflammation of the intrahepatic bile ducts, causing progressive ductopenia, cholestasis, and if not effectively treated, leading to fibrosis, cirrhosis and liver failure. Nowadays almost all patients with PBC are diagnosed at an early disease stage and receive treatment with ursodeoxycholic acid (UDCA) which is currently the only drug approved for the treatment of patients with PBC. However, approximately 20% to 30% (up to >50% in patients presenting under the age of 40 years) still does not have a benefit from UDCA and has a reduced prognosis as compared to healthy individuals. Major steps forwards in the field of PBC have been done in the last decade, however there are still significant areas of unmet clinical need in PBC: the lack of knowledge on etiopathogenesis; a poor understanding of disease sub-phenotypes; the lack of biomarkers of disease progression that allow risk stratification needed in clinical management and trials design, among the others. In the current evolving research landscape with the availability of the -omics technologies generating libraries of genome-wide data, metabolomics and proteomics data, among the others, the prospects of discovering the gene and molecular underpinnings of PBC are more promising than ever. Scientists envision an era of "personalized medicine" when more and more people will obtain their own genetic and metabolic maps, enabling them to identify their status as carriers of specific risk profiles.

Based on these premises, the current project aims to build up a research, nation-wide infrastructure (around 60 Italian participating centres will be involved) to study the biology of PBC and, in particular, to explore why a significant group of, typically young patients fail primary therapy with UDCA, placing them at risk of developing progressive disease and needing liver transplantation (LT). The investigators will recruit patients and organise the collection of important clinical information and laboratory investigation, together with biological samples. Data will be collected in the form of electronic Case Report Forms (REDCap cloud) that will be completed by clinicians at baseline and thereafter on an annual basis. The clinical information will allow us to identify patients' clinical profiles. The biological samples will allow to understand key aspects of people's make up, including patient genes and the way their immune system works, and the differences in make up between people with different clinical phenotypes.

This research infrastructure would represent an invaluable resource for successful translational research in this field. Specifically, it would serve investigators conducting research; clinicians treating patients; epidemiologists gathering demographic data; and the drug and device industry seeking new markets. It also can represent a necessary infrastructure for the implementation of the European Reference Networks (ERN) for rare diseases, main pillars of the current EU policy framework on National Plans for research and development. The Italian PBC database would also be crucial for drug development, specifically to assess the feasibility of clinical trials, to facilitate the planning of appropriate clinical trials, to support the enrolment of patients and to assess the impact of new interventions.

Study Type

Observational

Enrollment (Estimated)

