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A randomized, double-blind, placebo-controlled clinical trial assessing the efficacy of combining pasireotide with aspiration sclerotherapy to improve volume reduction of dominant hepatic cysts

The primary objective is to minimize fluid reaccumulation in the hepatic cyst after aspiration sclerotherapy in order to reduce cyst size. The secondary objectives are to reduce symptoms, improve health-related quality of life (HRQL), and reduce…

Screening of patients with Crigler-Najjar

Screening of patients with the inherited liver disease Crigler-Najjar.

An explorative study determining the hepatic cyst penetration of cefazolin and factors affecting penetration.

The primary objective is to determine the cefazolin penetration into hepatic cysts. Secondary objective are to identify factors affecting antibiotic penetration and to evaluate the association between antibiotic penetration and development of…

The effect of one mutation in the ATP8B1 gene on hearing.

The aim of this study is to investigate the effect of ATP8B1 carrier on hearing in human.

Genetic and Radiological Screening of First Degree Family Members of Patients with Familial Liver Adenomatosis

To determine if LA is a familial disease. To identify germline mutations in HNF-1 alpha, ß-catenin and CYP1B1 genes in LA patients. To screen first degree relatives of patients diagnosed with liver adenomatosis for the presence of liver adenomas and…

A Phase 3 Double-blind, Randomized, Placebo-controlled Study of the Safety and Efficacy of Odevixibat (A4250) in Patients with Alagille Syndrome (ASSERT)

Primary Objective:To demonstrate the efficacy of repeated daily doses of 120 µg/kg/day odevixibat in relieving pruritus in patients with ALGS.Secondary Objectives:To assess the impact of odevixibat on serum bile acid levels in patients with ALGS.To…

An Open-label Extension Study to Evaluate Long-term Efficacy and Safety of A4250 in Children with Progressive Familial Intrahepatic Cholestasis Types 1 and 2 (PEDFIC 2)

Primary Objective (Cohort 1)To demonstrate a sustained effect of A4250 on serum bile acids and pruritus in children with progressive familial intrahepatic cholestasis (PFIC) Types 1 and 2.Primary Objective (Cohort 2)To evaluate the effect of A4250…

A Double-Blind, Randomized, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Odevixibat (A4250) in Children with Biliary Atresia Who Have Undergone a Kasai Hepatoportoenterostomy (BOLD)

This study has been transitioned to CTIS with ID 2024-512086-14-00 check the CTIS register for the current data. Primary Objective:The primary objective is to evaluate the efficacy of repeated once-daily doses of odevixibat versus placebo in…

An Open Label Study to Evaluate the Long-term Safety and Efficacy of Odevixibat (A4250) in Patients with Alagille Syndrome (ASSERT-EXT)

This study has been transitioned to CTIS with ID 2023-509028-17-00 check the CTIS register for the current data. To demonstrate a sustained effect of odevixibat on pruritus in patients with ALGS who have completed study A4250-012 (ASSERT)

A Multicenter, Open-Label, Single- and Multiple-Dose, Dose-Finding Study, with an Optional Open-Label Extension to Assess the Safety, Tolerability, and Pharmacokinetics of Obeticholic Acid in Pediatric Subjects with Biliary Atresia.;Trial Acronym: ObetiCholic Acid in Pediatric Subjects with BiliaRy AtrEsia (CARE)

Primary Objectives• Safety and tolerability• PK of OCA and its conjugates o SD PK Phase: To assess the PK of OCA and its conjugates after a single low dose of OCA on Day 1 and determine subject eligibility for the MD Phase o MD Phase: To assess the…

A Double-Blind, Randomized, Placebo-Controlled, Phase 3 Study to Demonstrate Efficacy and Safety of A4250 in Children with Progressive Familial Intrahepatic Cholestasis Types 1 and 2 (PEDFIC 1)

To demonstrate the efficacy of repeated daily doses of 40 µg/kg/day and 120 µg/kg/day A4250 in children with progressive familial intrahepatic cholestasis (PFIC) Types 1 and 2, as determined by the following:* Proportion of patients experiencing at…

The identification of new polycystic liver disease genes.

The identification of a new gene(s) that causes autosomal dominant polycystic liver disease.

The Effect of UrsodeoXyCholicacid (UDCA) and ezetimibe on total faecal steRol ExcreTion and plasma lipid lEvels

The primary objective is to evaluate the effect of UDCA and ezetimibe on cholesterol elimination assessed as total faecal sterol concentration. Secondary objective is to assess the effect of UDCA and ezetimibe on plasma lipid profile/composition.