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Trial record 4 of 19 for:    MIPOMERSEN

A Study of the Safety and Efficacy of Two Different Regimens of Mipomersen in Patients With Familial Hypercholesterolemia and Inadequately Controlled Low-Density Lipoprotein Cholesterol (FOCUS FH)

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: NCT01475825
Recruitment Status : Completed
First Posted : November 21, 2011
Results First Posted : March 14, 2019
Last Update Posted : March 26, 2019
Sponsor:
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
Heterozygous Familial
Interventions Drug: mipomersen sodium 200 mg
Drug: Placebo
Drug: mipomersen sodium 70 mg
Enrollment 309
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Regimen A: Mipomersen, 200 mg, Once Weekly Regimen A: Placebo, Once Weekly Regimen B: Mipomersen, 70 mg, Thrice Weekly Regimen B: Placebo, Thrice Weekly
Hide Arm/Group Description Participants received once weekly subcutaneous (SC) injections of mipomersen sodium 200 milligrams (mg) during the 60-week Blinded Treatment Period, continued the dosing regimen during the 26-week Open-Label Continuation Period, and then entered the 24-week Post-Treatment Period. Participants received once weekly SC injections of Placebo during the 60-week Blinded Treatment Period, continued the dosing regimen during the 26-week Open-Label Continuation Period, and then entered the 24-week Post-Treatment Period. Participants received thrice weekly SC injections of mipomersen sodium 70 mg during the 60-week Blinded Treatment Period, continued the dosing regimen during the 26-week Open-Label Continuation Period, and then entered the 24-week Post-Treatment Period. Participants received thrice weekly SC injections of placebo during the 60-week Blinded Treatment Period, continued the dosing regimen during the 26-week Open-Label Continuation Period, and then entered the 24-week Post-Treatment Period.
Period Title: Overall Study
Started 104 51 102 52
Randomized and Treated 104 51 102 52
Completed Blinded Treatment Period 60 42 59 40
Entered Open-Label Continuation 45 31 44 29
Completed Open-Label Continuation 37 19 38 22
Completed 37 19 38 22
Not Completed 67 32 64 30
Reason Not Completed
Adverse Event             41             15             27             11
Death             1             0             1             0
Lack of Efficacy             0             0             1             0
Lost to Follow-up             0             0             3             1
Non-compliance with Study Drug             1             2             4             0
Other: Not available             0             0             2             0
Protocol Violation             1             0             0             0
Physician Decision             0             1             0             0
Withdrawal by Subject             8             3             11             7
Did not enter OLC             15             11             15             11
Arm/Group Title Regimen A: Mipomersen, 200 mg, Once Weekly Regimen A: Placebo, Once Weekly Regimen B: Mipomersen, 70 mg, Thrice Weekly Regimen B: Placebo, Thrice Weekly Total
Hide Arm/Group Description Participants received once weekly SC injections of mipomersen sodium 200 mg during the 60-week Blinded Treatment Period, continued the dosing regimen during the 26-week Open-Label Continuation Period, and then entered the 24-week Post-Treatment Period. Participants received once weekly SC injections of Placebo during the 60-week Blinded Treatment Period, continued the dosing regimen during the 26-week Open-Label Continuation Period, and then entered the 24-week Post-Treatment Period. Participants received thrice weekly SC injections of mipomersen sodium 70 mg during the 60-week Blinded Treatment Period, continued the dosing regimen during the 26-week Open-Label Continuation Period, and then entered the 24-week Post-Treatment Period. Participants received thrice weekly SC injections of placebo during the 60-week Blinded Treatment Period, continued the dosing regimen during the 26-week Open-Label Continuation Period, and then entered the 24-week Post-Treatment Period. Total of all reporting groups
Overall Number of Baseline Participants 104 51 102 52 309
Hide Baseline Analysis Population Description
The full analysis set included all randomized participants who took at least 1 dose of study drug (mipomersen or placebo), had a valid baseline low-density lipoprotein cholesterol (LDL-C) measurement, and had at least 1 post-baseline LDL-C measurement.
