National Database on Primary Biliary Cholangitis

NCT ID: NCT05151809

Last Updated: 2025-07-25

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

RECRUITING

Total Enrollment

6000 participants

Study Classification

OBSERVATIONAL

Study Start Date

2019-09-19

Study Completion Date

2029-01-24

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

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.

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

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.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Primary Biliary Cholangitis

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Observational Model Type

COHORT

Study Time Perspective

PROSPECTIVE

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

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.

Clinical information

Intervention Type OTHER

The investigators will recruit PBC patients and collect important clinical information and laboratory investigation, together with biological samples.

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

Clinical information

The investigators will recruit PBC patients and collect important clinical information and laboratory investigation, together with biological samples.

Intervention Type OTHER

Other Intervention Names

Discover alternative or legacy names that may be used to describe the listed interventions across different sources.

Laboratory information Biological samples

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

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
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

Meet the organizations funding or collaborating on the study and learn about their roles.

University of Milano Bicocca

OTHER

Sponsor Role lead

Responsible Party

Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.

Responsibility Role SPONSOR

Locations

Explore where the study is taking place and check the recruitment status at each participating site.

Ospedali riuniti di Ancona

Ancona, , Italy

Site Status RECRUITING

A.O.U. Consorziale - Policlinico Bari- UO Gastroenterologia

Bari, , Italy

Site Status RECRUITING

UO di gastroenterologia, Asst Papa Giovanni XXIII

Bergamo, , Italy

Site Status RECRUITING

Policlinico S. Orsola Malpighi- UO Medicina Interna

Bologna, , Italy

Site Status RECRUITING

Policlinico S.Orsola Malpighi- Ambulatorio di epatologia e gestione trapianto epatico

Bologna, , Italy

Site Status NOT_YET_RECRUITING

Gastroenterologia, fisiopatologia e endoscopia digestiva, Azienda sanitaria dell'alto adige

Bolzano, , Italy

Site Status RECRUITING

Unità epatologica ed ecografia internistica a valenza dipartimentale, Fondazione Ospedaliera Poliambulanza

Brescia, , Italy

Site Status RECRUITING

UO di gastroenterologia, Spedali civili di Brescia

Brescia, , Italy

Site Status NOT_YET_RECRUITING

UO epatologia, Azienda ospedaliero università di Cagliari Policlinico universitario Monserrato

Cagliari, , Italy

Site Status RECRUITING

Ospedale IRCCS Saverio De Bellis- UOC di Gastroenterologia 1

Castellana Grotte, , Italy

Site Status RECRUITING

UO epatologia Ospedale Garibaldi

Catania, , Italy

Site Status RECRUITING

UO di fisiopatologia digestiva, Azienda Ospedaliera Universitaria Mater Domini

Catanzaro, , Italy

Site Status RECRUITING

UO di gastroenterologia, Ospedale Valduce

Como, , Italy

Site Status RECRUITING

Azienda Socio Sanitaria Territoriale (ASST) di Cremona- UO Medicina Interna - Ambulatori di Epatologia

Cremona, , Italy

Site Status RECRUITING

Presidio Ospedaliero di Faenza- UO Medicina Interna

Faenza, , Italy

Site Status RECRUITING

UO Clinica Medica, Azienda Ospedaliera Universitaria Careggi Firenze

Florence, , Italy

Site Status RECRUITING

Centro C.U.R.E. Centro Universitario per la ricerca e la Cura delle Malattie Epatiche

Foggia, , Italy

Site Status RECRUITING

Ambulatorio di Malattie del Fegato, U.O. Medicina, ASST Valle Olona, Presidio di Gallarate

Gallarate, , Italy

Site Status RECRUITING

UO Clinica Gastroenterologica, Policlinico San Martino

Genova, , Italy

Site Status RECRUITING

Centro Medicina del Viaggiatore e delle Migrazioni, ASP Catanzaro, Presìdio Ospedaliero "Giovanni Paolo II"

Lamezia Terme, , Italy

Site Status RECRUITING

UO di epatologia clinica e biomolecolare, Università degli studi di Messina

Messina, , Italy

Site Status NOT_YET_RECRUITING

University of Milano-Bicocca

Milan, , Italy

Site Status RECRUITING

Policlinico di Milano

Milan, , Italy

Site Status RECRUITING

UO di epatologia, Ospedale Niguarda Ca' Granda

Milan, , Italy

Site Status RECRUITING

UO di Epatologia, Ospedale San Giuseppe

Milan, , Italy

Site Status RECRUITING

UO epatologia, Istituto scientifico universitario San Raffaele

Milan, , Italy

Site Status RECRUITING

AOU di Modena- U.O. Gastroenterologia

Modena, , Italy

Site Status RECRUITING

Struttura Complessa di Medicina ad Indirizzo Metabolico Nutrizionale, Dipartimento Ospedaliero di Medicina Interna

