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Trial record 12 of 19 for:    MIPOMERSEN

Efficacy and Safety Study of ISIS 301012 (Mipomersen) as Add-on in Familial Hypercholesterolemic Patients With Coronary Artery Disease (RADICHOL II)

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ClinicalTrials.gov Identifier: NCT00706849
Recruitment Status : Completed
First Posted : June 30, 2008
Results First Posted : March 21, 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 Heterozygous Familial Hypercholesterolemia
Coronary Artery Disease
Interventions Drug: mipomersen sodium
Drug: placebo
Enrollment 124
Recruitment Details  
Pre-assignment Details Two hundred and twenty-five patients were screened and 124 participants were randomized on Day 1 in a 2:1 ratio to receive mipomersen or placebo once a week for 26 weeks.
Arm/Group Title Placebo Mipomersen
Hide Arm/Group Description Participants received a 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 41 83
Full Analysis Set 41 [1] 82 [2]
Completed 41 [3] 73 [4]
Not Completed 0 10
Reason Not Completed
Adverse Event             0             9
Other             0             1
[1]
Treated patients with a valid baseline & post-baseline LDL-C measurement
[2]
Treated patients with a valid baseline & post-baseline LDL-C measurement.
[3]
38 patients entered the open-label extension study (301012-CS6; NCT00694109).
[4]
55 patients entered the open-label extension study (301012-CS6; NCT00694109).
Period Title: Follow-up Period
Started 3 [1] 28 [1]
Completed 3 25
Not Completed 0 3
Reason Not Completed
Withdrawal by Subject             0             2
Protocol non-compliance             0             1
[1]
Participants enrolled in the open-label extension (NCT00694109) or continued in the follow-up period
Arm/Group Title Placebo Mipomersen Total
Hide Arm/Group Description Participants received a 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 41 83 124
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 41 participants 83 participants 124 participants
55.9  (9.3) 56.2  (9.7) 56.1  (9.5)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 41 participants 83 participants 124 participants
Female
13
  31.7%
33
  39.8%
46
  37.1%
Male
28
  68.3%
50
  60.2%
78
  62.9%
Race/Ethnicity, Customized  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 41 participants 83 participants 124 participants
Hispanic or Latino 2 2 4
Not Hispanic or Latino 39 81 120
Race/Ethnicity, Customized  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 41 participants 83 participants 124 participants
White 38 81 119
Black 1 2 3
Other race 2 0 2
Body Mass Index (BMI)  
Mean (Standard Deviation)
Unit of measure:  Kg/m^2
Number Analyzed 41 participants 83 participants 124 participants
30.25  (3.79) 28.66  (4.23) 29.18  (4.14)
Waist/hip ratio  
Mean (Standard Deviation)
Unit of measure:  Ratio
Number Analyzed 41 participants 83 participants 124 participants
0.95  (0.07) 0.93  (0.08) 0.93  (0.08)
Metabolic syndrome   [1] 
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 41 participants 83 participants 124 participants
No 30 48 78
Yes 11 35 46
[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 41 participants 83 participants 124 participants
Current 4 13 17
non-current 17 32 49
Never 20 38 58
Alcohol use  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 41 participants 83 participants 124 participants
Current 31 64 95
Non-current 7 10 17
Never 3 9 12
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. For patients with triglycerides <400 mg/dL, LDL-C was obtained using Friedewald’s calculation; and for patients with triglycerides ≥400 mg/dL, LDL-C was directly measured by the central laboratory using ultracentrifugation. 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 a 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 41 82
Mean (Standard Deviation)
Unit of Measure: percentage of Baseline
5.17  (18.02) -28.02  (26.99)
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 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 treatment 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 t-test, 2 sided
Comments [Not Specified]
2.Primary Outcome
Title LDL 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 a 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 41 82
Mean (Standard Deviation)
Unit of Measure: mg/dL
Baseline 142.9  (51.6) 152.9  (48.7)
Primary efficacy time point 146.4  (43.4) 103.9  (33.0)
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 a 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 41 82
Mean (Standard Deviation)
Unit of Measure: percentage of baseline
7.02  (16.52) -26.31  (22.16)
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]