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 104 participants 51 participants 102 participants 52 participants 309 participants
56.36  (9.766) 55.49  (10.481) 53.15  (11.918) 54.38  (9.939) 54.85  (10.53)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 104 participants 51 participants 102 participants 52 participants 309 participants
Female
58
  55.8%
29
  56.9%
54
  52.9%
27
  51.9%
168
  54.4%
Male
46
  44.2%
22
  43.1%
48
  47.1%
25
  48.1%
141
  45.6%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 104 participants 51 participants 102 participants 52 participants 309 participants
Hispanic or Latino
4
   3.8%
4
   7.8%
4
   3.9%
6
  11.5%
18
   5.8%
Not Hispanic or Latino
99
  95.2%
45
  88.2%
95
  93.1%
46
  88.5%
285
  92.2%
Unknown or Not Reported
1
   1.0%
2
   3.9%
3
   2.9%
0
   0.0%
6
   1.9%
LDL-C Baseline Values   [1] 
Mean (Standard Deviation)
Unit of measure:  mg/dL
Cohort 1 Number Analyzed 67 participants 34 participants 66 participants 33 participants 200 participants
262  (103.1) 255  (75.0) 274  (75.1) 263  (82.9) NA [2]   (NA)
Cohort 2 Number Analyzed 37 participants 17 participants 36 participants 19 participants 109 participants
177  (20.8) 179  (25.6) 178  (17.6) 169  (26.6) NA [2]   (NA)
[1]
Measure Analysis Population Description: The full analysis set was analyzed in two cohorts, shown below.
[2]
The total baseline values were not originally calculated, and data were no longer available for further analysis
Apolipoprotein B Baseline Values   [1] 
Mean (Standard Deviation)
Unit of measure:  mg/dL
Cohort 1 Number Analyzed 67 participants 34 participants 66 participants 33 participants 200 participants
172  (52.1) 170  (35.0) 182  (37.9) 174  (45.2) NA [2]   (NA)
Cohort 2 Number Analyzed 37 participants 17 participants 36 participants 19 participants 109 participants
131  (17.3) 134  (17.0) 135  (16.3) 128  (23.4) NA [2]   (NA)
[1]
Measure Analysis Population Description: The full analysis set was analyzed in two cohorts, shown below.
[2]
The total baseline values were not originally calculated, and data were no longer available for further analysis
Lipoprotein (a) Baseline Values   [1] 
Mean (Standard Deviation)
Unit of measure:  mg/dL
Cohort 1 Number Analyzed 67 participants 34 participants 66 participants 33 participants 200 participants
43  (39.5) 50  (45.6) 38  (40.3) 51  (50.8) NA [2]   (NA)
Cohort 2 Number Analyzed 37 participants 17 participants 36 participants 19 participants 109 participants
52  (58.4) 48  (65.6) 59  (49.3) 27  (28.2) NA [2]   (NA)
[1]
Measure Analysis Population Description: The full analysis set was analyzed in two cohorts, shown below.
[2]
The total baseline values were not originally calculated, and data were no longer available for further analysis
1.Primary Outcome
Title Percent Change From Baseline To Primary Endpoint Visit (PET) In LDL-C In Cohort 1
Hide Description The percent change from baseline to PET in LDL-C was measured in Cohort 1, which included participants with severe heterozygous familial hypercholesterolemia (HeFH). Severe HeFH was defined as LDL-C levels ≥200 mg/deciliter (dL) plus the presence of coronary heart disease (CHD)/risk equivalents or LDL-C levels ≥300 mg/dL regardless of the presence of CHD/risk equivalents.
Time Frame Baseline and Week 61
Hide Outcome Measure Data
Hide Analysis Population Description
The full analysis set for Cohort 1 included all randomized participants who took at least 1 dose of study drug in Cohort 1 (mipomersen or placebo), had a valid baseline LDL-C measurement, and had at least 1 post-baseline LDL-C measurement.
Arm/Group Title Regimen A: Mipomersen, 200 mg, Once Weekly Regimen A: Placebo, Once Weekly Regimen B: Mipomersen, 70 mg, Thrice Weekly Regimen B: Placebo, Thrice Weekly
Hide Arm/Group Description:
Participants received once weekly SC injections of mipomersen sodium 200 mg during the 60-week Blinded Treatment Period, continued the dosing regimen during the 26-week Open-Label Continuation Period, and then entered the 24-week Post-Treatment Period.
Participants received once weekly SC injections of Placebo during the 60-week Blinded Treatment Period, continued the dosing regimen during the 26-week Open-Label Continuation Period, and then entered the 24-week Post-Treatment Period.
Participants received thrice weekly SC injections of mipomersen sodium 70 mg during the 60-week Blinded Treatment Period, continued the dosing regimen during the 26-week Open-Label Continuation Period, and then entered the 24-week Post-Treatment Period.
Participants received thrice weekly SC injections of placebo during the 60-week Blinded Treatment Period, continued the dosing regimen during the 26-week Open-Label Continuation Period, and then entered the 24-week Post-Treatment Period.
Overall Number of Participants Analyzed 67 34 66 33
Least Squares Mean (Standard Error)
Unit of Measure: Percent
-27.17  (5.653) -6.77  (6.749) -22.96  (5.362) -10.62  (5.765)
2.Secondary Outcome
Title Percent Change From Baseline To PET In LDL-C In Cohort 2
Hide Description The percent change from baseline to PET in LDL-C was measured in Cohort 2, which included participants with HeFH with LDL-C levels ≥160 mg/dL and <200 mg/dL, plus the presence of CHD/risk equivalents.
