Trial Outcomes & Findings for Octreotide in Severe Polycystic Liver Disease (NCT NCT00426153)
NCT ID: NCT00426153
Last Updated: 2012-11-21
Results Overview
Percent change from baseline in liver volume, measured in milliliters by Magnetic Resonance Imaging (MRI)or Computed Tomography (CT) scans
Recruitment status
COMPLETED
Study phase
PHASE2/PHASE3
Target enrollment
42 participants
Primary outcome timeframe
Baseline, 12 months
Results posted on
2012-11-21
Participant Flow
42 participants were enrolled at Mayo Clinic in Rochester, Minnesota from 1/1/2007 to 5/19/2008.
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.
Participant milestones
| Measure |
Octreotide
Participants received Octreotide LAR® Depot injections intramuscularly every 28 days (+/- 5 days) for one year
|
Placebo
Participants received an injection of placebo (sham) medication intramuscularly every 28 days (+/- 5 day) for one year
|
|---|---|---|
|
Overall Study
STARTED
|
28
|
14
|
|
Overall Study
COMPLETED
|
28
|
14
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Octreotide in Severe Polycystic Liver Disease
Baseline characteristics by cohort
| Measure |
Octreotide
n=28 Participants
Participants received Octreotide LAR® Depot injections intramuscularly every 28 days (+/- 5 days) for one year
|
Placebo
n=14 Participants
Participants received an injection of placebo (sham) medication intramuscularly every 28 days (+/- 5 day) for one year
|
Total
n=42 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age Continuous
|
49.7 years
STANDARD_DEVIATION 9 • n=93 Participants
|
50.3 years
STANDARD_DEVIATION 7.3 • n=4 Participants
|
49.9 years
STANDARD_DEVIATION 8.4 • n=27 Participants
|
|
Sex: Female, Male
Female
|
23 Participants
n=93 Participants
|
13 Participants
n=4 Participants
|
36 Participants
n=27 Participants
|
|
Sex: Female, Male
Male
|
5 Participants
n=93 Participants
|
1 Participants
n=4 Participants
|
6 Participants
n=27 Participants
|
|
Region of Enrollment
United States
|
28 participants
n=93 Participants
|
14 participants
n=4 Participants
|
42 participants
n=27 Participants
|
|
Glomerular Filtration Rate
|
70 milliliters/minute per 1.73 m^2
STANDARD_DEVIATION 27 • n=93 Participants
|
71 milliliters/minute per 1.73 m^2
STANDARD_DEVIATION 27 • n=4 Participants
|
70 milliliters/minute per 1.73 m^2
STANDARD_DEVIATION 27 • n=27 Participants
|
|
Body Mass Index
|
26.3 kilograms/meter^2
STANDARD_DEVIATION 5.77 • n=93 Participants
|
24.4 kilograms/meter^2
STANDARD_DEVIATION 2.98 • n=4 Participants
|
25.7 kilograms/meter^2
STANDARD_DEVIATION 5.0 • n=27 Participants
|
|
Body Weight
|
76.0 kilograms
STANDARD_DEVIATION 20.2 • n=93 Participants
|
70.9 kilograms
STANDARD_DEVIATION 10.9 • n=4 Participants
|
74.3 kilograms
STANDARD_DEVIATION 17.8 • n=27 Participants
|
|
Serum creatinine
|
1.1 milligrams/deciliter
STANDARD_DEVIATION 0.4 • n=93 Participants
|
1.1 milligrams/deciliter
STANDARD_DEVIATION 0.5 • n=4 Participants
|
1.1 milligrams/deciliter
STANDARD_DEVIATION 0.5 • n=27 Participants
|
|
Fasting plasma glucose
|
93.4 milligrams/deciliter
STANDARD_DEVIATION 11.2 • n=93 Participants
|
93.6 milligrams/deciliter
STANDARD_DEVIATION 7.8 • n=4 Participants
|
93.5 milligrams/deciliter
STANDARD_DEVIATION 10.1 • n=27 Participants
|
|
Urine Albumin
|
65 milligrams/24 hours
STANDARD_DEVIATION 123 • n=93 Participants
|
130 milligrams/24 hours
STANDARD_DEVIATION 237 • n=4 Participants
|
87 milligrams/24 hours
STANDARD_DEVIATION 171 • n=27 Participants
|
|
Systolic Blood Pressure
