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Trial record 15 of 19 for:    MIPOMERSEN

Measure Liver Fat Content After ISIS 301012 (Mipomersen) Administration

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT00362180
Recruitment Status : Completed
First Posted : August 9, 2006
Results First Posted : June 24, 2015
Last Update Posted : October 21, 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 Lipid Metabolism, Inborn Errors
Hyperlipidemias
Metabolic Diseases
Hypolipoproteinemia
Hypolipoproteinemias
Hypobetalipoproteinemias
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Infant, Newborn, Diseases
Congenital Abnormalities
Metabolic Disorder
Hypercholesterolemia
Dyslipidemias
Lipid Metabolism Disorders
Interventions Drug: mipomersen
Drug: Placebo
Enrollment 38
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Cohort A: Mipomersen Cohort A: Placebo Cohort D: Mipomersen Cohort D: Placebo Cohort E: Placebo Cohort E: Mipomersen Cohort F: no Study Intervention Cohort G: Mipomersen Cohort G: Placebo Followed by Mipomersen
Hide Arm/Group Description Healthy volunteers treated with 6 injections of mipomersen 200 mg over the course of 4 weeks. Healthy volunteers treated with 6 injections of placebo over the course of 4 weeks. Participants with impaired fasting glucose and mixed dyslipidemia who were treated with 6 injections of mipomersen 200 mg over the course of 4 weeks. Participants with impaired fasting glucose and mixed dyslipidemia who were treated with 6 injections of placebo over the course of 4 weeks. Participants with uncomplicated heterozygous familial hypercholesterolemia (HeFH) treated with weekly placebo injections for 13 weeks. Participants with uncomplicated heterozygous familial hypercholesterolemia (HeFH) treated with weekly mipomersen 200 mg injections for 13 weeks. A reference group of participants with familial hypobetalipoproteinemia (FBHL) who did not receive a study intervention. Data gathered for 15 weeks. Participants with well-controlled Type 2 diabetes mellitus, hypercholesterolemia, and normal triglyceride levels were treated with mipomersen 200 mg weekly for 26 weeks, followed by open-label mipomersen 200 mg weekly for an additional 26 weeks. Participants with well-controlled Type 2 diabetes mellitus, hypercholesterolemia, and normal triglyceride levels were treated with placebo weekly for 26 weeks, followed by open-label mipomersen 200 mg weekly for an additional 26 weeks.
Period Title: Treated Period
Started 4 2 1 0 11 10 6 3 1
Completed 3 2 1 0 11 9 6 2 1
Not Completed 1 0 0 0 0 1 0 1 0
Reason Not Completed
Withdrawal by Subject             1             0             0             0             0             0             0             0             0
Adverse Event             0             0             0             0             0             1             0             1             0
Period Title: 20 Week Safety Follow-up
Started 3 2 1 0 11 9 0 [1] 3 1
Completed 3 2 1 0 11 9 0 3 1
Not Completed 0 0 0 0 0 0 0 0 0
[1]
This observational cohort did not include a safety follow-up period.
Arm/Group Title Cohort A: Mipomersen Cohort A: Placebo Cohort D: Mipomersen Cohort D: Placebo Cohort E: Placebo Cohort E: Mipomersen Cohort F: no Study Intervention Cohort G: Mipomersen Cohort G: Placebo Followed by Mipomersen Total
Hide Arm/Group Description Healthy volunteers treated with 6 injections of mipomersen 200 mg over the course of 4 weeks. Healthy volunteers treated with 6 injections of placebo over the course of 4 weeks. Participants with impaired fasting glucose and mixed dyslipidemia who were treated with 6 injections of mipomersen 200 mg over the course of 4 weeks. Participants with impaired fasting glucose and mixed dyslipidemia who were treated with 6 injections of placebo over the course of 4 weeks. Participants with uncomplicated heterozygous familial hypercholesterolemia (HeFH) treated with weekly placebo injections for 13 weeks. Participants with uncomplicated heterozygous familial hypercholesterolemia (HeFH) treated with weekly mipomersen 200 mg injections for 13 weeks. A reference group of participants with familial hypobetalipoproteinemia (FBHL) who did not receive a study intervention. Data gathered for 15 weeks. Participants with well-controlled Type 2 diabetes mellitus, hypercholesterolemia, and normal triglyceride levels were treated with mipomersen 200 mg weekly for 26 weeks, followed by open-label mipomersen 200 mg weekly for an additional 26 weeks. Participants with well-controlled Type 2 diabetes mellitus, hypercholesterolemia, and normal triglyceride levels were treated with placebo weekly for 26 weeks, followed by open-label mipomersen 200 mg weekly for an additional 26 weeks. Total of all reporting groups
Overall Number of Baseline Participants 4 2 1 0 11 10 6 3 1 38
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Median (Full Range)
Unit of measure:  Years
Number Analyzed 4 participants 2 participants 1 participants 0 participants 11 participants 10 participants 6 participants 3 participants 1 participants 38 participants
58.5
(57 to 65)
40.5
(24 to 57)
59.0
(59 to 59)
46.0
(28 to 59)
50.0
(23 to 63)
49.5
(29 to 71)
62.0
(49 to 64)
58.0
(58 to 58)
53.5
(23 to 71)
Gender  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 4 participants 2 participants 1 participants 0 participants 11 participants 10 participants 6 participants 3 participants 1 participants 38 participants
Female 2 1 1 8 4 1 2 1 20
Male 2 1 0 3 6 5 1 0 18
Race/Ethnicity, Customized  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 4 participants 2 participants 1 participants 0 participants 11 participants 10 participants 6 participants 3 participants 1 participants 38 participants
White 4 1 1 11 10 6 3 1 37
Black or African American 0 1 0 0 0 0 0 0 1
1.Primary Outcome
Title Change From Baseline in Liver Triglyceride (TG) Content As Measured by Magnetic Resonance Spectroscopy (MRS)
Hide Description Localized proton MRS was used to quantify liver TG concentration. All MRS imaging of the liver was performed using the same 1.5T scanner. The instructions were to cover the entire liver (from just above the dome to just below the inferior tip) using a qualified imaging technique (Yokoo, 2009, Radiology). Liver fat quantification was performed by determining the liver fat fraction (%) derived from MRS using selected Regions of Interest (ROI). The analyst typically looked for the branching of the right portal vein and mapped slices from one scan to the next. There were typically 2 ROIs in the right lobe and 1 in the left lobe. Magnetic resonance spectroscopy imaging ROIs between different scans were selected using anatomical landmarks for both time points.