Time Frame Baseline, PET (up to 60 weeks)
Hide Outcome Measure Data
Hide Analysis Population Description
The full analysis set included all randomized participants who took at least 1 dose of study drug (mipomersen or placebo), had a valid baseline LDL-C measurement, and had at least 1 post-baseline LDL-C measurement.
Arm/Group Title Regimen A: Mipomersen, 200 mg, Once Weekly Regimen A: Placebo, Once Weekly Regimen B: Mipomersen, 70 mg, Thrice Weekly Regimen B: Placebo, Thrice Weekly
Hide Arm/Group Description:
Participants received once weekly SC injections of mipomersen sodium 200 mg during the 60-week Blinded Treatment Period, continued the dosing regimen during the 26-week Open-Label Continuation Period, and then entered the 24-week Post-Treatment Period.
Participants received once weekly SC injections of Placebo during the 60-week Blinded Treatment Period, continued the dosing regimen during the 26-week Open-Label Continuation Period, and then entered the 24-week Post-Treatment Period.
Participants received thrice weekly SC injections of mipomersen sodium 70 mg during the 60-week Blinded Treatment Period, continued the dosing regimen during the 26-week Open-Label Continuation Period, and then entered the 24-week Post-Treatment Period.
Participants received thrice weekly SC injections of placebo during the 60-week Blinded Treatment Period, continued the dosing regimen during the 26-week Open-Label Continuation Period, and then entered the 24-week Post-Treatment Period.
Overall Number of Participants Analyzed 37 17 36 19
Least Squares Mean (Standard Error)
Unit of Measure: Percent
-31.20  (8.927) -9.25  (10.621) -43.60  (8.342) -13.57  (9.066)
3.Secondary Outcome
Title Percent Change From Baseline To PET In Apolipoprotein B (Apo B) In Cohort 1
Hide Description The percent change from baseline to PET in Apo B was measured in participants in Cohort 1 with HeFH during the Blinded Treatment Period.
Time Frame Baseline and Week 61
Hide Outcome Measure Data
Hide Analysis Population Description
The full analysis set included all randomized participants who took at least 1 dose of study drug (mipomersen or placebo), had a valid baseline LDL-C measurement, and had at least 1 post-baseline LDL-C measurement.
Arm/Group Title Regimen A: Mipomersen, 200 mg, Once Weekly Regimen A: Placebo, Once Weekly Regimen B: Mipomersen, 70 mg, Thrice Weekly Regimen B: Placebo, Thrice Weekly
Hide Arm/Group Description:
Participants received once weekly SC injections of mipomersen sodium 200 mg during the 60-week Blinded Treatment Period, continued the dosing regimen during the 26-week Open-Label Continuation Period, and then entered the 24-week Post-Treatment Period.
Participants received once weekly SC injections of Placebo during the 60-week Blinded Treatment Period, continued the dosing regimen during the 26-week Open-Label Continuation Period, and then entered the 24-week Post-Treatment Period.
Participants received thrice weekly SC injections of mipomersen sodium 70 mg during the 60-week Blinded Treatment Period, continued the dosing regimen during the 26-week Open-Label Continuation Period, and then entered the 24-week Post-Treatment Period.
Participants received thrice weekly SC injections of placebo during the 60-week Blinded Treatment Period, continued the dosing regimen during the 26-week Open-Label Continuation Period, and then entered the 24-week Post-Treatment Period.
Overall Number of Participants Analyzed 67 34 66 33
Least Squares Mean (Standard Error)
Unit of Measure: Percent
-24.14  (5.058) -2.83  (6.115) -21.43  (4.861) -7.28  (5.309)
4.Secondary Outcome
Title Percent Change From Baseline To PET In Apolipoprotein B (Apo B) In Cohort 2
Hide Description The percent change from baseline to PET in Apo B was measured in participants in Cohort 2 with HeFH during the Blinded Treatment Period.
Time Frame Baseline and Week 61
Hide Outcome Measure Data
Hide Analysis Population Description
The full analysis set included all randomized participants who took at least 1 dose of study drug (mipomersen or placebo), had a valid baseline LDL-C measurement, and had at least 1 post-baseline LDL-C measurement.
Arm/Group Title Regimen A: Mipomersen, 200 mg, Once Weekly Regimen A: Placebo, Once Weekly Regimen B: Mipomersen, 70 mg, Thrice Weekly Regimen B: Placebo, Thrice Weekly
Hide Arm/Group Description:
Participants received once weekly SC injections of mipomersen sodium 200 mg during the 60-week Blinded Treatment Period, continued the dosing regimen during the 26-week Open-Label Continuation Period, and then entered the 24-week Post-Treatment Period.
