An observational study in Primary Sclerosing Cholangitis, sponsored by University of Milano Bicocca. Recruiting at 63 sites in Italy. Open to participants aged 17 Years and older. Per ClinicalTrials.gov, last updated 2024-01-02.
Sponsored by University of Milano Bicocca · Observational
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.
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.
149 studies on the registry are indexed under Cholangitis, Sclerosing; 39 are open to participants now.
This study's planned enrollment of 6,000 is above the median of 200 across 46 observational studies indexed under Cholangitis, Sclerosing.
Browse Cholangitis, Sclerosing studies →University of Milano Bicocca is the lead sponsor of 126 studies on the registry; 47 are open to participants now.
Counted across the registry records on this site, refreshed daily.
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).
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
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.
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.
Time frame: Overall duration of the study (10 years)
Identification of factors associated with response to therapies;
Identification of factors associated with response to therapies;
Time frame: Overall duration of the study (10 years)
Identification of biomarkers influencing the progression of PSC and affecting mortality
Identification of biomarkers influencing the progression of PSC and affecting mortality
Time frame: Overall duration of the study (10 years)
Assessment of safety and long-term efficacy of novel therapies
Assessment of safety and long-term efficacy of novel therapies
Time frame: Overall duration of the study (10 years)
Started Oct 2022 (actual). Primary completion is estimated for Oct 2032, 120 months after the start.
How these figures are worked out, and which studies are left out of them.
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