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Trial record 9 of 13 for:    "Polycystic Liver Disease" | "Liver Extracts"

Octreotide in Severe Polycystic Liver Disease

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ClinicalTrials.gov Identifier: NCT00426153
Recruitment Status : Completed
First Posted : January 24, 2007
Results First Posted : November 21, 2012
Last Update Posted : November 21, 2012
Sponsor:
Collaborators:
Novartis
National Center for Research Resources (NCRR)
Information provided by (Responsible Party):
Marie Hogan, Mayo Clinic

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Triple (Participant, Care Provider, Investigator);   Primary Purpose: Treatment
Conditions Polycystic Kidney, Autosomal Dominant
Polycystic Liver Disease
Hepatomegaly
Liver Diseases
Kidney, Polycystic
Abdominal Pain
Interventions Drug: Octreotide
Drug: Placebo
Enrollment 42
Recruitment Details 42 participants were enrolled at Mayo Clinic in Rochester, Minnesota from 1/1/2007 to 5/19/2008.
Pre-assignment Details 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.
Arm/Group Title Octreotide Placebo
Hide Arm/Group Description Participants received Octreotide LAR® Depot injections intramuscularly every 28 days (+/- 5 days) for one year Participants received an injection of placebo (sham) medication intramuscularly every 28 days (+/- 5 day) for one year
Period Title: Overall Study
Started 28 [1] 14
Completed 28 14
Not Completed 0 0
[1]
Participants were randomized in a 2:1 ratio, Octreotide LAR depot: Placebo
Arm/Group Title Octreotide Placebo Total
Hide Arm/Group Description Participants received Octreotide LAR® Depot injections intramuscularly every 28 days (+/- 5 days) for one year Participants received an injection of placebo (sham) medication intramuscularly every 28 days (+/- 5 day) for one year Total of all reporting groups
Overall Number of Baseline Participants 28 14 42
Hide Baseline Analysis Population Description
[Not Specified]
Age Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 28 participants 14 participants 42 participants
49.7  (9) 50.3  (7.3) 49.9  (8.4)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 28 participants 14 participants 42 participants
Female
23
  82.1%
13
  92.9%
36
  85.7%
Male
5
  17.9%
1
   7.1%
6
  14.3%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 28 participants 14 participants 42 participants
28 14 42
Glomerular Filtration Rate   [1] 
Mean (Standard Deviation)
Unit of measure:  Milliliters/minute per 1.73 m^2
Number Analyzed 28 participants 14 participants 42 participants
70  (27) 71  (27) 70  (27)
[1]
Measure Description: 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.
Body Mass Index   [1] 
Mean (Standard Deviation)
Unit of measure:  Kilograms/meter^2
Number Analyzed 28 participants 14 participants 42 participants
26.3  (5.77) 24.4  (2.98) 25.7  (5.0)
[1]
Measure Description: 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.
Body Weight  
Mean (Standard Deviation)
Unit of measure:  Kilograms
Number Analyzed 28 participants 14 participants 42 participants
76.0  (20.2) 70.9  (10.9) 74.3  (17.8)
Serum creatinine   [1] 
Mean (Standard Deviation)
Unit of measure:  Milligrams/deciliter
Number Analyzed 28 participants 14 participants 42 participants
1.1  (0.4) 1.1  (0.5) 1.1  (0.5)
[1]
Measure Description: Creatinine in the serum is a blood test, and is an indicator of kidney function.
Fasting plasma glucose  
Mean (Standard Deviation)
Unit of measure:  Milligrams/deciliter
Number Analyzed 28 participants 14 participants 42 participants
93.4  (11.2) 93.6  (7.8) 93.5  (10.1)
Urine Albumin   [1] 
Mean (Standard Deviation)
Unit of measure:  Milligrams/24 hours
Number Analyzed 28 participants 14 participants 42 participants
65  (123) 130  (237) 87  (171)
[1]
Measure Description: 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.
Systolic Blood Pressure   [1] 
Mean (Standard Deviation)
Unit of measure:  Millimeters of mercury (mmHg)
Number Analyzed 28 participants 14 participants 42 participants
122.1  (13.2) 120.5  (13.5) 121.6  (13.2)
[1]
Measure Description: Systolic blood pressure is the upper number on a blood pressure reading and measures contraction of the ventricles, or lower chambers of the heart
Diastolic Blood Pressure   [1] 
Mean (Standard Deviation)
Unit of measure:  Millimeters of mercury (mmHg)
Number Analyzed 28 participants 14 participants 42 participants
79.8  (8.9) 79.1  (9.2) 79.6  (8.9)
[1]
Measure Description: Diastolic blood pressure is the lower number on a blood pressure reading and measures dilation of the ventricles, or lower chambers of the heart.
Liver volume   [1] 
Mean (Standard Deviation)
Unit of measure:  Milliliters (ml)
Number Analyzed 28 participants 14 participants 42 participants
5907.7  (2915.0) 5373.9  (3565.4) 5729.8  (3113.1)
[1]
Measure Description: Liver volume was measured by magnetic resonance imaging (MRI) or computed tomography [CT].
Kidney volume   [1] 
Mean (Standard Deviation)
Unit of measure:  Milliliters (ml)
Number Analyzed 28 participants 14 participants 42 participants
1142.9  (826.9) 803.0  (269.1) 1049.2  (728.3)
[1]
Measure Description:

