Phase 3 N=42 Randomized Triple-blind Treatment

Octreotide in Severe Polycystic Liver Disease

Polycystic Kidney, Autosomal Dominant · Polycystic Liver Disease · Hepatomegaly · Liver Diseases · Kidney, Polycystic

Bottom Line

View on ClinicalTrials.gov: NCT00426153 ↗

Enrolled (actual)

Serious AEs

7.1%

Results posted

Nov 2012

Primary outcome: Primary: Percent Change in Liver Volume — -5.0; 0.9 percent change — p=0.048

Study Design & Population

Study type: Interventional
Phase: Phase 3
Interventions: Octreotide (Drug); Placebo (Drug)
Age: Adult, Older Adult · 18+ yrs
Sex: All
Sponsor: Mayo Clinic
Primary completion: Oct 2008

Outcome Measures

Outcome	Result	p-value
PRIMARY Percent Change in Liver Volume	-5.0; 0.9	0.048 sig
SECONDARY Percent Change in Renal Volume	0.25; 8.61	0.045 sig
SECONDARY Percent Change in Glomerular Filtration Rate (GFR)	-5.1; -7.2	0.98
SECONDARY Change in Subject Reported Outcomes Using Mean Health Related Quality of Life (HRQoL) Scores	74.8; 80.4; 77.0; 82.1; 59.8; 76.8	—

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.

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

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT00426153). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.