National Database on Primary Sclerosing Cholangitis (PSC)

Primary Sclerosing Cholangitis Clinical Trial

Official title:

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05618145
• Other study ID #: PSC_Database
• Status: Recruiting
• Start date: October 21, 2022
• Est. completion date: October 21, 2032

Study information

• Verified date: December 2023
• Source: University of Milano Bicocca
• Contact: Pietro Invernizzi, MD
• Phone: 039 233 2187
• Email: pietro.invernizzi[@]unimib.it
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

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 information / eligibility

• Status: Recruiting
• Enrollment: 6000
• Est. completion date: October 21, 2032
• Est. primary completion date: October 21, 2032
• Accepts healthy volunteers: No
• Gender: All
• Age group: 17 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Cholangitis
• Cholangitis, Sclerosing
• Primary Sclerosing Cholangitis

Intervention

Other:
• Clinical information
The investigators will recruit PSC patients and collect important clinical information and laboratory investigation, together with biological samples.

Locations

Country	Name	City	State
Italy	Ospedali riuniti di Ancona	Ancona
Italy	Policlinico di Bari	Bari
Italy	ASST Bergamo ovest	Bergamo
Italy	ASST Papa Giovanni XXIII	Bergamo
Italy	Ospedale Sant'Orsola Malpighi	Bologna
Italy	Policlinico S. Orsola Malpighi	Bologna
Italy	Azienda sanitaria dell'Alto Adige	Bolzano
Italy	Spedali civili	Brescia
Italy	Unità epatologica ed ecografia internistica a valenza dipartimentale, Fondazione Ospedaliera Poliambulanza	Brescia
Italy	Policlinico universitario Monserrato	Cagliari
Italy	Presidio Ospedaliero San Michele ARNAS G. Brotzu	Cagliari
Italy	Osp. "S de Bellis" - IRCCS	Castellana Grotte
Italy	UO epatologia Ospedale Garibaldi	Catania
Italy	Azienda Ospedaliera Universitaria Mater Domini	Catanzaro
Italy	Azienda Ospedaliera Universitaria- Mater Domini	Catanzaro
Italy	Ospedale Valduce	Como
Italy	Unità operativa complessa di medicina interna- Azienda ospedaliera di Cosenza	Cosenza
Italy	ASST di Cremona	Cremona
Italy	Ospedale santa croce	Cuneo
Italy	Presidio Ospedaliero di Faenza (RA)	Faenza
Italy	Azienda Ospedaliero-Universitaria di Ferrara	Ferrara
Italy	Azienda ospedaliera universitaria Careggi	Firenze
Italy	Azienda ospedaliera universitaria careggi Firenze	Firenze
Italy	Centro C.U.R.E. Centro Universitario per la ricerca e la Cura delle Malattie Epatiche	Foggia
Italy	Ospedale Casa Sollievo della Sofferenza	Foggia
Italy	ASST Valle Olona	Gallarate
Italy	Policlinico San Martino	Genova
Italy	Presìdio Ospedaliero "Giovanni Paolo II"	Lamezia Terme
Italy	ASST Lecco	Lecco
Italy	UO di epatologia clinica e biomolecolare. Università degli studi di Messina	Messina
Italy	Az. Ospedaliera San Paolo- Polo universitario	Milano
Italy	Istituto scientifico universitario San Raffaele	Milano
Italy	Ospedale Niguarda Ca' Granda	Milano
Italy	Ospedale San Giuseppe	Milano
Italy	Policlinico di Milano	Milano
Italy	Dipartimento Ospedaliero di Medicina Interna	Modena
Italy	ASST Monza	Monza
Italy	Fondazione Evangelica Villa Betania, Ospedale generale di Zona	Napoli
Italy	Ospedale policlinico Federico II di Napoli	Napoli
Italy	Università della Campania Luigi Vanvitelli	Napoli
Italy	Ospedale maggiore della carità	Novara
Italy	Azienda Ospedaliera di Padova	Padova
Italy	UO di Gastroenterologia Azienda ospedaliera di Padova	Padova
Italy	Policlinico Paolo Giaccone	Palermo
Italy	Azienda ospedaliero universitaria di Parma	Parma
Italy	Istituto Clinico Città di Pavia-Gruppo Sandonato	Pavia
Italy	Azienda ospedaliera di Perugia	Perugia
Italy	Day Hospital Internistico e ambulatorio di Epatologia, ASL Pescara	Pescara
Italy	Aou pisana Cisanello	Pisa
Italy	Ospedale di Pordenone AS FO	Pordenone
Italy	Ospedale San Carlo	Potenza
Italy	Azienda unità sanitaria locale della Romagna	Rimini
Italy	Ospedale A. Gemelli	Roma
Italy	Policlinico Umberto I	Roma
Italy	Policlinico universitario campus bio-medico	Roma
Italy	Universita' di Roma Tor Vergata	Roma
Italy	Istituto clinico Humanitas	Rozzano
Italy	Università degli studi di Salerno	Salerno
Italy	Azienda Ospedaliero-Universitaria Molinette	Torino
Italy	Azienda provinciale per i servizi sanitari	Trento
Italy	Ospedale di Cattinara, ASU GI	Trieste
Italy	ASU FC	Udine
Italy	ASST Sette Laghi	Varese

Sponsors (1)

Lead Sponsor	Collaborator
University of Milano Bicocca

Country where clinical trial is conducted

Italy, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Identify and define distinct phenotypes and sub-phenotypes of PSC patients at higher risk of disease progression.	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	Identification of factors associated with response to therapies;	Identification of factors associated with response to therapies;	Overall duration of the study (10 years)
Secondary	Identification of biomarkers influencing the progression of PSC and affecting mortality	Identification of biomarkers influencing the progression of PSC and affecting mortality	Overall duration of the study (10 years)
Secondary	Assessment of safety and long-term efficacy of novel therapies	Assessment of safety and long-term efficacy of novel therapies	Overall duration of the study (10 years)