Not provided
| ID | Type | Description | Link |
|---|---|---|---|
| UL1RR024150 | U.S. NIH Grant/Contract | View source |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Name | Class |
|---|---|
| Novartis | INDUSTRY |
| National Center for Research Resources (NCRR) | NIH |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
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.
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.
Not provided
Not provided
Not provided
Not provided
Not provided
| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Octreotide | Active Comparator | Participants received Octreotide LAR® Depot injections (up to 40 mg) intramuscularly every 28 days (+/- 5 days) for one year |
|
| Placebo | Placebo Comparator | Participants received an injection of placebo (sham) medication intramuscularly every 28 days (+/- 5 day) for one year |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Octreotide | Drug | Participants received Octreotide LAR® Depot injections (up to 40 mg)intramuscularly every 28 days (+/- 5 days) for one year |
|
| Measure | Description | Time Frame |
|---|---|---|
| Percent Change in Liver Volume | Percent change from baseline in liver volume, measured in milliliters by Magnetic Resonance Imaging (MRI)or Computed Tomography (CT) scans | Baseline, 12 months |
| Measure | Description | Time Frame |
|---|---|---|
| Percent Change in Renal Volume | Percent change from baseline in renal volume, measured in milliliters by MRI or CT scans | Baseline, 12 months |
| Percent Change in Glomerular Filtration Rate (GFR) |
Not provided
Inclusion Criteria:
Exclusion Criteria:
Not provided
Not provided
Not provided
Not provided
Not provided
| Name | Affiliation | Role |
|---|---|---|
| Marie C. Hogan, M.D., Ph.D. | Mayo Clinic | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Mayo Clinic | Rochester | Minnesota | 55905 | United States |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 22773240 | Result | 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. | |
| 39356039 | Derived | St Pierre K, Cashmore BA, Bolignano D, Zoccali C, Ruospo M, Craig JC, Strippoli GF, Mallett AJ, Green SC, Tunnicliffe DJ. Interventions for preventing the progression of autosomal dominant polycystic kidney disease. Cochrane Database Syst Rev. 2024 Oct 2;10(10):CD010294. doi: 10.1002/14651858.CD010294.pub3. |
Not provided
Not provided
Following consent a tolerability test dose of 100 micrograms of short-acting octreotide was administered subcutaneously to subjects, followed by 4 hours of observation/vital signs. Randomized intramuscular dosing began the next day.
42 participants were enrolled at Mayo Clinic in Rochester, Minnesota from 1/1/2007 to 5/19/2008.
Not provided
| ID | Title | Description |
|---|---|---|
| FG000 | Octreotide | Participants received Octreotide LAR® Depot injections intramuscularly every 28 days (+/- 5 days) for one year |
| FG001 | Placebo | Participants received an injection of placebo (sham) medication intramuscularly every 28 days (+/- 5 day) for one year |
| Title | Milestones | Reasons Not Completed | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
Not provided
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | Octreotide | Participants received Octreotide LAR® Depot injections intramuscularly every 28 days (+/- 5 days) for one year |
| BG001 | Placebo | Participants received an injection of placebo (sham) medication intramuscularly every 28 days (+/- 5 day) for one year |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age Continuous | Mean |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Percent Change in Liver Volume | Percent change from baseline in liver volume, measured in milliliters by Magnetic Resonance Imaging (MRI)or Computed Tomography (CT) scans | Posted | Mean | Standard Deviation | percent change | Baseline, 12 months |
|
1 year
Not provided
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Octreotide | Participants received Octreotide LAR® Depot injections intramuscularly every 28 days (+/- 5 days) for one year |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Abdominal hernia, incarcerated | Gastrointestinal disorders | Non-systematic Assessment | Hernia surgically repaired |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Alopecia, moderate | Skin and subcutaneous tissue disorders | Non-systematic Assessment |