Time Frame Baseline, Day 26, Day 99
Hide Outcome Measure Data
Hide Analysis Population Description
Full analysis set. In Cohort E: Placebo, Day 99 N=11 instead of 10 because participant did not have a post treatment MRS by Day 26.
Arm/Group Title Cohort A: Placebo Cohort A: Mipomersen Cohort D Cohort E: Placebo Cohort E: Mipomersen Cohort F: no Study Intervention Cohort G: Placebo Followed by Mipomersen Cohort G: Mipomersen
Hide Arm/Group Description:
Healthy volunteers treated with 6 injections of placebo over the course of 4 weeks.
Healthy volunteers treated with 6 injections of mipomersen 200 mg over the course of 4 weeks.
Participants with impaired fasting glucose and mixed dyslipidemia who were treated with 6 injections of mipomersen 200 mg over the course of 4 weeks.
Participants with uncomplicated heterozygous familial hypercholesterolemia (HeFH) treated with weekly placebo injections for 13 weeks.
Participants with uncomplicated heterozygous familial hypercholesterolemia (HeFH) treated with weekly mipomersen 200 mg injections for 13 weeks.
Participants with a diagnosis of Familial Hypobetalipoproteinemia (FHBL) and were not treated with mipomersen or placebo.
Participants with well-controlled Type 2 diabetes mellitus, hypercholesterolemia, and normal triglyceride levels were treated with placebo weekly for 26 weeks, followed by open-label mipomersen 200 mg weekly for an additional 26 weeks.
Participants with well-controlled Type 2 diabetes mellitus, hypercholesterolemia, and normal triglyceride levels were treated with mipomersen 200 mg weekly for 26 weeks, followed by open-label mipomersen 200 mg weekly for an additional 26 weeks.
Overall Number of Participants Analyzed 2 4 1 11 10 6 1 3
Median (Full Range)
Unit of Measure: percentage of total liver content
Baseline (Cohort E: Placebo n=11)
0.2
(0.1 to 0.3)
1.2
(0.5 to 1.8)
6.4
(6.4 to 6.4)
0.8
(0.2 to 3.2)
1.1
(0.3 to 3.8)
21.4
(13.2 to 30.1)
2.6
(2.6 to 2.6)
2.5
(1.7 to 3.2)
Day 26 (Cohort E: Placebo n=10)
0.3
(0.0 to 0.6)
0.3
(0.0 to 0.5)
4.45
(4.45 to 4.45)
0.1
(-0.4 to 0.8)
0.1
(-2.5 to 0.5)
-1.2
(-2.6 to 4.3)
NA [1] 
(NA to NA)
NA [1] 
(NA to NA)
Day 99 (Cohort E: Placebo n=11)
NA [2] 
(NA to NA)
NA [2] 
(NA to NA)
NA [3] 
(NA to NA)
-0.0
(-1.5 to 1.3)
0.4
(-1.7 to 5.1)
0.8
(-2.0 to 4.3)
-1.0
(-1.0 to -1.0)
7.8
(1.1 to 14.5)
[1]
Liver TG not collected at day 26 as per protocol
[2]
Liver TG not collected at day 99 as per protocol
[3]
Only 1 patient was enrolled in cohort D, there was no day 99 visit for cohort D
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Cohort E: Placebo, Cohort E: Mipomersen
Comments The p-value is a comparison between the placebo group and the mipomersen group in Cohort E as a change from Baseline to Day 26.
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.7244
Comments [Not Specified]
Method exact Wilcoxon Rank-sum test
Comments [Not Specified]
Show Statistical Analysis 2 Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Cohort E: Placebo, Cohort E: Mipomersen
Comments The p-value is a comparison between the placebo group and the mipomersen group in Cohort E as a change from Baseline to Day 99.
