National Database on Primary Sclerosing Cholangitis (PSC)
- Conditions
- Primary Sclerosing Cholangitis
- Interventions
- Other: Clinical information
- Registration Number
- NCT05618145
- Lead Sponsor
- University of Milano Bicocca
- Brief Summary
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.
- 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.
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 6000
- 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);
- Subject unwilling to participate at the study
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Arm && Interventions
Group Intervention Description PSC population residing in Italy Clinical information All PSC patients living in Italy and aged at least 17 years can be included in the database.
- Primary Outcome Measures
Name Time Method Identify and define distinct phenotypes and sub-phenotypes of PSC patients at higher risk of disease progression. Overall duration of the study (10 years) Identify and define distinct phenotypes and sub-phenotypes of PSC patients at higher risk of disease progression.
- Secondary Outcome Measures
Name Time Method Identification of factors associated with response to therapies; Overall duration of the study (10 years) Identification of factors associated with response to therapies;
Identification of biomarkers influencing the progression of PSC and affecting mortality Overall duration of the study (10 years) Identification of biomarkers influencing the progression of PSC and affecting mortality
Assessment of safety and long-term efficacy of novel therapies Overall duration of the study (10 years) Assessment of safety and long-term efficacy of novel therapies
Trial Locations
- Locations (63)
Presidio Ospedaliero San Michele ARNAS G. Brotzu
🇮🇹Cagliari, Italy
Azienda Ospedaliera Universitaria- Mater Domini
🇮🇹Catanzaro, Italy
Ospedale Casa Sollievo della Sofferenza
🇮🇹Foggia, Italy
Ospedali riuniti di Ancona
🇮🇹Ancona, Italy
ASST Bergamo ovest
🇮🇹Bergamo, Italy
Azienda sanitaria dell'Alto Adige
🇮🇹Bolzano, Italy
Policlinico di Bari
🇮🇹Bari, Italy
ASST Papa Giovanni XXIII
🇮🇹Bergamo, Italy
Spedali civili
🇮🇹Brescia, Italy
UO epatologia Ospedale Garibaldi
🇮🇹Catania, Italy
Azienda ospedaliera universitaria careggi Firenze
🇮🇹Firenze, Italy
Unità epatologica ed ecografia internistica a valenza dipartimentale, Fondazione Ospedaliera Poliambulanza
🇮🇹Brescia, Italy
Osp. "S de Bellis" - IRCCS
🇮🇹Castellana Grotte, Italy
Ospedale Valduce
🇮🇹Como, Italy
Ospedale santa croce
🇮🇹Cuneo, Italy
Presidio Ospedaliero di Faenza (RA)
🇮🇹Faenza, Italy
Ospedale Sant'Orsola Malpighi
🇮🇹Bologna, Italy
Policlinico S. Orsola Malpighi
🇮🇹Bologna, Italy
Unità operativa complessa di medicina interna- Azienda ospedaliera di Cosenza
🇮🇹Cosenza, Italy
Centro C.U.R.E. Centro Universitario per la ricerca e la Cura delle Malattie Epatiche
🇮🇹Foggia, Italy
ASST Lecco
🇮🇹Lecco, Italy
Ospedale policlinico Federico II di Napoli
🇮🇹Napoli, Italy
Azienda unità sanitaria locale della Romagna
🇮🇹Rimini, Italy
Policlinico Umberto I
🇮🇹Roma, Italy
Policlinico universitario Monserrato
🇮🇹Cagliari, Italy
Azienda Ospedaliera Universitaria Mater Domini
🇮🇹Catanzaro, Italy
ASST di Cremona
🇮🇹Cremona, Italy
Azienda Ospedaliero-Universitaria di Ferrara
🇮🇹Ferrara, Italy
Azienda ospedaliera universitaria Careggi
🇮🇹Firenze, Italy
Policlinico San Martino
🇮🇹Genova, Italy
ASST Monza
🇮🇹Monza, Italy
Università della Campania Luigi Vanvitelli
🇮🇹Napoli, Italy
Ospedale maggiore della carità
🇮🇹Novara, Italy
Policlinico Paolo Giaccone
🇮🇹Palermo, Italy
Day Hospital Internistico e ambulatorio di Epatologia, ASL Pescara
🇮🇹Pescara, Italy
Azienda Ospedaliera di Padova
🇮🇹Padova, Italy
UO di Gastroenterologia Azienda ospedaliera di Padova
🇮🇹Padova, Italy
Azienda ospedaliera di Perugia
🇮🇹Perugia, Italy
Aou pisana Cisanello
🇮🇹Pisa, Italy
Ospedale San Carlo
🇮🇹Potenza, Italy
Ospedale di Cattinara, ASU GI
🇮🇹Trieste, Italy
Ospedale San Giuseppe
🇮🇹Milano, Italy
ASST Valle Olona
🇮🇹Gallarate, Italy
Presìdio Ospedaliero "Giovanni Paolo II"
🇮🇹Lamezia Terme, Italy
UO di epatologia clinica e biomolecolare. Università degli studi di Messina
🇮🇹Messina, Italy
Istituto scientifico universitario San Raffaele
🇮🇹Milano, Italy
Ospedale Niguarda Ca' Granda
🇮🇹Milano, Italy
Dipartimento Ospedaliero di Medicina Interna
🇮🇹Modena, Italy
Istituto Clinico Città di Pavia-Gruppo Sandonato
🇮🇹Pavia, Italy
Istituto clinico Humanitas
🇮🇹Rozzano, Italy
Università degli studi di Salerno
🇮🇹Salerno, Italy
Policlinico universitario campus bio-medico
🇮🇹Roma, Italy
Universita' di Roma Tor Vergata
🇮🇹Roma, Italy
Az. Ospedaliera San Paolo- Polo universitario
🇮🇹Milano, Italy
Policlinico di Milano
🇮🇹Milano, Italy
Fondazione Evangelica Villa Betania, Ospedale generale di Zona
🇮🇹Napoli, Italy
Azienda ospedaliero universitaria di Parma
🇮🇹Parma, Italy
Ospedale di Pordenone AS FO
🇮🇹Pordenone, Italy
Azienda Ospedaliero-Universitaria Molinette
🇮🇹Torino, Italy
Azienda provinciale per i servizi sanitari
🇮🇹Trento, Italy
ASU FC
🇮🇹Udine, Italy
ASST Sette Laghi
🇮🇹Varese, Italy
Ospedale A. Gemelli
🇮🇹Roma, Italy