Primary Biliary CholangitisPBC322NCT05151809
National Database on Primary Biliary Cholangitis
An observational study of Primary Biliary Cholangitis, run by University of Milano Bicocca at 55 sites in Italy.
Trial summary
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. 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?
- Clinical information
- How many people
- 6,000 participants
- Where
- 55 sitesItaly
- Study dates
- 19 Sep 2019to 24 Jan 2029
- How long will I take part?
- 10 Years
- 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: 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.
Who may be able to join
- 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.
Who cannot join
- The patient has explicitly declared his/her unwillingness to participate to the study
Trial locations and contacts
55 sites in 1 country. Contact the study team or a site near you to ask about taking part.
55 sites in 1 country
Italy55 sites, 55 recruiting
- Recruiting
Ospedali riuniti di Ancona
Ancona
- Recruiting
A.O.U. Consorziale - Policlinico Bari- UO Gastroenterologia
Bari
- Recruiting
UO di gastroenterologia, Asst Papa Giovanni XXIII
Bergamo
- Recruiting
Policlinico S. Orsola Malpighi- UO Medicina Interna
Bologna
Luigi Muratori, luigi.muratori@unibo.it
- Not yet recruiting
Policlinico S.Orsola Malpighi- Ambulatorio di epatologia e gestione trapianto epatico
Bologna
- Recruiting
Gastroenterologia, fisiopatologia e endoscopia digestiva, Azienda sanitaria dell'alto adige
Bolzano
- Recruiting
Unità epatologica ed ecografia internistica a valenza dipartimentale, Fondazione Ospedaliera Poliambulanza
Brescia
- Not yet recruiting
UO di gastroenterologia, Spedali civili di Brescia
Brescia
- Recruiting
UO epatologia, Azienda ospedaliero università di Cagliari Policlinico universitario Monserrato
Cagliari
- Recruiting
Ospedale IRCCS Saverio De Bellis- UOC di Gastroenterologia 1
Castellana Grotte
- Recruiting
UO epatologia Ospedale Garibaldi
Catania
- Recruiting
UO di fisiopatologia digestiva, Azienda Ospedaliera Universitaria Mater Domini
Catanzaro
- Recruiting
UO di gastroenterologia, Ospedale Valduce
Como
- Recruiting
Azienda Socio Sanitaria Territoriale (ASST) di Cremona- UO Medicina Interna - Ambulatori di Epatologia
Cremona
- Recruiting
Presidio Ospedaliero di Faenza- UO Medicina Interna
Faenza
- Recruiting
UO Clinica Medica, Azienda Ospedaliera Universitaria Careggi Firenze
Florence
- Recruiting
Centro C.U.R.E. Centro Universitario per la ricerca e la Cura delle Malattie Epatiche
Foggia
- Recruiting
Ambulatorio di Malattie del Fegato, U.O. Medicina, ASST Valle Olona, Presidio di Gallarate
Gallarate
- Recruiting
UO Clinica Gastroenterologica, Policlinico San Martino
Genova
- Recruiting
Centro Medicina del Viaggiatore e delle Migrazioni, ASP Catanzaro, Presìdio Ospedaliero "Giovanni Paolo II"
Lamezia Terme
- Not yet recruiting
UO di epatologia clinica e biomolecolare, Università degli studi di Messina
Messina
- Recruiting
University of Milano-Bicocca
Milan, 20126
Pietro Invernizzi, pietro.invernizzi@unimib.it
Marco Carbone, marco.carbone@unimib.it
- Recruiting
Policlinico di Milano
Milan
- Recruiting
UO di epatologia, Ospedale Niguarda Ca' Granda
Milan
- Recruiting
UO di Epatologia, Ospedale San Giuseppe
Milan
- Recruiting
UO epatologia, Istituto scientifico universitario San Raffaele
Milan
- Recruiting
AOU di Modena- U.O. Gastroenterologia
Modena
- Recruiting
Struttura Complessa di Medicina ad Indirizzo Metabolico Nutrizionale, Dipartimento Ospedaliero di Medicina Interna
Modena
- Not yet recruiting
