National Database on Primary Sclerosing Cholangitis (PSC)

December 29, 2023 updated by: University of Milano Bicocca

Multicenter, Nationwide, Observational, Prospective Study Based on the Development of a Primary Sclerosing Cholangitis Patient's Database Linked to a Biological Sample Storage

Primary sclerosing cholangitis (PSC) a rare, chronic fibroinflammatory disease of the liver.

No data about the disease epidemiology exist in Italy. Therefore this study aims to develop a national PSC patient database linked to a biological sample storage.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Detailed Description

Primary sclerosing cholangitis is a chronic fibroinflammatory disease of the liver characterized by chronic inflammation and sclerosis of the intrahepatic and/or extrahepatic bile ducts, and a risk for progression to liver failure and development of colorectal and hepatobiliary cancer 1. Both children and adults are affected. Patients with PSC have a diminished life expectancy with a median survival of 17 years after diagnosis. PSC is the leading indication for transplantation in some European countries. Epidemiological studies have found the highest prevalence rates of PSC in Northern European countries and North America (United States and Canada) ranging between 3.85 to 16.2 per 100,000 persons. In Italy, the estimates is of 0.8 per 100,000 persons but these figures underestimate the real disease burden 2.

Liver transplantation is currently the only life-extending accepted therapy for patients with end-stage liver disease (ESLD) secondary to PSC, and patients with PSC complicated by CCA who meet specific criteria. PSC recurs in the transplanted liver in up to 40% of patients. Taken together and combined with patients' debilitating quality of life issues, these data highlight the considerable disease burden and clinical impact of PSC on patients' outcomes. There is a strong, yet poorly understood, relationship between PSC and IBD; nearly 70%-80% of PSC patients have IBD, mainly ulcerative colitis.

Despite the high mortality associated with PSC and the efforts to optimize its management, there is no medical therapy proven to halt the progression of PSC or prevent its serious complications.

There have been no epidemiologic studies in PSC carried out in Italy, with the exception of the report of prevalence from the National registry of rare disease which provided an underestimated prevalence of 500 patients across the country. However, this study is limited by the nature of the registry which has administrative purposes and has a high rate of underreported cases.

The aim of the study is to implement a nationwide data collection on this rare disease to describe incidence and prevalence of PSC in Italy; identify and define distinct phenotypes and sub-phenotypes of PSC patients; identify factors influencing the progression of PSC and affecting mortality; assess safety and long-term efficacy of novel therapies.

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.