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 a 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 41 82
Mean (Standard Deviation)
Unit of Measure: mg/dL
Baseline 126.8  (33.2) 132.8  (33.9)
Primary efficacy time point 133.8  (32.6) 95.0  (29.7)
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 a 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 41 82
Mean (Standard Deviation)
Unit of Measure: percentage of baseline
3.85  (12.84) -19.43  (19.25)
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 a 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 41 82
Mean (Standard Deviation)
Unit of Measure: mg/dL
Baseline 213.4  (54.6) 225.3  (51.5)
Primary efficacy time point 219.0  (49.0) 176.0  (35.9)
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 a 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 41 82
Mean (Standard Deviation)
Unit of Measure: percentage of baseline
3.74  (16.04) -25.05  (25.71)
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]
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 a 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 41 82
Mean (Standard Deviation)
Unit of Measure: mg/dL
Baseline 165.3  (54.5) 175.5  (51.1)
Primary efficacy time point 168.2  (47.5) 125.2  (37.8)
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 a 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 41 82
Median (Inter-Quartile Range)
Unit of Measure: percentage of baseline
0.5
(-16.2 to 17.9)
-14.3
(-32.7 to 9.7)
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 0.042
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 a 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 41 82
Median (Inter-Quartile Range)
Unit of Measure: mg/dL
Baseline
100
(74 to 137)
107
(85 to 137)
Primary efficacy time point
101
(76 to 139)
89
(70 to 127)
11.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 a 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 41 82
Median (Inter-Quartile Range)
Unit of Measure: percentage of baseline
0.0
(-8.0 to 13.0)
-21.1
(-37.9 to 0.0)
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]
12.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 a 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 41 82
Median (Inter-Quartile Range)
Unit of Measure: mg/dL
Baseline
53
(17 to 108)
45
(13 to 93)
Primary efficacy time point
51
(18 to 108)
35
(9 to 56)
13.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 a 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 41 82
Median (Inter-Quartile Range)
Unit of Measure: percentage of baseline
0.0
(-15.4 to 15.0)
-13.8
(-33.3 to 11.8)
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 0.023
Comments No adjustments were made for tertiary parameters.
Method Wilcoxon (Mann-Whitney)
Comments [Not Specified]
14.Other Pre-specified Outcome
Title Very Low Density Lipoprotein 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 a 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 41 82
Median (Inter-Quartile Range)
Unit of Measure: mg/dL
Baseline
20
(15 to 28)
21
(17 to 27)
Primary efficacy time point
20
(15 to 28)
18
(14 to 25)
15.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 a 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 41 82
Median (Inter-Quartile Range)
Unit of Measure: percentage of baseline
-2.8
(-13.1 to 13.1)
-29.2
(-46.8 to -13.7)
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 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 a 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 41 82
Median (Inter-Quartile Range)
Unit of Measure: ratio
Baseline
2.72
(2.51 to 3.34)
2.99
(2.56 to 4.00)
Primary efficacy time point
2.95
(2.19 to 3.49)
2.09
(1.52 to 3.00)
17.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 a 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 41 82
Mean (Standard Deviation)
Unit of Measure: percentage of baseline
3.71  (8.70) -2.44  (14.22)
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 0.004
Comments No adjustments were made for tertiary parameters.
Method t-test, 2 sided
Comments [Not Specified]
18.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 a 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 41 82
Mean (Standard Deviation)
Unit of Measure: mg/dL
Baseline 146.1  (24.9) 150.7  (28.1)
Primary efficacy time point 151.5  (29.1) 145.4  (27.9)
19.Other Pre-specified Outcome
Title Percent Change From Baseline in High-Density Lipoprotein Cholesterol 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 a 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 41 82
Median (Inter-Quartile Range)
Unit of Measure: percentage of baseline
5.8
(0.0 to 11.5)
2.5
(-10.3 to 11.7)
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 0.207
Comments No adjustments were made for tertiary parameters.
