Octreotide in Severe Polycystic Liver Disease

Mayo Clinic

Status and phase

Completed

Phase 3

Phase 2

Conditions

Polycystic Liver Disease

Hepatomegaly

Polycystic Kidney, Autosomal Dominant

Kidney, Polycystic

Abdominal Pain

Liver Diseases

Treatments

Drug: Placebo

Drug: Octreotide

Study type

Interventional

Funder types

Other

Industry

NIH

Identifiers

NCT00426153

UL1RR024150 (U.S. NIH Grant/Contract)

06-004128

Details and patient eligibility

About

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.

Full 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.

Enrollment

42 patients

Sex

All

Ages

18 to 80 years old

Volunteers

No 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