Participants received once weekly SC injections of Placebo during the 60-week Blinded Treatment Period, continued the dosing regimen during the 26-week Open-Label Continuation Period, and then entered the 24-week Post-Treatment Period.
Participants received thrice weekly SC injections of mipomersen sodium 70 mg during the 60-week Blinded Treatment Period, continued the dosing regimen during the 26-week Open-Label Continuation Period, and then entered the 24-week Post-Treatment Period.
Participants received thrice weekly SC injections of placebo during the 60-week Blinded Treatment Period, continued the dosing regimen during the 26-week Open-Label Continuation Period, and then entered the 24-week Post-Treatment Period.
Overall Number of Participants Analyzed 37 17 36 19
Least Squares Mean (Standard Error)
Unit of Measure: Percent
-30.76  (7.309) -6.67  (8.758) -36.34  (7.039) -3.77  (8.109)
5.Secondary Outcome
Title Percent Change From Baseline To PET in Lipoprotein (a) In Cohort 1
Hide Description The percent change from baseline to PET in Lipoprotein A1 was measured in participants in Cohort 1 with HeFH during the Blinded Treatment Period.
Time Frame Baseline and Week 61
Hide Outcome Measure Data
Hide Analysis Population Description
The full analysis set included all randomized participants who took at least 1 dose of study drug (mipomersen or placebo), had a valid baseline LDL-C measurement, and had at least 1 post-baseline LDL-C measurement.
Arm/Group Title Regimen A: Mipomersen, 200 mg, Once Weekly Regimen A: Placebo, Once Weekly Regimen B: Mipomersen, 70 mg, Thrice Weekly Regimen B: Placebo, Thrice Weekly
Hide Arm/Group Description:
Participants received once weekly SC injections of mipomersen sodium 200 mg during the 60-week Blinded Treatment Period, continued the dosing regimen during the 26-week Open-Label Continuation Period, and then entered the 24-week Post-Treatment Period.
Participants received once weekly SC injections of Placebo during the 60-week Blinded Treatment Period, continued the dosing regimen during the 26-week Open-Label Continuation Period, and then entered the 24-week Post-Treatment Period.
Participants received thrice weekly SC injections of mipomersen sodium 70 mg during the 60-week Blinded Treatment Period, continued the dosing regimen during the 26-week Open-Label Continuation Period, and then entered the 24-week Post-Treatment Period.
Participants received thrice weekly SC injections of placebo during the 60-week Blinded Treatment Period, continued the dosing regimen during the 26-week Open-Label Continuation Period, and then entered the 24-week Post-Treatment Period.
Overall Number of Participants Analyzed 67 34 66 33
Least Squares Mean (Standard Error)
Unit of Measure: Percent
-18.84  (7.870) -16.85  (9.959) -27.18  (5.497) -10.08  (5.992)
6.Secondary Outcome
Title Percent Change From Baseline To PET in Lipoprotein (a) In Cohort 2
Hide Description The percent change from baseline to PET in Lipoprotein A1 was measured in participants in Cohort 2 with HeFH during the Blinded Treatment Period.
Time Frame Baseline and Week 61
Hide Outcome Measure Data
Hide Analysis Population Description
The full analysis set included all randomized participants who took at least 1 dose of study drug (mipomersen or placebo), had a valid baseline LDL-C measurement, and had at least 1 post-baseline LDL-C measurement.
Arm/Group Title Regimen A: Mipomersen, 200 mg, Once Weekly Regimen A: Placebo, Once Weekly Regimen B: Mipomersen, 70 mg, Thrice Weekly Regimen B: Placebo, Thrice Weekly
Hide Arm/Group Description:
Participants received once weekly SC injections of mipomersen sodium 200 mg during the 60-week Blinded Treatment Period, continued the dosing regimen during the 26-week Open-Label Continuation Period, and then entered the 24-week Post-Treatment Period.
Participants received once weekly SC injections of Placebo during the 60-week Blinded Treatment Period, continued the dosing regimen during the 26-week Open-Label Continuation Period, and then entered the 24-week Post-Treatment Period.
Participants received thrice weekly SC injections of mipomersen sodium 70 mg during the 60-week Blinded Treatment Period, continued the dosing regimen during the 26-week Open-Label Continuation Period, and then entered the 24-week Post-Treatment Period.
Participants received thrice weekly SC injections of placebo during the 60-week Blinded Treatment Period, continued the dosing regimen during the 26-week Open-Label Continuation Period, and then entered the 24-week Post-Treatment Period.
