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Trial record 2 of 19 for:    MIPOMERSEN

Open Label Extension of ISIS 301012 (Mipomersen) to Treat Familial Hypercholesterolemia

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: NCT00477594
Recruitment Status : Completed
First Posted : May 24, 2007
Results First Posted : April 5, 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: None (Open Label);   Primary Purpose: Treatment
Conditions Lipid Metabolism, Inborn Errors
Hypercholesterolemia, Autosomal Dominant
Hyperlipidemias
Metabolic Diseases
Hyperlipoproteinemia Type II
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Infant, Newborn, Diseases
Metabolic Disorder
Congenital Abnormalities
Hypercholesterolemia
Hyperlipoproteinemias
Dyslipidemias
Lipid Metabolism Disorders
Intervention Drug: mipomersen sodium
Enrollment 21
Recruitment Details Patients who completed dosing in study 301012-CS8 (NCT00280995) or 301012-CS9 (NCT00281008) at a site in the U.S. with an acceptable safety profile, per Investigator judgment, were eligible to enroll in this study.
Pre-assignment Details Patients who participated in Cohorts A, B, or C in study 301012-CS9 were randomized in a 1:1 ratio to mipomersen 200 mg once a week (QW) or 200 mg mipomersen every other week (QOW). Patients who participated in study 301012-CS8 or Cohort D of study 301012-CS9 received 200 mg mipomersen QW.
Arm/Group Title Mipomersen 200 mg Per Week Mipomersen 200 mg Every Other Week
Hide Arm/Group Description Participants received 200 mg mipomersen once a week by subcutaneous injection, for up to 3 years. Participants received 200 mg mipomersen every other week by subcutaneous injection, for up to 3 years. Participants could receive mipomersen 200 mg once a week at the Investigator’s discretion after the first 52 weeks of the treatment period.
Period Title: Year 1 and Year 2
Started 16 5
Completed 7 1
Not Completed 9 4
Reason Not Completed
Adverse Event             4             2
Physician Decision             2             1
Withdrawal by Subject             2             1
Other             1             0
Period Title: Year 3
Started 4 0
Completed 2 0
Not Completed 2 0
Reason Not Completed
Adverse Event             1             0
Withdrawal by Subject             1             0
Period Title: Follow-up Period
Started 16 5
Completed 13 4
Not Completed 3 1
Reason Not Completed
Withdrawal by Subject             2             1
Other             1             0
Arm/Group Title Mipomersen 200 mg Per Week Mipomersen 200 mg Every Other Week Total
Hide Arm/Group Description Participants received 200 mg mipomersen once a week by subcutaneous injection, for up to 3 years. Participants received 200 mg mipomersen every other week by subcutaneous injection, for up to 3 years. Participants could receive mipomersen 200 mg once a week at the Investigator’s discretion after the first 52 weeks of the treatment period. Total of all reporting groups
Overall Number of Baseline Participants 16 5 21
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 16 participants 5 participants 21 participants
47.1  (11.6) 54.6  (15.7) 48.9  (12.7)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 16 participants 5 participants 21 participants
Female
4
  25.0%
2
  40.0%
6
  28.6%
Male
12
  75.0%
3
  60.0%
15
  71.4%
Race/Ethnicity, Customized  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 16 participants 5 participants 21 participants
Hispanic or Latino 1 0 1
Not Hispanic or Latino 15 5 20
Race/Ethnicity, Customized  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 16 participants 5 participants 21 participants
White 14 5 19
Other race 1 0 1
Multiple 1 0 1
Body Mass Index (BMI)  
Mean (Standard Deviation)
Unit of measure:  Kg/m^2
Number Analyzed 16 participants 5 participants 21 participants
28.9  (5.60) 27.4  (4.81) 28.6  (5.34)
Metabolic syndrome   [1] 
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 16 participants 5 participants 21 participants
No 8 3 11
Yes 8 2 10
[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 16 participants 5 participants 21 participants
Current 1 0 1
Non-current 5 2 7
Never 10 3 13
Alcohol use  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 16 participants 5 participants 21 participants
Current 13 3 16
Non-current 1 0 1
Never 2 2 4
1.Primary Outcome
Title Percent Change From Baseline in Low-density Lipoprotein Cholesterol (LDL-C)
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. For patients who were on placebo in the index study or who took their last dose of mipomersen ≥6 months prior to first dose in this study, Baseline is defined as the last measurement prior to first dose in this study. For participants who took their last dose of mipomersen less than 6 months before starting this study, Baseline is defined as the last measurement taken prior to receiving a first dose in the index study.