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0513
Comments [Not Specified]
Method exact Wilcoxon Rank-sum test
Comments [Not Specified]
2.Secondary Outcome
Title Baseline Apolipoprotein B
Hide Description Samples were taken following an overnight fast.
Time Frame Baseline
Hide Outcome Measure Data
Hide Analysis Population Description
Full analysis set
Arm/Group Title Cohort A: Placebo Cohort A: Mipomersen Cohort D Cohort E: Placebo Cohort E: Mipomersen Cohort F: no Study Intervention Cohort G: Placebo Followed by Mipomersen Cohort G: Mipomersen
Hide Arm/Group Description:
Healthy volunteers treated with 6 injections of placebo over the course of 4 weeks.
Healthy volunteers treated with 6 injections of mipomersen 200 mg over the course of 4 weeks.
Participants with impaired fasting glucose and mixed dyslipidemia who were treated with 6 injections of mipomersen 200 mg over the course of 4 weeks.
Participants with uncomplicated heterozygous familial hypercholesterolemia (HeFH) treated with weekly placebo injections for 13 weeks.
Participants with uncomplicated heterozygous familial hypercholesterolemia (HeFH) treated with weekly mipomersen 200 mg injections for 13 weeks.
Participants with a diagnosis of Familial Hypobetalipoproteinemia (FHBL) and were not treated with mipomersen or placebo.
Participants with well-controlled Type 2 diabetes mellitus, hypercholesterolemia, and normal triglyceride levels were treated with placebo weekly for 26 weeks, followed by open-label mipomersen 200 mg weekly for an additional 26 weeks.
Participants with well-controlled Type 2 diabetes mellitus, hypercholesterolemia, and normal triglyceride levels were treated with mipomersen 200 mg weekly for 26 weeks, followed by open-label mipomersen 200 mg weekly for an additional 26 weeks.
Overall Number of Participants Analyzed 2 4 1 11 10 6 1 3
Median (Inter-Quartile Range)
Unit of Measure: mg/wk
86
(70 to 102)
100.5
(76.5 to 102.0)
155
(155 to 155)
122.0
(106.0 to 148.0)
126.5
(109.0 to 153.0)
31.5
(28.0 to 41.0)
120.0
(120.0 to 120.0)
113.0
(90.0 to 132.0)
3.Secondary Outcome
Title Percent Change in Apolipoprotein B From Baseline to Day 99
Hide Description Samples were taken following an overnight fast.
Time Frame Day 26 and Day 99
Hide Outcome Measure Data
Hide Analysis Population Description
Full analysis set with last observation carried forward.
Arm/Group Title Cohort A: Placebo Cohort A: Mipomersen Cohort D Cohort E: Placebo Cohort E: Mipomersen Cohort F: No Study Intervention Cohort G: Placebo Followed by Mipomersen Cohort G: Mipomersen
Hide Arm/Group Description:
Healthy volunteers treated with 6 injections of placebo over the course of 4 weeks.
Healthy volunteers treated with 6 injections of mipomersen 200 mg over the course of 4 weeks.
Participants with impaired fasting glucose and mixed dyslipidemia who were treated with 6 injections of mipomersen 200 mg over the course of 4 weeks.
Participants with uncomplicated heterozygous familial hypercholesterolemia (HeFH) treated with weekly placebo injections for 13 weeks.
Participants with uncomplicated heterozygous familial hypercholesterolemia (HeFH) treated with weekly mipomersen 200 mg injections for 13 weeks.
A reference group of participants with familial hypobetalipoproteinemia (FBHL) who did not receive a study intervention. Data gathered for 15 weeks.
Participants with well-controlled Type 2 diabetes mellitus, hypercholesterolemia, and normal triglyceride levels were treated with placebo weekly for 26 weeks, followed by open-label mipomersen 200 mg weekly for an additional 26 weeks.
Participants with well-controlled Type 2 diabetes mellitus, hypercholesterolemia, and normal triglyceride levels were treated with mipomersen 200 mg weekly for 26 weeks, followed by open-label mipomersen 200 mg weekly for an additional 26 weeks.
Overall Number of Participants Analyzed 2 4 1 11 10 6 1 3
Median (Inter-Quartile Range)
Unit of Measure: percent change
Day 26
-3.3
(-8.6 to 2.0)
-20.8
(-24.8 to -13.6)
-41.6149
(-41.6149 to -41.6149)
-1.1
(-1.8 to 0.0)
-1.8
(-16.3 to 13.4)
3.6
(-4.9 to 14.3)
NA [1] 
(NA to NA)
NA [1] 
(NA to NA)
Day 99
NA [2] 
(NA to NA)
NA [2] 
(NA to NA)
NA [3] 
(NA to NA)
6.0
(-2.7 to 15.3)
-16.3
(-29.3 to -5.6)
4.1
(0.0 to 5.9)
-25.8
(-25.8 to -25.8)
-44.2
(-55.6 to -18.2)
[1]
Apolipoprotein B was not collected at day 26 as per protocol
[2]
Apolipoprotein B was not collected at day 99 as per protocol
[3]
Only 1 patient was enrolled in cohort D, there was no day 99 visit for cohort D
4.Secondary Outcome
Title Baseline Low-Density Lipoprotein Cholesterol
Hide Description Samples were taken following overnight fast.