Method Wilcoxon (Mann-Whitney)
Comments [Not Specified]
20.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 a 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 41 82
Median (Inter-Quartile Range)
Unit of Measure: mg/dL
Baseline
48
(41 to 53)
47
(40 to 58)
Primary efficacy time point
51
(42 to 58)
48
(40 to 58)
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 a 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   2/41 (4.88%)   6/83 (7.23%) 
Cardiac disorders     
Acute myocardial infarction  1  0/41 (0.00%)  1/83 (1.20%) 
Angina pectoris  1  0/41 (0.00%)  1/83 (1.20%) 
Coronary artery disease  1  1/41 (2.44%)  0/83 (0.00%) 
Supraventricular tachycardia  1  1/41 (2.44%)  0/83 (0.00%) 
General disorders     
Chest pain  1  0/41 (0.00%)  1/83 (1.20%) 
Non-cardiac chest pain  1  0/41 (0.00%)  1/83 (1.20%) 
Neoplasms benign, malignant and unspecified (incl cysts and polyps)     
Basal cell carcinoma  1  0/41 (0.00%)  1/83 (1.20%) 
Respiratory, thoracic and mediastinal disorders     
Pulmonary embolism  1  0/41 (0.00%)  1/83 (1.20%) 
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   38/41 (92.68%)   83/83 (100.00%) 
Blood and lymphatic system disorders     
Anaemia  1  0/41 (0.00%)  1/83 (1.20%) 
Lymphadenopathy  1  1/41 (2.44%)  1/83 (1.20%) 
Cardiac disorders     
Angina pectoris  1  0/41 (0.00%)  2/83 (2.41%) 
Coronary artery disease  1  0/41 (0.00%)  1/83 (1.20%) 
Myocardial ischaemia  1  1/41 (2.44%)  1/83 (1.20%) 
Palpitations  1  0/41 (0.00%)  2/83 (2.41%) 
Sinus bradycardia  1  1/41 (2.44%)  0/83 (0.00%) 
Ear and labyrinth disorders     
Ear pain  1  1/41 (2.44%)  0/83 (0.00%) 
Tinnitus  1  1/41 (2.44%)  0/83 (0.00%) 
Vertigo  1  1/41 (2.44%)  1/83 (1.20%) 
Eye disorders     
Cataract  1  0/41 (0.00%)  1/83 (1.20%) 
Conjunctivitis  1  0/41 (0.00%)  1/83 (1.20%) 
Dry eye  1  0/41 (0.00%)  1/83 (1.20%) 
Eye irritation  1  0/41 (0.00%)  1/83 (1.20%) 
Eyelid cyst  1  0/41 (0.00%)  1/83 (1.20%) 
Presbyopia  1  1/41 (2.44%)  0/83 (0.00%) 
Gastrointestinal disorders     
Abdominal distension  1  1/41 (2.44%)  1/83 (1.20%) 
Abdominal pain  1  1/41 (2.44%)  2/83 (2.41%) 
Abdominal pain lower  1  1/41 (2.44%)  0/83 (0.00%) 
Abdominal pain upper  1  1/41 (2.44%)  6/83 (7.23%) 
Bezoar  1  0/41 (0.00%)  1/83 (1.20%) 
Constipation  1  3/41 (7.32%)  4/83 (4.82%) 
Dental caries  1  2/41 (4.88%)  0/83 (0.00%) 
Diarrhoea  1  5/41 (12.20%)  9/83 (10.84%) 
Diverticulum  1  1/41 (2.44%)  1/83 (1.20%) 
Dry mouth  1  0/41 (0.00%)  1/83 (1.20%) 
Dyspepsia  1  0/41 (0.00%)  3/83 (3.61%) 
Dysphagia  1  0/41 (0.00%)  1/83 (1.20%) 
Gastric polyps  1  0/41 (0.00%)  1/83 (1.20%) 
