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Trial record 6 of 19 for:    MIPOMERSEN

Safety and Efficacy of Mipomersen (ISIS 301012) As Add-on Therapy in High Risk Hypercholesterolemic Patients

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ClinicalTrials.gov Identifier: NCT00770146
Recruitment Status : Completed
First Posted : October 9, 2008
Results First Posted : March 20, 2013
Last Update Posted : September 9, 2016
Sponsor:
Collaborator:
Ionis Pharmaceuticals, Inc.
Information provided by (Responsible Party):
Kastle Therapeutics, LLC

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Conditions Hypercholesterolemia
Coronary Heart Disease
Interventions Drug: Mipomersen sodium
Drug: Placebo
Enrollment 158
Recruitment Details  
Pre-assignment Details Five hundred and seventy-seven patients were screened and 158 patients were randomized at 43 study centers in a 2:1 ratio to receive mipomersen or placebo once a week for 26 weeks. Participants who finished treatment or who discontinued prematurely from the study for any reason were assessed for safety for 24 weeks after the last study drug dose.
Arm/Group Title Placebo Mipomersen
Hide Arm/Group Description Participants received placebo subcutaneous injection once a week for 26 weeks. Participants received mipomersen 200 mg as a subcutaneous injection once a week for 26 weeks.
Period Title: Treatment Period
Started 53 105
Treated 52 105
Full Analysis Set 50 [1] 101 [1]
Completed 44 60
Not Completed 9 45
Reason Not Completed
Withdrawal by Subject             6             13
Adverse Event             2             26
Other             1             5
Protocol non-compliance             0             1
[1]
Treated patients with a valid baseline & post-baseline LDL-C measurement
Period Title: Follow-up Period
Started 53 105
Completed 42 85
Not Completed 11 20
Reason Not Completed
Withdrawal by Subject             7             13
Adverse Event             0             2
Other             4             5
Arm/Group Title Placebo Mipomersen Total
Hide Arm/Group Description Participants received placebo subcutaneous injection once a week for 26 weeks. Participants received mipomersen 200 mg as a subcutaneous injection once a week for 26 weeks. Total of all reporting groups
Overall Number of Baseline Participants 52 105 157
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous   [1] 
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 52 participants 105 participants 157 participants
59.3  (9.5) 59.3  (10.0) 59.3  (9.8)
[1]
Measure Description: Baseline data are provided for the Safety Set.
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 52 participants 105 participants 157 participants
Female
23
  44.2%
53
  50.5%
76
  48.4%
Male
29
  55.8%
52
  49.5%
81
  51.6%
Race/Ethnicity, Customized  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 52 participants 105 participants 157 participants
White 40 83 123
Black 11 20 31
American Indian or Alaskan Native 1 1 2
Other race 0 1 1
Race/Ethnicity, Customized  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 52 participants 105 participants 157 participants
Hispanic or Latino 9 16 25
Not Hispanic or Latino 43 89 132
Body Mass Index (BMI)  
Mean (Standard Deviation)
Unit of measure:  Kg/m^2
Number Analyzed 52 participants 105 participants 157 participants
30.0  (4.42) 30.7  (4.61) 30.4  (4.55)
Waist/hip ratio  
Mean (Standard Deviation)
Unit of measure:  Ratio
Number Analyzed 52 participants 105 participants 157 participants
0.94  (0.07) 0.94  (0.06) 0.94  (0.06)
Metabolic syndrome   [1] 
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 52 participants 105 participants 157 participants
No 12 32 44
Yes 40 73 113
[1]
Measure Description:

A patient was classified as having metabolic syndrome if any 3 of the following risk factors existed: 1) Waist circumference ≥102 cm (men) or ≥88 cm (women); 2) Triglycerides ≥150 mg/dL*; 3) High density lipoprotein cholesterol <40 mg/dL (men) or <50 mg/dL (women)*; 4) Systolic blood pressure ≥130 or diastolic ≥85 mmHg *; 5) Fasting glucose ≥100 mg/dL *.

* = or on medication for the specified risk factor.

Tobacco use  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 52 participants 105 participants 157 participants
Current 11 18 29
Non-current 19 31 50
Never 22 56 78
Alcohol use  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 52 participants 105 participants 157 participants
Current 20 50 70
Non-current 12 24 36
Never 20 31 51
Diabetic status at screening  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 52 participants 105 participants 157 participants
Yes 30 58 88
No 22 47 69
Cardiovascular history   [1] 
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 52 participants 105 participants 157 participants
Angina 5 9 14
Coronary heart disease (CHD) 21 52 73
Myocardial infarction 11 17 28
Coronary artery bypass graft surgery 4 14 18
Percutaneous coronary intervention 4 18 22
Coronary artery disease without event 6 14 20
Other clinical atherosclerotic disease 3 11 14
Peripheral artery disease 1 5 6
Abdominal aortic aneurysm 1 0 1
Carotid 1 8 9
CHD or other atherosclerotic disease 24 58 82
[1]
Measure Description: Participants may be counted in more than one category.
1.Primary Outcome
Title Percent Change From Baseline in Low-density Lipoprotein Cholesterol (LDL-C) at the Primary Efficacy Time Point
Hide Description LDL cholesterol was measured in mg/dL. Samples were taken following an overnight fast. LDL-C was obtained using Friedewald’s calculation for patients with triglycerides ≤400 mg/dL and was directly measured by the central laboratory using ultracentrifugation for patients with triglycerides >400 mg/dL. The primary efficacy time point was the post-baseline visit closest to 14 days after the last dose of study treatment.
Time Frame Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET; Week 28 or the post-baseline visit closest to 14 days after the last dose).
Hide Outcome Measure Data
Hide Analysis Population Description
The Full Analysis Set, which consisted of all randomized patients who received at least 1 injection of study drug (mipomersen or placebo) and with a valid baseline and at least 1 post-baseline LDL-C measurement.
Arm/Group Title Placebo Mipomersen
Hide Arm/Group Description:
Participants received placebo subcutaneous injection once a week for 26 weeks.
Participants received mipomersen 200 mg as a subcutaneous injection once a week for 26 weeks.
Overall Number of Participants Analyzed 50 101
Median (Inter-Quartile Range)
Unit of Measure: percentage of baseline
-3.4
(-14.8 to 11.8)
-40.2
(-56.4 to -19.1)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo, Mipomersen
Comments Based upon prior clinical study experience with mipomersen, it was estimated that the standard deviation of the percent change in LDL-C is approximately 22%. With at least 20 patients in the control group and 40 patients in the mipomersen-treated group, this study would have at least 90% power to detect a 20% difference between the 2 groups.