Modena, , Italy

Site Status RECRUITING

Day Hospital e Ambulatorio di Epatoogia e Nutrizione Clinica, Fondazione Evangelica Villa Betania, Ospedale generale di Zona

Napoli, , Italy

Site Status NOT_YET_RECRUITING

Ospedale policlinico Federico II di Napoli

Napoli, , Italy

Site Status RECRUITING

Università della Campania Luigi Vanvitelli di Napoli- Dipartimento di epato-gastroenterologia

Napoli, , Italy

Site Status RECRUITING

UO medicina ed epatologia, Ospedale maggiore della carità

Novara, , Italy

Site Status RECRUITING

UO di Gastroenterologia, Azienda ospedaliera di Padova

Padua, , Italy

Site Status RECRUITING

UO di Medicina Interna, Azienda Ospedaliera di Padova

Padua, , Italy

Site Status RECRUITING

UO gastroenterologia, Policlinico Paolo Giaccone

Palermo, , Italy

Site Status RECRUITING

Malattie infettive ed epatologia, Azienda ospedaliero universitaria di Parma

Parma, , Italy

Site Status RECRUITING

Day Hospital Internistico e ambulatorio di Epatologia, ASL Pescara

Pescara, , Italy

Site Status RECRUITING

UO epatologia, AOU pisana Cisanello

Pisa, , Italy

Site Status RECRUITING

S.C. Gastroenterologia, Ospedale di Pordenone, AS FO

Pordenone, , Italy

Site Status RECRUITING

Medicina clinica ed epatologia, Policlinico universitario campus bio-medico

Roma, , Italy

Site Status RECRUITING

Policlinico Universitario Fondazione Agostino Gemelli- UO di medicina interna

Roma, , Italy

Site Status RECRUITING

UO di Epatologia, Universita' di Roma Tor Vergata

Roma, , Italy

Site Status RECRUITING

UO di Gastroenterologia, Policlinico Umberto I

Roma, , Italy

Site Status RECRUITING

Medicina interna ed epatologia, Istituto clinico Humanitas

Rozzano, , Italy

Site Status RECRUITING

Università degli studi di Salerno

Salerno, , Italy

Site Status RECRUITING

SSD Epatologia, Fondazione Casa Sollievo della Sofferenza

San Giovanni Rotondo, , Italy

Site Status NOT_YET_RECRUITING

UO di Gastroenterologia e Endoscopia Digestiva, Ospedale Casa Sollievo della Sofferenza

San Giovanni Rotondo, , Italy

Site Status RECRUITING

Azienda Ospedale Universitaria di Sassari- UOC Medicina Interna

Sassari, , Italy

Site Status RECRUITING

S.C. Gastroenterologia, Azienda Ospedaliero-Universitaria Molinette

Torino, , Italy

Site Status RECRUITING

Gastroenterologia ed endoscopia digestiva, Azienda provinciale per i servizi sanitari

Trento, , Italy

Site Status RECRUITING

Ospedale regionale Ca' Foncello di Treviso- Unità operativa complessa di Gastroenterologia

Treviso, , Italy

Site Status RECRUITING

SC (UCO) Clinica Patologie del Fegato, Ospedale di Cattinara, ASU GI

Trieste, , Italy

Site Status RECRUITING

SOS di DPT Epatologia e Trapianti di Fegato, ASU FC

Udine, , Italy

Site Status RECRUITING

UO di gastroenterologia ed endoscopia digestiva, Azienda macchi, Asst sette laghi

Varese, , Italy

Site Status RECRUITING

Azienda Ospedaliera Universitaria Integrata Verona- UOC Gastroeneterologia A

Verona, , Italy

Site Status RECRUITING

Countries

Review the countries where the study has at least one active or historical site.

Italy

Central Contacts

Reach out to these primary contacts for questions about participation or study logistics.

Pietro Invernizzi, MD

Role: CONTACT

+39 039 233 2187

Marco Carbone, MD

Role: CONTACT

02 6448 8139

Facility Contacts

Find local site contact details for specific facilities participating in the trial.

Luigi Muratori

Role: primary

Pietro Invernizzi

Role: primary

Marco Carbone

Role: backup

Other Identifiers

Review additional registry numbers or institutional identifiers associated with this trial.

PBC322

Identifier Type: -

Identifier Source: org_study_id

More Related Trials

Additional clinical trials that may be relevant based on similarity analysis.

Percutaneous Cholangiopancreatoscopy Registry
NCT05210322 ACTIVE_NOT_RECRUITING