Time Frame Baseline and Weeks 52 and 104
Hide Outcome Measure Data
Hide Analysis Population Description
The Safety Set included all enrolled patients who received at least 1 injection of study drug. "N" indicates the number of participants with available data at the specified time point.
Arm/Group Title Mipomersen 200 mg Per Week Mipomersen 200 mg Every Other Week
Hide Arm/Group Description:
Participants received 200 mg mipomersen once a week by subcutaneous injection, for up to 3 years.
Participants received 200 mg mipomersen every other week by subcutaneous injection, for up to 3 years. Participants could receive mipomersen 200 mg once a week at the Investigator’s discretion after the first 52 weeks of the treatment period.
Overall Number of Participants Analyzed 16 5
Median (Inter-Quartile Range)
Unit of Measure: percentage of baseline
Week 52 [N=13, 4]
-16.2
(-41.8 to -7.8)
-2.6
(-10.2 to 14.8)
Week 104 [N=7, 1]
-32.4
(-38.8 to -16.6)
30.6
(30.6 to 30.6)
Week 104/Early Termination [N=16, 5]
-13.8
(-31.4 to -3.0)
9.5
(3.4 to 11.5)
2.Primary Outcome
Title Low-density Lipoprotein Cholesterol (LDL-C) Over Time
Hide Description 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. For patients who were on placebo in the index study or who took their last dose of mipomersen ≥6 months prior to first dose in this study, Baseline is defined as the last measurement prior to first dose in this study. For participants who took their last dose of mipomersen less than 6 months before starting this study, Baseline is defined as the last measurement taken prior to receiving a first dose in the index study.
Time Frame Baseline and Weeks 52 and 104.
Hide Outcome Measure Data
Hide Analysis Population Description
The Safety Set included all enrolled patients who received at least 1 injection of study drug. "N" indicates the number of participants with available data at the specified time point.
Arm/Group Title Mipomersen 200 mg Per Week Mipomersen 200 mg Every Other Week
Hide Arm/Group Description:
Participants received 200 mg mipomersen once a week by subcutaneous injection, for up to 3 years.
Participants received 200 mg mipomersen every other week by subcutaneous injection, for up to 3 years. Participants could receive mipomersen 200 mg once a week at the Investigator’s discretion after the first 52 weeks of the treatment period.
Overall Number of Participants Analyzed 16 5
Median (Inter-Quartile Range)
Unit of Measure: mg/dL
Baseline [N=16, 5]
177
(134 to 205)
131
(124 to 147)
Week 52 [N=13, 4]
145
(106 to 173)
127
(124 to 139)
Week 104 [N=7, 1]
121
(82 to 156)
162
(162 to 162)
Week 104/Early Termination [N=16, 5}
144
(116 to 213)
161
(146 to 162)
3.Secondary Outcome
Title Percent Change From Baseline in Apolipoprotein B
Hide Description Apolipoprotein B was measured in mg/dL. Samples were taken following an overnight fast. For patients who were on placebo in the index study or who took their last dose of mipomersen ≥6 months prior to first dose in this study, Baseline is defined as the last measurement prior to first dose in this study. For participants who took their last dose of mipomersen less than 6 months before starting this study, Baseline is defined as the last measurement taken prior to receiving a first dose in the index study.
Time Frame Baseline and Weeks 52 and 104
Hide Outcome Measure Data
Hide Analysis Population Description
The Safety Set included all enrolled patients who received at least 1 injection of study drug. "N" indicates the number of participants with available data at the specified time point.
Arm/Group Title Mipomersen 200 mg Per Week Mipomersen 200 mg Every Other Week
Hide Arm/Group Description:
Participants received 200 mg mipomersen once a week by subcutaneous injection, for up to 3 years.
Participants received 200 mg mipomersen every other week by subcutaneous injection, for up to 3 years. Participants could receive mipomersen 200 mg once a week at the Investigator’s discretion after the first 52 weeks of the treatment period.
