National Database on Primary Sclerosing Cholangitis (PSC)

Recruiting

• Conditions: Primary Sclerosing Cholangitis

• 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.

Study Timeline

• Study Start: 2022-10-21
• Study First Submitted: 2022-11-08
• Study First Submitted QC: 2022-11-08
• Study First Posted: 2022-11-16
• Status Verified: 2023-12
• Last Update Submitted: 2023-12-29
• Last Update Posted: 2024-01-02
• Primary Completion: 2032-10-21
• Study Completion: 2032-10-21

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 6000

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 & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

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)

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

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