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).

Genotypes and genetic mutations   [1] 
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 28 participants 14 participants 42 participants
ADPKD genotype with PKD1 gene mutation 16 9 25
ADPKD genotype with PKD2 gene mutation 5 1 6
ADPKD genotype with no genetic mutation detected 3 0 3
ADPLD genotype with PRKCSH gene mutation 3 1 4
ADPLD genotype with SEC63 gene mutation 0 1 1
ADPLD genotype with no genetic mutation detected 1 2 3
[1]
Measure Description: 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.
1.Primary Outcome
Title Percent Change in Liver Volume
Hide Description Percent change from baseline in liver volume, measured in milliliters by Magnetic Resonance Imaging (MRI)or Computed Tomography (CT) scans
Time Frame Baseline, 12 months
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Octreotide Placebo
Hide Arm/Group Description:
Participants received Octreotide LAR® Depot injections intramuscularly every 28 days (+/- 5 days) for one year
Participants received an injection of placebo (sham) medication intramuscularly every 28 days (+/- 5 day) for one year
Overall Number of Participants Analyzed 28 14
Mean (Standard Deviation)
Unit of Measure: percent change
-5.0  (6.77) 0.9  (8.33)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Octreotide, Placebo
Comments P values <0.05 were considered statistically significant.
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.048
Comments [Not Specified]
Method Rank-sum test
Comments [Not Specified]
2.Secondary Outcome
Title Percent Change in Renal Volume
Hide Description Percent change from baseline in renal volume, measured in milliliters by MRI or CT scans
Time Frame Baseline, 12 months
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Octreotide Placebo
Hide Arm/Group Description:
Participants received Octreotide LAR® Depot injections intramuscularly every 28 days (+/- 5 days) for one year
Participants received an injection of placebo (sham) medication intramuscularly every 28 days (+/- 5 day) for one year
Overall Number of Participants Analyzed 21 8
Mean (Standard Deviation)
Unit of Measure: percent change
0.25  (7.53) 8.61  (10.07)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Octreotide, Placebo
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.045
Comments P values < 0.05 were considered statistically significant
Method Rank sum
Comments [Not Specified]
3.Secondary Outcome
Title Percent Change in Glomerular Filtration Rate (GFR)
Hide Description Percent change from baseline in renal function/GFR, measured by clearance of iothalamate with monitoring of bladder emptying using ultrasound
Time Frame Baseline, 12 months
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Octreotide Placebo
Hide Arm/Group Description:
Participants received Octreotide LAR® Depot injections intramuscularly every 28 days (+/- 5 days) for one year
Participants received an injection of placebo (sham) medication intramuscularly every 28 days (+/- 5 day) for one year
Overall Number of Participants Analyzed 21 8
Mean (Standard Deviation)
Unit of Measure: percent change
-5.1  (15.46) -7.2  (13.21)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Octreotide, Placebo
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.98
Comments P Values < 0.05 were considered statistically significant
Method Rank sum
Comments [Not Specified]
4.Secondary Outcome
Title Change in Subject Reported Outcomes Using Mean Health Related Quality of Life (HRQoL) Scores
Hide Description 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).
Time Frame Baseline, 12 months
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Octreotide Placebo
Hide Arm/Group Description:
Participants received Octreotide LAR® Depot injections intramuscularly every 28 days (+/- 5 days) for one year