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.001
Comments Statistical significance was concluded if p ≤ 0.05.
Method Wilcoxon (Mann-Whitney)
Comments [Not Specified]
2.Primary Outcome
Title Low-density Lipoprotein Cholesterol (LDL-C) at Baseline and at the Primary Efficacy Time Point
Hide Description The primary efficacy time point (PET) was the post-baseline visit closest to 14 days after the last dose of study treatment.
Time Frame Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET; Week 28 or the post-baseline visit closest to 14 days after the last dose).
Hide Outcome Measure Data
Hide Analysis Population Description
Full analysis set
Arm/Group Title Placebo Mipomersen
Hide Arm/Group Description:
Participants received placebo subcutaneous injection once a week for 26 weeks.
Participants received mipomersen 200 mg as a subcutaneous injection once a week for 26 weeks.
Overall Number of Participants Analyzed 50 101
Median (Inter-Quartile Range)
Unit of Measure: mg/dL
Baseline
112
(101 to 134)
116
(106 to 137)
Primary efficacy time point
109
(90 to 128)
69
(51 to 98)
3.Secondary Outcome
Title Percent Change From Baseline in Apolipoprotein B at the Primary Efficacy Time Point
Hide Description Apolipoprotein B was measured in mg/dL. Samples were taken following an overnight fast. The primary efficacy time point was the post-baseline visit closest to 14 days after the last dose of study treatment.
Time Frame Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET; Week 28 or the post-baseline visit closest to 14 days after the last dose).
Hide Outcome Measure Data
Hide Analysis Population Description
Full analysis set
Arm/Group Title Placebo Mipomersen
Hide Arm/Group Description:
Participants received placebo subcutaneous injection once a week for 26 weeks.
Participants received mipomersen 200 mg as a subcutaneous injection once a week for 26 weeks.
Overall Number of Participants Analyzed 50 101
Median (Inter-Quartile Range)
Unit of Measure: percentage of baseline
-1.7
(-12.6 to 7.5)
-40.6
(-53.9 to -22.6)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo, Mipomersen
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.001
Comments Inflation of type 1 error was controlled by specifying a small number of secondary parameters and a sequential inferential approach in which inferential conclusions about each successive parameter required statistical significance of the prior one.
Method Wilcoxon (Mann-Whitney)
Comments [Not Specified]
4.Secondary Outcome
Title Apolipoprotein B at Baseline and at the Primary Efficacy Time Point
Hide Description The primary efficacy time point (PET) was the post-baseline visit closest to 14 days after the last dose of study treatment.
Time Frame Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET; Week 28 or the post-baseline visit closest to 14 days after the last dose).
Hide Outcome Measure Data
Hide Analysis Population Description
Full analysis set
Arm/Group Title Placebo Mipomersen
Hide Arm/Group Description:
Participants received placebo subcutaneous injection once a week for 26 weeks.
Participants received mipomersen 200 mg as a subcutaneous injection once a week for 26 weeks.
Overall Number of Participants Analyzed 50 101
Median (Inter-Quartile Range)
Unit of Measure: mg/dL
Baseline
106
(98 to 132)
114
(102 to 129)
Primary efficacy time point
108
(91 to 122)
64
(52 to 95)
5.Secondary Outcome
Title Percent Change From Baseline in Total Cholesterol at the Primary Efficacy Time Point
Hide Description Total cholesterol was measured in mg/dL. Samples were taken following an overnight fast. The primary efficacy time point was the post-baseline visit closest to 14 days after the last dose of study treatment.
Time Frame Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET; Week 28 or the post-baseline visit closest to 14 days after the last dose).
Hide Outcome Measure Data
Hide Analysis Population Description
Full analysis set
Arm/Group Title Placebo Mipomersen
Hide Arm/Group Description:
Participants received placebo subcutaneous injection once a week for 26 weeks.
Participants received mipomersen 200 mg as a subcutaneous injection once a week for 26 weeks.
Overall Number of Participants Analyzed 50 101
Mean (Standard Deviation)
Unit of Measure: percentage of baseline
-2.7  (14.58) -26.4  (18.65)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo, Mipomersen
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.001
Comments Inflation of type 1 error was controlled by specifying a small number of secondary parameters and a sequential inferential approach in which inferential conclusions about each successive parameter required statistical significance of the prior one.
Method t-test, 2 sided
Comments [Not Specified]
6.Secondary Outcome
Title Total Cholesterol at Baseline and at the Primary Efficacy Time Point
Hide Description The primary efficacy time point (PET) was the post-baseline visit closest to 14 days after the last dose of study treatment.
Time Frame Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET; Week 28 or the post-baseline visit closest to 14 days after the last dose).
Hide Outcome Measure Data
Hide Analysis Population Description
Full analysis set
Arm/Group Title Placebo Mipomersen
Hide Arm/Group Description:
Participants received placebo subcutaneous injection once a week for 26 weeks.
Participants received mipomersen 200 mg as a subcutaneous injection once a week for 26 weeks.
Overall Number of Participants Analyzed 50 101
Mean (Standard Deviation)
Unit of Measure: mg/dL
Baseline 200.0  (42.1) 202.6  (36.8)
Primary efficacy time point 192.2  (38.3) 147.4  (39.9)
7.Secondary Outcome
Title Percent Change From Baseline in Non-High-Density Lipoprotein Cholesterol at the Primary Efficacy Time Point
Hide Description Non-high-density lipoprotein cholesterol was measured in mg/dL. Samples were taken following an overnight fast. The primary efficacy time point was the post-baseline visit closest to 14 days after the last dose of study treatment.
Time Frame Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET; Week 28 or the post-baseline visit closest to 14 days after the last dose).
Hide Outcome Measure Data
Hide Analysis Population Description
Full analysis set
Arm/Group Title Placebo Mipomersen
Hide Arm/Group Description:
Participants received placebo subcutaneous injection once a week for 26 weeks.
Participants received mipomersen 200 mg as a subcutaneous injection once a week for 26 weeks.
Overall Number of Participants Analyzed 50 101
Median (Inter-Quartile Range)
Unit of Measure: percentage of baseline
-1.2
(-13.6 to 11.5)
-38.7
(-54.0 to -24.2)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo, Mipomersen
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.001
Comments Inflation of type 1 error was controlled by specifying a small number of secondary parameters and a sequential inferential approach in which inferential conclusions about each successive parameter required statistical significance of the prior one.
