Clinical Trial: Octreotide in Severe Polycystic Liver Disease

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

Official Title: Pilot Study Of Long-Acting Octreotide (Octreotide LAR® Depot) In The Treatment Of Patients With Severe Polycystic Liver Disease

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.

Detailed Summary:

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.


Sponsor: Mayo Clinic

Current Primary Outcome: 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


Original Primary Outcome: Percent change in liver volume as measured by MRI at baseline and 12 months.

Current Secondary Outcome:

  • 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
  • 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
  • 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).


Original Secondary Outcome:

  • Percent change in liver cyst volume, renal volume, and renal cyst volume as measured by MRI at baseline and 12 months.
  • Response rates, based on changes in liver and renal volume.
  • Adverse events.
  • Changes in quality of life.


Information By: Mayo Clinic

Dates:
Date Received: January 22, 2007
Date Started: January 2007
Date Completion:
Last Updated: October 22, 2012
Last Verified: October 2012