|
122.1 millimeters of mercury (mmHg)
STANDARD_DEVIATION 13.2 • n=93 Participants
|
120.5 millimeters of mercury (mmHg)
STANDARD_DEVIATION 13.5 • n=4 Participants
|
121.6 millimeters of mercury (mmHg)
STANDARD_DEVIATION 13.2 • n=27 Participants
|
|
Diastolic Blood Pressure
|
79.8 millimeters of mercury (mmHg)
STANDARD_DEVIATION 8.9 • n=93 Participants
|
79.1 millimeters of mercury (mmHg)
STANDARD_DEVIATION 9.2 • n=4 Participants
|
79.6 millimeters of mercury (mmHg)
STANDARD_DEVIATION 8.9 • n=27 Participants
|
|
Liver volume
|
5907.7 milliliters (ml)
STANDARD_DEVIATION 2915.0 • n=93 Participants
|
5373.9 milliliters (ml)
STANDARD_DEVIATION 3565.4 • n=4 Participants
|
5729.8 milliliters (ml)
STANDARD_DEVIATION 3113.1 • n=27 Participants
|
|
Kidney volume
|
1142.9 milliliters (ml)
STANDARD_DEVIATION 826.9 • n=93 Participants
|
803.0 milliliters (ml)
STANDARD_DEVIATION 269.1 • n=4 Participants
|
1049.2 milliliters (ml)
STANDARD_DEVIATION 728.3 • n=27 Participants
|
|
Genotypes and genetic mutations
ADPKD genotype with PKD1 gene mutation
|
16 Participants
n=93 Participants
|
9 Participants
n=4 Participants
|
25 Participants
n=27 Participants
|
|
Genotypes and genetic mutations
ADPKD genotype with PKD2 gene mutation
|
5 Participants
n=93 Participants
|
1 Participants
n=4 Participants
|
6 Participants
n=27 Participants
|
|
Genotypes and genetic mutations
ADPKD genotype with no genetic mutation detected
|
3 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
3 Participants
n=27 Participants
|
|
Genotypes and genetic mutations
ADPLD genotype with PRKCSH gene mutation
|
3 Participants
n=93 Participants
|
1 Participants
n=4 Participants
|
4 Participants
n=27 Participants
|
|
Genotypes and genetic mutations
ADPLD genotype with SEC63 gene mutation
|
0 Participants
n=93 Participants
|
1 Participants
n=4 Participants
|
1 Participants
n=27 Participants
|
|
Genotypes and genetic mutations
ADPLD genotype with no genetic mutation detected
|
1 Participants
n=93 Participants
|
2 Participants
n=4 Participants
|
3 Participants
n=27 Participants
|
PRIMARY outcome
Timeframe: Baseline, 12 monthsPercent change from baseline in liver volume, measured in milliliters by Magnetic Resonance Imaging (MRI)or Computed Tomography (CT) scans
Outcome measures
| Measure |
Octreotide
n=28 Participants
Participants received Octreotide LAR® Depot injections intramuscularly every 28 days (+/- 5 days) for one year
|
Placebo
n=14 Participants
Participants received an injection of placebo (sham) medication intramuscularly every 28 days (+/- 5 day) for one year
|
|---|---|---|
|
Percent Change in Liver Volume
|
-5.0 percent change
Standard Deviation 6.77
|
0.9 percent change
Standard Deviation 8.33
|
SECONDARY outcome
Timeframe: Baseline, 12 monthsPopulation: 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.
Percent change from baseline in renal volume, measured in milliliters by MRI or CT scans
Outcome measures
| Measure |
Octreotide
n=21 Participants
Participants received Octreotide LAR® Depot injections intramuscularly every 28 days (+/- 5 days) for one year
|
Placebo
n=8 Participants
Participants received an injection of placebo (sham) medication intramuscularly every 28 days (+/- 5 day) for one year
|
|---|---|---|
|
Percent Change in Renal Volume
|
0.25 percent change
Standard Deviation 7.53
|
8.61 percent change
Standard Deviation 10.07
|
SECONDARY outcome
Timeframe: Baseline, 12 monthsPopulation: 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.
