Patients suffering from symptomatic polycystic liver disease (PLD) with underlying diagnosis of isolated polycystic liver disease (PCLD) or autosomal dominant kidney disease (ADPKD)
Conditions
Interventions
Trade Name: URSOCHOL 300
URSOCHOL 450
Pharmaceutical Form: Pillules
Sponsors
Radboud University Nijmegen Medical Center
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: • 18 = age = 80 years • Polycystic liver disease with underlying diagnosis of (PCLD or ADPKD), defined as = 20 liver cysts • Total liver volume = 2500 mL • Symptomatic defined as ECOG-PS = 1, and having at least three out of ten PCLD symptoms: - Abdominal pain - Abdominal distension - Abdominal fullness - Dyspnea - Early satiety - Back pain - Nausea/vomiting - Anorexia - Weight loss - Jaundice • Informed consent, patients are willing and able to comply with the study drug regimen and all other study requirements. Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 32 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 2
Exclusion criteria
Exclusion criteria: • Use of oral anticonceptives or estrogen supplementation • Use of UDCA in 3 months before baseline • Females who are pregnant or breast-feeding or patients of reproductive potential not employing an effective method of birth control. • Intervention (aspiration or surgical intervention) within six months before baseline • Treatment with somatostatin analogues within months before baseline • Renal dysfunction (MDRD-GFR < 30 ml/min/1.73m2) • Patients with a kidney transplant • Hypersensitivity reaction to UDCA or patients with galactose-intolerance, lactase deficiency or glucose-galactose malabsorption • Acute cholecystitis or frequent biliary colic attacks • Acute stomach or duodenal ulcers • Inflammation of small intestine or colon • Use of drugs that can interact with UDCA, such as colestyramine or aluminium hydroxide • Enrolment in another clinical trial of an investigational agent while participating in this study • History or other evidence of severe illness or any other conditions which would make the patient, in the opinion of the investigator, unsuitable for the study • Mental illness that interferes with the patient ability to comply with the protocol
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: First, to demonstrate whether UDCA-therapy is effective in reducing total liver volume in PLD patients. ;Secondary Objective: Second, we want to assess if UDCA modifies quality of life. Finally, we want to assess safety and tolerability;Primary end point(s): The main outcome measure will be the proportional change of total liver volume from baseline to 6 months as determined by CT. ;Timepoint(s) of evaluation of this end point: Baseline liver volume will be compared to liver volume at 24 weeks. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): • Change in absolute total liver volume • Change in symptoms, measured by GI-questionnaire • Change in quality of life, measured by SF-36 questionnaire • Proportion of patients having any reduction in total liver volume after 24 weeks • (Serious )Adverse events that occur in these 24 weeks • TKV at baseline and after 24 weeks ;Timepoint(s) of evaluation of this end point: For all secondary time points baseline values will be compared to values at week 24. | — |
Countries
Netherlands, Spain
Contacts
Public ContactDep.of Gastroenterology&Hepatology
Radboud University Nijmegen Medical Center
Outcome results
None listed