Time Frame Baseline
Hide Outcome Measure Data
Hide Analysis Population Description
Full analysis set
Arm/Group Title Cohort A: Placebo Cohort A: Mipomersen Cohort D Cohort E: Placebo Cohort E: Mipomersen Cohort F: No Study Intervention Cohort G: Placebo Followed by Mipomersen Cohort G: Mipomersen
Hide Arm/Group Description:
Healthy volunteers treated with 6 injections of placebo over the course of 4 weeks.
Healthy volunteers treated with 6 injections of mipomersen 200 mg over the course of 4 weeks.
Participants with impaired fasting glucose and mixed dyslipidemia who were treated with 6 injections of mipomersen 200 mg over the course of 4 weeks.
Participants with uncomplicated heterozygous familial hypercholesterolemia (HeFH) treated with weekly placebo injections for 13 weeks.
Participants with uncomplicated heterozygous familial hypercholesterolemia (HeFH) treated with weekly mipomersen 200 mg injections for 13 weeks.
A reference group of participants with familial hypobetalipoproteinemia (FBHL) who did not receive a study intervention. Data gathered for 15 weeks.
Participants with well-controlled Type 2 diabetes mellitus, hypercholesterolemia, and normal triglyceride levels were treated with placebo weekly for 26 weeks, followed by open-label mipomersen 200 mg weekly for an additional 26 weeks.
Participants with well-controlled Type 2 diabetes mellitus, hypercholesterolemia, and normal triglyceride levels were treated with mipomersen 200 mg weekly for 26 weeks, followed by open-label mipomersen 200 mg weekly for an additional 26 weeks.
Overall Number of Participants Analyzed 2 4 1 11 10 6 1 3
Median (Inter-Quartile Range)
Unit of Measure: mg/wk
103.5
(77.0 to 130.0)
126.0
(93.0 to 139.5)
156.0
(156.0 to 156.0)
148.0
(131.0 to 182.0)
151.5
(127.0 to 161.0)
41.5
(37.0 to 55.0)
147.0
(147.0 to 147.0)
121.0
(94.0 to 131.0)
5.Secondary Outcome
Title Percent Change in Low-Density Lipoprotein Cholesterol From Baseline to Day 99
Hide Description Samples were taken following an overnight fast.
Time Frame Day 26 and Day 99
Hide Outcome Measure Data
Hide Analysis Population Description
Full analysis set with last observation carried forward.
Arm/Group Title Cohort A: Placebo Cohort A: Mipomersen Cohort D: Mipomersen Cohort E: Placebo Cohort E: Mipomersen Cohort F: No Study Intervention Cohort G: Placebo Follwed by Mipomersen Cohort G: Mipomersen
Hide Arm/Group Description:
Healthy volunteers treated with 6 injections of placebo over the course of 4 weeks.
Healthy volunteers treated with 6 injections of mipomersen 200 mg over the course of 4 weeks.
Participants with impaired fasting glucose and mixed dyslipidemia who were treated with 6 injections of mipomersen 200 mg over the course of 4 weeks.
Participants with uncomplicated heterozygous familial hypercholesterolemia (HeFH) treated with weekly placebo injections for 13 weeks.
Participants with uncomplicated heterozygous familial hypercholesterolemia (HeFH) treated with weekly mipomersen 200 mg injections for 13 weeks.
A reference group of participants with familial hypobetalipoproteinemia (FBHL) who did not receive a study intervention. Data gathered for 15 weeks.
Participants with well-controlled Type 2 diabetes mellitus, hypercholesterolemia, and normal triglyceride levels were treated with placebo weekly for 26 weeks, followed by open-label mipomersen 200 mg weekly for an additional 26 weeks.
Participants with well-controlled Type 2 diabetes mellitus, hypercholesterolemia, and normal triglyceride levels were treated with mipomersen 200 mg weekly for 26 weeks, followed by open-label mipomersen 200 mg weekly for an additional 26 weeks.
Overall Number of Participants Analyzed 2 4 1 11 10 6 1 3
Median (Inter-Quartile Range)
Unit of Measure: percent change
Day 26
1.1
(-11.7 to 13.8)
-14.3
(-20.5 to -6.0)
-41.7143
(-41.7143 to -41.7143)
-2.7
(-5.0 to 2.4)
-5.4
(-20.9 to 11.4)
0.0
(-4.9 to 8.0)
NA [1] 
(NA to NA)
NA [1] 
(NA to NA)
Day 99
NA [2] 
(NA to NA)
NA [2] 
(NA to NA)
NA [3] 
(NA to NA)
0.7
(-9.8 to 13.5)
-15.9
(-36.1 to -12.4)
2.6
(-5.1 to 9.0)
-19.0
(-19.0 to -19.0)
-33.9
(-63.8 to -18.3)
[1]
Low-density lipoprotein was not collected at day 26 as per protocol
[2]
Low-density lipoprotein was not collected at day 99 as per protocol
[3]
Only 1 patient was enrolled in cohort D, there was no day 99 visit for cohort D
6.Secondary Outcome
Title Baseline Total Cholesterol
Hide Description Samples were taken following an overnight fast.