Gastric ulcer  1  0/41 (0.00%)  1/83 (1.20%) 
Gastritis  1  0/41 (0.00%)  1/83 (1.20%) 
Gastrooesophageal reflux disease  1  0/41 (0.00%)  1/83 (1.20%) 
Haematochezia  1  1/41 (2.44%)  0/83 (0.00%) 
Haemorrhoids  1  1/41 (2.44%)  0/83 (0.00%) 
Hiatus hernia  1  0/41 (0.00%)  1/83 (1.20%) 
Hypoaesthesia oral  1  0/41 (0.00%)  1/83 (1.20%) 
Irritable bowel syndrome  1  1/41 (2.44%)  0/83 (0.00%) 
Lip pain  1  0/41 (0.00%)  1/83 (1.20%) 
Lip swelling  1  0/41 (0.00%)  1/83 (1.20%) 
Mouth ulceration  1  1/41 (2.44%)  0/83 (0.00%) 
Nausea  1  6/41 (14.63%)  14/83 (16.87%) 
Oesophageal dilatation  1  0/41 (0.00%)  1/83 (1.20%) 
Paraesthesia oral  1  0/41 (0.00%)  1/83 (1.20%) 
Periodontal disease  1  0/41 (0.00%)  1/83 (1.20%) 
Retching  1  0/41 (0.00%)  1/83 (1.20%) 
Tooth disorder  1  1/41 (2.44%)  0/83 (0.00%) 
Toothache  1  0/41 (0.00%)  1/83 (1.20%) 
Vomiting  1  0/41 (0.00%)  5/83 (6.02%) 
General disorders     
Adverse drug reaction  1  0/41 (0.00%)  1/83 (1.20%) 
Asthenia  1  2/41 (4.88%)  1/83 (1.20%) 
Chills  1  1/41 (2.44%)  6/83 (7.23%) 
Cyst  1  1/41 (2.44%)  0/83 (0.00%) 
Discomfort  1  1/41 (2.44%)  1/83 (1.20%) 
Facial pain  1  0/41 (0.00%)  1/83 (1.20%) 
Fatigue  1  4/41 (9.76%)  22/83 (26.51%) 
Feeling jittery  1  0/41 (0.00%)  1/83 (1.20%) 
Influenza like illness  1  3/41 (7.32%)  15/83 (18.07%) 
Injection site discolouration  1  1/41 (2.44%)  13/83 (15.66%) 
Injection site discomfort  1  0/41 (0.00%)  10/83 (12.05%) 
Injection site eczema  1  0/41 (0.00%)  1/83 (1.20%) 
Injection site erythema  1  3/41 (7.32%)  56/83 (67.47%) 
Injection site haematoma  1  9/41 (21.95%)  45/83 (54.22%) 
Injection site haemorrhage  1  1/41 (2.44%)  9/83 (10.84%) 
Injection site induration  1  0/41 (0.00%)  11/83 (13.25%) 
Injection site inflammation  1  0/41 (0.00%)  3/83 (3.61%) 
Injection site lymphadenopathy  1  0/41 (0.00%)  1/83 (1.20%) 
Injection site macule  1  0/41 (0.00%)  3/83 (3.61%) 
Injection site nodule  1  0/41 (0.00%)  8/83 (9.64%) 
Injection site oedema  1  0/41 (0.00%)  6/83 (7.23%) 
Injection site pain  1  8/41 (19.51%)  54/83 (65.06%) 
Injection site papule  1  0/41 (0.00%)  5/83 (6.02%) 
Injection site paraesthesia  1  0/41 (0.00%)  2/83 (2.41%) 
Injection site pruritus  1  2/41 (4.88%)  23/83 (27.71%) 
Injection site rash  1  0/41 (0.00%)  11/83 (13.25%) 
Injection site reaction  1  1/41 (2.44%)  5/83 (6.02%) 
Injection site recall reaction  1  0/41 (0.00%)  15/83 (18.07%) 
Injection site swelling  1  0/41 (0.00%)  13/83 (15.66%) 
Injection site urticaria  1  0/41 (0.00%)  3/83 (3.61%) 
Injection site vesicles  1  0/41 (0.00%)  2/83 (2.41%) 