Percent change from baseline in renal function/GFR, measured by clearance of iothalamate with monitoring of bladder emptying using ultrasound
Outcome measures
| Measure |
Octreotide
n=21 Participants
Participants received Octreotide LAR® Depot injections intramuscularly every 28 days (+/- 5 days) for one year
|
Placebo
n=8 Participants
Participants received an injection of placebo (sham) medication intramuscularly every 28 days (+/- 5 day) for one year
|
|---|---|---|
|
Percent Change in Glomerular Filtration Rate (GFR)
|
-5.1 percent change
Standard Deviation 15.46
|
-7.2 percent change
Standard Deviation 13.21
|
SECONDARY outcome
Timeframe: Baseline, 12 monthsScores 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).
Outcome measures
| Measure |
Octreotide
n=24 Participants
Participants received Octreotide LAR® Depot injections intramuscularly every 28 days (+/- 5 days) for one year
|
Placebo
n=12 Participants
Participants received an injection of placebo (sham) medication intramuscularly every 28 days (+/- 5 day) for one year
|
|---|---|---|
|
Change in Subject Reported Outcomes Using Mean Health Related Quality of Life (HRQoL) Scores
Physical functioning at baseline
|
74.8 units on a scale
Standard Deviation 21.96
|
80.4 units on a scale
Standard Deviation 23.73
|
|
Change in Subject Reported Outcomes Using Mean Health Related Quality of Life (HRQoL) Scores
Physical functioning at 12 months
|
77.0 units on a scale
Standard Deviation 21.32
|
82.1 units on a scale
Standard Deviation 18.58
|
|
Change in Subject Reported Outcomes Using Mean Health Related Quality of Life (HRQoL) Scores
Social functioning at baseline
|
79.0 units on a scale
Standard Deviation 19.56
|
75.0 units on a scale
Standard Deviation 27.30
|
|
Change in Subject Reported Outcomes Using Mean Health Related Quality of Life (HRQoL) Scores
Emotional role at 12 month
|
91.4 units on a scale
Standard Deviation 23.74
|
81.0 units on a scale
Standard Deviation 38.60
|
|
Change in Subject Reported Outcomes Using Mean Health Related Quality of Life (HRQoL) Scores
Standardized mental component at baseline
|
50.8 units on a scale
Standard Deviation 7.74
|
48.4 units on a scale
Standard Deviation 10.96
|
|
Change in Subject Reported Outcomes Using Mean Health Related Quality of Life (HRQoL) Scores
Physical role at baseline
|
59.8 units on a scale
Standard Deviation 42.13
|
76.8 units on a scale
Standard Deviation 39.79
|
|
Change in Subject Reported Outcomes Using Mean Health Related Quality of Life (HRQoL) Scores
Physical role at 12 months
|
74.1 units on a scale
Standard Deviation 35.00
|
75.0 units on a scale
Standard Deviation 41.60
|
|
Change in Subject Reported Outcomes Using Mean Health Related Quality of Life (HRQoL) Scores
Bodily Pain at baseline
|
67.8 units on a scale
Standard Deviation 15.29
|
65.5 units on a scale
Standard Deviation 24.25
|
|
Change in Subject Reported Outcomes Using Mean Health Related Quality of Life (HRQoL) Scores
Bodily pain at 12 months
|
75.7 units on a scale
Standard Deviation 19.02
|
68.7 units on a scale
Standard Deviation 25.51
|
|
Change in Subject Reported Outcomes Using Mean Health Related Quality of Life (HRQoL) Scores
General health at baseline
|
55.9 units on a scale
Standard Deviation 21.29
|
58.0 units on a scale
Standard Deviation 23.63
|
|
Change in Subject Reported Outcomes Using Mean Health Related Quality of Life (HRQoL) Scores
General health at 12 months
|
53.5 units on a scale
Standard Deviation 17.32
|
63.4 units on a scale
Standard Deviation 23.96
|
|
Change in Subject Reported Outcomes Using Mean Health Related Quality of Life (HRQoL) Scores
Vitality at baseline
|
49.6 units on a scale
Standard Deviation 21.81
|
53.9 units on a scale
Standard Deviation 25.96
|
|
Change in Subject Reported Outcomes Using Mean Health Related Quality of Life (HRQoL) Scores
Vitality at 12 months
|
54.4 units on a scale
Standard Deviation 24.23