Method Wilcoxon (Mann-Whitney)
Comments [Not Specified]
8.Secondary Outcome
Title Non-High-Density Lipoprotein Cholesterol at Baseline and at the Primary Efficacy Time Point
Hide Description The primary efficacy time point (PET) was the post-baseline visit closest to 14 days after the last dose of study treatment.
Time Frame Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET; Week 28 or the post-baseline visit closest to 14 days after the last dose).
Hide Outcome Measure Data
Hide Analysis Population Description
Full analysis set
Arm/Group Title Placebo Mipomersen
Hide Arm/Group Description:
Participants received placebo subcutaneous injection once a week for 26 weeks.
Participants received mipomersen 200 mg as a subcutaneous injection once a week for 26 weeks.
Overall Number of Participants Analyzed 50 101
Median (Inter-Quartile Range)
Unit of Measure: mg/dL
Baseline
144
(125 to 175)
144
(132 to 171)
Primary efficacy time point
140
(115 to 165)
90
(67 to 116)
9.Other Pre-specified Outcome
Title Percent Change From Baseline in Triglycerides at the Primary Efficacy Time Point
Hide Description Triglycerides were measured in mg/dL. Samples were taken following an overnight fast. The primary efficacy time point was the post-baseline visit closest to 14 days after the last dose of study treatment.
Time Frame Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET; Week 28 or the post-baseline visit closest to 14 days after the last dose).
Hide Outcome Measure Data
Hide Analysis Population Description
Full analysis set
Arm/Group Title Placebo Mipomersen
Hide Arm/Group Description:
Participants received placebo subcutaneous injection once a week for 26 weeks.
Participants received mipomersen 200 mg as a subcutaneous injection once a week for 26 weeks.
Overall Number of Participants Analyzed 50 101
Median (Inter-Quartile Range)
Unit of Measure: percentage of baseline
2.7
(-24.0 to 24.2)
-26.2
(-48.1 to -8.8)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo, Mipomersen
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.001
Comments No adjustments were made for tertiary parameters.
Method Wilcoxon (Mann-Whitney)
Comments [Not Specified]
10.Other Pre-specified Outcome
Title Triglycerides at Baseline and at the Primary Efficacy Time Point
Hide Description The primary efficacy time point (PET) was the post-baseline visit closest to 14 days after the last dose of study treatment.
Time Frame Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET; Week 28 or the post-baseline visit closest to 14 days after the last dose).
Hide Outcome Measure Data
Hide Analysis Population Description
Full analysis set
Arm/Group Title Placebo Mipomersen
Hide Arm/Group Description:
Participants received placebo subcutaneous injection once a week for 26 weeks.
Participants received mipomersen 200 mg as a subcutaneous injection once a week for 26 weeks.
Overall Number of Participants Analyzed 50 101
Median (Inter-Quartile Range)
Unit of Measure: mg/dL
Baseline
139
(98 to 176)
143
(105 to 175)
Primary efficacy time point
135
(96 to 177)
88
(67 to 128)
11.Other Pre-specified Outcome
Title Percent Change From Baseline in High Density Lipoprotein Cholesterol (HDL-C) at the Primary Efficacy Time Point
Hide Description High-density lipoprotein cholesterol (HDL-C) was measured in mg/dL. Samples were taken following an overnight fast. The primary efficacy time point was the post-baseline visit closest to 14 days after the last dose of study treatment.
Time Frame Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET; Week 28 or the post-baseline visit closest to 14 days after the last dose).
Hide Outcome Measure Data
Hide Analysis Population Description
Full analysis set
Arm/Group Title Placebo Mipomersen
Hide Arm/Group Description:
Participants received placebo subcutaneous injection once a week for 26 weeks.
Participants received mipomersen 200 mg as a subcutaneous injection once a week for 26 weeks.
Overall Number of Participants Analyzed 50 101
Mean (Standard Deviation)
Unit of Measure: percentage of baseline
2.2  (16.44) 2.2  (17.99)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo, Mipomersen
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.977
Comments No adjustments were made for tertiary parameters.
Method t-test, 2 sided
Comments [Not Specified]
12.Other Pre-specified Outcome
Title High-Density Lipoprotein Cholesterol at Baseline and at the Primary Efficacy Time Point
Hide Description The primary efficacy time point (PET) was the post-baseline visit closest to 14 days after the last dose of study treatment.
Time Frame Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET; Week 28 or the post-baseline visit closest to 14 days after the last dose).
Hide Outcome Measure Data
Hide Analysis Population Description
Full analysis set
Arm/Group Title Placebo Mipomersen
Hide Arm/Group Description:
Participants received placebo subcutaneous injection once a week for 26 weeks.
Participants received mipomersen 200 mg as a subcutaneous injection once a week for 26 weeks.
Overall Number of Participants Analyzed 50 101
Mean (Standard Deviation)
Unit of Measure: mg/dL
Baseline 48.4  (15.9) 50.8  (12.0)
Primary efficacy time point 48.9  (16.1) 51.1  (12.3)
13.Other Pre-specified Outcome
Title Percent Change From Baseline in Lipoprotein (a) at the Primary Efficacy Time Point
Hide Description Lipoprotein (a) was measured in mg/dL. Samples were taken following an overnight fast. The primary efficacy time point was the post-baseline visit closest to 14 days after the last dose of study treatment.
Time Frame Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET; Week 28 or the post-baseline visit closest to 14 days after the last dose).
Hide Outcome Measure Data
Hide Analysis Population Description
Full analysis set
Arm/Group Title Placebo Mipomersen
Hide Arm/Group Description:
Participants received placebo subcutaneous injection once a week for 26 weeks.
Participants received mipomersen 200 mg as a subcutaneous injection once a week for 26 weeks.
Overall Number of Participants Analyzed 50 101
Mean (Standard Deviation)
Unit of Measure: percentage of baseline
2.3  (28.09) -24.0  (24.47)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo, Mipomersen
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.001
Comments No adjustments were made for tertiary parameters.
Method t-test, 2 sided
Comments [Not Specified]
14.Other Pre-specified Outcome
Title Lipoprotein (a) at Baseline and at the Primary Efficacy Time Point
Hide Description The primary efficacy time point (PET) was the post-baseline visit closest to 14 days after the last dose of study treatment.
Time Frame Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET; Week 28 or the post-baseline visit closest to 14 days after the last dose).
Hide Outcome Measure Data
Hide Analysis Population Description
Full analysis set
Arm/Group Title Placebo Mipomersen
Hide Arm/Group Description:
Participants received placebo subcutaneous injection once a week for 26 weeks.
Participants received mipomersen 200 mg as a subcutaneous injection once a week for 26 weeks.
