CompletedPhase 2

Ursodeoxycholic Acid as Treatment for Polycystic Liver Disease

Netherlands, Spain34 participantsStarted 2013-12

Plain-language summary

Rationale: Polycystic liver disease (PLD) is a rare disorder characterized by \>20 fluid-filled hepatic cysts. Polycystic livers are present in the combination with renal cysts as a manifestation of autosomal dominant polycystic kidney disease (ADPKD), or isolated in the absence of renal cysts as autosomal dominant polycystic liver disease (ADPLD or PCLD). PLD patients are confronted with symptoms caused by the mass effect of their polycystic liver every day for the rest of their life. There is no standard therapeutic option for symptomatic PLD patients. Current options are fairly invasive or their efficacy is only moderate. Preliminary data in our research lab have shown that ursodeoxycholic acid (UDCA) inhibited the proliferation of polycystic human cholangiocytes in vitro through the normalization of the intracellular calcium levels in cystic cholangiocytes. The investigators also found that daily oral administration of UDCA for 5 months to polycystic kidney disease (PCK) rats, an animal model of ARPKD that spontaneously develops hepato-renal cystogenesis, resulted in inhibition of hepatic cystogenesis. The investigators hypothesize that UDCA is an effective therapeutic tool in reducing liver volume in PLD. Objective: First, to demonstrate whether UDCA-therapy is effective in reducing total liver volume in PLD patients. Second, the investigators want to assess if UDCA modifies quality of life. Finally, the investigators want to assess safety and tolerability. Study design: International, multicenter, randomized, controlled trial Study population: 34 subjects (18 ≤age ≤ 80 years) suffering from symptomatic polycystic liver disease with underlying diagnosis of (PCLD or ADPKD), defined as ≥ 20 liver cysts on CT-scan and liver volume of ≥ 2500. Symptomatic is defined as Eastern Cooperative Oncology Group- Performance Score (ECOG-PS) ≥ 1 and having at least three out of ten PLD symptoms. Intervention: The patients will be randomized (1:1) into two groups. One group of patients will receive 15-20mg/kg/day UDCA for 24 weeks. The other group will receive standard care. Main study endpoint: Proportional change of total liver volume in UDCA treated patients versus non treated patients, as assessed by CT at baseline and 6 months.

Who can participate

Age range

18 Years – 80 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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 (2), and having at least three out of ten PCLD symptoms: * Informed consent, patients are willing and able to comply with the study drug regimen and all other study requirements. 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 six months before baseline * Renal dysfunction (MDRD-Glomerular filtration rate\< 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, aluminium hydroxide or cyclosporin * 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 o…

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Effect of UDCA on total liver volume

Timeframe: Baseline to week 24

Trial details

NCT IDNCT02021110

SponsorRadboud University Medical Center

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2015-10

View on ClinicalTrials.gov More Polycystic Liver Disease trials