Octreotide in Severe Polycystic Liver Disease

Liver Diseases Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00426153
• Other study ID #: 06-004128
• Secondary ID: UL1RR024150
• Status: Completed
• Phase: Phase 2/Phase 3
• First received: January 22, 2007
• Last updated: October 22, 2012
• Start date: January 2007
• Est. completion date: October 2008

Study information

• Verified date: October 2012
• Source: Mayo Clinic
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Institutional Review Board
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 42
• Est. completion date: October 2008
• Est. primary completion date: October 2008
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 18 Years to 80 Years

Eligibility

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

Study Design

Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Caregiver, Investigator), Primary Purpose: Treatment

Related Conditions & MeSH terms

• Abdominal Pain
• Cysts
• Hepatomegaly
• Kidney, Polycystic
• Liver Diseases
• Polycystic Kidney Diseases
• Polycystic Kidney, Autosomal Dominant
• Polycystic Liver Disease

Intervention

Drug:
• Octreotide
Participants received Octreotide LAR® Depot injections (up to 40 mg)intramuscularly every 28 days (+/- 5 days) for one year
• Placebo
Participants received an injection of placebo (sham) medication intramuscularly every 28 days (+/- 5 day) for one year

Locations

Country	Name	City	State
United States	Mayo Clinic	Rochester	Minnesota

Sponsors (3)

Lead Sponsor	Collaborator
Mayo Clinic	National Center for Research Resources (NCRR), Novartis

Country where clinical trial is conducted

United States, 

References & Publications (1)

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( — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	Baseline, 12 months	No
Secondary	Percent Change in Renal Volume	Percent change from baseline in renal volume, measured in milliliters by MRI or CT scans	Baseline, 12 months	No
Secondary	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	Baseline, 12 months	No
Secondary	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).	Baseline, 12 months	No

External Links

•   Mayo Clinic Clinical Trials