Primary Sclerosing CholangitisNCT05618145
National Database on Primary Sclerosing Cholangitis (PSC)
An observational study of Primary Sclerosing Cholangitis, run by University of Milano Bicocca at 63 sites in Italy.
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. Read more about this study
- Who is it for?
- Ages 17 years and older
- Conditions the study is for
- Primary Sclerosing Cholangitis
- What is being tested?
- Clinical information
- How many people
- 6,000 participants
- Where
- 63 sitesItaly
- Study dates
- 21 Oct 2022to 21 Oct 2032
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 17 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
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).
Who may be able to join
- 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);
Who cannot join
- Subject unwilling to participate at the study
Trial locations and contacts
63 sites in 1 country. Contact the study team or a site near you to ask about taking part.
63 sites in 1 country
Italy63 sites, 63 recruiting
- Not yet recruiting
Ospedali riuniti di Ancona
Ancona
Marco Marzioni
- Not yet recruiting
Policlinico di Bari
Bari
Antonino Castellaneta
- Not yet recruiting
ASST Bergamo ovest
Bergamo
Silvia Colombo
- Not yet recruiting
ASST Papa Giovanni XXIII
Bergamo
Stefano Fagiuoli
- Not yet recruiting
Ospedale Sant'Orsola Malpighi
Bologna
Luigi Muratori
- Not yet recruiting
Policlinico S. Orsola Malpighi
Bologna
Francesco Azzaroli
- Not yet recruiting
Azienda sanitaria dell'Alto Adige
Bolzano
Andrea Mega
- Not yet recruiting
Spedali civili
Brescia
Chiara Ricci
- Not yet recruiting
Unità epatologica ed ecografia internistica a valenza dipartimentale, Fondazione Ospedaliera Poliambulanza
Brescia
Massimo Graffeo
- Not yet recruiting
Policlinico universitario Monserrato
Cagliari
Luchino Chessa
- Not yet recruiting
Presidio Ospedaliero San Michele ARNAS G. Brotzu
Cagliari
Teresa Zolfino
- Not yet recruiting
Osp. "S de Bellis" - IRCCS
Castellana Grotte
Francesco Losito
- Not yet recruiting
UO epatologia Ospedale Garibaldi
Catania
Maurizio Russello
- Not yet recruiting
Azienda Ospedaliera Universitaria Mater Domini
Catanzaro
Ludovico Abenavoli, MD
- Not yet recruiting
Azienda Ospedaliera Universitaria- Mater Domini
Catanzaro
Benedetto Caroleo
- Not yet recruiting
Ospedale Valduce
Como
Natalia Terreni
- Not yet recruiting
Unità operativa complessa di medicina interna- Azienda ospedaliera di Cosenza
Cosenza
Rossana De Marco
- Not yet recruiting
ASST di Cremona
Cremona
Ilaria Cavalli
- Not yet recruiting
Ospedale santa croce
Cuneo
Alberto Mattalia
- Not yet recruiting
Presidio Ospedaliero di Faenza (RA)
Faenza
Lucia Napoli
- Not yet recruiting
Azienda Ospedaliero-Universitaria di Ferrara
Ferrara
Loredana Simone
- Not yet recruiting
Azienda ospedaliera universitaria careggi Firenze
Florence
Fabio Marra
- Not yet recruiting
Azienda ospedaliera universitaria Careggi
Florence
Andrea Galli
- Not yet recruiting
Centro C.U.R.E. Centro Universitario per la ricerca e la Cura delle Malattie Epatiche
Foggia
Francesco Bellanti
- Not yet recruiting
Ospedale Casa Sollievo della Sofferenza
Foggia
Grazia Niro
- Not yet recruiting
ASST Valle Olona
Gallarate
Cinzia Simoni
- Not yet recruiting
Policlinico San Martino
Genova
Edoardo Giovanni Giannini
- Not yet recruiting
Presìdio Ospedaliero "Giovanni Paolo II"
Lamezia Terme
Lorenzo Antonio Surace
- Not yet recruiting
ASST Lecco
Lecco
Roberta Pozzi
- Not yet recruiting
UO di epatologia clinica e biomolecolare. Università degli studi di Messina
Messina
Giovanni Raimondo
- Not yet recruiting
Az. Ospedaliera San Paolo- Polo universitario
Milan
Piermaria Battezzati
- Not yet recruiting
Istituto scientifico universitario San Raffaele
Milan
Massimo Memoli
- Not yet recruiting
Ospedale Niguarda Ca' Granda
Milan
Luca Belli
- Not yet recruiting
Ospedale San Giuseppe
Milan
Mauro Viganò
- Not yet recruiting
Policlinico di Milano
Milan
Pietro Lampertico
- Not yet recruiting
Dipartimento Ospedaliero di Medicina Interna
Modena
Pietro Andreone, MD
- Recruiting
ASST Monza
Monza, 20900
Pietro Invernizzi, MD, pietro.invernizzi@unimib.it
Pietro Invernizzi, MD
- Not yet recruiting
Fondazione Evangelica Villa Betania, Ospedale generale di Zona
Naples
Ernesto Claar
- Not yet recruiting
Ospedale policlinico Federico II di Napoli
Naples
Filomena Morisco
- Not yet recruiting
Università della Campania Luigi Vanvitelli
Naples
Alessandro Federico
- Not yet recruiting
Ospedale maggiore della carità
Novara
Cristina Rigamonti
- Not yet recruiting
Azienda Ospedaliera di Padova
Padova
Luca Fabris
- Not yet recruiting
UO di Gastroenterologia Azienda ospedaliera di Padova
Padova
Nora Cazzagon
- Not yet recruiting
Policlinico Paolo Giaccone
Palermo
Vincenza Calvaruso
- Not yet recruiting
Azienda ospedaliero universitaria di Parma
Parma
Gabriele Missale
- Not yet recruiting
Istituto Clinico Città di Pavia-Gruppo Sandonato
Pavia
Guido Poggi
- Not yet recruiting
Azienda ospedaliera di Perugia
Perugia
Olivia Morelli
- Not yet recruiting
Day Hospital Internistico e ambulatorio di Epatologia, ASL Pescara
Pescara
Valeria Pace Palitti
- Not yet recruiting
Aou pisana Cisanello
Pisa
Maurizia Brunetto
- Not yet recruiting