Overall Number of Participants Analyzed 37 17 36 19
Least Squares Mean (Standard Error)
Unit of Measure: Percent
-23.41  (10.002) -11.86  (11.871) -35.56  (11.846) 18.79  (15.271)
Time Frame Adverse events were monitored from the time of the administration of the participant's first dose of study drug (mipomersen or placebo) through the participant’s last visit of the Post-Treatment Period, up to Week 110.
Adverse Event Reporting Description Adverse events were collected for all randomized participants who received at least 1 dose of study drug (mipomersen or placebo).
 
Arm/Group Title Regimen A: Mipomersen, 200 mg, Once Weekly Regimen A: Placebo, Once Weekly Regimen B: Mipomersen, 70 mg, Thrice Weekly Regimen B: Placebo, Thrice Weekly
Hide Arm/Group Description Participants received once weekly SC injections of mipomersen sodium 200 mg during the 60-week Blinded Treatment Period, continued the dosing regimen during the 26-week Open-Label Continuation Period, and then entered the 24-week Post-Treatment Period. Participants received once weekly SC injections of Placebo during the 60-week Blinded Treatment Period, continued the dosing regimen during the 26-week Open-Label Continuation Period, and then entered the 24-week Post-Treatment Period. Participants received thrice weekly SC injections of mipomersen sodium 70 mg during the 60-week Blinded Treatment Period, continued the dosing regimen during the 26-week Open-Label Continuation Period, and then entered the 24-week Post-Treatment Period. Participants received thrice weekly SC injections of placebo during the 60-week Blinded Treatment Period, continued the dosing regimen during the 26-week Open-Label Continuation Period, and then entered the 24-week Post-Treatment Period.
All-Cause Mortality
Regimen A: Mipomersen, 200 mg, Once Weekly Regimen A: Placebo, Once Weekly Regimen B: Mipomersen, 70 mg, Thrice Weekly Regimen B: Placebo, Thrice Weekly
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   1/104 (0.96%)   0/51 (0.00%)   1/102 (0.98%)   0/52 (0.00%) 
Show Serious Adverse Events Hide Serious Adverse Events
Regimen A: Mipomersen, 200 mg, Once Weekly Regimen A: Placebo, Once Weekly Regimen B: Mipomersen, 70 mg, Thrice Weekly Regimen B: Placebo, Thrice Weekly
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   17/104 (16.35%)   13/51 (25.49%)   23/102 (22.55%)   11/52 (21.15%) 
Blood and lymphatic system disorders         
Anaemia  1  1/104 (0.96%)  1/51 (1.96%)  0/102 (0.00%)  0/52 (0.00%) 
Cardiac disorders         
Angina pectoris  1  7/104 (6.73%)  4/51 (7.84%)  0/102 (0.00%)  4/52 (7.69%) 
Angina unstable  1  1/104 (0.96%)  3/51 (5.88%)  4/102 (3.92%)  3/52 (5.77%) 
Acute myocardial infarction  1  0/104 (0.00%)  1/51 (1.96%)  2/102 (1.96%)  2/52 (3.85%) 
Coronary artery disease  1  3/104 (2.88%)  0/51 (0.00%)  0/102 (0.00%)  1/52 (1.92%) 
Myocardial infarction  1  0/104 (0.00%)  0/51 (0.00%)  3/102 (2.94%)  0/52 (0.00%) 
Acute coronary syndrome  1  0/104 (0.00%)  2/51 (3.92%)  0/102 (0.00%)  0/52 (0.00%) 
Atrial fibrillation  1  0/104 (0.00%)  1/51 (1.96%)  1/102 (0.98%)  0/52 (0.00%) 
Tachycardia  1  0/104 (0.00%)  1/51 (1.96%)  1/102 (0.98%)  0/52 (0.00%) 
Atrial flutter  1  0/104 (0.00%)  1/51 (1.96%)  0/102 (0.00%)  0/52 (0.00%) 
