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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 : September 23, 2021
Sponsor:
Collaborator:
Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)
Information provided by (Responsible Party):
Radboud University Medical Center

Study Description
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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 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.


Condition or disease Intervention/treatment Phase
Polycystic Liver Disease Polycystic Kidney, Autosomal Dominant Drug: Ursodeoxycholic Acid Phase 2

Detailed Description:
We investigated whether ursodeoxycholic acid was able to reduce total liver volume in polycystic liver disease.
Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 34 participants
Allocation: Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: An International, Multicenter, Randomized Controlled Clinical Trial Assessing the Efficacy of Ursodeoxycholic Acid as a Volume Reducing Treatment in Symptomatic Polycystic Liver Disease
Study Start Date : December 2013
Actual Primary Completion Date : October 2015
Actual Study Completion Date : October 2015

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Liver Diseases
Drug Information available for: Ursodiol

Arms and Interventions
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Arm Intervention/treatment
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
 Drug: Ursodeoxycholic Acid
The intervention group will receive 15-20mg/kg/day UDCA for 24 weeks
Other Name: Ursochol


Outcome Measures
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Primary Outcome Measures :
  1. 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 and week 24


Secondary Outcome Measures :
  1. Effect of UDCA-therapy on absolute total liver volume [ Time Frame: Baseline to week 24 ]
    Absolute total liver volume at baseline and end of treatment (week 24) will be measured

  2. Effect of UDCA on gastro-intestinal symptoms measured by a GI-questionnaire [ Time Frame: Baseline to week 24 ]
    Improvement in Gastrointestinal Symptom score

  3. Effect of UDCA on health related quality of life as measured by Study Form -36 [ Time Frame: Baseline to week 24 ]
    Improvement in Study Form -36 score

  4. Proportion of patients with any reduction in total liver volume after 24 weeks [ Time Frame: Baseline to week 24 ]
    Proportion reduction in total liver volume

  5. Effect of UDCA on absolute total kidney volume [ Time Frame: Baseline to week 24 ]
    Change in total kidney volume after 24 weeks


Other Outcome Measures:
  1. Adverse events as a measure of tolerability and safety of UDCA [ Time Frame: Baseline to week 24 ]
    All adverse events


Eligibility Criteria
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Ages Eligible for Study:   18 Years to 80 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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 of the investigator, unsuitable for the study
  • Mental illness that interferes with the patient ability to comply with the protocol
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02021110


Locations
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Netherlands
Radboud University Medical Centre Nijmegen
Nijmegen, Gelderland, Netherlands
Academic Medical Centre Amsterdam
Amsterdam, Netherlands
Spain
Donostia University Hospital
San Sebastian, Spain
Sponsors and Collaborators
Radboud University Medical Center
Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)
Investigators
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Principal Investigator: Joost PH Drenth, dr. Radboud University Medical Centre Nijmegen, the Netherlands
More Information
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Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

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Responsible Party: Radboud University Medical Center
ClinicalTrials.gov Identifier: NCT02021110    
Other Study ID Numbers: PLD 11-01
First Posted: December 27, 2013    Key Record Dates
Last Update Posted: September 23, 2021
Last Verified: April 2016
Additional relevant MeSH terms:
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Cysts
Liver Diseases
Polycystic Kidney Diseases
Polycystic Kidney, Autosomal Dominant
Digestive System Diseases
Kidney Diseases, Cystic
Kidney Diseases
Urologic Diseases
Abnormalities, Multiple
 Congenital Abnormalities
Ciliopathies
Genetic Diseases, Inborn
Neoplasms
Pathological Conditions, Anatomical
Ursodeoxycholic Acid
Cholagogues and Choleretics
Gastrointestinal Agents