Overall Number of Participants Analyzed 50 101
Mean (Standard Deviation)
Unit of Measure: mg/dL
Baseline 51.1  (48.6) 54.3  (57.0)
Primary efficacy time point 49.5  (47.3) 39.6  (47.0)
15.Other Pre-specified Outcome
Title Percent Change From Baseline in Very Low Density Lipoprotein Cholesterol at the Primary Efficacy Time Point
Hide Description Very low density lipoprotein (VLDL) cholesterol was measured in mg/dL. Samples were taken following an overnight fast. The primary efficacy time point was the post-baseline visit closest to 14 days after the last dose of study treatment.
Time Frame Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET; Week 28 or the post-baseline visit closest to 14 days after the last dose).
Hide Outcome Measure Data
Hide Analysis Population Description
Full analysis set
Arm/Group Title Placebo Mipomersen
Hide Arm/Group Description:
Participants received placebo subcutaneous injection once a week for 26 weeks.
Participants received mipomersen 200 mg as a subcutaneous injection once a week for 26 weeks.
Overall Number of Participants Analyzed 50 101
Median (Inter-Quartile Range)
Unit of Measure: percentage of baseline
1.9
(-23.1 to 26.3)
-26.7
(-46.8 to -9.1)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo, Mipomersen
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.001
Comments No adjustments were made for tertiary parameters.
Method Wilcoxon (Mann-Whitney)
Comments [Not Specified]
16.Other Pre-specified Outcome
Title Very Low Density Lipoprotein Cholesterol at Baseline and at the Primary Efficacy Time Point
Hide Description The primary efficacy time point (PET) was the post-baseline visit closest to 14 days after the last dose of study treatment.
Time Frame Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET; Week 28 or the post-baseline visit closest to 14 days after the last dose).
Hide Outcome Measure Data
Hide Analysis Population Description
Full analysis set
Arm/Group Title Placebo Mipomersen
Hide Arm/Group Description:
Participants received placebo subcutaneous injection once a week for 26 weeks.
Participants received mipomersen 200 mg as a subcutaneous injection once a week for 26 weeks.
Overall Number of Participants Analyzed 50 101
Median (Inter-Quartile Range)
Unit of Measure: mg/dL
Baseline
28
(20 to 35)
29
(21 to 35)
Primary efficacy time point
27
(19 to 35)
18
(13 to 26)
17.Other Pre-specified Outcome
Title Percent Change From Baseline in the Ratio of LDL Cholesterol to HDL Cholesterol at the Primary Efficacy Time Point
Hide Description The ratio of low-density lipoprotein (LDL) cholesterol to high-density lipoprotein (HDL) cholesterol was measured at Baseline and the post-baseline visit closest to 14 days after the last dose of study treatment.
Time Frame Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET; Week 28 or the post-baseline visit closest to 14 days after the last dose).
Hide Outcome Measure Data
Hide Analysis Population Description
Full analysis set
Arm/Group Title Placebo Mipomersen
Hide Arm/Group Description:
Participants received placebo subcutaneous injection once a week for 26 weeks.
Participants received mipomersen 200 mg as a subcutaneous injection once a week for 26 weeks.
Overall Number of Participants Analyzed 50 101
Mean (Standard Deviation)
Unit of Measure: percentage of baseline
-5.3  (25.31) -37.4  (27.24)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo, Mipomersen
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.001
Comments No adjustments were made for tertiary parameters.
Method t-test, 2 sided
Comments [Not Specified]
18.Other Pre-specified Outcome
Title Ratio of LDL Cholesterol to HDL Cholesterol at Baseline and at the Primary Efficacy Time Point
Hide Description The primary efficacy time point (PET) was the post-baseline visit closest to 14 days after the last dose of study treatment.
Time Frame Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET; Week 28 or the post-baseline visit closest to 14 days after the last dose).
Hide Outcome Measure Data
Hide Analysis Population Description
Full analysis set
Arm/Group Title Placebo Mipomersen
Hide Arm/Group Description:
Participants received placebo subcutaneous injection once a week for 26 weeks.
Participants received mipomersen 200 mg as a subcutaneous injection once a week for 26 weeks.
Overall Number of Participants Analyzed 50 101
Mean (Standard Deviation)
Unit of Measure: ratio
Baseline 2.82  (1.383) 2.54  (0.854)
Primary efficacy time point 2.54  (1.147) 1.54  (0.761)
19.Other Pre-specified Outcome
Title Percent Change From Baseline in Apolipoprotein A1 at the Primary Efficacy Time Point
Hide Description Apolipoprotein A1 was measured in mg/dL. Samples were taken following an overnight fast. The primary efficacy time point was the post-baseline visit closest to 14 days after the last dose of study treatment.
Time Frame Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET; Week 28 or the post-baseline visit closest to 14 days after the last dose).
Hide Outcome Measure Data
Hide Analysis Population Description
Full analysis set
Arm/Group Title Placebo Mipomersen
Hide Arm/Group Description:
Participants received placebo subcutaneous injection once a week for 26 weeks.
Participants received mipomersen 200 mg as a subcutaneous injection once a week for 26 weeks.
Overall Number of Participants Analyzed 50 101
Mean (Standard Deviation)
Unit of Measure: percentage of baseline
-1.0  (11.16) -5.6  (12.56)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo, Mipomersen
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.032
Comments No adjustments were made for tertiary parameters.
Method t-test, 2 sided
Comments [Not Specified]
20.Other Pre-specified Outcome
Title Apolipoprotein A1 at Baseline and at the Primary Efficacy Time Point
Hide Description The primary efficacy time point (PET) was the post-baseline visit closest to 14 days after the last dose of study treatment.
Time Frame Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET; Week 28 or the post-baseline visit closest to 14 days after the last dose).
Hide Outcome Measure Data
Hide Analysis Population Description
Full analysis set
Arm/Group Title Placebo Mipomersen
Hide Arm/Group Description:
Participants received placebo subcutaneous injection once a week for 26 weeks.
Participants received mipomersen 200 mg as a subcutaneous injection once a week for 26 weeks.
Overall Number of Participants Analyzed 50 101
Mean (Standard Deviation)
Unit of Measure: mg/dL
Baseline 150.8  (30.5) 156.8  (25.4)
Primary efficacy time point 147.8  (27.3) 146.8  (24.5)
Time Frame Up to Week 28 (2 weeks after last dose)
Adverse Event Reporting Description In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
 
Arm/Group Title Placebo Mipomersen
Hide Arm/Group Description Participants received placebo subcutaneous injection once a week for 26 weeks. Participants received mipomersen 200 mg as a subcutaneous injection once a week for 26 weeks.
