Clinical trial NCT00426153: 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
Overall status: COMPLETED
Phase: PHASE2
PHASE3
Conditions: Polycystic Kidney, Autosomal Dominant
Polycystic Liver Disease
Hepatomegaly
Liver Diseases
Kidney, Polycystic
Abdominal Pain
Lead sponsor: Mayo Clinic
Sponsor class: OTHER
Collaborators: name: Novartis
name: National Center for Research Resources (NCRR)
First posted: 2007-01-24
Enrollment: 42
Sex: ALL
Minimum age (years): 18
Maximum age (years): 80
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.

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

Primary outcomes:
measure: Percent Change in Liver Volume
timeFrame: Baseline, 12 months
Secondary outcomes:
measure: Percent Change in Renal Volume
timeFrame: Baseline, 12 months
measure: Percent Change in Glomerular Filtration Rate (GFR)
timeFrame: Baseline, 12 months
measure: Change in Subject Reported Outcomes Using Mean Health Related Quality of Life (HRQoL) Scores
timeFrame: Baseline, 12 months
Other outcomes:
Locations:
city: Rochester
country: United States
facility: Mayo Clinic
state: Minnesota
Study officials:
affiliation: Mayo Clinic
name: Marie C. Hogan, M.D., Ph.D.
role: PRINCIPAL_INVESTIGATOR
References:
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.
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.