Phase 3
Completed N=42
Octreotide in Severe Polycystic Liver Disease
Polycystic Kidney, Autosomal Dominant · Polycystic Liver Disease · Hepatomegaly · Liver disease
Source: ClinicalTrials.gov NCT00426153 ↗
Enrolled (actual)
42
Serious AEs
7.1%
Results posted
Nov 2012
Primary outcomePrimary: Percent Change in Liver Volume — -5.0; 0.9 percent change — p=0.048
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.
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 | — |
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
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. Informational only — not medical advice.