|
54.6 units on a scale
Standard Deviation 27.91
|
|
Change in Subject Reported Outcomes Using Mean Health Related Quality of Life (HRQoL) Scores
Social functioning at 12 months
|
82.9 units on a scale
Standard Deviation 19.35
|
81.3 units on a scale
Standard Deviation 21.79
|
|
Change in Subject Reported Outcomes Using Mean Health Related Quality of Life (HRQoL) Scores
Emotional role at baseline
|
82.1 units on a scale
Standard Deviation 32.05
|
73.8 units on a scale
Standard Deviation 37.39
|
|
Change in Subject Reported Outcomes Using Mean Health Related Quality of Life (HRQoL) Scores
Mental health at baseline
|
76.0 units on a scale
Standard Deviation 14.12
|
75.4 units on a scale
Standard Deviation 18.75
|
|
Change in Subject Reported Outcomes Using Mean Health Related Quality of Life (HRQoL) Scores
Mental health at 12 months
|
76.9 units on a scale
Standard Deviation 16.54
|
80.7 units on a scale
Standard Deviation 18.76
|
|
Change in Subject Reported Outcomes Using Mean Health Related Quality of Life (HRQoL) Scores
Standardized physical component at baseline
|
42.6 units on a scale
Standard Deviation 9.73
|
46.0 units on a scale
Standard Deviation 10.16
|
|
Change in Subject Reported Outcomes Using Mean Health Related Quality of Life (HRQoL) Scores
Standardized physical component at 12 months
|
44.7 units on a scale
Standard Deviation 10.00
|
46.1 units on a scale
Standard Deviation 9.51
|
|
Change in Subject Reported Outcomes Using Mean Health Related Quality of Life (HRQoL) Scores
Standardized mental component at 12 month
|
52.2 units on a scale
Standard Deviation 8.03
|
51.3 units on a scale
Standard Deviation 11.15
|
Adverse Events
Octreotide
Serious events: 3 serious events
Other events: 21 other events
Deaths: 0 deaths
Placebo
Serious events: 0 serious events
Other events: 4 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Octreotide
n=28 participants at risk
Participants received Octreotide LAR® Depot injections intramuscularly every 28 days (+/- 5 days) for one year
|
Placebo
n=14 participants at risk
Participants received an injection of placebo (sham) medication intramuscularly every 28 days (+/- 5 day) for one year
|
|---|---|---|
|
Gastrointestinal disorders
Abdominal hernia, incarcerated
|
3.6%
1/28 • Number of events 1 • 1 year
|
0.00%
0/14 • 1 year
|
|
Renal and urinary disorders
Bacteremia
|
3.6%
1/28 • Number of events 1 • 1 year
|
0.00%
0/14 • 1 year
|
|
Renal and urinary disorders
Urinary tract infection
|
3.6%
1/28 • Number of events 1 • 1 year
|
0.00%
0/14 • 1 year
|
Other adverse events
| Measure |
Octreotide
n=28 participants at risk
Participants received Octreotide LAR® Depot injections intramuscularly every 28 days (+/- 5 days) for one year
|
Placebo
n=14 participants at risk
Participants received an injection of placebo (sham) medication intramuscularly every 28 days (+/- 5 day) for one year
|
|---|---|---|
|
Skin and subcutaneous tissue disorders
Alopecia, moderate
|
3.6%
1/28 • Number of events 1 • 1 year
|
0.00%
0/14 • 1 year
|
|
Gastrointestinal disorders
Diarrhea, Grade 1
|
60.7%
17/28 • Number of events 17 • 1 year
|
28.6%
4/14 • Number of events 4 • 1 year
|
|
Skin and subcutaneous tissue disorders
Injection site granuloma
|
17.9%
5/28 • Number of events 5 • 1 year
|
0.00%
0/14 • 1 year
|
|
Skin and subcutaneous tissue disorders
Injection site pain
|
75.0%
21/28 • Number of events 21 • 1 year
|
21.4%
3/14 • Number of events 3 • 1 year
|
|
Gastrointestinal disorders
Steatorrhea & weight loss
|
3.6%
1/28 • Number of events 1 • 1 year
|
0.00%
0/14 • 1 year
|
|
Gastrointestinal disorders
abdominal cramping, bloating and gas
|
50.0%
14/28 • Number of events 14 • 1 year
|
28.6%
4/14 • Number of events 4 • 1 year
|
Additional Information
Marie Hogan, MD, PhD, Assistant Prof of Medicine, College of Medicine
Mayo Clinic
Phone: 507-266-9364
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place