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Ursodeoxycholic Acid as Treatment for Polycystic Liver Disease (CURSOR)

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ClinicalTrials.gov Identifier: NCT02021110
Recruitment Status : Completed
First Posted : December 27, 2013
Last Update Posted : April 7, 2016
Sponsor:
Collaborators:
Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)
Biodonostia Health Research Institute
Information provided by (Responsible Party):
Radboud University

December 12, 2013
December 27, 2013
April 7, 2016
December 2013
October 2015   (Final data collection date for primary outcome measure)
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
Same as current
Complete list of historical versions of study NCT02021110 on ClinicalTrials.gov Archive Site
  • 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 ]
Same as current
Adverse events as a measure of tolerability and safety of UDCA [ Time Frame: Baseline to week 24 ]
Same as current
 
Ursodeoxycholic Acid as Treatment for Polycystic Liver Disease
An International, Multicenter, Randomized Controlled Clinical Trial Assessing the Efficacy of Ursodeoxycholic Acid as a Volume Reducing Treatment in Symptomatic Polycystic Liver Disease

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 of ≥ 2500. Symptomatic is defined as 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.

Main secondary outcome variables:

  • To demonstrate whether UDCA-therapy changes absolute total liver volume
  • To demonstrate whether UDCA-therapy changes gastro-intestinal symptoms measured by a GI-questionnaire
  • To demonstrate whether UDCA-therapy changes quality of life as measured by SF-36
  • To demonstrate which proportion of patients has any reduction in total liver volume after 24 weeks
  • To demonstrate whether UDCA is well tolerated
  • To demonstrate whether UDCA-therapy changes absolute total kidney volume (TKV).
Not Provided
Interventional
Phase 2
Allocation: Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
  • Polycystic Liver Disease (PLD):
  • Polycystic Kidney, Autosomal Dominant
  • Polycystic Liver Disease
Drug: Ursodeoxycholic Acid
The intervention group will receive 15-20mg/kg/day UDCA for 24 weeks
Other Name: Ursochol
  • No Intervention: Control group
    This group will receive standard care (no treatment)
  • Experimental: Ursodeoxycholic Acid
    The intervention group will receive 15-20mg/kg/day UDCA for 24 weeks
    Intervention: Drug: Ursodeoxycholic Acid
D'Agnolo HM, Kievit W, Takkenberg RB, Riaño I, Bujanda L, Neijenhuis MK, Brunenberg EJ, Beuers U, Banales JM, Drenth JP. Ursodeoxycholic acid in advanced polycystic liver disease: A phase 2 multicenter randomized controlled trial. J Hepatol. 2016 Sep;65(3):601-7. doi: 10.1016/j.jhep.2016.05.009. Epub 2016 May 17.

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Completed
34
Same as current
October 2015
October 2015   (Final data collection date for primary outcome measure)

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-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, 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 of the investigator, unsuitable for the study
  • Mental illness that interferes with the patient ability to comply with the protocol
Sexes Eligible for Study: All
18 Years to 80 Years   (Adult, Older Adult)
No
Contact information is only displayed when the study is recruiting subjects
Netherlands,   Spain
 
 
NCT02021110
PLD 11-01
Yes
Not Provided
Not Provided
Radboud University
Radboud University
  • Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)
  • Biodonostia Health Research Institute
Principal Investigator: Joost PH Drenth, dr. Radboud University Medical Centre Nijmegen, the Netherlands
Radboud University
April 2016

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP