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NCT Number: NCT00426153

Octreotide in Severe Polycystic Liver Disease

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.

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Key information

Conditions

Polycystic Kidney, Autosomal Dominant Abdominal Pain Abnormalities, Multiple Ciliopathies Congenital Abnormalities Congenital, Hereditary, and Neonatal Diseases and Abnormalities Digestive System Diseases Female Urogenital Diseases Female Urogenital Diseases and Pregnancy Complications Genetic Diseases, Inborn Hepatomegaly Hypertrophy Kidney Diseases Kidney Diseases, Cystic Kidney, Polycystic Liver Diseases Male Urogenital Diseases Neurologic Manifestations Pain Pathological Conditions, Anatomical Pathological Conditions, Signs and Symptoms Polycystic Kidney Diseases Polycystic Liver Disease Signs and Symptoms Signs and Symptoms, Digestive Urogenital Diseases Urologic Diseases

Age range

18 year–80 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2 / Phase 3

Primary location

Mayo Clinic

Rochester, Minnesota, 55905, United States

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Octreotide

Drug

Participants received Octreotide LAR® Depot injections (up to 40 mg)intramuscularly every 28 days (+/- 5 days) for one year

Other names: Octreotide LAR® Depot

Placebo

Drug

Participants received an injection of placebo (sham) medication intramuscularly every 28 days (+/- 5 day) for one year

Other names: Placebo injection

Primary outcomes

  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 outcomes

  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).

Sponsors and collaborators

Lead sponsor

Mayo Clinic

Other

Collaborators

  • National Center for Research Resources (NCRR)
  • Novartis

Registry information

Official study title

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

Important dates

Study start
2007
Primary completion
2008
Study completion
2008
First posted
Jan 24, 2007
Registry last updated
Nov 21, 2012

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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