Overall Number of Participants Analyzed 16 5
Median (Inter-Quartile Range)
Unit of Measure: percentage of baseline
Week 52 [N=13, 4]
-25.9
(-39.1 to -11.0)
-7.3
(-19.7 to 7.7)
Week 104 [N=7, 1]
-27.2
(-39.7 to -22.4)
20.8
(20.8 to 20.8)
Week 104/Early Termination [N=16, 5]
-15.8
(-26.2 to -3.5)
-3.7
(-14.9 to -1.6)
4.Secondary Outcome
Title Apolipoprotein B Over Time
Hide Description For patients who were on placebo in the index study or who took their last dose of mipomersen ≥6 months prior to first dose in this study, Baseline is defined as the last measurement prior to first dose in this study. For participants who took their last dose of mipomersen less than 6 months before starting this study, Baseline is defined as the last measurement taken prior to receiving a first dose in the index study.
Time Frame Baseline and Weeks 52 and 104.
Hide Outcome Measure Data
Hide Analysis Population Description
The Safety Set included all enrolled patients who received at least 1 injection of study drug. "N" indicates the number of participants with available data at the specified time point.
Arm/Group Title Mipomersen 200 mg Per Week Mipomersen 200 mg Every Other Week
Hide Arm/Group Description:
Participants received 200 mg mipomersen once a week by subcutaneous injection, for up to 3 years.
Participants received 200 mg mipomersen every other week by subcutaneous injection, for up to 3 years. Participants could receive mipomersen 200 mg once a week at the Investigator’s discretion after the first 52 weeks of the treatment period.
Overall Number of Participants Analyzed 16 5
Median (Inter-Quartile Range)
Unit of Measure: mg/dL
Baseline [N=16, 5]
148
(127 to 181)
124
(109 to 154)
Week 52 [N=13, 4]
136
(102 to 146)
110
(104 to 123)
Week 104 [N=7, 1]
110
(73 to 146)
128
(128 to 128)
Week 104/Early Termination [N=16, 5]
132
(104 to 175)
128
(122 to 131)
5.Secondary Outcome
Title Percent Change From Baseline in Total Cholesterol
Hide Description Total cholesterol was measured in mg/dL. Samples were taken following an overnight fast.
Time Frame Baseline and Weeks 52 and 104.
Hide Outcome Measure Data
Hide Analysis Population Description
The Safety Set included all enrolled patients who received at least 1 injection of study drug. "N" indicates the number of participants with available data at the specified time point.
Arm/Group Title Mipomersen 200 mg Per Week Mipomersen 200 mg Every Other Week
Hide Arm/Group Description:
Participants received 200 mg mipomersen once a week by subcutaneous injection, for up to 3 years.
Participants received 200 mg mipomersen every other week by subcutaneous injection, for up to 3 years. Participants could receive mipomersen 200 mg once a week at the Investigator’s discretion after the first 52 weeks of the treatment period.
Overall Number of Participants Analyzed 16 5
Median (Inter-Quartile Range)
Unit of Measure: percentage of baseline
Week 52 [N=13, 4]
-10.5
(-26.8 to -6.4)
-6.5
(-13.4 to 8.6)
Week 104 [N=7, 1]
-25.3
(-29.2 to -18.0)
-1.5
(-1.5 to -1.5)
Week 104/Early Termination [N=16, 5]
-11.2
(-24.2 to -2.4)
-1.5
(-1.7 to 2.6)
6.Secondary Outcome
Title Total Cholesterol Over Time
Hide Description For patients who were on placebo in the index study or who took their last dose of mipomersen ≥6 months prior to first dose in this study, Baseline is defined as the last measurement prior to first dose in this study. For participants who took their last dose of mipomersen less than 6 months before starting this study, Baseline is defined as the last measurement taken prior to receiving a first dose in the index study.
Time Frame Baseline and Weeks 52 and 104.
Hide Outcome Measure Data
Hide Analysis Population Description
The Safety Set included all enrolled patients who received at least 1 injection of study drug. "N" indicates the number of participants with available data at the specified time point.
Arm/Group Title Mipomersen 200 mg Per Week Mipomersen 200 mg Every Other Week
Hide Arm/Group Description:
Participants received 200 mg mipomersen once a week by subcutaneous injection, for up to 3 years.