Cardiac arrest  1  0/104 (0.00%)  1/51 (1.96%)  0/102 (0.00%)  0/52 (0.00%) 
Cardiac failure congestive  1  1/104 (0.96%)  0/51 (0.00%)  0/102 (0.00%)  0/52 (0.00%) 
Coronary artery stenosis  1  0/104 (0.00%)  0/51 (0.00%)  0/102 (0.00%)  1/52 (1.92%) 
Myocardial ischaemia  1  0/104 (0.00%)  0/51 (0.00%)  1/102 (0.98%)  0/52 (0.00%) 
Supraventricular tachycardia  1  0/104 (0.00%)  0/51 (0.00%)  1/102 (0.98%)  0/52 (0.00%) 
Eye disorders         
Eye haemorrhage  1  0/104 (0.00%)  0/51 (0.00%)  1/102 (0.98%)  0/52 (0.00%) 
Gastrointestinal disorders         
Abdominal hernia  1  0/104 (0.00%)  0/51 (0.00%)  1/102 (0.98%)  0/52 (0.00%) 
Constipation  1  0/104 (0.00%)  0/51 (0.00%)  1/102 (0.98%)  0/52 (0.00%) 
Gastric ulcer  1  0/104 (0.00%)  0/51 (0.00%)  0/102 (0.00%)  1/52 (1.92%) 
Gastritis erosive  1  0/104 (0.00%)  1/51 (1.96%)  0/102 (0.00%)  0/52 (0.00%) 
Large intestine perforation  1  0/104 (0.00%)  0/51 (0.00%)  0/102 (0.00%)  1/52 (1.92%) 
Obstruction gastric  1  0/104 (0.00%)  1/51 (1.96%)  0/102 (0.00%)  0/52 (0.00%) 
Pancreatic cyst  1  0/104 (0.00%)  0/51 (0.00%)  1/102 (0.98%)  0/52 (0.00%) 
General disorders         
Non-cardiac chest pain  1  1/104 (0.96%)  2/51 (3.92%)  2/102 (1.96%)  1/52 (1.92%) 
Chest pain  1  0/104 (0.00%)  0/51 (0.00%)  2/102 (1.96%)  0/52 (0.00%) 
Death  1  2/104 (1.92%)  0/51 (0.00%)  0/102 (0.00%)  0/52 (0.00%) 
Asthenia  1  1/104 (0.96%)  0/51 (0.00%)  0/102 (0.00%)  0/52 (0.00%) 
Drug intolerance  1  0/104 (0.00%)  0/51 (0.00%)  0/102 (0.00%)  1/52 (1.92%) 
Influenza like illness  1  1/104 (0.96%)  0/51 (0.00%)  0/102 (0.00%)  0/52 (0.00%) 
Pyrexia  1  0/104 (0.00%)  1/51 (1.96%)  0/102 (0.00%)  0/52 (0.00%) 
Hepatobiliary disorders         
Cholelithiasis  1  1/104 (0.96%)  0/51 (0.00%)  0/102 (0.00%)  0/52 (0.00%) 
Infections and infestations         
Diverticulitis  1  0/104 (0.00%)  1/51 (1.96%)  1/102 (0.98%)  0/52 (0.00%) 
Cellulitis  1  0/104 (0.00%)  0/51 (0.00%)  1/102 (0.98%)  0/52 (0.00%) 
Device related infection  1  0/104 (0.00%)  0/51 (0.00%)  0/102 (0.00%)  1/52 (1.92%) 
Escherichia pyelonephritis  1  1/104 (0.96%)  0/51 (0.00%)  0/102 (0.00%)  0/52 (0.00%) 
Gastroenteritis  1  0/104 (0.00%)  0/51 (0.00%)  1/102 (0.98%)  0/52 (0.00%) 
Pneumonia  1  0/104 (0.00%)  0/51 (0.00%)  1/102 (0.98%)  0/52 (0.00%) 
Injury, poisoning and procedural complications         
Coronary artery restenosis  1  0/104 (0.00%)  0/51 (0.00%)  1/102 (0.98%)  0/52 (0.00%) 
Post procedural haemorrhage  1  0/104 (0.00%)  0/51 (0.00%)  0/102 (0.00%)  1/52 (1.92%) 
Investigations         
Helicobacter test negative  1  0/104 (0.00%)  0/51 (0.00%)  0/102 (0.00%)  1/52 (1.92%) 
Low density lipoprotein increased  1  0/104 (0.00%)  0/51 (0.00%)  1/102 (0.98%)  0/52 (0.00%) 
Musculoskeletal and connective tissue disorders         
Musculoskeletal pain  1  0/104 (0.00%)  1/51 (1.96%)  1/102 (0.98%)  0/52 (0.00%) 
Arthralgia  1  0/104 (0.00%)  1/51 (1.96%)  0/102 (0.00%)  0/52 (0.00%) 
Back pain  1  0/104 (0.00%)  1/51 (1.96%)  0/102 (0.00%)  0/52 (0.00%) 
Joint contracture  1  0/104 (0.00%)  0/51 (0.00%)  1/102 (0.98%)  0/52 (0.00%) 
Osteoarthritis  1  0/104 (0.00%)  0/51 (0.00%)  1/102 (0.98%)  0/52 (0.00%) 
Spinal osteoarthritis  1  0/104 (0.00%)  1/51 (1.96%)  0/102 (0.00%)  0/52 (0.00%) 