Injection site warmth  1  0/41 (0.00%)  12/83 (14.46%) 
Injury associated with device  1  0/41 (0.00%)  1/83 (1.20%) 
Irritability  1  0/41 (0.00%)  2/83 (2.41%) 
Local swelling  1  0/41 (0.00%)  1/83 (1.20%) 
Localised oedema  1  0/41 (0.00%)  1/83 (1.20%) 
Malaise  1  1/41 (2.44%)  0/83 (0.00%) 
Non-cardiac chest pain  1  0/41 (0.00%)  2/83 (2.41%) 
Oedema peripheral  1  1/41 (2.44%)  7/83 (8.43%) 
Pain  1  3/41 (7.32%)  5/83 (6.02%) 
Pyrexia  1  0/41 (0.00%)  10/83 (12.05%) 
Tenderness  1  0/41 (0.00%)  1/83 (1.20%) 
Vessel puncture site haematoma  1  0/41 (0.00%)  1/83 (1.20%) 
Hepatobiliary disorders     
Hepatic cyst  1  0/41 (0.00%)  1/83 (1.20%) 
Hepatic lesion  1  1/41 (2.44%)  0/83 (0.00%) 
Hepatic steatosis  1  0/41 (0.00%)  5/83 (6.02%) 
Hepatomegaly  1  0/41 (0.00%)  1/83 (1.20%) 
Infections and infestations     
Abscess oral  1  1/41 (2.44%)  0/83 (0.00%) 
Asymptomatic bacteriuria  1  0/41 (0.00%)  1/83 (1.20%) 
Bronchitis  1  1/41 (2.44%)  0/83 (0.00%) 
Ear infection  1  1/41 (2.44%)  0/83 (0.00%) 
Eye infection  1  1/41 (2.44%)  0/83 (0.00%) 
Eye infection viral  1  1/41 (2.44%)  0/83 (0.00%) 
Furuncle  1  1/41 (2.44%)  0/83 (0.00%) 
Gastrointestinal infection  1  1/41 (2.44%)  0/83 (0.00%) 
Gastrointestinal viral infection  1  0/41 (0.00%)  2/83 (2.41%) 
H1N1 influenza  1  0/41 (0.00%)  1/83 (1.20%) 
Influenza  1  2/41 (4.88%)  5/83 (6.02%) 
Kidney infection  1  0/41 (0.00%)  2/83 (2.41%) 
Laryngitis  1  1/41 (2.44%)  0/83 (0.00%) 
Localised infection  1  0/41 (0.00%)  1/83 (1.20%) 
Lower respiratory tract infection  1  0/41 (0.00%)  1/83 (1.20%) 
Lyme disease  1  1/41 (2.44%)  0/83 (0.00%) 
Nasopharyngitis  1  3/41 (7.32%)  9/83 (10.84%) 
Pneumonia  1  0/41 (0.00%)  1/83 (1.20%) 
Respiratory tract infection  1  1/41 (2.44%)  0/83 (0.00%) 
Rhinitis  1  0/41 (0.00%)  1/83 (1.20%) 
Sinusitis  1  4/41 (9.76%)  1/83 (1.20%) 
Upper respiratory tract infection  1  3/41 (7.32%)  4/83 (4.82%) 
Urinary tract infection  1  4/41 (9.76%)  6/83 (7.23%) 
Vaginal infection  1  0/41 (0.00%)  1/83 (1.20%) 
Viral upper respiratory tract infection  1  0/41 (0.00%)  1/83 (1.20%) 
Vulvovaginal mycotic infection  1  1/41 (2.44%)  0/83 (0.00%) 
Injury, poisoning and procedural complications     
Animal bite  1  1/41 (2.44%)  0/83 (0.00%) 
Arthropod bite  1  1/41 (2.44%)  0/83 (0.00%) 
Arthropod sting  1  1/41 (2.44%)  0/83 (0.00%) 
Brain contusion  1  0/41 (0.00%)  1/83 (1.20%) 
Contusion  1  1/41 (2.44%)  5/83 (6.02%) 
Epicondylitis  1  0/41 (0.00%)  1/83 (1.20%) 
Face injury  1  0/41 (0.00%)  1/83 (1.20%) 
Fall  1  0/41 (0.00%)  1/83 (1.20%) 
Injury  1  0/41 (0.00%)  1/83 (1.20%) 