Participants received an injection of placebo (sham) medication intramuscularly every 28 days (+/- 5 day) for one year
Overall Number of Participants Analyzed 24 12
Mean (Standard Deviation)
Unit of Measure: units on a scale
Physical functioning at baseline 74.8  (21.96) 80.4  (23.73)
Physical functioning at 12 months 77.0  (21.32) 82.1  (18.58)
Physical role at baseline 59.8  (42.13) 76.8  (39.79)
Physical role at 12 months 74.1  (35.00) 75.0  (41.60)
Bodily Pain at baseline 67.8  (15.29) 65.5  (24.25)
Bodily pain at 12 months 75.7  (19.02) 68.7  (25.51)
General health at baseline 55.9  (21.29) 58.0  (23.63)
General health at 12 months 53.5  (17.32) 63.4  (23.96)
Vitality at baseline 49.6  (21.81) 53.9  (25.96)
Vitality at 12 months 54.4  (24.23) 54.6  (27.91)
Social functioning at baseline 79.0  (19.56) 75.0  (27.30)
Social functioning at 12 months 82.9  (19.35) 81.3  (21.79)
Emotional role at baseline 82.1  (32.05) 73.8  (37.39)
Emotional role at 12 month 91.4  (23.74) 81.0  (38.60)
Mental health at baseline 76.0  (14.12) 75.4  (18.75)
Mental health at 12 months 76.9  (16.54) 80.7  (18.76)
Standardized physical component at baseline 42.6  (9.73) 46.0  (10.16)
Standardized physical component at 12 months 44.7  (10.00) 46.1  (9.51)
Standardized mental component at baseline 50.8  (7.74) 48.4  (10.96)
Standardized mental component at 12 month 52.2  (8.03) 51.3  (11.15)
Time Frame 1 year
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Octreotide Placebo
Hide Arm/Group Description Participants received Octreotide LAR® Depot injections intramuscularly every 28 days (+/- 5 days) for one year Participants received an injection of placebo (sham) medication intramuscularly every 28 days (+/- 5 day) for one year
All-Cause Mortality
Octreotide Placebo
Affected / at Risk (%) Affected / at Risk (%)
Total   --/--      --/--    
Show Serious Adverse Events Hide Serious Adverse Events
Octreotide Placebo
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   3/28 (10.71%)      0/14 (0.00%)    
Gastrointestinal disorders     
Abdominal hernia, incarcerated * [1]  1/28 (3.57%)  1 0/14 (0.00%)  0
Renal and urinary disorders     
Bacteremia * [2]  1/28 (3.57%)  1 0/14 (0.00%)  0
Urinary tract infection * [3]  1/28 (3.57%)  1 0/14 (0.00%)  0
*
Indicates events were collected by non-systematic assessment
[1]
Hernia surgically repaired
[2]
Bacteremia associated with nephrolithiasis
[3]
UTI, abdominal pain and fever responded to antibiotic therapy
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Octreotide Placebo
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   21/28 (75.00%)      4/14 (28.57%)    
Gastrointestinal disorders     
Diarrhea, Grade 1 *  17/28 (60.71%)  17 4/14 (28.57%)  4
Steatorrhea & weight loss * [1]  1/28 (3.57%)  1 0/14 (0.00%)  0
abdominal cramping, bloating and gas *  14/28 (50.00%)  14 4/14 (28.57%)  4
Skin and subcutaneous tissue disorders     
Alopecia, moderate *  1/28 (3.57%)  1 0/14 (0.00%)  0
Injection site granuloma *  5/28 (17.86%)  5 0/14 (0.00%)  0
Injection site pain *  21/28 (75.00%)  21 3/14 (21.43%)  3
*
Indicates events were collected by non-systematic assessment
[1]
Subject withdrew from study due to steatorrhea & weight loss
Certain Agreements
All Principal Investigators ARE employed by the organization sponsoring the study.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Marie Hogan, MD, PhD, Assistant Prof of Medicine, College of Medicine
Organization: Mayo Clinic
Phone: 507-266-9364
EMail: hogan.marie@mayo.edu
Layout table for additonal information
Responsible Party: Marie Hogan, Mayo Clinic
ClinicalTrials.gov Identifier: NCT00426153     History of Changes
Other Study ID Numbers: 06-004128
UL1RR024150 ( U.S. NIH Grant/Contract )
First Submitted: January 22, 2007
First Posted: January 24, 2007
Results First Submitted: October 22, 2012
Results First Posted: November 21, 2012
Last Update Posted: November 21, 2012