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Recruiting NCT07317193

NCT07317193 DEFINING THE GENETIC DRIVERS OF ADULT-ONSET CHOLESTATIC LIVER DISEASE

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Clinical Trial Summary
NCT ID NCT07317193
Status Recruiting
Phase
Sponsor Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico
Condition Cholestatic Liver Disease
Study Type INTERVENTIONAL
Enrollment 60 participants
Start Date 2025-11-01
Primary Completion 2026-01-31

Eligibility & Interventions

Sex All sexes
Min Age 18 Years
Max Age 65 Years
Study Type INTERVENTIONAL
Interventions
Advanced Whole Genome Sequencing to Identify Rare Pathogenic Variants in Unexplained Cholestatic Liver Disease

Eligibility Fast-Check

Enter your details for a quick preliminary check. This does not replace medical advice.

What to Expect as a Participant

You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.

This trial targets 60 participants in total. It began in 2025-11-01 with a primary completion date of 2026-01-31.

⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.

Brief Summary

Cholestatic disease in adults comprises a heterogeneous group of conditions characterized by intra- or extrahepatic alterations of bile flow that can lead to fibrosis or hepatic decompensation. Due to the heterogeneity of clinical manifestation, which is sometimes very subtle, diagnosis based on clinical, histological, and radiological evaluation is often very complicated. Genetic testing can be helpful in identifying the cause of the clinical phenotype, thereby allowing for targeted follow-up adequate to the patient's specific characteristics and risk factors. Although the utility of genetic analysis has been well documented for other liver diseases or in pediatric cohorts of children with cholestatic disease, the use and benefits of genetic testing in adults with cholestatic disease are still little explored and investigated. In this context, through the use of whole-genome sequencing (WGS), the FIRST project aims to evaluate the role of rare genetic variants in the pathogenesis of cholestatic disease and the utility of WGS in defining a genetic diagnosis.

Eligibility Criteria

Inclusion Criteria: Cases: * Adults, aged \> 18 years with: 1. persistent or intermittent elevations in serum alkaline phosphatase (ALP) or gamma-glutamyltransferase (GGT) for at least six months not explained following standard diagnostic assessment adhering to the guidelines of the European Association for the Study of the Liver (EASL), or with a positive family history of unexplained cholestasis or hepato-biliary cancer, negative to previous genetic tests (targeted panel for PFIC genes or WES); 2. primary sclerosing cholangitis (PSC) with unusual features: small-duct PSC, non-typical radiological findings according to radiological guidelines on PSC, absence of concomitant inflammatory bowel disease, negative to previous genetic tests (targeted panel for PFIC genes or WES) or who didn't perform previous genetic test; 3. primary biliary cholangitis (PBC) without specific anti-mitochondrial antibodies, negative to previous genetic tests (targeted panel for PFIC genes or WES) or who didn't perform previous genetic test. * Signature of informed consent Controls: -Blood donors (age 18-65 years) without clinical signs of liver diseases based on the collected clinical parameters: anthropometric (BMI\>18 and \<25), haematological (Hb, white blood cells, platelets within the reference range), biochemical traits (albumin, bilirubin, AST, ALT, GGT, ALP within the reference range), medical history (negative for chronic or concomitant diseases, including immunological diseases) Exclusion Criteria: Cases: * Patients who do not possess the above inclusion criteria or have at least one of the following exclusion criteria: * an already known genetic diagnosis explaining the clinical phenotype * affected by other causes of liver disease such as viral or autoimmune hepatitis Controls: -Blood donors with clinical signs of liver diseases

Contact & Investigator

Central Contact

Luisa Ronzoni, Doctor

✉ luisa.ronzoni@policlinico.mi.it

📞 02.5503.4101

Frequently Asked Questions

Who can join the NCT07317193 clinical trial?

This trial is open to participants of all sexes, aged 18 Years or older, up to 65 Years, studying Cholestatic Liver Disease. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.

Is NCT07317193 currently recruiting?

Yes, NCT07317193 is actively recruiting participants. Contact the research team at luisa.ronzoni@policlinico.mi.it for enrollment information.

Where is the NCT07317193 trial being conducted?

This trial is being conducted at Milan, Italy.

Who is sponsoring the NCT07317193 clinical trial?

NCT07317193 is sponsored by Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico. The trial plans to enroll 60 participants.

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