Joint sprain  1  1/41 (2.44%)  0/83 (0.00%) 
Muscle injury  1  1/41 (2.44%)  0/83 (0.00%) 
Muscle strain  1  1/41 (2.44%)  1/83 (1.20%) 
Post procedural haematoma  1  0/41 (0.00%)  1/83 (1.20%) 
Procedural pain  1  1/41 (2.44%)  1/83 (1.20%) 
Road traffic accident  1  1/41 (2.44%)  0/83 (0.00%) 
Scratch  1  0/41 (0.00%)  1/83 (1.20%) 
Skin laceration  1  1/41 (2.44%)  0/83 (0.00%) 
Sunburn  1  1/41 (2.44%)  0/83 (0.00%) 
Thermal burn  1  0/41 (0.00%)  1/83 (1.20%) 
Tooth fracture  1  0/41 (0.00%)  1/83 (1.20%) 
Investigations     
Alanine aminotransferase increased  1  0/41 (0.00%)  6/83 (7.23%) 
Aspartate aminotransferase increased  1  1/41 (2.44%)  3/83 (3.61%) 
Bacterial test positive  1  0/41 (0.00%)  1/83 (1.20%) 
Blood creatine phosphokinase increased  1  2/41 (4.88%)  0/83 (0.00%) 
Blood pressure increased  1  1/41 (2.44%)  0/83 (0.00%) 
Body temperature increased  1  0/41 (0.00%)  2/83 (2.41%) 
Carotid bruit  1  1/41 (2.44%)  1/83 (1.20%) 
Computerised tomogram abdomen abnormal  1  0/41 (0.00%)  1/83 (1.20%) 
Electrocardiogram T wave inversion  1  0/41 (0.00%)  1/83 (1.20%) 
Epstein-Barr virus antibody positive  1  0/41 (0.00%)  1/83 (1.20%) 
Haematocrit decreased  1  0/41 (0.00%)  1/83 (1.20%) 
Haemoglobin decreased  1  0/41 (0.00%)  1/83 (1.20%) 
Hepatic enzyme increased  1  0/41 (0.00%)  4/83 (4.82%) 
Herpes simplex serology positive  1  0/41 (0.00%)  1/83 (1.20%) 
International normalised ratio increased  1  0/41 (0.00%)  2/83 (2.41%) 
Liver function test abnormal  1  1/41 (2.44%)  5/83 (6.02%) 
Multiple gated acquisition scan abnormal  1  0/41 (0.00%)  1/83 (1.20%) 
Nitrite urine present  1  0/41 (0.00%)  1/83 (1.20%) 
Nuclear magnetic resonance imaging abnormal  1  0/41 (0.00%)  1/83 (1.20%) 
Platelet count decreased  1  0/41 (0.00%)  2/83 (2.41%) 
Protein urine present  1  0/41 (0.00%)  1/83 (1.20%) 
Pulse abnormal  1  0/41 (0.00%)  1/83 (1.20%) 
Red blood cells urine positive  1  0/41 (0.00%)  1/83 (1.20%) 
Transaminases increased  1  0/41 (0.00%)  2/83 (2.41%) 
Urine leukocyte esterase positive  1  0/41 (0.00%)  1/83 (1.20%) 
Weight decreased  1  1/41 (2.44%)  1/83 (1.20%) 
Weight increased  1  1/41 (2.44%)  1/83 (1.20%) 
White blood cells urine positive  1  1/41 (2.44%)  1/83 (1.20%) 
Metabolism and nutrition disorders     
Decreased appetite  1  3/41 (7.32%)  2/83 (2.41%) 
Dehydration  1  0/41 (0.00%)  1/83 (1.20%) 
Vitamin B12 deficiency  1  0/41 (0.00%)  1/83 (1.20%) 
Musculoskeletal and connective tissue disorders     
Arthralgia  1  4/41 (9.76%)  6/83 (7.23%) 
Axillary mass  1  1/41 (2.44%)  0/83 (0.00%) 
Back pain  1  4/41 (9.76%)  7/83 (8.43%) 
Bursitis  1  1/41 (2.44%)  0/83 (0.00%) 
