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Octreotide in Severe Polycystic Liver Disease

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT00426153
Recruitment Status : Completed
First Posted : January 24, 2007
Results First Posted : November 21, 2012
Last Update Posted : November 21, 2012
Sponsor:
Collaborators:
Novartis
National Center for Research Resources (NCRR)
Information provided by (Responsible Party):
Marie Hogan, Mayo Clinic

Study Description
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Brief Summary:
This study will evaluate the effect of Octreotide LAR® on the liver volumes of patients with severe polycystic liver disease who are not candidates or decline surgical treatments such as liver cyst fenestration, liver resection or liver transplantation. A total of 42 patients will be recruited -14 who will receive placebo and 28 the study drug. Preliminary evidence indicates that this drug is safe and non-toxic in other disease states. Treatment with this drug holds promise not only for individuals with liver involvement, but also for many more patients with polycystic kidney disease.

Condition or disease Intervention/treatment Phase
Polycystic Kidney, Autosomal Dominant Polycystic Liver Disease Hepatomegaly Liver Diseases Kidney, Polycystic Abdominal Pain Drug: Octreotide Drug: Placebo Phase 2 Phase 3

Detailed Description:

The primary aim of this study is to compare the effect of Octreotide LAR® Depot on the liver volume of patients with severe polycystic liver disease who are not candidates or decline surgical treatments such as liver cyst fenestration, liver resection or liver transplantation compared with placebo. The secondary aims of the study are: (1)Assess the effect of Octreotide LAR® Depot on the total kidney volume and iothalamate clearance in patients with polycystic kidney disease associated with severe polycystic liver disease who are not candidates or decline surgical treatments such as liver cyst fenestration, liver resection or liver transplantation. (2)Evaluate quality of life changes associated with the administration of Octreotide LAR® Depot in these patients. (3)Assess toxicity of Octreotide LAR® Depot in patients with polycystic liver disease (PLD).

Note: Subjects who completed this 1 year randomized trial were offered enrollment into an open-label (all subjects received Octreotide) extension trial for an additional two years of treatment.

Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 42 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple (Participant, Care Provider, Investigator)
Primary Purpose: Treatment
Official Title: Pilot Study Of Long-Acting Octreotide (Octreotide LAR® Depot) In The Treatment Of Patients With Severe Polycystic Liver Disease
Study Start Date : January 2007
Actual Primary Completion Date : October 2008
Actual Study Completion Date : October 2008

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Liver Diseases

Arms and Interventions
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Arm Intervention/treatment
Active Comparator: Octreotide
Participants received Octreotide LAR® Depot injections (up to 40 mg) intramuscularly every 28 days (+/- 5 days) for one year
 Drug: Octreotide
Participants received Octreotide LAR® Depot injections (up to 40 mg)intramuscularly every 28 days (+/- 5 days) for one year
Other Name: Octreotide LAR® Depot
Placebo Comparator: Placebo
Participants received an injection of placebo (sham) medication intramuscularly every 28 days (+/- 5 day) for one year
 Drug: Placebo
Participants received an injection of placebo (sham) medication intramuscularly every 28 days (+/- 5 day) for one year
Other Name: Placebo injection


Outcome Measures
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Primary Outcome Measures :
  1. Percent Change in Liver Volume [ Time Frame: Baseline, 12 months ]
    Percent change from baseline in liver volume, measured in milliliters by Magnetic Resonance Imaging (MRI)or Computed Tomography (CT) scans


Secondary Outcome Measures :
  1. Percent Change in Renal Volume [ Time Frame: Baseline, 12 months ]
    Percent change from baseline in renal volume, measured in milliliters by MRI or CT scans

  2. Percent Change in Glomerular Filtration Rate (GFR) [ Time Frame: Baseline, 12 months ]
    Percent change from baseline in renal function/GFR, measured by clearance of iothalamate with monitoring of bladder emptying using ultrasound

  3. Change in Subject Reported Outcomes Using Mean Health Related Quality of Life (HRQoL) Scores [ Time Frame: Baseline, 12 months ]
    Scores on the Medical Outcomes Study 36-Item Short-Form General Health Survey (SF-36), version 2. Subjects completed the SF-36 which consists of 8 sub-scales which are additionally summarized into 2 summary components (physical and mental). The subscales and the summary scales both range from 0 to 100, with (0 = worst imaginable, 100 = best imaginable).


Eligibility Criteria
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Information from the National Library of Medicine

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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:

  • Age - 18 years and older
  • Diagnosis of Polycystic Liver Disease (PLD) associated with ADPKD or isolated Autosomal Dominant Polycystic liver Disease (ADPLD)
  • Severe PLD defined as a liver volume greater than 4000 mL or symptomatic disease due to mass effects from hepatic cysts
  • Not a candidate for or declining surgical intervention

Exclusion Criteria:

  • Women of childbearing potential or their sexual partners who are unwilling to employ adequate contraception
  • Creatinine greater than 3mg/dL or hemodialysis dependent
  • Cancer or major systemic disease that could prevent completion of the planned follow-up or interfere with data collection or interpretation
  • Uncontrolled diabetes mellitus as defined by blood glucose levels of greater than or equal to 250 mg/dL on 2 or more consecutive daily readings despite antidiabetic therapy
  • Neurologic/psychologic conditions preventing appropriate informed consent
  • Symptomatic gallstones or biliary sludge
  • Variceal bleeding or hepatic encephalopathy within prior 30 days
  • Uncontrolled hypertension (Systolic blood pressure greater than 160 mmHg; Diastolic blood pressure greater than 100 mmHg)
  • Current, or prior use of somatostatin analogue (octreotide, lanreotide) in past 6 months
  • History of significant adverse reaction to a somatostatin analogue
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): NCT00426153


Locations
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United States, Minnesota
Mayo Clinic
Rochester, Minnesota, United States, 55905
Sponsors and Collaborators
Mayo Clinic
Novartis
National Center for Research Resources (NCRR)
Investigators
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Principal Investigator: Marie C. Hogan, M.D., Ph.D. Mayo Clinic
More Information
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Additional Information:

Publications of Results:

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

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Responsible Party: Marie Hogan, MD, PhD, Assistant Professor of Medicine, College of Medicine, Mayo Clinic
ClinicalTrials.gov Identifier: NCT00426153    
Other Study ID Numbers: 06-004128
UL1RR024150 ( U.S. NIH Grant/Contract )
First Posted: January 24, 2007    Key Record Dates
Results First Posted: November 21, 2012
Last Update Posted: November 21, 2012
Last Verified: October 2012
Additional relevant MeSH terms:
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Cysts
Liver Diseases
Hepatomegaly
Polycystic Kidney Diseases
Polycystic Kidney, Autosomal Dominant
Abdominal Pain
Digestive System Diseases
Pain
Neurologic Manifestations
Signs and Symptoms, Digestive
Kidney Diseases, Cystic
Kidney Diseases
 Urologic Diseases
Abnormalities, Multiple
Congenital Abnormalities
Ciliopathies
Genetic Diseases, Inborn
Neoplasms
Pathological Conditions, Anatomical
Hypertrophy
Octreotide
Gastrointestinal Agents
Antineoplastic Agents, Hormonal
Antineoplastic Agents