Time Frame Baseline
Hide Outcome Measure Data
Hide Analysis Population Description
Full analysis set
Arm/Group Title Cohort A: Placebo Cohort A: Mipomersen Cohort D Cohort E: Placebo Cohort E: Mipomersen Cohort F: No Study Intervention Cohort G: Placebo Followed by Mipomersen Cohort G: Mipomersen
Hide Arm/Group Description:
Healthy volunteers treated with 6 injections of placebo over the course of 4 weeks.
Healthy volunteers treated with 6 injections of mipomersen 200 mg over the course of 4 weeks.
Participants with impaired fasting glucose and mixed dyslipidemia who were treated with 6 injections of mipomersen 200 mg over the course of 4 weeks.
Participants with uncomplicated heterozygous familial hypercholesterolemia (HeFH) treated with weekly placebo injections for 13 weeks.
Participants with uncomplicated heterozygous familial hypercholesterolemia (HeFH) treated with weekly mipomersen 200 mg injections for 13 weeks.
A reference group of participants with familial hypobetalipoproteinemia (FBHL) who did not receive a study intervention. Data gathered for 15 weeks.
Participants with well-controlled Type 2 diabetes mellitus, hypercholesterolemia, and normal triglyceride levels were treated with placebo weekly for 26 weeks, followed by open-label mipomersen 200 mg weekly for an additional 26 weeks.
Participants with well-controlled Type 2 diabetes mellitus, hypercholesterolemia, and normal triglyceride levels were treated with mipomersen 200 mg weekly for 26 weeks, followed by open-label mipomersen 200 mg weekly for an additional 26 weeks.
Overall Number of Participants Analyzed 2 4 1 11 10 6 1 3
Median (Inter-Quartile Range)
Unit of Measure: mg/wk
201.0
(165.0 to 237.0)
207.0
(176.0 to 213.0)
263.0
(263.0 to 263.0)
208.0
(191.0 to 245.0)
220.5
(189.0 to 248.0)
107.0
(93.0 to 115.0)
231.0
(231.0 to 231.0)
199.0
(171.0 to 208.0)
7.Secondary Outcome
Title Percent Change in Total Cholesterol From Baseline to Day 99
Hide Description Samples were taken following an overnight fast.
Time Frame Day 26 and Day 99
Hide Outcome Measure Data
Hide Analysis Population Description
Full analysis set with last observation carried forward.
Arm/Group Title Cohort A: Placebo Cohort A: Mipomersen Cohort D: Mipomersen Cohort E: Mipomersen Cohort E: Placebo Cohort F: No Study Intervention Cohort G: Placebo Follwed by Mipomersen Cohort G: Mipomersen
Hide Arm/Group Description:
Healthy volunteers treated with 6 injections of placebo over the course of 4 weeks.
Healthy volunteers treated with 6 injections of mipomersen 200 mg over the course of 4 weeks.
Participants with impaired fasting glucose and mixed dyslipidemia who were treated with 6 injections of mipomersen 200 mg over the course of 4 weeks.
Participants with uncomplicated heterozygous familial hypercholesterolemia (HeFH) treated with weekly mipomersen 200 mg injections for 13 weeks.
Participants with uncomplicated heterozygous familial hypercholesterolemia (HeFH) treated with weekly placebo injections for 13 weeks.
A reference group of participants with familial hypobetalipoproteinemia (FBHL) who did not receive a study intervention. Data gathered for 15 weeks.
Participants with well-controlled Type 2 diabetes mellitus, hypercholesterolemia, and normal triglyceride levels were treated with placebo weekly for 26 weeks, followed by open-label mipomersen 200 mg weekly for an additional 26 weeks.
Participants with well-controlled Type 2 diabetes mellitus, hypercholesterolemia, and normal triglyceride levels were treated with mipomersen 200 mg weekly for 26 weeks, followed by open-label mipomersen 200 mg weekly for an additional 26 weeks.