All-Cause Mortality
Placebo Mipomersen
Affected / at Risk (%) Affected / at Risk (%)
Total   --/--   --/-- 
Show Serious Adverse Events Hide Serious Adverse Events
Placebo Mipomersen
Affected / at Risk (%) Affected / at Risk (%)
Total   4/52 (7.69%)   7/105 (6.67%) 
Cardiac disorders     
Acute coronary syndrome  1  1/52 (1.92%)  0/105 (0.00%) 
Acute myocardial infarction  1  1/52 (1.92%)  0/105 (0.00%) 
Angina pectoris  1  0/52 (0.00%)  1/105 (0.95%) 
Angina unstable  1  0/52 (0.00%)  1/105 (0.95%) 
Cardiogenic shock  1  1/52 (1.92%)  0/105 (0.00%) 
Coronary artery disease  1  0/52 (0.00%)  1/105 (0.95%) 
General disorders     
Non-cardiac chest pain  1  1/52 (1.92%)  1/105 (0.95%) 
Investigations     
Electrocardiogram abnormal  1  1/52 (1.92%)  0/105 (0.00%) 
Neoplasms benign, malignant and unspecified (incl cysts and polyps)     
Non-small cell lung cancer  1  0/52 (0.00%)  1/105 (0.95%) 
Nervous system disorders     
Hypoaesthesia  1  0/52 (0.00%)  1/105 (0.95%) 
Respiratory, thoracic and mediastinal disorders     
Dyspnoea exertional  1  1/52 (1.92%)  0/105 (0.00%) 
Vascular disorders     
Hypertension  1  0/52 (0.00%)  1/105 (0.95%) 
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA 13.0
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Placebo Mipomersen
Affected / at Risk (%) Affected / at Risk (%)
Total   42/52 (80.77%)   97/105 (92.38%) 
Blood and lymphatic system disorders     
Anaemia  1  1/52 (1.92%)  5/105 (4.76%) 
Iron deficiency anaemia  1  1/52 (1.92%)  0/105 (0.00%) 
Lymphadenopathy  1  1/52 (1.92%)  4/105 (3.81%) 
Thrombocytopenia  1  1/52 (1.92%)  0/105 (0.00%) 
Cardiac disorders     
Angina pectoris  1  1/52 (1.92%)  2/105 (1.90%) 
Atrial flutter  1  1/52 (1.92%)  0/105 (0.00%) 
Atrioventricular block  1  1/52 (1.92%)  0/105 (0.00%) 
Bradycardia  1  0/52 (0.00%)  1/105 (0.95%) 
Cardiac discomfort  1  1/52 (1.92%)  0/105 (0.00%) 
Left ventricular hypertrophy  1  0/52 (0.00%)  1/105 (0.95%) 
Palpitations  1  0/52 (0.00%)  4/105 (3.81%) 
Tachycardia  1  1/52 (1.92%)  1/105 (0.95%) 
Ear and labyrinth disorders     
Cerumen impaction  1  1/52 (1.92%)  0/105 (0.00%) 
Vertigo positional  1  0/52 (0.00%)  1/105 (0.95%) 
Endocrine disorders     
Adrenal mass  1  0/52 (0.00%)  1/105 (0.95%) 
Goitre  1  0/52 (0.00%)  1/105 (0.95%) 
Hypothyroidism  1  1/52 (1.92%)  0/105 (0.00%) 
Eye disorders     
Blepharitis  1  0/52 (0.00%)  1/105 (0.95%) 
Cataract nuclear  1  0/52 (0.00%)  1/105 (0.95%) 
Ectropion  1  0/52 (0.00%)  1/105 (0.95%) 
Eyelid oedema  1  0/52 (0.00%)  1/105 (0.95%) 
Keratopathy  1  0/52 (0.00%)  1/105 (0.95%) 
Visual impairment  1  2/52 (3.85%)  0/105 (0.00%) 
Gastrointestinal disorders     
Abdominal discomfort  1  2/52 (3.85%)  2/105 (1.90%) 
Abdominal distension  1  1/52 (1.92%)  2/105 (1.90%) 
Abdominal hernia  1  0/52 (0.00%)  1/105 (0.95%) 
Abdominal pain  1  0/52 (0.00%)  2/105 (1.90%) 
Abdominal pain lower  1  2/52 (3.85%)  1/105 (0.95%) 
Abdominal pain upper  1  2/52 (3.85%)  0/105 (0.00%) 
Abdominal tenderness  1  0/52 (0.00%)  2/105 (1.90%) 
Aphthous stomatitis  1  1/52 (1.92%)  0/105 (0.00%) 
Barrett's oesophagus  1  0/52 (0.00%)  1/105 (0.95%) 
Colitis  1  1/52 (1.92%)  1/105 (0.95%) 
Colonic polyp  1  1/52 (1.92%)  0/105 (0.00%) 
Constipation  1  2/52 (3.85%)  2/105 (1.90%) 
Diarrhoea  1  3/52 (5.77%)  7/105 (6.67%) 
Diverticulum  1  2/52 (3.85%)  0/105 (0.00%) 
Diverticulum intestinal  1  1/52 (1.92%)  0/105 (0.00%) 
Dry mouth  1  0/52 (0.00%)  1/105 (0.95%) 
Duodenitis  1  0/52 (0.00%)  1/105 (0.95%) 
Dyspepsia  1  3/52 (5.77%)  4/105 (3.81%) 
Dysphagia  1  1/52 (1.92%)  0/105 (0.00%) 
Flatulence  1  2/52 (3.85%)  1/105 (0.95%) 
Gastric ulcer  1  1/52 (1.92%)  0/105 (0.00%) 
Gastritis  1  1/52 (1.92%)  1/105 (0.95%) 
Gastritis erosive  1  0/52 (0.00%)  1/105 (0.95%) 
Gastrooesophageal reflux disease  1  2/52 (3.85%)  3/105 (2.86%) 
Haemorrhoids  1  2/52 (3.85%)  0/105 (0.00%) 
Hiatus hernia  1  1/52 (1.92%)  1/105 (0.95%) 
Nausea  1  3/52 (5.77%)  11/105 (10.48%) 
Oesophageal ulcer  1  1/52 (1.92%)  0/105 (0.00%) 
Oesophagitis  1  1/52 (1.92%)  2/105 (1.90%) 
Pancreatic atrophy  1  1/52 (1.92%)  0/105 (0.00%) 
Pancreatic cyst  1  1/52 (1.92%)  1/105 (0.95%) 
Pancreatic duct dilatation  1  2/52 (3.85%)  0/105 (0.00%) 