Not provided
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Marie Hogan, MD, PhD, Assistant Prof of Medicine, College of Medicine | Mayo Clinic | 507-266-9364 | hogan.marie@mayo.edu |
Not provided
| ID | Term |
|---|---|
| D016891 | Polycystic Kidney, Autosomal Dominant |
| C536330 | Polycystic liver disease |
| D006529 | Hepatomegaly |
| D008107 | Liver Diseases |
| D007690 | Polycystic Kidney Diseases |
| D015746 | Abdominal Pain |
| ID | Term |
|---|---|
| D052177 | Kidney Diseases, Cystic |
| D007674 | Kidney Diseases |
| D014570 | Urologic Diseases |
| D052776 | Female Urogenital Diseases |
Not provided
Not provided
| ID | Term |
|---|---|
| D015282 | Octreotide |
| ID | Term |
|---|---|
| D010456 | Peptides, Cyclic |
| D047028 | Macrocyclic Compounds |
| D011083 | Polycyclic Compounds |
| D010455 | Peptides |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Placebo | Drug | Participants received an injection of placebo (sham) medication intramuscularly every 28 days (+/- 5 day) for one year |
|
|
Percent change from baseline in renal function/GFR, measured by clearance of iothalamate with monitoring of bladder emptying using ultrasound
| Baseline, 12 months |
| Change in Subject Reported Outcomes Using Mean Health Related Quality of Life (HRQoL) Scores | 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). | Baseline, 12 months |
| 26166166 | Derived | 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. |
| BG002 | Total | Total of all reporting groups |
| years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Region of Enrollment | Number | participants |
|
| Glomerular Filtration Rate | Glomerular Filtration Rate measures how fast the kidneys are filtering. 13 subjects were excluded from the analysis of the kidney data: 4 subjects had renal transplant before enrollment (3 in octreotide and 1 in placebo groups), 8 subjects had a diagnosis of ADPLD (4 in each group), 1 placebo subject has missing kidney data due to incomplete image coverage. | Mean | Standard Deviation | milliliters/minute per 1.73 m^2 |
|
| Body Mass Index | Body Mass Index (BMI) is a health index for comparing weight to height. BMI is a person's weight in kilograms (kg) divided by his or her height in meters squared. The body mass index is an indication if a person is at a suitable weight for his height on an approximation of body fat. A body mass index of under 20 is considered to be underweight, while a body mass index between 20 to 25 is considered healthy. A body mass index in the range of 25 to 30 is regarded as overweight. A body mass index over 30 is regarded as obese. | Mean | Standard Deviation | kilograms/meter^2 |
|
| Body Weight | Mean | Standard Deviation | kilograms |
|
| Serum creatinine | Creatinine in the serum is a blood test, and is an indicator of kidney function. | Mean | Standard Deviation | milligrams/deciliter |
|
| Fasting plasma glucose | Mean | Standard Deviation | milligrams/deciliter |
|
| Urine Albumin | A protein urine test measures the amount of proteins, such as albumin, found in a urine sample. Urine albumin is an indicator of kidney function. | Mean | Standard Deviation | milligrams/24 hours |
|
| Systolic Blood Pressure | Systolic blood pressure is the upper number on a blood pressure reading and measures contraction of the ventricles, or lower chambers of the heart | Mean | Standard Deviation | millimeters of mercury (mmHg) |
|
| Diastolic Blood Pressure | Diastolic blood pressure is the lower number on a blood pressure reading and measures dilation of the ventricles, or lower chambers of the heart. | Mean | Standard Deviation | millimeters of mercury (mmHg) |
|
| Liver volume | Liver volume was measured by magnetic resonance imaging (MRI) or computed tomography [CT]. | Mean | Standard Deviation | milliliters (ml) |
|
| Kidney volume | Kidney volume was measured by magnetic resonance imaging (MRI) or computed tomography [CT]. 13 subjects were excluded from the analysis of the kidney data: 4 subjects had renal transplant before enrollment (3 in octreotide and 1 in placebo groups), 8 subjects had a diagnosis of ADPLD (4 in each group), 1 placebo subject has missing kidney data due to incomplete image coverage. (Octreotide group n = 21 and placebo group n = 8). | Mean | Standard Deviation | milliliters (ml) |