Overall Number of Participants Analyzed 2 4 1 10 11 6 1 3
Median (Inter-Quartile Range)
Unit of Measure: percent change
Day 26
-3.5
(-7.9 to 0.8)
-11.7
(-12.5 to -0.7)
-29.5374
(-29.5374 to -29.5374)
-3.1
(-16.9 to 10.1)
0.1
(-2.1 to 3.0)
1.4
(0.0 to 2.7)
NA [1] 
(NA to NA)
NA [1] 
(NA to NA)
Day 99
NA [2] 
(NA to NA)
NA [2] 
(NA to NA)
NA [3] 
(NA to NA)
-11.8
(-24.2 to -9.7)
-0.8
(-8.9 to 12.7)
-0.4
(-6.9 to 4.3)
-21.6
(-33.9 to -14.1)
-21.6
(-33.9 to -14.1)
[1]
Total cholesterol was not collected at day 26 as per protocol
[2]
Total cholesterol was not collected at day 99 as per protocol
[3]
Only 1 patient was enrolled in cohort D, there was no day 99 visit for cohort D
Time Frame [Not Specified]
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 Not Treated
Hide Arm/Group Description Placebo Mipomersen Not Treated
All-Cause Mortality
Placebo Mipomersen Not Treated
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   --/--   --/--   --/-- 
Show Serious Adverse Events Hide Serious Adverse Events
Placebo Mipomersen Not Treated
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   0/14 (0.00%)   1/19 (5.26%)   0/6 (0.00%) 
Respiratory, thoracic and mediastinal disorders       
Nasal septum disorder  1  0/14 (0.00%)  1/19 (5.26%)  0/6 (0.00%) 
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 Not Treated
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   14/14 (100.00%)   19/19 (100.00%)   3/6 (50.00%) 
Blood and lymphatic system disorders       
Anaemia  1  0/14 (0.00%)  1/19 (5.26%)  0/6 (0.00%) 
Eosinophilia  1  0/14 (0.00%)  1/19 (5.26%)  0/6 (0.00%) 
Thrombocytopenia  1  0/14 (0.00%)  1/19 (5.26%)  0/6 (0.00%) 
Cardiac disorders       
Palpitations  1  0/14 (0.00%)  1/19 (5.26%)  0/6 (0.00%) 
Ear and labyrinth disorders       
Ear pain  1  0/14 (0.00%)  1/19 (5.26%)  0/6 (0.00%) 
Eustachian tube disorder  1  0/14 (0.00%)  1/19 (5.26%)  0/6 (0.00%) 
Eye disorders       
Eye inflammation  1  0/14 (0.00%)  1/19 (5.26%)  0/6 (0.00%) 
Vitreous floaters  1  0/14 (0.00%)  1/19 (5.26%)  0/6 (0.00%) 
Gastrointestinal disorders       
Abdominal discomfort  1  0/14 (0.00%)  1/19 (5.26%)  0/6 (0.00%) 
Abdominal pain  1  0/14 (0.00%)  1/19 (5.26%)  0/6 (0.00%) 
Abdominal pain upper  1  0/14 (0.00%)  2/19 (10.53%)  0/6 (0.00%) 
Constipation  1  1/14 (7.14%)  0/19 (0.00%)  0/6 (0.00%) 
Dental caries  1  0/14 (0.00%)  1/19 (5.26%)  0/6 (0.00%) 
Diarrhoea  1  0/14 (0.00%)  1/19 (5.26%)  0/6 (0.00%) 
Dry mouth  1  0/14 (0.00%)  1/19 (5.26%)  0/6 (0.00%) 
Dyspepsia  1  0/14 (0.00%)  1/19 (5.26%)  0/6 (0.00%) 
Flatulence  1  1/14 (7.14%)  0/19 (0.00%)  0/6 (0.00%) 
Gastrooesophageal reflux disease  1  1/14 (7.14%)  2/19 (10.53%)  1/6 (16.67%) 
Haematochezia  1  2/14 (14.29%)  0/19 (0.00%)  0/6 (0.00%) 
Irritable bowel syndrome  1  2/14 (14.29%)  1/19 (5.26%)  0/6 (0.00%) 
Nausea  1  1/14 (7.14%)  4/19 (21.05%)  0/6 (0.00%) 
Tooth disorder  1  1/14 (7.14%)  0/19 (0.00%)  0/6 (0.00%) 
Toothache  1  1/14 (7.14%)  0/19 (0.00%)  0/6 (0.00%) 
Vomiting  1  2/14 (14.29%)  2/19 (10.53%)  0/6 (0.00%) 
General disorders       
Chest discomfort  1  1/14 (7.14%)  1/19 (5.26%)  0/6 (0.00%) 
Chills  1  0/14 (0.00%)  1/19 (5.26%)  0/6 (0.00%) 
Fatigue  1  5/14 (35.71%)  2/19 (10.53%)  0/6 (0.00%) 
Feeling cold  1  0/14 (0.00%)  1/19 (5.26%)  0/6 (0.00%) 
Influenza like illness  1  2/14 (14.29%)  9/19 (47.37%)  0/6 (0.00%) 
Injection site discolouration  1  0/14 (0.00%)  4/19 (21.05%)  0/6 (0.00%) 
Injection site discomfort  1  0/14 (0.00%)  5/19 (26.32%)  0/6 (0.00%) 
