An international, multicenter, randomized controlled clinical trial assessing the efficacy of Ursodeoxycholic acid as a volume reducing treatment for symptomatic polycystic livers
- Conditions
- polycystic liver disease10019813
- Registration Number
- NL-OMON38971
- Lead Sponsor
- niversitair Medisch Centrum Sint Radboud
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Completed
- Sex
- Not specified
- Target Recruitment
- 22
• 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 (2), 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.
• 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
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method <p>Proportional change of total liver volume in UDCA treated patients versus non<br /><br>treated patients, as assessed by CT at baseline and 6 months.</p><br>
- Secondary Outcome Measures
Name Time Method <p>Main secondary outcome variables:<br /><br>• To demonstrate whether UDCA-therapy changes absolute total liver volume<br /><br>• To demonstrate whether UDCA-therapy changes gastro-intestinal symptoms<br /><br>measured by a GI-questionnaire<br /><br>• To demonstrate whether UDCA-therapy changes quality of life as measured by<br /><br>SF-36<br /><br>• To demonstrate which proportion of patients has any reduction in total liver<br /><br>volume after 24 weeks<br /><br>• To demonstrate whether UDCA is well tolerated<br /><br>• To demonstrate whether UDCA-therapy changes absolute total kidney volume<br /><br>(TKV).</p><br>