Neoplasms benign, malignant and unspecified (incl cysts and polyps)         
Lymphoma  1  0/104 (0.00%)  1/51 (1.96%)  0/102 (0.00%)  0/52 (0.00%) 
Nervous system disorders         
Syncope  1  0/104 (0.00%)  0/51 (0.00%)  1/102 (0.98%)  1/52 (1.92%) 
Transient ischaemic attack  1  1/104 (0.96%)  0/51 (0.00%)  1/102 (0.98%)  0/52 (0.00%) 
Aphasia  1  0/104 (0.00%)  0/51 (0.00%)  1/102 (0.98%)  0/52 (0.00%) 
Carotid artery stenosis  1  0/104 (0.00%)  0/51 (0.00%)  1/102 (0.98%)  0/52 (0.00%) 
Cerebral infarction  1  0/104 (0.00%)  0/51 (0.00%)  1/102 (0.98%)  0/52 (0.00%) 
Hemiparesis  1  0/104 (0.00%)  0/51 (0.00%)  1/102 (0.98%)  0/52 (0.00%) 
Ischaemic stroke  1  0/104 (0.00%)  0/51 (0.00%)  1/102 (0.98%)  0/52 (0.00%) 
Pregnancy, puerperium and perinatal conditions         
Abortion spontaneous  1  0/104 (0.00%)  1/51 (1.96%)  0/102 (0.00%)  0/52 (0.00%) 
Psychiatric disorders         
Major depression  1  0/104 (0.00%)  0/51 (0.00%)  0/102 (0.00%)  1/52 (1.92%) 
Suicide attempt  1  0/104 (0.00%)  0/51 (0.00%)  0/102 (0.00%)  1/52 (1.92%) 
Renal and urinary disorders         
Haematuria  1  1/104 (0.96%)  1/51 (1.96%)  0/102 (0.00%)  0/52 (0.00%) 
Acute kidney injury  1  1/104 (0.96%)  0/51 (0.00%)  0/102 (0.00%)  0/52 (0.00%) 
Renal cyst  1  1/104 (0.96%)  0/51 (0.00%)  0/102 (0.00%)  0/52 (0.00%) 
Reproductive system and breast disorders         
Benign prostatic hyperplasia  1  1/104 (0.96%)  0/51 (0.00%)  0/102 (0.00%)  0/52 (0.00%) 
Cervical polyp  1  0/104 (0.00%)  0/51 (0.00%)  0/102 (0.00%)  1/52 (1.92%) 
Fibrocystic breast disease  1  1/104 (0.96%)  0/51 (0.00%)  0/102 (0.00%)  0/52 (0.00%) 
Ovarian cyst torsion  1  0/104 (0.00%)  0/51 (0.00%)  1/102 (0.98%)  0/52 (0.00%) 
Respiratory, thoracic and mediastinal disorders         
Asthma  1  1/104 (0.96%)  0/51 (0.00%)  0/102 (0.00%)  0/52 (0.00%) 
Dyspnoea exertional  1  0/104 (0.00%)  0/51 (0.00%)  1/102 (0.98%)  0/52 (0.00%) 
Pleuritic pain  1  0/104 (0.00%)  0/51 (0.00%)  0/102 (0.00%)  1/52 (1.92%) 
Pulmonary embolism  1  1/104 (0.96%)  0/51 (0.00%)  0/102 (0.00%)  0/52 (0.00%) 
Pulmonary oedema  1  0/104 (0.00%)  0/51 (0.00%)  0/102 (0.00%)  1/52 (1.92%) 
Vascular disorders         
Embolism venous  1  0/104 (0.00%)  0/51 (0.00%)  1/102 (0.98%)  0/52 (0.00%) 
Hypertension  1  1/104 (0.96%)  0/51 (0.00%)  0/102 (0.00%)  0/52 (0.00%) 
Hypertensive crisis  1  0/104 (0.00%)  0/51 (0.00%)  0/102 (0.00%)  1/52 (1.92%) 
Peripheral arterial occlusive disease  1  0/104 (0.00%)  1/51 (1.96%)  0/102 (0.00%)  0/52 (0.00%) 
Peripheral artery stenosis  1  1/104 (0.96%)  0/51 (0.00%)  0/102 (0.00%)  0/52 (0.00%) 
1
Term from vocabulary, MedDRA 18.1
Indicates events were collected by systematic assessment
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Regimen A: Mipomersen, 200 mg, Once Weekly Regimen A: Placebo, Once Weekly Regimen B: Mipomersen, 70 mg, Thrice Weekly Regimen B: Placebo, Thrice Weekly
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   82/104 (78.85%)   27/51 (52.94%)   78/102 (76.47%)   33/52 (63.46%) 
Cardiac disorders         
Angina pectoris  1  8/104 (7.69%)  5/51 (9.80%)  4/102 (3.92%)  6/52 (11.54%) 
Palpitations  1  2/104 (1.92%)  0/51 (0.00%)  2/102 (1.96%)  3/52 (5.77%) 
Gastrointestinal disorders         
Nausea  1  9/104 (8.65%)  3/51 (5.88%)  7/102 (6.86%)  2/52 (3.85%) 