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
        • Not yet recruiting
        • Ospedali Riuniti di Ancona
        • Contact:
          • Marco Marzioni
      • Bari, Italy
        • Not yet recruiting
        • Policlinico di Bari
        • Contact:
          • Antonino Castellaneta
      • Bergamo, Italy
        • Not yet recruiting
        • ASST Papa Giovanni XXIII
        • Contact:
          • Stefano Fagiuoli
      • Bergamo, Italy
        • Not yet recruiting
        • ASST Bergamo Ovest
        • Contact:
          • Silvia Colombo
      • Bologna, Italy
        • Not yet recruiting
        • Ospedale Sant'Orsola Malpighi
        • Contact:
          • Luigi Muratori
      • Bologna, Italy
        • Not yet recruiting
        • Policlinico S. Orsola Malpighi
        • Contact:
          • Francesco Azzaroli
      • Bolzano, Italy
        • Not yet recruiting
        • Azienda Sanitaria dell'Alto Adige
        • Contact:
          • Andrea Mega
      • Brescia, Italy
        • Not yet recruiting
        • Spedali Civili
        • Contact:
          • Chiara Ricci
      • Brescia, Italy
        • Not yet recruiting
        • Unità epatologica ed ecografia internistica a valenza dipartimentale, Fondazione Ospedaliera Poliambulanza
        • Contact:
          • Massimo Graffeo
      • Cagliari, Italy
        • Not yet recruiting
        • Policlinico Universitario Monserrato
        • Contact:
          • Luchino Chessa
      • Cagliari, Italy
        • Not yet recruiting
        • Presidio Ospedaliero San Michele ARNAS G. Brotzu
        • Contact:
          • Teresa Zolfino
      • Castellana Grotte, Italy
        • Not yet recruiting
        • Osp. "S de Bellis" - IRCCS
        • Contact:
          • Francesco Losito
      • Catania, Italy
        • Not yet recruiting
        • UO epatologia Ospedale Garibaldi
        • Contact:
          • Maurizio Russello
      • Catanzaro, Italy
        • Not yet recruiting
        • Azienda Ospedaliera Universitaria Mater Domini
        • Contact:
          • Ludovico Abenavoli, MD
      • Catanzaro, Italy
        • Not yet recruiting
        • Azienda Ospedaliera Universitaria- Mater Domini
        • Contact:
          • Benedetto Caroleo
      • Como, Italy
        • Not yet recruiting
        • Ospedale Valduce
        • Contact:
          • Natalia Terreni
      • Cosenza, Italy
        • Not yet recruiting
        • Unità operativa complessa di medicina interna- Azienda ospedaliera di Cosenza
        • Contact:
          • Rossana De Marco
      • Cremona, Italy
        • Not yet recruiting
        • ASST di Cremona
        • Contact:
          • Ilaria Cavalli
      • Cuneo, Italy
        • Not yet recruiting
        • Ospedale Santa Croce
        • Contact:
          • Alberto Mattalia
      • Faenza, Italy
        • Not yet recruiting
        • Presidio Ospedaliero di Faenza (RA)
        • Contact:
          • Lucia Napoli
      • Ferrara, Italy
        • Not yet recruiting
        • Azienda Ospedaliero-Universitaria di Ferrara
        • Contact:
          • Loredana Simone
      • Firenze, Italy
        • Not yet recruiting
        • Azienda Ospedaliera Universitaria Careggi
        • Contact:
          • Andrea Galli
      • Firenze, Italy
        • Not yet recruiting
        • Azienda ospedaliera universitaria careggi Firenze
        • Contact:
          • Fabio Marra
      • Foggia, Italy
        • Not yet recruiting
        • Centro C.U.R.E. Centro Universitario per la ricerca e la Cura delle Malattie Epatiche
        • Contact:
          • Francesco Bellanti
      • Foggia, Italy
        • Not yet recruiting
        • Ospedale Casa Sollievo della Sofferenza
        • Contact:
          • Grazia Niro
      • Gallarate, Italy
        • Not yet recruiting
        • ASST Valle Olona
        • Contact:
          • Cinzia Simoni
      • Genova, Italy
        • Not yet recruiting
        • Policlinico San Martino
        • Contact:
          • Edoardo Giovanni Giannini
      • Lamezia Terme, Italy
        • Not yet recruiting
        • Presìdio Ospedaliero "Giovanni Paolo II"
        • Contact:
          • Lorenzo Antonio Surace
      • Lecco, Italy
        • Not yet recruiting
        • ASST Lecco
        • Contact:
          • Roberta Pozzi
      • Messina, Italy
        • Not yet recruiting
        • UO di epatologia clinica e biomolecolare. Università degli studi di Messina
        • Contact:
          • Giovanni Raimondo
      • Milano, Italy
        • Not yet recruiting
        • Az. Ospedaliera San Paolo- Polo universitario
        • Contact:
          • Piermaria Battezzati
      • Milano, Italy
        • Not yet recruiting
        • Istituto scientifico universitario San Raffaele
        • Contact:
          • Massimo Memoli
      • Milano, Italy
        • Not yet recruiting
        • Ospedale Niguarda Ca' Granda
        • Contact:
          • Luca Belli
      • Milano, Italy
        • Not yet recruiting
        • Ospedale San Giuseppe
        • Contact:
          • Mauro Viganò
      • Milano, Italy
        • Not yet recruiting
        • Policlinico di Milano
        • Contact:
          • Pietro Lampertico
      • Modena, Italy
        • Not yet recruiting
        • Dipartimento Ospedaliero di Medicina Interna
        • Contact:
          • Pietro Andreone, MD
      • Monza, Italy, 20900
        • Recruiting
        • ASST Monza
        • Contact:
        • Principal Investigator:
          • Pietro Invernizzi, MD
      • Napoli, Italy
        • Not yet recruiting
        • Fondazione Evangelica Villa Betania, Ospedale generale di Zona
        • Contact:
          • Ernesto Claar
      • Napoli, Italy
        • Not yet recruiting
        • Ospedale policlinico Federico II di Napoli
        • Contact:
          • Filomena Morisco
      • Napoli, Italy
        • Not yet recruiting
        • Università della Campania Luigi Vanvitelli
        • Contact:
          • Alessandro Federico
      • Novara, Italy
        • Not yet recruiting
        • Ospedale Maggiore della Carità
        • Contact:
          • Cristina Rigamonti
      • Padova, Italy
        • Not yet recruiting
        • Azienda Ospedaliera Di Padova
        • Contact:
          • Luca Fabris
      • Padova, Italy
        • Not yet recruiting
        • UO di Gastroenterologia Azienda ospedaliera di Padova
        • Contact:
          • Nora Cazzagon
      • Palermo, Italy
        • Not yet recruiting
        • Policlinico Paolo Giaccone
        • Contact:
          • Vincenza Calvaruso
      • Parma, Italy
        • Not yet recruiting
        • Azienda Ospedaliero Universitaria di Parma
        • Contact:
          • Gabriele Missale
      • Pavia, Italy
        • Not yet recruiting
        • Istituto Clinico Città di Pavia-Gruppo Sandonato
        • Contact:
          • Guido Poggi
      • Perugia, Italy
        • Not yet recruiting
        • Azienda Ospedaliera di Perugia
        • Contact:
          • Olivia Morelli
      • Pescara, Italy
        • Not yet recruiting
        • Day Hospital Internistico e ambulatorio di Epatologia, ASL Pescara
        • Contact:
          • Valeria Pace Palitti
      • Pisa, Italy
        • Not yet recruiting
        • AOU Pisana Cisanello
        • Contact:
          • Maurizia Brunetto
      • Pordenone, Italy
        • Not yet recruiting
        • Ospedale di Pordenone AS FO
        • Contact:
          • Anna Baragiotta
      • Potenza, Italy
        • Not yet recruiting
        • Ospedale San Carlo
        • Contact:
          • Nello Buccianti
      • Rimini, Italy
        • Not yet recruiting
        • Azienda Unità Sanitaria Locale della Romagna
        • Contact:
          • Giorgio Bellardini
      • Roma, Italy
        • Not yet recruiting
        • Ospedale A. Gemelli
        • Contact:
          • Luca Miele
      • Roma, Italy
        • Not yet recruiting
        • Policlinico Umberto I
        • Contact:
          • Domentico MD Alvaro
      • Roma, Italy
        • Not yet recruiting
        • Policlinico Universitario Campus Bio-Medico
        • Contact:
          • Umberto Vespasiani Gentilucci
      • Roma, Italy
        • Not yet recruiting
        • Universita' di Roma Tor Vergata
        • Contact:
          • Leonardo Baiocchi
      • Rozzano, Italy
        • Not yet recruiting
        • Istituto Clinico Humanitas
        • Contact:
          • Ana Lleo De Nalda
      • Salerno, Italy
        • Not yet recruiting
        • Università degli studi di Salerno
        • Contact:
          • Marcello Persico
      • Torino, Italy
        • Not yet recruiting
        • Azienda Ospedaliero-Universitaria Molinette
        • Contact:
          • Ester Vanni
      • Trento, Italy
        • Not yet recruiting
        • Azienda provinciale per i servizi sanitari
        • Contact:
          • Giovanni Vettori
      • Trieste, Italy
        • Not yet recruiting
        • Ospedale di Cattinara, ASU GI
        • Contact:
          • Saveria Crocè
      • Udine, Italy
        • Not yet recruiting
        • ASU FC
        • Contact:
          • Pierluigi Tonitutto
      • Varese, Italy
        • Not yet recruiting
        • ASST Sette Laghi
        • Contact:
          • Sergio Segato