Day Hospital e Ambulatorio di Epatoogia e Nutrizione Clinica, Fondazione Evangelica Villa Betania, Ospedale generale di Zona
Naples
- Recruiting
Ospedale policlinico Federico II di Napoli
Naples
- Recruiting
Università della Campania Luigi Vanvitelli di Napoli- Dipartimento di epato-gastroenterologia
Naples
- Recruiting
UO medicina ed epatologia, Ospedale maggiore della carità
Novara
- Recruiting
UO di Gastroenterologia, Azienda ospedaliera di Padova
Padova
- Recruiting
UO di Medicina Interna, Azienda Ospedaliera di Padova
Padova
- Recruiting
UO gastroenterologia, Policlinico Paolo Giaccone
Palermo
- Recruiting
Malattie infettive ed epatologia, Azienda ospedaliero universitaria di Parma
Parma
- Recruiting
Day Hospital Internistico e ambulatorio di Epatologia, ASL Pescara
Pescara
- Recruiting
UO epatologia, AOU pisana Cisanello
Pisa
- Recruiting
S.C. Gastroenterologia, Ospedale di Pordenone, AS FO
Pordenone
- Recruiting
Medicina clinica ed epatologia, Policlinico universitario campus bio-medico
Roma
- Recruiting
Policlinico Universitario Fondazione Agostino Gemelli- UO di medicina interna
Roma
- Recruiting
UO di Epatologia, Universita' di Roma Tor Vergata
Roma
- Recruiting
UO di Gastroenterologia, Policlinico Umberto I
Roma
- Recruiting
Medicina interna ed epatologia, Istituto clinico Humanitas
Rozzano
- Recruiting
Università degli studi di Salerno
Salerno
- Not yet recruiting
SSD Epatologia, Fondazione Casa Sollievo della Sofferenza
San Giovanni Rotondo
- Recruiting
UO di Gastroenterologia e Endoscopia Digestiva, Ospedale Casa Sollievo della Sofferenza
San Giovanni Rotondo
- Recruiting
Azienda Ospedale Universitaria di Sassari- UOC Medicina Interna
Sassari
- Recruiting
S.C. Gastroenterologia, Azienda Ospedaliero-Universitaria Molinette
Torino
- Recruiting
Gastroenterologia ed endoscopia digestiva, Azienda provinciale per i servizi sanitari
Trento
- Recruiting
Ospedale regionale Ca' Foncello di Treviso- Unità operativa complessa di Gastroenterologia
Treviso
- Recruiting
SC (UCO) Clinica Patologie del Fegato, Ospedale di Cattinara, ASU GI
Trieste
- Recruiting
SOS di DPT Epatologia e Trapianti di Fegato, ASU FC
Udine
- Recruiting
UO di gastroenterologia ed endoscopia digestiva, Azienda macchi, Asst sette laghi
Varese
- Recruiting
Azienda Ospedaliera Universitaria Integrata Verona- UOC Gastroeneterologia A
Verona
About this study
Official title: 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.
Read the 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.
- Conditions
- Primary Biliary Cholangitis
- Being tested
- Clinical information
- Keywords
- Primary Biliary Cholangitis, Disease phenotypes, National Database
- 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.
Clinical informationOther
Also called Laboratory information, Biological samples
The investigators will recruit PBC patients and collect important clinical information and laboratory investigation, together with biological samples.
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: Other: Clinical information |
What is being measured
The primary outcome is the main question the study is designed to answer.
Phenotypes and sub-phenotypes of PBC Italian patients
Identify and define distinct phenotypes and sub-phenotypes of PBC patients at higher risk of disease progression.
Measured: Overall duration of the study (10 years)
Secondary outcomes (3)
Response to UDCA therapy
Defined as Alkaline Phosphatase Level<1 x upper limit of normal. The investigators will fit a multivariate analysis using logistic regression using baseline variables.