6000

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact Backup

Study Locations

  • Italy
      • Ancona, Italy
        • Recruiting
        • Ospedali Riuniti Di Ancona
      • Bari, Italy
        • Recruiting
        • A.O.U. Consorziale - Policlinico Bari- UO Gastroenterologia
      • Bergamo, Italy
        • Recruiting
        • UO di gastroenterologia, Asst Papa Giovanni XXIII
      • Bologna, Italy
        • Recruiting
        • Policlinico S. Orsola Malpighi- UO Medicina Interna
        • Contact:
      • Bologna, Italy
        • Not yet recruiting
        • Policlinico S.Orsola Malpighi- Ambulatorio di epatologia e gestione trapianto epatico
      • Bolzano, Italy
        • Recruiting
        • Gastroenterologia, fisiopatologia e endoscopia digestiva, Azienda sanitaria dell'alto adige
      • Brescia, Italy
        • Recruiting
        • Unità epatologica ed ecografia internistica a valenza dipartimentale, Fondazione Ospedaliera Poliambulanza
      • Brescia, Italy
        • Not yet recruiting
        • UO di gastroenterologia, Spedali civili di Brescia
      • Cagliari, Italy
        • Recruiting
        • UO epatologia, Azienda ospedaliero università di Cagliari Policlinico universitario Monserrato
      • Castellana Grotte, Italy
        • Recruiting
        • Ospedale IRCCS Saverio De Bellis- UOC di Gastroenterologia 1
      • Catania, Italy
        • Recruiting
        • UO epatologia Ospedale Garibaldi
      • Catanzaro, Italy
        • Recruiting
        • UO di fisiopatologia digestiva, Azienda Ospedaliera Universitaria Mater Domini
      • Como, Italy
        • Recruiting
        • UO di gastroenterologia, Ospedale Valduce
      • Cremona, Italy
        • Recruiting
        • Azienda Socio Sanitaria Territoriale (ASST) di Cremona- UO Medicina Interna - Ambulatori di Epatologia
      • Faenza, Italy
        • Recruiting
        • Presidio Ospedaliero di Faenza- UO Medicina Interna
      • Firenze, Italy
        • Recruiting
        • UO Clinica Medica, Azienda Ospedaliera Universitaria Careggi Firenze
      • Foggia, Italy
        • Recruiting
        • Centro C.U.R.E. Centro Universitario per la ricerca e la Cura delle Malattie Epatiche
      • Gallarate, Italy
        • Recruiting
        • Ambulatorio di Malattie del Fegato, U.O. Medicina, ASST Valle Olona, Presidio di Gallarate
      • Genova, Italy
        • Recruiting
        • UO Clinica Gastroenterologica, Policlinico San Martino
      • Lamezia Terme, Italy
        • Recruiting
        • Centro Medicina del Viaggiatore e delle Migrazioni, ASP Catanzaro, Presìdio Ospedaliero "Giovanni Paolo II"
      • Messina, Italy
        • Not yet recruiting
        • UO di epatologia clinica e biomolecolare, Università degli studi di Messina
      • Milan, Italy, 20126
      • Milano, Italy
        • Recruiting
        • Policlinico di Milano
      • Milano, Italy
        • Recruiting
        • UO di epatologia, Ospedale Niguarda Ca' Granda
      • Milano, Italy
        • Recruiting
        • UO epatologia, Istituto scientifico universitario San Raffaele
      • Milano, Italy
        • Recruiting
        • UO di Epatologia, Ospedale San Giuseppe
      • Modena, Italy
        • Recruiting
        • Struttura Complessa di Medicina ad Indirizzo Metabolico Nutrizionale, Dipartimento Ospedaliero di Medicina Interna
      • Modena, Italy
        • Recruiting
        • AOU di Modena- U.O. Gastroenterologia
      • Napoli, Italy
        • Not yet recruiting
        • Day Hospital e Ambulatorio di Epatoogia e Nutrizione Clinica, Fondazione Evangelica Villa Betania, Ospedale generale di Zona
      • Napoli, Italy
        • Recruiting
        • Ospedale policlinico Federico II di Napoli
      • Napoli, Italy
        • Recruiting
        • Università della Campania Luigi Vanvitelli di Napoli- Dipartimento di epato-gastroenterologia
      • Novara, Italy
        • Recruiting
        • UO medicina ed epatologia, Ospedale maggiore della carità
      • Padova, Italy
        • Recruiting
        • UO di Gastroenterologia, Azienda ospedaliera di Padova
      • Padova, Italy
        • Recruiting
        • UO di Medicina Interna, Azienda Ospedaliera di Padova
      • Palermo, Italy
        • Recruiting
        • UO gastroenterologia, Policlinico Paolo Giaccone
      • Parma, Italy
        • Recruiting
        • Malattie infettive ed epatologia, Azienda ospedaliero universitaria di Parma
      • Pescara, Italy
        • Recruiting
        • Day Hospital Internistico e ambulatorio di Epatologia, ASL Pescara
      • Pisa, Italy
        • Recruiting
        • UO epatologia, AOU pisana Cisanello
      • Pordenone, Italy
        • Recruiting
        • S.C. Gastroenterologia, Ospedale di Pordenone, AS FO
      • Roma, Italy
        • Recruiting
        • Medicina clinica ed epatologia, Policlinico universitario campus bio-medico
      • Roma, Italy
        • Recruiting
        • UO di Epatologia, Universita' di Roma Tor Vergata
      • Roma, Italy
        • Recruiting
        • UO di Gastroenterologia, Policlinico Umberto I
      • Roma, Italy
        • Recruiting
        • Policlinico Universitario Fondazione Agostino Gemelli- UO di medicina interna
      • Rozzano, Italy
        • Recruiting
        • Medicina interna ed epatologia, Istituto clinico Humanitas
      • Salerno, Italy
        • Recruiting
        • Università degli studi di salerno
      • San Giovanni Rotondo, Italy
        • Recruiting
        • UO di Gastroenterologia e Endoscopia Digestiva, Ospedale Casa Sollievo della Sofferenza
      • San Giovanni Rotondo, Italy
        • Not yet recruiting
        • SSD Epatologia, Fondazione Casa Sollievo della Sofferenza
      • Sassari, Italy
        • Recruiting
        • Azienda Ospedale Universitaria di Sassari- UOC Medicina Interna
      • Torino, Italy
        • Recruiting
        • S.C. Gastroenterologia, Azienda Ospedaliero-Universitaria Molinette
      • Trento, Italy
        • Recruiting
        • Gastroenterologia ed endoscopia digestiva, Azienda provinciale per i servizi sanitari
      • Treviso, Italy
        • Recruiting
        • Ospedale regionale Ca' Foncello di Treviso- Unità operativa complessa di Gastroenterologia
      • Trieste, Italy
        • Recruiting
        • SC (UCO) Clinica Patologie del Fegato, Ospedale di Cattinara, ASU GI
      • Udine, Italy
        • Recruiting
        • SOS di DPT Epatologia e Trapianti di Fegato, ASU FC
      • Varese, Italy
        • Recruiting
        • UO di gastroenterologia ed endoscopia digestiva, Azienda macchi, Asst sette laghi
      • Verona, Italy
        • Recruiting
        • Azienda Ospedaliera Universitaria Integrata Verona- UOC Gastroeneterologia A