Regurgitation  1  0/52 (0.00%)  1/105 (0.95%) 
Toothache  1  1/52 (1.92%)  0/105 (0.00%) 
Vomiting  1  1/52 (1.92%)  4/105 (3.81%) 
General disorders     
Asthenia  1  0/52 (0.00%)  2/105 (1.90%) 
Chest discomfort  1  1/52 (1.92%)  0/105 (0.00%) 
Chills  1  0/52 (0.00%)  8/105 (7.62%) 
Fatigue  1  4/52 (7.69%)  13/105 (12.38%) 
Feeling jittery  1  1/52 (1.92%)  0/105 (0.00%) 
Influenza like illness  1  1/52 (1.92%)  7/105 (6.67%) 
Injection site discolouration  1  2/52 (3.85%)  19/105 (18.10%) 
Injection site discomfort  1  1/52 (1.92%)  2/105 (1.90%) 
Injection site dryness  1  0/52 (0.00%)  1/105 (0.95%) 
Injection site erythema  1  2/52 (3.85%)  56/105 (53.33%) 
Injection site exfoliation  1  0/52 (0.00%)  1/105 (0.95%) 
Injection site haematoma  1  3/52 (5.77%)  17/105 (16.19%) 
Injection site haemorrhage  1  1/52 (1.92%)  6/105 (5.71%) 
Injection site induration  1  0/52 (0.00%)  5/105 (4.76%) 
Injection site inflammation  1  0/52 (0.00%)  2/105 (1.90%) 
Injection site nodule  1  4/52 (7.69%)  13/105 (12.38%) 
Injection site oedema  1  0/52 (0.00%)  6/105 (5.71%) 
Injection site pain  1  11/52 (21.15%)  57/105 (54.29%) 
Injection site pallor  1  0/52 (0.00%)  1/105 (0.95%) 
Injection site papule  1  0/52 (0.00%)  1/105 (0.95%) 
Injection site pruritus  1  0/52 (0.00%)  31/105 (29.52%) 
Injection site rash  1  0/52 (0.00%)  6/105 (5.71%) 
Injection site reaction  1  0/52 (0.00%)  5/105 (4.76%) 
Injection site recall reaction  1  0/52 (0.00%)  2/105 (1.90%) 
Injection site swelling  1  0/52 (0.00%)  17/105 (16.19%) 
Injection site urticaria  1  0/52 (0.00%)  3/105 (2.86%) 
Injection site vesicles  1  0/52 (0.00%)  5/105 (4.76%) 
Injection site warmth  1  0/52 (0.00%)  9/105 (8.57%) 
Malaise  1  0/52 (0.00%)  1/105 (0.95%) 
Non-cardiac chest pain  1  0/52 (0.00%)  1/105 (0.95%) 
Oedema  1  1/52 (1.92%)  0/105 (0.00%) 
Oedema peripheral  1  1/52 (1.92%)  3/105 (2.86%) 
Pain  1  1/52 (1.92%)  6/105 (5.71%) 
Pyrexia  1  3/52 (5.77%)  5/105 (4.76%) 
Vessel puncture site haemorrhage  1  1/52 (1.92%)  0/105 (0.00%) 
Hepatobiliary disorders     
Biliary dilatation  1  0/52 (0.00%)  1/105 (0.95%) 
Cholelithiasis  1  0/52 (0.00%)  1/105 (0.95%) 
Hepatic cyst  1  3/52 (5.77%)  0/105 (0.00%) 
Hepatic lesion  1  1/52 (1.92%)  0/105 (0.00%) 
Hepatic steatosis  1  2/52 (3.85%)  11/105 (10.48%) 
Hepatitis  1  1/52 (1.92%)  0/105 (0.00%) 
Hepatomegaly  1  0/52 (0.00%)  1/105 (0.95%) 
Immune system disorders     
Drug hypersensitivity  1  0/52 (0.00%)  1/105 (0.95%) 
Hypersensitivity  1  0/52 (0.00%)  1/105 (0.95%) 
Seasonal allergy  1  0/52 (0.00%)  4/105 (3.81%) 
Infections and infestations     
Acarodermatitis  1  1/52 (1.92%)  0/105 (0.00%) 
Bronchitis  1  2/52 (3.85%)  2/105 (1.90%) 
Cellulitis  1  0/52 (0.00%)  1/105 (0.95%) 
Conjunctivitis viral  1  1/52 (1.92%)  0/105 (0.00%) 
Fungal infection  1  0/52 (0.00%)  1/105 (0.95%) 
Influenza  1  2/52 (3.85%)  6/105 (5.71%) 
Labyrinthitis  1  0/52 (0.00%)  1/105 (0.95%) 
Laryngitis  1  1/52 (1.92%)  0/105 (0.00%) 
Nasopharyngitis  1  2/52 (3.85%)  6/105 (5.71%) 
Oral herpes  1  0/52 (0.00%)  1/105 (0.95%) 
Otitis externa  1  1/52 (1.92%)  0/105 (0.00%) 
Otitis media  1  1/52 (1.92%)  2/105 (1.90%) 
Pneumonia  1  0/52 (0.00%)  1/105 (0.95%) 
Rhinitis  1  1/52 (1.92%)  0/105 (0.00%) 
Sinusitis  1  3/52 (5.77%)  4/105 (3.81%) 
Tinea pedis  1  0/52 (0.00%)  1/105 (0.95%) 
Tooth abscess  1  1/52 (1.92%)  0/105 (0.00%) 
Tooth infection  1  0/52 (0.00%)  1/105 (0.95%) 
Upper respiratory tract infection  1  6/52 (11.54%)  10/105 (9.52%) 
Urinary tract infection  1  5/52 (9.62%)  12/105 (11.43%) 
Viral infection  1  1/52 (1.92%)  1/105 (0.95%) 
Viral upper respiratory tract infection  1  0/52 (0.00%)  1/105 (0.95%) 
Injury, poisoning and procedural complications     
Arthropod sting  1  1/52 (1.92%)  0/105 (0.00%) 
Concussion  1  0/52 (0.00%)  1/105 (0.95%) 
Contusion  1  1/52 (1.92%)  1/105 (0.95%) 
Excoriation  1  0/52 (0.00%)  1/105 (0.95%) 
Fall  1  0/52 (0.00%)  2/105 (1.90%) 
Fibula fracture  1  0/52 (0.00%)  1/105 (0.95%) 
Foot fracture  1  1/52 (1.92%)  0/105 (0.00%) 
Joint sprain  1  0/52 (0.00%)  1/105 (0.95%) 
Meniscus lesion  1  0/52 (0.00%)  1/105 (0.95%) 
Muscle strain  1  2/52 (3.85%)  1/105 (0.95%) 
Periorbital haematoma  1  0/52 (0.00%)  1/105 (0.95%) 