Injection site erythema  1  0/14 (0.00%)  8/19 (42.11%)  0/6 (0.00%) 
Injection site haematoma  1  0/14 (0.00%)  4/19 (21.05%)  0/6 (0.00%) 
Injection site induration  1  0/14 (0.00%)  3/19 (15.79%)  0/6 (0.00%) 
Injection site inflammation  1  0/14 (0.00%)  1/19 (5.26%)  0/6 (0.00%) 
Injection site pain  1  0/14 (0.00%)  9/19 (47.37%)  0/6 (0.00%) 
Injection site pallor  1  0/14 (0.00%)  2/19 (10.53%)  0/6 (0.00%) 
Injection site pruritus  1  0/14 (0.00%)  3/19 (15.79%)  0/6 (0.00%) 
Injection site reaction  1  8/14 (57.14%)  10/19 (52.63%)  0/6 (0.00%) 
Injection site swelling  1  0/14 (0.00%)  2/19 (10.53%)  0/6 (0.00%) 
Malaise  1  0/14 (0.00%)  1/19 (5.26%)  0/6 (0.00%) 
Non-cardiac chest pain  1  0/14 (0.00%)  1/19 (5.26%)  0/6 (0.00%) 
Pain  1  0/14 (0.00%)  2/19 (10.53%)  0/6 (0.00%) 
Pyrexia  1  0/14 (0.00%)  2/19 (10.53%)  0/6 (0.00%) 
Hepatobiliary disorders       
Hepatic fibrosis  1  0/14 (0.00%)  1/19 (5.26%)  0/6 (0.00%) 
Hepatic pain  1  0/14 (0.00%)  1/19 (5.26%)  0/6 (0.00%) 
Hepatic steatosis  1  0/14 (0.00%)  4/19 (21.05%)  0/6 (0.00%) 
Liver disorder  1  0/14 (0.00%)  1/19 (5.26%)  0/6 (0.00%) 
Immune system disorders       
Allergy to arthropod bite  1  1/14 (7.14%)  0/19 (0.00%)  0/6 (0.00%) 
Infections and infestations       
Fungal skin infection  1  0/14 (0.00%)  1/19 (5.26%)  0/6 (0.00%) 
Influenza  1  3/14 (21.43%)  4/19 (21.05%)  0/6 (0.00%) 
Nasopharyngitis  1  5/14 (35.71%)  7/19 (36.84%)  1/6 (16.67%) 
Pertussis  1  1/14 (7.14%)  0/19 (0.00%)  0/6 (0.00%) 
Pharyngitis  1  1/14 (7.14%)  2/19 (10.53%)  0/6 (0.00%) 
Pneumonia  1  1/14 (7.14%)  0/19 (0.00%)  0/6 (0.00%) 
Sinusitis  1  1/14 (7.14%)  3/19 (15.79%)  0/6 (0.00%) 
Urinary tract infection  1  1/14 (7.14%)  2/19 (10.53%)  0/6 (0.00%) 
Injury, poisoning and procedural complications       
Arthropod bite  1  1/14 (7.14%)  0/19 (0.00%)  0/6 (0.00%) 
Joint injury  1  1/14 (7.14%)  0/19 (0.00%)  0/6 (0.00%) 
Post procedural complication  1  2/14 (14.29%)  0/19 (0.00%)  0/6 (0.00%) 
Post-traumatic pain  1  0/14 (0.00%)  1/19 (5.26%)  0/6 (0.00%) 
Road traffic accident  1  1/14 (7.14%)  1/19 (5.26%)  0/6 (0.00%) 
Investigations       
Alanine aminotransferase increased  1  0/14 (0.00%)  4/19 (21.05%)  2/6 (33.33%) 
Aspartate aminotransferase increased  1  0/14 (0.00%)  4/19 (21.05%)  1/6 (16.67%) 
Blood creatine phosphokinase increased  1  0/14 (0.00%)  1/19 (5.26%)  0/6 (0.00%) 
C-reactive protein increased  1  0/14 (0.00%)  0/19 (0.00%)  1/6 (16.67%) 
Gamma-glutamyltransferase increased  1  0/14 (0.00%)  2/19 (10.53%)  1/6 (16.67%) 
Hepatic enzyme increased  1  0/14 (0.00%)  1/19 (5.26%)  0/6 (0.00%) 
Protein urine present  1  0/14 (0.00%)  1/19 (5.26%)  0/6 (0.00%) 
Urine analysis abnormal  1  0/14 (0.00%)  1/19 (5.26%)  0/6 (0.00%) 
Metabolism and nutrition disorders       
Hyperglycaemia  1  0/14 (0.00%)  1/19 (5.26%)  0/6 (0.00%) 
Musculoskeletal and connective tissue disorders       
Arthralgia  1  1/14 (7.14%)  0/19 (0.00%)  0/6 (0.00%) 
Back pain  1  0/14 (0.00%)  2/19 (10.53%)  1/6 (16.67%) 
Bursitis  1  0/14 (0.00%)  1/19 (5.26%)  0/6 (0.00%) 
Groin pain  1  0/14 (0.00%)  1/19 (5.26%)  0/6 (0.00%) 
Musculoskeletal chest pain  1  0/14 (0.00%)  2/19 (10.53%)  0/6 (0.00%) 
Musculoskeletal stiffness  1  0/14 (0.00%)  2/19 (10.53%)  0/6 (0.00%) 
Myalgia  1  1/14 (7.14%)  2/19 (10.53%)  0/6 (0.00%) 
Neck pain  1  0/14 (0.00%)  1/19 (5.26%)  0/6 (0.00%) 
Pain in extremity  1  1/14 (7.14%)  2/19 (10.53%)  0/6 (0.00%) 
Synovial cyst  1  0/14 (0.00%)  1/19 (5.26%)  0/6 (0.00%) 