|
| Genotypes and genetic mutations | Autosomal dominant polycystic kidney disease (ADPKD) is caused by mutations in one of two genes: PKD1 or PKD2, and is characterized by the development of kidney cysts and other manifestations, of which polycystic live disease (PLD) is most common. Autosomal dominant PLD (ADPLD) also exists as a genetically distinct disease with few or absent kidney cysts. Like ADPKD, ADPLD is genetically heterogeneous, with the 1st two genes identified (PRKCSH & SEC63) accounting for approximately 1/3 to 1/2 of isolated ADPLD cases. | Number | Participants |
|
| Units | Counts |
|---|
| Participants |
|
|
|
| Secondary | Percent Change in Renal Volume | Percent change from baseline in renal volume, measured in milliliters by MRI or CT scans | 13 subjects were excluded from the analysis of the kidney data: 4 subjects had renal transplant before enrollment (3 in octreotide and 1 in placebo groups), 8 subjects had a diagnosis of ADPLD (4 in each group), 1 placebo subject has missing kidney data due to incomplete image coverage. | Posted | Mean | Standard Deviation | percent change | Baseline, 12 months |
|
|
|
|
| Secondary | Percent Change in Glomerular Filtration Rate (GFR) | Percent change from baseline in renal function/GFR, measured by clearance of iothalamate with monitoring of bladder emptying using ultrasound | 13 subjects were excluded from the analysis of the kidney data: 4 subjects had renal transplant before enrollment (3 in octreotide and 1 in placebo groups), 8 subjects had a diagnosis of ADPLD (4 in each group), 1 placebo subject has missing kidney data due to incomplete image coverage. | Posted | Mean | Standard Deviation | percent change | Baseline, 12 months |
|
|
|
|
| Secondary | Change in Subject Reported Outcomes Using Mean Health Related Quality of Life (HRQoL) Scores | 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). | Posted | Mean | Standard Deviation | units on a scale | Baseline, 12 months |
|
|
|
| 3 |
| 28 |
| 21 |
| 28 |
| EG001 | Placebo | Participants received an injection of placebo (sham) medication intramuscularly every 28 days (+/- 5 day) for one year | 0 | 14 | 4 | 14 |
|
| Bacteremia | Renal and urinary disorders | Non-systematic Assessment | Bacteremia associated with nephrolithiasis |
|
| Urinary tract infection | Renal and urinary disorders | Non-systematic Assessment | UTI, abdominal pain and fever responded to antibiotic therapy |
|
| Diarrhea, Grade 1 | Gastrointestinal disorders | Non-systematic Assessment |
|
| Injection site granuloma | Skin and subcutaneous tissue disorders | Non-systematic Assessment |
|
| Injection site pain | Skin and subcutaneous tissue disorders | Non-systematic Assessment |
|
| Steatorrhea & weight loss | Gastrointestinal disorders | Non-systematic Assessment | Subject withdrew from study due to steatorrhea & weight loss |
|
| abdominal cramping, bloating and gas | Gastrointestinal disorders | Non-systematic Assessment |
|
Not provided
Not provided
| D005261 |
| Female Urogenital Diseases and Pregnancy Complications |
| D000091642 | Urogenital Diseases |
| D052801 | Male Urogenital Diseases |
| D000015 | Abnormalities, Multiple |
| D000013 | Congenital Abnormalities |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D000072661 | Ciliopathies |
| D030342 | Genetic Diseases, Inborn |
| D004066 | Digestive System Diseases |
| D006984 | Hypertrophy |
| D020763 | Pathological Conditions, Anatomical |
| D013568 | Pathological Conditions, Signs and Symptoms |
| D010146 | Pain |
| D009461 | Neurologic Manifestations |
| D012816 | Signs and Symptoms |
| D012817 | Signs and Symptoms, Digestive |
| D000602 |
| Amino Acids, Peptides, and Proteins |
| Physical role at baseline |
|
| Physical role at 12 months |
|
| Bodily Pain at baseline |
|
| Bodily pain at 12 months |
|
| General health at baseline |
|
| General health at 12 months |
|
| Vitality at baseline |
|
| Vitality at 12 months |
|
| Social functioning at baseline |
|
| Social functioning at 12 months |
|
| Emotional role at baseline |
|
| Emotional role at 12 month |
|
| Mental health at baseline |
|
| Mental health at 12 months |
|
| Standardized physical component at baseline |
|
| Standardized physical component at 12 months |
|
| Standardized mental component at baseline |
|
| Standardized mental component at 12 month |
|