Octreotide in Severe Polycystic Liver Disease

This study has been completed.
Sponsor:
Collaborators:
Novartis
Information provided by (Responsible Party):
Marie Hogan, Mayo Clinic
ClinicalTrials.gov Identifier:
NCT00426153
First received: January 22, 2007
Last updated: October 22, 2012
Last verified: October 2012
  Purpose

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 Intervention Phase
Polycystic Kidney, Autosomal Dominant
Polycystic Liver Disease
Hepatomegaly
Liver Diseases
Kidney, Polycystic
Abdominal Pain
Drug: Octreotide
Drug: Placebo
Phase 2
Phase 3

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, 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

Resource links provided by NLM:


Further study details as provided by Mayo Clinic:

Primary Outcome Measures:
  • Percent Change in Liver Volume [ Time Frame: Baseline, 12 months ] [ Designated as safety issue: No ]
    Percent change from baseline in liver volume, measured in milliliters by Magnetic Resonance Imaging (MRI)or Computed Tomography (CT) scans


Secondary Outcome Measures:
  • Percent Change in Renal Volume [ Time Frame: Baseline, 12 months ] [ Designated as safety issue: No ]
    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 ] [ Designated as safety issue: No ]
    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 ] [ Designated as safety issue: No ]
    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).


Enrollment: 42
Study Start Date: January 2007
Study Completion Date: October 2008
Primary Completion Date: October 2008 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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

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.

  Eligibility

Ages Eligible for Study:   18 Years to 80 Years
Genders Eligible for Study:   Both
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
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00426153

Locations
United States, Minnesota
Mayo Clinic
Rochester, Minnesota, United States, 55905
Sponsors and Collaborators
Mayo Clinic
Novartis
Investigators
Principal Investigator: Marie C. Hogan, M.D., Ph.D. Mayo Clinic
  More Information

Additional Information:
Publications:
Responsible Party: Marie Hogan, MD, PhD, Assistant Professor of Medicine, College of Medicine, Mayo Clinic
ClinicalTrials.gov Identifier: NCT00426153     History of Changes
Other Study ID Numbers: 06-004128, UL1RR024150
Study First Received: January 22, 2007
Results First Received: October 22, 2012
Last Updated: October 22, 2012
Health Authority: United States: Institutional Review Board

Additional relevant MeSH terms:
Liver Diseases
Abdominal Pain
Cysts
Hepatomegaly
Multicystic Dysplastic Kidney
Polycystic Kidney Diseases
Polycystic Kidney, Autosomal Dominant
Congenital Abnormalities
Digestive System Diseases
Hypertrophy
Kidney Diseases
Kidney Diseases, Cystic
Neoplasms
Pain
Pathological Conditions, Anatomical
Signs and Symptoms
Signs and Symptoms, Digestive
Urogenital Abnormalities
Urologic Diseases
Octreotide
Antineoplastic Agents
Antineoplastic Agents, Hormonal
Gastrointestinal Agents
Pharmacologic Actions
Therapeutic Uses

ClinicalTrials.gov processed this record on October 30, 2014