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

15 years and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Sampling Method

Probability Sample

Study Population

All PSC patients living in Italy and aged at least 17 years can be included in the database. Willing and able to give informed consent prior to any study specific procedure being performed; Diagnosis of PSC according to the most recent published guidelines (EASL).

Description

Inclusion Criteria:

  • Willing and able to give informed consent prior to any study specific procedure being performed;
  • Diagnosis of PSC according to the most recent published guidelines (EASL);

Exclusion Criteria:

  • Subject unwilling to participate at 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
PSC population residing in Italy
All PSC patients living in Italy and aged at least 17 years can be included in the database.
Other: Clinical information
The investigators will recruit PSC patients and collect important clinical information and laboratory investigation, together with biological samples.

What is the study measuring?

Primary Outcome Measures

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

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Identification of factors associated with response to therapies;
Time Frame: Overall duration of the study (10 years)
Identification of factors associated with response to therapies;
Overall duration of the study (10 years)
Identification of biomarkers influencing the progression of PSC and affecting mortality
Time Frame: Overall duration of the study (10 years)
Identification of biomarkers influencing the progression of PSC and affecting mortality
Overall duration of the study (10 years)
Assessment of safety and long-term efficacy of novel therapies
Time Frame: Overall duration of the study (10 years)
Assessment of safety and long-term efficacy of novel therapies
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

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)

October 21, 2022

Primary Completion (Estimated)

October 21, 2032

Study Completion (Estimated)

October 21, 2032

Study Registration Dates

First Submitted

November 8, 2022

First Submitted That Met QC Criteria

November 8, 2022

First Posted (Actual)

November 16, 2022

Study Record Updates

Last Update Posted (Estimated)

January 1, 2024

Last Update Submitted That Met QC Criteria

December 29, 2023

Last Verified

December 1, 2023

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • PSC_Database

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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