Clinical Trial: Ursodeoxycholic Acid as Treatment for Polycystic Liver Disease

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

Official Title: An International, Multicenter, Randomized Controlled Clinical Trial Assessing the Efficacy of Ursodeoxycholic Acid as a Volume Reducing Treatment in Symptomatic Polycystic Liver

Brief 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 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

Detailed Summary:
Sponsor: Radboud University

Current Primary Outcome: Effect of UDCA on total liver volume [ Time Frame: Baseline to week 24 ]

Proportional change of total liver volume in UDCA treated patients versus non treated patients, as assessed by CT at baseline andweek 24


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Effect of UDCA-therapy on absolute total liver volume [ Time Frame: Baseline to week 24 ]
    Absolute TLV at baseline and end of treatment (week 24) will be measured
  • Effect of UDCA on gastro-intestinal symptoms measured by a GI-questionnaire [ Time Frame: Baseline to week 24 ]
  • Effect of UDCA on health related quality of life as measured by SF-36 [ Time Frame: Baseline to week 24 ]
  • Proportion of patients with any reduction in total liver volume after 24 weeks [ Time Frame: Baseline to week 24 ]
  • Effect of UDCA on abslute total kidney volume [ Time Frame: Baseline to week 24 ]


Original Secondary Outcome: Same as current

Information By: Radboud University

Dates:
Date Received: December 12, 2013
Date Started: December 2013
Date Completion:
Last Updated: April 6, 2016
Last Verified: April 2016