A Phase 2 interventional study of Ursodeoxycholic Acid in Polycystic Liver Disease and Polycystic Kidney, Autosomal Dominant, sponsored by Radboud University Medical Center. Completed at 3 sites in 2 countries. Open to participants aged 18 Years to 80 Years. Per ClinicalTrials.gov, last updated 2021-09-23.
Sponsored by Radboud University Medical Center · Phase 2, Interventional, and Treatment
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.
We investigated whether ursodeoxycholic acid was able to reduce total liver volume in polycystic liver disease.
118 studies on the registry are indexed under Cysts; 22 are open to participants now.
This study's enrollment of 34 is below the median of 51 across 72 interventional studies indexed under Cysts.
Browse Cysts studies →Radboud University Medical Center is the lead sponsor of 959 studies on the registry; 134 are open to participants now.
Of its 6 completed or terminated interventional studies of FDA-regulated products, 0 (0%) have results posted.
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Exclusion Criteria:
This group will receive standard care (no treatment)
The intervention group will receive 15-20mg/kg/day UDCA for 24 weeks
Drug: Ursodeoxycholic Acid
The intervention group will receive 15-20mg/kg/day UDCA for 24 weeks
Also known as: Ursochol
Effect of UDCA on total liver volume
Proportional change of total liver volume in UDCA treated patients versus non treated patients, as assessed by CT at baseline and week 24
Time frame: Baseline to week 24
Effect of UDCA-therapy on absolute total liver volume
Absolute total liver volume at baseline and end of treatment (week 24) will be measured
Time frame: Baseline to week 24
Effect of UDCA on gastro-intestinal symptoms measured by a GI-questionnaire
Improvement in Gastrointestinal Symptom score
Time frame: Baseline to week 24
Effect of UDCA on health related quality of life as measured by Study Form -36
Improvement in Study Form -36 score
Time frame: Baseline to week 24
Proportion of patients with any reduction in total liver volume after 24 weeks
Proportion reduction in total liver volume
Time frame: Baseline to week 24
Effect of UDCA on absolute total kidney volume
Change in total kidney volume after 24 weeks
Time frame: Baseline to week 24
Adverse events as a measure of tolerability and safety of UDCA
All adverse events
Time frame: Baseline to week 24
This study is completed, as verified in Apr 2016. You cannot join it, but the record below documents what was studied.
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Radboud University Medical Center