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Octreotide in Severe Polycystic Liver Disease

Primary Purpose

Polycystic Kidney, Autosomal Dominant, Polycystic Liver Disease, Hepatomegaly

Status
Completed
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
Octreotide
Placebo
Sponsored by
Mayo Clinic
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Polycystic Kidney, Autosomal Dominant

Eligibility Criteria

18 Years - 80 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • Mayo Clinic

Arms of the Study

Arm 1

Arm 2

Arm Type

Active Comparator

Placebo Comparator

Arm Label

Octreotide

Placebo

Arm Description

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

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

Outcomes

Primary Outcome Measures

Percent Change in Liver Volume
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
Percent change from baseline in renal volume, measured in milliliters by MRI or CT scans
Percent Change in Glomerular Filtration Rate (GFR)
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
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).

Full Information

First Posted
January 22, 2007
Last Updated
October 22, 2012
Sponsor
Mayo Clinic
Collaborators
Novartis, National Center for Research Resources (NCRR)
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1. Study Identification

Unique Protocol Identification Number
NCT00426153
Brief Title
Octreotide in Severe Polycystic Liver Disease
Official Title
Pilot Study Of Long-Acting Octreotide (Octreotide LAR® Depot) In The Treatment Of Patients With Severe Polycystic Liver Disease
Study Type
Interventional

2. Study Status

Record Verification Date
October 2012
Overall Recruitment Status
Completed
Study Start Date
January 2007 (undefined)
Primary Completion Date
October 2008 (Actual)
Study Completion Date
October 2008 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Mayo Clinic
Collaborators
Novartis, National Center for Research Resources (NCRR)

4. Oversight

Data Monitoring Committee
No

5. Study Description

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 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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Polycystic Kidney, Autosomal Dominant, Polycystic Liver Disease, Hepatomegaly, Liver Diseases, Kidney, Polycystic, Abdominal Pain

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2, Phase 3
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigator
Allocation
Randomized
Enrollment
42 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Octreotide
Arm Type
Active Comparator
Arm Description
Participants received Octreotide LAR® Depot injections (up to 40 mg) intramuscularly every 28 days (+/- 5 days) for one year
Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Description
Participants received an injection of placebo (sham) medication intramuscularly every 28 days (+/- 5 day) for one year
Intervention Type
Drug
Intervention Name(s)
Octreotide
Other Intervention Name(s)
Octreotide LAR® Depot
Intervention Description
Participants received Octreotide LAR® Depot injections (up to 40 mg)intramuscularly every 28 days (+/- 5 days) for one year
Intervention Type
Drug
Intervention Name(s)
Placebo
Other Intervention Name(s)
Placebo injection
Intervention Description
Participants received an injection of placebo (sham) medication intramuscularly every 28 days (+/- 5 day) for one year
Primary Outcome Measure Information:
Title
Percent Change in Liver Volume
Description
Percent change from baseline in liver volume, measured in milliliters by Magnetic Resonance Imaging (MRI)or Computed Tomography (CT) scans
Time Frame
Baseline, 12 months
Secondary Outcome Measure Information:
Title
Percent Change in Renal Volume
Description
Percent change from baseline in renal volume, measured in milliliters by MRI or CT scans
Time Frame
Baseline, 12 months
Title
Percent Change in Glomerular Filtration Rate (GFR)
Description
Percent change from baseline in renal function/GFR, measured by clearance of iothalamate with monitoring of bladder emptying using ultrasound
Time Frame
Baseline, 12 months
Title
Change in Subject Reported Outcomes Using Mean Health Related Quality of Life (HRQoL) Scores
Description
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).
Time Frame
Baseline, 12 months

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
80 Years
Accepts Healthy Volunteers
No
Eligibility 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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Marie C. Hogan, M.D., Ph.D.
Organizational Affiliation
Mayo Clinic
Official's Role
Principal Investigator
Facility Information:
Facility Name
Mayo Clinic
City
Rochester
State/Province
Minnesota
ZIP/Postal Code
55905
Country
United States

12. IPD Sharing Statement

Citations:
PubMed Identifier
22773240
Citation
Hogan MC, Masyuk TV, Page L, Holmes DR 3rd, Li X, Bergstralh EJ, Irazabal MV, Kim B, King BF, Glockner JF, Larusso NF, Torres VE. Somatostatin analog therapy for severe polycystic liver disease: results after 2 years. Nephrol Dial Transplant. 2012 Sep;27(9):3532-9. doi: 10.1093/ndt/gfs152. Epub 2012 Jul 6.
Results Reference
result
PubMed Identifier
26166166
Citation
Hogan MC, Masyuk T, Bergstralh E, Li B, Kremers WK, Vaughan LE, Ihrke A, Severson AL, Irazabal MV, Glockner J, LaRusso NF, Torres VE. Efficacy of 4 Years of Octreotide Long-Acting Release Therapy in Patients With Severe Polycystic Liver Disease. Mayo Clin Proc. 2015 Aug;90(8):1030-7. doi: 10.1016/j.mayocp.2015.05.011. Epub 2015 Jul 9.
Results Reference
derived
Links:
URL
http://clinicaltrials.mayo.edu
Description
Mayo Clinic Clinical Trials

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Octreotide in Severe Polycystic Liver Disease

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