Ospedale di Pordenone AS FO
Pordenone
Anna Baragiotta
- Not yet recruiting
Ospedale San Carlo
Potenza
Nello Buccianti
- Not yet recruiting
Azienda unità sanitaria locale della Romagna
Rimini
Giorgio Bellardini
- Not yet recruiting
Ospedale A. Gemelli
Roma
Luca Miele
- Not yet recruiting
Policlinico Umberto I
Roma
Domentico MD Alvaro
- Not yet recruiting
Policlinico universitario campus bio-medico
Roma
Umberto Vespasiani Gentilucci
- Not yet recruiting
Universita' di Roma Tor Vergata
Roma
Leonardo Baiocchi
- Not yet recruiting
Istituto clinico Humanitas
Rozzano
Ana Lleo De Nalda
- Not yet recruiting
Università degli studi di Salerno
Salerno
Marcello Persico
- Not yet recruiting
Azienda Ospedaliero-Universitaria Molinette
Torino
Ester Vanni
- Not yet recruiting
Azienda provinciale per i servizi sanitari
Trento
Giovanni Vettori
- Not yet recruiting
Ospedale di Cattinara, ASU GI
Trieste
Saveria Crocè
- Not yet recruiting
ASU FC
Udine
Pierluigi Tonitutto
- Not yet recruiting
ASST Sette Laghi
Varese
Sergio Segato
About this study
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
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.
Read the 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.
- Conditions
- Primary Sclerosing Cholangitis
- Being tested
- Clinical information
- Keywords
- Primary Sclerosing Cholangitis, Disease phenotypes, National Database
- Medical subject headings
- Cholangitis, Sclerosing
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Clinical informationOther
The investigators will recruit PSC patients and collect important clinical information and laboratory investigation, together with biological samples.
| Study group | Role | What they receive |
|---|---|---|
| PSC population residing in Italy | All PSC patients living in Italy and aged at least 17 years can be included in the database. Receives: Other: Clinical information |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: Overall duration of the study (10 years)
Secondary outcomes (3)
Identification of factors associated with response to therapies;
Identification of factors associated with response to therapies;
Measured: 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
Measured: 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
Measured: Overall duration of the study (10 years)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Nov 2022
- Study start21 Oct 2022
- Primary completion21 Oct 2032 (estimated)
- Study completion21 Oct 2032 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Primary Sclerosing CholangitisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 6,000 participants (estimated)
Sponsor and investigators
- Lead sponsor
- University of Milano Bicocca
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05618145
- Sponsor's protocol ID
- PSC_Database
- Status last verified
- Dec 2023
- First submitted
- 8 Nov 2022
- Last updated
- 2 Jan 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 2 Jan 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT05618145
- Acronym / sponsor ID
- PSC_Database
- Conditions
- Primary Sclerosing Cholangitis
- Keywords
- Primary Sclerosing Cholangitis, Disease phenotypes, National Database
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 6,000 participants (estimated)
- Lead sponsor
- University of Milano BicoccaUniversity, hospital or other
- Study start
- 21 Oct 2022 (actual)
- Primary completion
- 21 Oct 2032 (estimated)
- Study completion
- 21 Oct 2032 (estimated)
- First posted
- 16 Nov 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| PSC population residing in Italy | – | Other: Clinical informationAll PSC patients living in Italy and aged at least 17 years can be included in the database. |
Who may qualify
- Ages
- 17 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- 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).
Key inclusion criteria (2 listed)
- 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);
Key exclusion criteria (1 listed)
- Subject unwilling to participate at the study
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Clinical informationThe investigators will recruit PSC patients and collect important clinical information and laboratory investigation, together with biological samples. | Other | PSC population residing in Italy |
Primary outcome
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.
3 secondary outcomes
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
Time frame: 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)
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 Jan 2024.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
63 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Primary Sclerosing Cholangitis, run by University of Milano Bicocca at 63 sites in Italy.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 2 Jan 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 63 sites in Italy. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Pietro Invernizzi, MD (039 233 2187, pietro.invernizzi@unimib.it), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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)). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 2 Jan 2024.