Abdominal pain lower  1  0/104 (0.00%)  3/51 (5.88%)  0/102 (0.00%)  1/52 (1.92%) 
General disorders         
Influenza like illness  1  43/104 (41.35%)  10/51 (19.61%)  26/102 (25.49%)  10/52 (19.23%) 
Injection site erythema  1  8/104 (7.69%)  1/51 (1.96%)  13/102 (12.75%)  1/52 (1.92%) 
Injection site pain  1  13/104 (12.50%)  1/51 (1.96%)  7/102 (6.86%)  0/52 (0.00%) 
Injection site swelling  1  4/104 (3.85%)  1/51 (1.96%)  8/102 (7.84%)  1/52 (1.92%) 
Injection site pruritus  1  3/104 (2.88%)  1/51 (1.96%)  7/102 (6.86%)  1/52 (1.92%) 
Hepatobiliary disorders         
Hepatic steatosis  1  10/104 (9.62%)  1/51 (1.96%)  9/102 (8.82%)  1/52 (1.92%) 
Infections and infestations         
Nasopharyngitis  1  7/104 (6.73%)  4/51 (7.84%)  7/102 (6.86%)  7/52 (13.46%) 
Influenza  1  5/104 (4.81%)  3/51 (5.88%)  9/102 (8.82%)  5/52 (9.62%) 
Upper respiratory tract infection  1  2/104 (1.92%)  4/51 (7.84%)  7/102 (6.86%)  2/52 (3.85%) 
Bronchitis  1  5/104 (4.81%)  3/51 (5.88%)  3/102 (2.94%)  3/52 (5.77%) 
Urinary tract infection  1  1/104 (0.96%)  0/51 (0.00%)  7/102 (6.86%)  1/52 (1.92%) 
Investigations         
Alanine aminotransferase increased  1  21/104 (20.19%)  2/51 (3.92%)  18/102 (17.65%)  0/52 (0.00%) 
Aspartate aminotransferase increased  1  17/104 (16.35%)  2/51 (3.92%)  11/102 (10.78%)  1/52 (1.92%) 
C-reactive protein increased  1  4/104 (3.85%)  3/51 (5.88%)  3/102 (2.94%)  3/52 (5.77%) 
Hepatic enzyme increased  1  8/104 (7.69%)  0/51 (0.00%)  4/102 (3.92%)  0/52 (0.00%) 
Blood creatine phosphokinase increased  1  1/104 (0.96%)  1/51 (1.96%)  4/102 (3.92%)  3/52 (5.77%) 
Musculoskeletal and connective tissue disorders         
Myalgia  1  8/104 (7.69%)  0/51 (0.00%)  5/102 (4.90%)  3/52 (5.77%) 
Back pain  1  6/104 (5.77%)  2/51 (3.92%)  3/102 (2.94%)  4/52 (7.69%) 
Musculoskeletal pain  1  4/104 (3.85%)  1/51 (1.96%)  3/102 (2.94%)  3/52 (5.77%) 
Pain in extremity  1  5/104 (4.81%)  3/51 (5.88%)  3/102 (2.94%)  0/52 (0.00%) 
Nervous system disorders         
Headache  1  8/104 (7.69%)  2/51 (3.92%)  9/102 (8.82%)  4/52 (7.69%) 
Dizziness  1  8/104 (7.69%)  1/51 (1.96%)  3/102 (2.94%)  1/52 (1.92%) 
Respiratory, thoracic and mediastinal disorders         
Cough  1  5/104 (4.81%)  0/51 (0.00%)  5/102 (4.90%)  3/52 (5.77%) 
Skin and subcutaneous tissue disorders         
Pruritus  1  0/104 (0.00%)  0/51 (0.00%)  2/102 (1.96%)  3/52 (5.77%) 
Vascular disorders         
Hypertension  1  3/104 (2.88%)  3/51 (5.88%)  4/102 (3.92%)  5/52 (9.62%) 
1
Term from vocabulary, MedDRA 18.1
Indicates events were collected by systematic assessment
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.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Medical Information
Organization: Kastle Therapeutics, LLC
Phone: 1-800-745-4447
EMail: medinfo@genzyme.com
Layout table for additonal information
Responsible Party: Kastle Therapeutics, LLC
ClinicalTrials.gov Identifier: NCT01475825     History of Changes
Other Study ID Numbers: MIPO3801011
2011-001480-42 ( EudraCT Number )
EFC12875 ( Other Identifier: Sanofi )
First Submitted: November 17, 2011
First Posted: November 21, 2011
Results First Submitted: November 13, 2018
Results First Posted: March 14, 2019
Last Update Posted: March 26, 2019