Post-traumatic pain  1  1/52 (1.92%)  0/105 (0.00%) 
Procedural pain  1  0/52 (0.00%)  1/105 (0.95%) 
Rib fracture  1  0/52 (0.00%)  1/105 (0.95%) 
Scapula fracture  1  0/52 (0.00%)  1/105 (0.95%) 
Scratch  1  0/52 (0.00%)  1/105 (0.95%) 
Skin laceration  1  2/52 (3.85%)  0/105 (0.00%) 
Thermal burn  1  0/52 (0.00%)  1/105 (0.95%) 
Wound  1  1/52 (1.92%)  1/105 (0.95%) 
Investigations     
Alanine aminotransferase increased  1  1/52 (1.92%)  9/105 (8.57%) 
Aspartate aminotransferase increased  1  1/52 (1.92%)  5/105 (4.76%) 
Beta 2 microglobulin urine increased  1  0/52 (0.00%)  1/105 (0.95%) 
Blood alkaline phosphatase increased  1  1/52 (1.92%)  0/105 (0.00%) 
Blood creatine phosphokinase increased  1  0/52 (0.00%)  2/105 (1.90%) 
Blood creatinine increased  1  2/52 (3.85%)  0/105 (0.00%) 
Blood glucose increased  1  0/52 (0.00%)  1/105 (0.95%) 
Blood lactate dehydrogenase increased  1  0/52 (0.00%)  1/105 (0.95%) 
Blood testosterone decreased  1  0/52 (0.00%)  1/105 (0.95%) 
Blood urea increased  1  2/52 (3.85%)  0/105 (0.00%) 
Body temperature increased  1  0/52 (0.00%)  1/105 (0.95%) 
C-reactive protein increased  1  0/52 (0.00%)  1/105 (0.95%) 
Eosinophil percentage increased  1  0/52 (0.00%)  1/105 (0.95%) 
Haematocrit decreased  1  0/52 (0.00%)  1/105 (0.95%) 
Haemoglobin decreased  1  0/52 (0.00%)  1/105 (0.95%) 
Heart rate irregular  1  0/52 (0.00%)  1/105 (0.95%) 
Helicobacter test positive  1  0/52 (0.00%)  1/105 (0.95%) 
Hepatic enzyme increased  1  0/52 (0.00%)  4/105 (3.81%) 
International normalised ratio increased  1  1/52 (1.92%)  2/105 (1.90%) 
Liver function test abnormal  1  0/52 (0.00%)  8/105 (7.62%) 
Platelet count decreased  1  0/52 (0.00%)  1/105 (0.95%) 
Protein urine present  1  0/52 (0.00%)  1/105 (0.95%) 
Prothrombin time prolonged  1  1/52 (1.92%)  1/105 (0.95%) 
QRS axis abnormal  1  0/52 (0.00%)  1/105 (0.95%) 
Red blood cell count decreased  1  0/52 (0.00%)  1/105 (0.95%) 
Red blood cells urine positive  1  0/52 (0.00%)  1/105 (0.95%) 
White blood cell count decreased  1  0/52 (0.00%)  1/105 (0.95%) 
Metabolism and nutrition disorders     
Decreased appetite  1  1/52 (1.92%)  2/105 (1.90%) 
Gout  1  0/52 (0.00%)  1/105 (0.95%) 
Hyperkalaemia  1  1/52 (1.92%)  0/105 (0.00%) 
Hypernatraemia  1  0/52 (0.00%)  1/105 (0.95%) 
Hyperuricaemia  1  0/52 (0.00%)  1/105 (0.95%) 
Hypokalaemia  1  1/52 (1.92%)  1/105 (0.95%) 
Vitamin B12 deficiency  1  1/52 (1.92%)  0/105 (0.00%) 
Vitamin D deficiency  1  0/52 (0.00%)  1/105 (0.95%) 
Musculoskeletal and connective tissue disorders     
Arthralgia  1  2/52 (3.85%)  2/105 (1.90%) 
Arthritis  1  1/52 (1.92%)  0/105 (0.00%) 
Back pain  1  2/52 (3.85%)  2/105 (1.90%) 
Bursitis  1  0/52 (0.00%)  1/105 (0.95%) 
Fibromyalgia  1  0/52 (0.00%)  1/105 (0.95%) 
Intervertebral disc degeneration  1  1/52 (1.92%)  0/105 (0.00%) 
Joint stiffness  1  2/52 (3.85%)  0/105 (0.00%) 
Muscle spasms  1  1/52 (1.92%)  3/105 (2.86%) 
Muscular weakness  1  2/52 (3.85%)  1/105 (0.95%) 
Musculoskeletal chest pain  1  0/52 (0.00%)  1/105 (0.95%) 
Musculoskeletal pain  1  1/52 (1.92%)  5/105 (4.76%) 
Musculoskeletal stiffness  1  0/52 (0.00%)  1/105 (0.95%) 
Myalgia  1  4/52 (7.69%)  7/105 (6.67%) 
Neck pain  1  1/52 (1.92%)  1/105 (0.95%) 
Osteoarthritis  1  0/52 (0.00%)  1/105 (0.95%) 
Osteopenia  1  0/52 (0.00%)  1/105 (0.95%) 
Pain in extremity  1  0/52 (0.00%)  9/105 (8.57%) 
Pain in jaw  1  0/52 (0.00%)  1/105 (0.95%) 
Rotator cuff syndrome  1  1/52 (1.92%)  0/105 (0.00%) 
Synovial cyst  1  0/52 (0.00%)  2/105 (1.90%) 
Tendonitis  1  1/52 (1.92%)  0/105 (0.00%) 
Neoplasms benign, malignant and unspecified (incl cysts and polyps)     
Angiomyolipoma  1  0/52 (0.00%)  1/105 (0.95%) 
Lung neoplasm  1  0/52 (0.00%)  1/105 (0.95%) 
Melanocytic naevus  1  0/52 (0.00%)  1/105 (0.95%) 
Seborrhoeic keratosis  1  0/52 (0.00%)  1/105 (0.95%) 
Nervous system disorders     
Carotid artery stenosis  1  0/52 (0.00%)  1/105 (0.95%) 
Cervicobrachial syndrome  1  1/52 (1.92%)  1/105 (0.95%) 
Dizziness  1  1/52 (1.92%)  5/105 (4.76%) 
Headache  1  3/52 (5.77%)  10/105 (9.52%) 
Hyporeflexia  1  0/52 (0.00%)  1/105 (0.95%) 
Lethargy  1  0/52 (0.00%)  2/105 (1.90%) 
Mental impairment  1  0/52 (0.00%)  1/105 (0.95%) 
Neuropathy peripheral  1  0/52 (0.00%)  1/105 (0.95%) 