Dupuytren's contracture  1  0/41 (0.00%)  1/83 (1.20%) 
Exostosis  1  0/41 (0.00%)  1/83 (1.20%) 
Groin pain  1  1/41 (2.44%)  1/83 (1.20%) 
Joint effusion  1  0/41 (0.00%)  1/83 (1.20%) 
Joint range of motion decreased  1  0/41 (0.00%)  1/83 (1.20%) 
Joint warmth  1  0/41 (0.00%)  1/83 (1.20%) 
Muscle fatigue  1  0/41 (0.00%)  1/83 (1.20%) 
Muscle spasms  1  0/41 (0.00%)  1/83 (1.20%) 
Muscular weakness  1  0/41 (0.00%)  1/83 (1.20%) 
Musculoskeletal discomfort  1  1/41 (2.44%)  0/83 (0.00%) 
Musculoskeletal pain  1  1/41 (2.44%)  3/83 (3.61%) 
Musculoskeletal stiffness  1  0/41 (0.00%)  1/83 (1.20%) 
Myalgia  1  4/41 (9.76%)  8/83 (9.64%) 
Neck pain  1  1/41 (2.44%)  0/83 (0.00%) 
Osteoarthritis  1  0/41 (0.00%)  1/83 (1.20%) 
Osteopenia  1  1/41 (2.44%)  0/83 (0.00%) 
Pain in extremity  1  3/41 (7.32%)  4/83 (4.82%) 
Pain in jaw  1  0/41 (0.00%)  1/83 (1.20%) 
Sensation of heaviness  1  0/41 (0.00%)  1/83 (1.20%) 
Spinal osteoarthritis  1  0/41 (0.00%)  1/83 (1.20%) 
Spondylitis  1  0/41 (0.00%)  1/83 (1.20%) 
Neoplasms benign, malignant and unspecified (incl cysts and polyps)     
Basal cell carcinoma  1  0/41 (0.00%)  1/83 (1.20%) 
Lipoma  1  0/41 (0.00%)  2/83 (2.41%) 
Morton's neuroma  1  0/41 (0.00%)  1/83 (1.20%) 
Nervous system disorders     
Amnesia  1  1/41 (2.44%)  0/83 (0.00%) 
Burning sensation  1  0/41 (0.00%)  1/83 (1.20%) 
Carotid artery stenosis  1  1/41 (2.44%)  0/83 (0.00%) 
Carpal tunnel syndrome  1  1/41 (2.44%)  0/83 (0.00%) 
Disturbance in attention  1  0/41 (0.00%)  1/83 (1.20%) 
Dizziness  1  4/41 (9.76%)  4/83 (4.82%) 
Dizziness postural  1  0/41 (0.00%)  2/83 (2.41%) 
Dysgeusia  1  0/41 (0.00%)  1/83 (1.20%) 
Headache  1  7/41 (17.07%)  15/83 (18.07%) 
Hyperaesthesia  1  1/41 (2.44%)  0/83 (0.00%) 
Hypoaesthesia  1  1/41 (2.44%)  0/83 (0.00%) 
Lethargy  1  0/41 (0.00%)  2/83 (2.41%) 
Migraine with aura  1  0/41 (0.00%)  1/83 (1.20%) 
Paraesthesia  1  0/41 (0.00%)  4/83 (4.82%) 
Sciatica  1  0/41 (0.00%)  2/83 (2.41%) 
Sinus headache  1  0/41 (0.00%)  1/83 (1.20%) 
Psychiatric disorders     
Anxiety  1  2/41 (4.88%)  3/83 (3.61%) 
Claustrophobia  1  0/41 (0.00%)  1/83 (1.20%) 
Insomnia  1  1/41 (2.44%)  2/83 (2.41%) 
Libido decreased  1  0/41 (0.00%)  1/83 (1.20%) 
Panic attack  1  0/41 (0.00%)  1/83 (1.20%) 
Stress  1  0/41 (0.00%)  1/83 (1.20%) 
Renal and urinary disorders     
Albuminuria  1  0/41 (0.00%)  1/83 (1.20%) 
Chromaturia  1  0/41 (0.00%)  1/83 (1.20%) 
Dysuria  1  0/41 (0.00%)  1/83 (1.20%) 
Haematuria  1  0/41 (0.00%)  1/83 (1.20%) 
Micturition urgency  1  0/41 (0.00%)  1/83 (1.20%) 
Pollakiuria  1  0/41 (0.00%)  2/83 (2.41%) 