Nervous system disorders       
Carotid artery stenosis  1  1/14 (7.14%)  0/19 (0.00%)  0/6 (0.00%) 
Disturbance in attention  1  1/14 (7.14%)  0/19 (0.00%)  0/6 (0.00%) 
Dizziness  1  1/14 (7.14%)  0/19 (0.00%)  0/6 (0.00%) 
Dizziness postural  1  1/14 (7.14%)  0/19 (0.00%)  0/6 (0.00%) 
Head discomfort  1  0/14 (0.00%)  1/19 (5.26%)  0/6 (0.00%) 
Headache  1  7/14 (50.00%)  5/19 (26.32%)  0/6 (0.00%) 
Hypoaesthesia  1  1/14 (7.14%)  0/19 (0.00%)  0/6 (0.00%) 
Migraine  1  1/14 (7.14%)  0/19 (0.00%)  0/6 (0.00%) 
Paraesthesia  1  1/14 (7.14%)  0/19 (0.00%)  0/6 (0.00%) 
Psychiatric disorders       
Affect lability  1  1/14 (7.14%)  0/19 (0.00%)  0/6 (0.00%) 
Depressed mood  1  0/14 (0.00%)  1/19 (5.26%)  0/6 (0.00%) 
Insomnia  1  0/14 (0.00%)  1/19 (5.26%)  0/6 (0.00%) 
Listless  1  0/14 (0.00%)  1/19 (5.26%)  0/6 (0.00%) 
Renal and urinary disorders       
Chromaturia  1  1/14 (7.14%)  0/19 (0.00%)  0/6 (0.00%) 
Microalbuminuria  1  0/14 (0.00%)  1/19 (5.26%)  0/6 (0.00%) 
Urine abnormality  1  1/14 (7.14%)  0/19 (0.00%)  0/6 (0.00%) 
Reproductive system and breast disorders       
Prostatomegaly  1  1/14 (7.14%)  0/19 (0.00%)  0/6 (0.00%) 
Respiratory, thoracic and mediastinal disorders       
Cough  1  1/14 (7.14%)  2/19 (10.53%)  0/6 (0.00%) 
Diaphragmatic disorder  1  1/14 (7.14%)  0/19 (0.00%)  0/6 (0.00%) 
Dysphonia  1  0/14 (0.00%)  1/19 (5.26%)  0/6 (0.00%) 
Dyspnoea  1  0/14 (0.00%)  1/19 (5.26%)  0/6 (0.00%) 
Epistaxis  1  1/14 (7.14%)  0/19 (0.00%)  0/6 (0.00%) 
Hiccups  1  0/14 (0.00%)  1/19 (5.26%)  0/6 (0.00%) 
Nasal congestion  1  1/14 (7.14%)  0/19 (0.00%)  0/6 (0.00%) 
Nasal septum disorder  1  0/14 (0.00%)  1/19 (5.26%)  0/6 (0.00%) 
Oropharyngeal pain  1  2/14 (14.29%)  0/19 (0.00%)  0/6 (0.00%) 
Skin and subcutaneous tissue disorders       
Alopecia  1  1/14 (7.14%)  0/19 (0.00%)  0/6 (0.00%) 
Eczema  1  0/14 (0.00%)  0/19 (0.00%)  1/6 (16.67%) 
Erythema  1  0/14 (0.00%)  1/19 (5.26%)  0/6 (0.00%) 
Increased tendency to bruise  1  0/14 (0.00%)  1/19 (5.26%)  0/6 (0.00%) 
Onychoclasis  1  0/14 (0.00%)  1/19 (5.26%)  0/6 (0.00%) 
Vascular disorders       
Hot flush  1  1/14 (7.14%)  0/19 (0.00%)  0/6 (0.00%) 
Hypertension  1  0/14 (0.00%)  1/19 (5.26%)  0/6 (0.00%) 
Subclavian artery stenosis  1  1/14 (7.14%)  0/19 (0.00%)  0/6 (0.00%) 
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA 13.0
The limitations to this study are 1) post-hoc amendments that caused early termination of cohorts and limited the accomplishment of study objectives and 2) post-hoc amendments which added cohorts into the study that were not previously specified.
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.
PI gives Sponsor a draft 90 days before publication. Sponsor has the right to demand that confidential information be removed, and can defer publication another 180 days upon notifying PI that it will file a patent application on inventions contained in the draft.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Christopher Bryant, Ph.D.
Organization: Kastle Therapeutics, LLC
Phone: 8152633913
EMail: cbryant@kastletx.com
Layout table for additonal information
Responsible Party: Kastle Therapeutics, LLC
ClinicalTrials.gov Identifier: NCT00362180     History of Changes
Other Study ID Numbers: 301012-CS10
2005-005783-90 ( EudraCT Number )
First Submitted: August 7, 2006
First Posted: August 9, 2006
Results First Submitted: February 25, 2013
Results First Posted: June 24, 2015
Last Update Posted: October 21, 2016