Octreotide in Severe Polycystic Liver Disease
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Purpose
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.
| Condition | Intervention | Phase |
|---|---|---|
|
Polycystic Kidney, Autosomal Dominant Polycystic Liver Disease Hepatomegaly Liver Diseases Kidney, Polycystic Abdominal Pain |
Drug: Octreotide Drug: Placebo |
Phase 2 Phase 3 |
| Study Type: | Interventional |
| Study Design: | Allocation: Randomized Endpoint Classification: Safety/Efficacy Study Intervention Model: Parallel Assignment Masking: Double Blind (Subject, Caregiver, Investigator) Primary Purpose: Treatment |
| Official Title: | Pilot Study Of Long-Acting Octreotide (Octreotide LAR® Depot) In The Treatment Of Patients With Severe Polycystic Liver Disease |
- Percent Change in Liver Volume [ Time Frame: Baseline, 12 months ] [ Designated as safety issue: No ]Percent change from baseline in liver volume, measured in milliliters by Magnetic Resonance Imaging (MRI)or Computed Tomography (CT) scans
- Percent Change in Renal Volume [ Time Frame: Baseline, 12 months ] [ Designated as safety issue: No ]Percent change from baseline in renal volume, measured in milliliters by MRI or CT scans
- Percent Change in Glomerular Filtration Rate (GFR) [ Time Frame: Baseline, 12 months ] [ Designated as safety issue: No ]Percent change from baseline in renal function/GFR, measured by clearance of iothalamate with monitoring of bladder emptying using ultrasound
- Change in Subject Reported Outcomes Using Mean Health Related Quality of Life (HRQoL) Scores [ Time Frame: Baseline, 12 months ] [ Designated as safety issue: No ]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).
| Enrollment: | 42 |
| Study Start Date: | January 2007 |
| Study Completion Date: | October 2008 |
| Primary Completion Date: | October 2008 (Final data collection date for primary outcome measure) |
| Arms | Assigned Interventions |
|---|---|
|
Active Comparator: Octreotide
Participants received Octreotide LAR® Depot injections (up to 40 mg) intramuscularly every 28 days (+/- 5 days) for one year
|
Drug: Octreotide
Participants received Octreotide LAR® Depot injections (up to 40 mg)intramuscularly every 28 days (+/- 5 days) for one year
Other Name: Octreotide LAR® Depot
|
|
Placebo Comparator: Placebo
Participants received an injection of placebo (sham) medication intramuscularly every 28 days (+/- 5 day) for one year
|
Drug: Placebo
Participants received an injection of placebo (sham) medication intramuscularly every 28 days (+/- 5 day) for one year
Other Name: Placebo injection
|
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| Ages Eligible for Study: | 18 Years to 80 Years |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
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
Contacts and Locations More Information
Additional Information:
Publications:
| Responsible Party: | Marie Hogan, MD, PhD, Assistant Professor of Medicine, College of Medicine, Mayo Clinic |
| ClinicalTrials.gov Identifier: | NCT00426153 History of Changes |
| Other Study ID Numbers: | 06-004128, UL1RR024150 |
| Study First Received: | January 22, 2007 |
| Results First Received: | October 22, 2012 |
| Last Updated: | October 22, 2012 |
| Health Authority: | United States: Institutional Review Board |
Additional relevant MeSH terms:
|
Abdominal Pain Hepatomegaly Polycystic Kidney Diseases Liver Diseases Polycystic Kidney, Autosomal Dominant Cysts Pain Signs and Symptoms Signs and Symptoms, Digestive Digestive System Diseases Hypertrophy |
Pathological Conditions, Anatomical Kidney Diseases, Cystic Kidney Diseases Urologic Diseases Neoplasms Octreotide Antineoplastic Agents, Hormonal Antineoplastic Agents Therapeutic Uses Pharmacologic Actions Gastrointestinal Agents |
ClinicalTrials.gov processed this record on May 19, 2013