Proteinuria  1  0/41 (0.00%)  3/83 (3.61%) 
Stress urinary incontinence  1  0/41 (0.00%)  1/83 (1.20%) 
Urge incontinence  1  0/41 (0.00%)  1/83 (1.20%) 
Reproductive system and breast disorders     
Menstruation delayed  1  1/41 (2.44%)  0/83 (0.00%) 
Prostatitis  1  1/41 (2.44%)  0/83 (0.00%) 
Prostatomegaly  1  1/41 (2.44%)  0/83 (0.00%) 
Respiratory, thoracic and mediastinal disorders     
Cough  1  2/41 (4.88%)  9/83 (10.84%) 
Dyspnoea  1  0/41 (0.00%)  2/83 (2.41%) 
Dyspnoea exertional  1  1/41 (2.44%)  0/83 (0.00%) 
Nasal congestion  1  1/41 (2.44%)  2/83 (2.41%) 
Nasal polyps  1  1/41 (2.44%)  0/83 (0.00%) 
Oropharyngeal pain  1  1/41 (2.44%)  7/83 (8.43%) 
Paranasal sinus hypersecretion  1  0/41 (0.00%)  1/83 (1.20%) 
Postnasal drip  1  0/41 (0.00%)  1/83 (1.20%) 
Productive cough  1  1/41 (2.44%)  2/83 (2.41%) 
Pulmonary congestion  1  1/41 (2.44%)  0/83 (0.00%) 
Rhinitis allergic  1  1/41 (2.44%)  0/83 (0.00%) 
Rhinorrhoea  1  1/41 (2.44%)  3/83 (3.61%) 
Sinus congestion  1  0/41 (0.00%)  2/83 (2.41%) 
Sinus disorder  1  0/41 (0.00%)  1/83 (1.20%) 
Upper respiratory tract congestion  1  1/41 (2.44%)  1/83 (1.20%) 
Skin and subcutaneous tissue disorders     
Actinic keratosis  1  1/41 (2.44%)  0/83 (0.00%) 
Cold sweat  1  0/41 (0.00%)  1/83 (1.20%) 
Dermatitis  1  0/41 (0.00%)  1/83 (1.20%) 
Dermatitis contact  1  1/41 (2.44%)  0/83 (0.00%) 
Dry skin  1  0/41 (0.00%)  1/83 (1.20%) 
Ecchymosis  1  0/41 (0.00%)  2/83 (2.41%) 
Eczema  1  0/41 (0.00%)  1/83 (1.20%) 
Erythema  1  0/41 (0.00%)  1/83 (1.20%) 
Hyperhidrosis  1  1/41 (2.44%)  1/83 (1.20%) 
Hypoaesthesia facial  1  0/41 (0.00%)  1/83 (1.20%) 
Pruritus  1  2/41 (4.88%)  2/83 (2.41%) 
Pruritus generalised  1  0/41 (0.00%)  2/83 (2.41%) 
Rash  1  4/41 (9.76%)  2/83 (2.41%) 
Rash maculo-papular  1  0/41 (0.00%)  1/83 (1.20%) 
Scab  1  0/41 (0.00%)  1/83 (1.20%) 
Skin induration  1  0/41 (0.00%)  1/83 (1.20%) 
Skin lesion  1  1/41 (2.44%)  0/83 (0.00%) 
Skin plaque  1  0/41 (0.00%)  1/83 (1.20%) 
Urticaria  1  0/41 (0.00%)  1/83 (1.20%) 
Vascular disorders     
Aortic aneurysm  1  0/41 (0.00%)  1/83 (1.20%) 
Flushing  1  1/41 (2.44%)  1/83 (1.20%) 
Hot flush  1  0/41 (0.00%)  3/83 (3.61%) 
Hypertension  1  1/41 (2.44%)  2/83 (2.41%) 
Hypotension  1  0/41 (0.00%)  1/83 (1.20%) 
Infarction  1  0/41 (0.00%)  1/83 (1.20%) 
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: NCT00706849     History of Changes
Other Study ID Numbers: 301012-CS7
First Submitted: June 26, 2008
First Posted: June 30, 2008
Results First Submitted: February 15, 2013
Results First Posted: March 21, 2013
Last Update Posted: September 9, 2016