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Sampling Method

Non-Probability Sample

Study Population

All the italian patients fulfilling the diagnostic criteria for Primary Biliary Cholangitis (European Association for the Study of the Liver. EASL Clinical Practice Guidelines: management of cholestatic liver diseases. J Hepatol. 2009 Aug;51(2):237-67) and aged at least 18.

Description

Inclusion Criteria:

All PBC patients living in Italy and aged at least 18 years can be included in the database. According to well-established criteria, PBC is diagnosed in subjects who fulfill two of the three of following criteria:

  • elevated alkaline phosphatase and /or GGT;
  • positive anti-mitochondrial autoantibodies (titer ≥ 1:40) or PBC-specific antinuclear antibodies (gp-210 and sp100);
  • characteristic histological features of florid bile ducts lesions and granulomatous lesion.

Exclusion Criteria:

The patient has explicitly declared his/her unwillingness to participate to the study

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Observational Models: Cohort
  • Time Perspectives: Prospective

Cohorts and Interventions

Group / Cohort
Intervention / Treatment
PBC population residing in Italy

All PBC patients living in Italy and aged at least 18 years can be included in the database. According to well-established criteria, PBC is diagnosed in subjects who fulfill two of the three of following criteria:

  1. elevated alkaline phosphatase and /or GGT;
  2. positive anti-mitochondrial autoantibodies (titer ≥ 1:40) or PBC-specific antinuclear antibodies (gp-210 and sp100);
  3. characteristic histological features of florid bile ducts lesions and granulomatous lesion.
Other: Clinical information
The investigators will recruit PBC patients and collect important clinical information and laboratory investigation, together with biological samples.
Other Names:
  • Laboratory information
  • Biological samples

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Phenotypes and sub-phenotypes of PBC Italian patients
Time Frame: Overall duration of the study (10 years)
Identify and define distinct phenotypes and sub-phenotypes of PBC patients at higher risk of disease progression.
Overall duration of the study (10 years)

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Response to UDCA therapy
Time Frame: Overall duration of the study (10 years)
Defined as Alkaline Phosphatase Level<1 x upper limit of normal. The investigators will fit a multivariate analysis using logistic regression using baseline variables.
Overall duration of the study (10 years)
Identification of factors influencing the progression of PBC
Time Frame: Overall duration of the study (10 years)
The investigators will calculate the time from the diagnosis of PBC to an event (liver decompensation, liver transplantation or death). They will then fit a multivariate analysis using Cox's proportional hazards regression model of diverse explanatory variables available at baseline.
Overall duration of the study (10 years)
Safety and long-term efficacy of novel therapies
Time Frame: Overall duration of the study (10 years)
The investigators will evaluate prospectively laboratory investigation and the treatment response to novel therapies that are entering the clinical practice, e.g. obeticholic acid, fibrates.
Overall duration of the study (10 years)

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

University of Milano Bicocca

Collaborators

Intercept Pharmaceuticals
Laboratoires Mayoly Spindler

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

September 19, 2019

Primary Completion (Estimated)

January 24, 2029

Study Completion (Estimated)

January 24, 2029

Study Registration Dates

First Submitted

November 15, 2021

First Submitted That Met QC Criteria

November 25, 2021

First Posted (Actual)

December 9, 2021

Study Record Updates

Last Update Posted (Actual)

July 25, 2025

Last Update Submitted That Met QC Criteria

July 24, 2025

Last Verified

July 1, 2025

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • PBC322

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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