Paraesthesia  1  1/52 (1.92%)  0/105 (0.00%) 
Presyncope  1  0/52 (0.00%)  1/105 (0.95%) 
Sinus headache  1  1/52 (1.92%)  1/105 (0.95%) 
Somnolence  1  0/52 (0.00%)  1/105 (0.95%) 
Syncope  1  0/52 (0.00%)  2/105 (1.90%) 
Tremor  1  1/52 (1.92%)  0/105 (0.00%) 
Trigeminal neuralgia  1  0/52 (0.00%)  1/105 (0.95%) 
Psychiatric disorders     
Anxiety  1  0/52 (0.00%)  3/105 (2.86%) 
Claustrophobia  1  0/52 (0.00%)  1/105 (0.95%) 
Depression  1  0/52 (0.00%)  3/105 (2.86%) 
Disorientation  1  0/52 (0.00%)  1/105 (0.95%) 
Dysphoria  1  0/52 (0.00%)  1/105 (0.95%) 
Insomnia  1  0/52 (0.00%)  5/105 (4.76%) 
Libido decreased  1  1/52 (1.92%)  0/105 (0.00%) 
Listless  1  0/52 (0.00%)  1/105 (0.95%) 
Renal and urinary disorders     
Azotaemia  1  0/52 (0.00%)  1/105 (0.95%) 
Dysuria  1  1/52 (1.92%)  0/105 (0.00%) 
Nocturia  1  1/52 (1.92%)  0/105 (0.00%) 
Pollakiuria  1  1/52 (1.92%)  0/105 (0.00%) 
Renal cyst  1  1/52 (1.92%)  3/105 (2.86%) 
Reproductive system and breast disorders     
Endometrial atrophy  1  0/52 (0.00%)  1/105 (0.95%) 
Genital lesion  1  0/52 (0.00%)  1/105 (0.95%) 
Premenstrual syndrome  1  0/52 (0.00%)  1/105 (0.95%) 
Vaginal discharge  1  0/52 (0.00%)  1/105 (0.95%) 
Respiratory, thoracic and mediastinal disorders     
Bronchitis chronic  1  0/52 (0.00%)  1/105 (0.95%) 
Cough  1  2/52 (3.85%)  3/105 (2.86%) 
Dysphonia  1  0/52 (0.00%)  1/105 (0.95%) 
Dyspnoea  1  0/52 (0.00%)  1/105 (0.95%) 
Increased upper airway secretion  1  1/52 (1.92%)  1/105 (0.95%) 
Nasal congestion  1  2/52 (3.85%)  2/105 (1.90%) 
Nasal turbinate abnormality  1  0/52 (0.00%)  1/105 (0.95%) 
Oropharyngeal pain  1  1/52 (1.92%)  3/105 (2.86%) 
Pharyngeal erythema  1  1/52 (1.92%)  1/105 (0.95%) 
Respiratory tract congestion  1  1/52 (1.92%)  0/105 (0.00%) 
Rhinitis allergic  1  2/52 (3.85%)  0/105 (0.00%) 
Rhinorrhoea  1  1/52 (1.92%)  0/105 (0.00%) 
Rhonchi  1  1/52 (1.92%)  0/105 (0.00%) 
Sinus congestion  1  4/52 (7.69%)  6/105 (5.71%) 
Upper respiratory tract congestion  1  1/52 (1.92%)  2/105 (1.90%) 
Wheezing  1  0/52 (0.00%)  2/105 (1.90%) 
Skin and subcutaneous tissue disorders     
Actinic keratosis  1  0/52 (0.00%)  1/105 (0.95%) 
Alopecia  1  0/52 (0.00%)  2/105 (1.90%) 
Dermal cyst  1  0/52 (0.00%)  2/105 (1.90%) 
Ecchymosis  1  0/52 (0.00%)  1/105 (0.95%) 
Erythema  1  0/52 (0.00%)  1/105 (0.95%) 
Hyperhidrosis  1  1/52 (1.92%)  0/105 (0.00%) 
Photosensitivity reaction  1  1/52 (1.92%)  0/105 (0.00%) 
Pruritus  1  1/52 (1.92%)  1/105 (0.95%) 
Rash  1  1/52 (1.92%)  1/105 (0.95%) 
Rash erythematous  1  1/52 (1.92%)  0/105 (0.00%) 
Rash maculo-papular  1  0/52 (0.00%)  1/105 (0.95%) 
Rash papular  1  0/52 (0.00%)  2/105 (1.90%) 
Skin lesion  1  0/52 (0.00%)  1/105 (0.95%) 
Urticaria  1  0/52 (0.00%)  1/105 (0.95%) 
Vascular disorders     
Aortic aneurysm  1  0/52 (0.00%)  1/105 (0.95%) 
Aortic stenosis  1  1/52 (1.92%)  0/105 (0.00%) 
Hot flush  1  1/52 (1.92%)  1/105 (0.95%) 
Hypertension  1  3/52 (5.77%)  11/105 (10.48%) 
Hypotension  1  0/52 (0.00%)  2/105 (1.90%) 
Intermittent claudication  1  1/52 (1.92%)  0/105 (0.00%) 
Peripheral arterial occlusive disease  1  1/52 (1.92%)  0/105 (0.00%) 
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA 13.0
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
After the multicenter publication or 12 months after completion of the study the PI may publish the results of his/her data from the study. The PI shall provide the sponsor with an advance copy at least 60 days prior to planned submission and the Sponsor shall have 60 days to review (contracts have variable timeframes; maximum times are stated here). The sponsor may request the deletion of any confidential information, or a delay in submission for an additional period not to exceed 90 days.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Genzyme Medical Information
Organization: Genzyme Corporation
Phone: 617-252-7832
Layout table for additonal information
Responsible Party: Kastle Therapeutics, LLC
ClinicalTrials.gov Identifier: NCT00770146     History of Changes
Other Study ID Numbers: 301012-CS12
First Submitted: October 8, 2008
First Posted: October 9, 2008
Results First Submitted: February 15, 2013
Results First Posted: March 20, 2013
Last Update Posted: September 9, 2016