Measured: Overall duration of the study (10 years)
Identification of factors influencing the progression of PBC
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.
Measured: Overall duration of the study (10 years)
Safety and long-term efficacy of novel therapies
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.
Measured: Overall duration of the study (10 years)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Dec 2021
- Study start19 Sep 2019
- Primary completion24 Jan 2029 (estimated)
- Study completion24 Jan 2029 (estimated)
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
- Prospective
- Enrolment
- 6,000 participants (estimated)
- Target duration
- 10 Years
Sponsor and investigators
- Lead sponsor
- University of Milano Bicocca
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05151809
- Sponsor's protocol ID
- PBC322
- Acronym
- PBC322
- Status last verified
- Jul 2025
- First submitted
- 15 Nov 2021
- Last updated
- 25 Jul 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 25 Jul 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Multicenter, Nationwide, Observational, Retrospective and Prospective Study Based on the Development of a Patients Database Linked to a Biological Sample Storage
- ClinicalTrials.gov ID
- NCT05151809
- Acronym / sponsor ID
- PBC322 / PBC322
- Conditions
- Primary Biliary Cholangitis
- Keywords
- Primary Biliary Cholangitis, Disease phenotypes, National Database
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 6,000 participants (estimated)
- Lead sponsor
- University of Milano BicoccaUniversity, hospital or other
- Study start
- 19 Sep 2019 (actual)
- Primary completion
- 24 Jan 2029 (estimated)
- Study completion
- 24 Jan 2029 (estimated)
- First posted
- 9 Dec 2021 (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) 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
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 design
- Observational model
- Cohort
- Time perspective
- Prospective
- Target follow-up
- 10 Years
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| PBC population residing in Italy | – | Other: Clinical informationAll 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. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- 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.
Key inclusion criteria (4 listed)
- 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.
Key exclusion criteria (1 listed)
- The patient has explicitly declared his/her unwillingness to participate to the study
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 |
|---|---|---|
| Clinical informationAlso: Laboratory information, Biological samplesThe investigators will recruit PBC patients and collect important clinical information and laboratory investigation, together with biological samples. | Other | PBC population residing in Italy |
Primary outcome
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.
3 secondary outcomes
Response to UDCA therapy
Time frame: Overall duration of the study (10 years)
Identification of factors influencing the progression of PBC
Time frame: Overall duration of the study (10 years)
Safety and long-term efficacy of novel therapies
Time frame: Overall duration of the study (10 years)
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 25 Jul 2025.
- Individual participant dataSharing planned: No
Related trials for Primary Biliary Cholangitis
RecruitingPhase 3
Seladelpar in Subjects With Primary Biliary Cholangitis (PBC) and Compensated Cirrhosis
Primary Biliary Cholangitis
RecruitingPhase 3
FIREFLY Trial: Fenofibrate Intervention---Randomized Evaluation in First-Line PBC Therapy
Primary Biliary Cholangitis (PBC)
RecruitingPhase 3
Fenofibrate for Patients With Primary Biliary Cirrhosis Who Had An Inadequate Response to Ursodeoxycholic Acid
Primary Biliary Cirrhosis
Not yet recruitingObservational study
A Study of Fatigue, Itching and Daily Activities in People With Primary Biliary Cholangitis Starting Elafibranor
Primary Biliary Cholangitis (PBC)
RecruitingPhase 2/3
Fenofibrate in Patients With Primary Biliary Cholangitis (PBC)
Primary Biliary Cholangitis
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.
55 sites 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 University of Milano Bicocca at 55 sites in Italy.
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 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 25 Jul 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 55 sites in Italy. 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 Pietro Invernizzi, MD (+39 039 233 2187, pietro.invernizzi@unimib.it), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Phenotypes and sub-phenotypes of PBC Italian patients (time frame: Overall duration of the study (10 years)). 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 25 Jul 2025.