Participants received 200 mg mipomersen every other week by subcutaneous injection, for up to 3 years. Participants could receive mipomersen 200 mg once a week at the Investigator’s discretion after the first 52 weeks of the treatment period.
Overall Number of Participants Analyzed 16 5
Median (Inter-Quartile Range)
Unit of Measure: mg/dL
Baseline [N=16, 5]
251
(206 to 293)
233
(199 to 235)
Week 52 [N=13, 4]
216
(173 to 245)
203
(192 to 220)
Week 104 [N=7, 1]
209
(143 to 209)
196
(196 to 196)
Week 104/Early Termination [N=16, 5]
209
(193 to 297)
231
(199 to 239)
7.Secondary Outcome
Title Percent Change From Baseline in Non-High-Density Lipoprotein Cholesterol
Hide Description Non-high-density lipoprotein cholesterol was measured in mg/dL. Samples were taken following an overnight fast.
Time Frame Baseline and Weeks 52 and 104.
Hide Outcome Measure Data
Hide Analysis Population Description
The Safety Set included all enrolled patients who received at least 1 injection of study drug. "N" indicates the number of participants with available data at the specified time point.
Arm/Group Title Mipomersen 200 mg Per Week Mipomersen 200 mg Every Other Week
Hide Arm/Group Description:
Participants received 200 mg mipomersen once a week by subcutaneous injection, for up to 3 years.
Participants received 200 mg mipomersen every other week by subcutaneous injection, for up to 3 years. Participants could receive mipomersen 200 mg once a week at the Investigator’s discretion after the first 52 weeks of the treatment period.
Overall Number of Participants Analyzed 16 5
Median (Inter-Quartile Range)
Unit of Measure: percentage of baseline
Week 52 [N=13, 4]
-24.7
(-40.5 to -12.6)
-6.7
(-15.0 to 9.5)
Week 104 [N=7, 1]
-34.3
(-37.9 to -21.6)
17.2
(17.2 to 17.2)
Week 104/Early Termination [N=16, 5]
-16.5
(-31.2 to -6.6)
3.7
(-3.0 to 14.1)
8.Secondary Outcome
Title Non-High-Density Lipoprotein Cholesterol Over Time
Hide Description For patients who were on placebo in the index study or who took their last dose of mipomersen ≥6 months prior to first dose in this study, Baseline is defined as the last measurement prior to first dose in this study. For participants who took their last dose of mipomersen less than 6 months before starting this study, Baseline is defined as the last measurement taken prior to receiving a first dose in the index study.
Time Frame Baseline and Weeks 52 and 104.
Hide Outcome Measure Data
Hide Analysis Population Description
The Safety Set included all enrolled patients who received at least 1 injection of study drug. "N" indicates the number of participants with available data at the specified time point.
Arm/Group Title Mipomersen 200 mg Per Week Mipomersen 200 mg Every Other Week
Hide Arm/Group Description:
Participants received 200 mg mipomersen once a week by subcutaneous injection, for up to 3 years.
Participants received 200 mg mipomersen every other week by subcutaneous injection, for up to 3 years. Participants could receive mipomersen 200 mg once a week at the Investigator’s discretion after the first 52 weeks of the treatment period.
Overall Number of Participants Analyzed 16 5
Median (Inter-Quartile Range)
Unit of Measure: mg/dL
Baseline [N=16, 5]
206
(167 to 253)
151
(149 to 203)
Week 52 [N=13, 4]
167
(134 to 208)
150
(140 to 164)
Week 104 [N=7, 1]
161
(95 to 174)
177
(177 to 177)
Week 104/Early Termination [N=16, 5]
164
(151 to 255)
177
(170 to 197)
9.Secondary Outcome
Title Number of Participants With Treatment-Emergent Adverse Events (AEs)
Hide Description AEs were considered as related if assessed by the Investigator as possibly, probably or definitely related to study drug. The severity of each event was assessed using the following categories: Mild (symptom(s) barely noticeable to the patient or do not make the patient uncomfortable); Moderate (symptom(s) of a sufficient severity to make the patient uncomfortable, performance of daily activities is influenced) or Severe (symptom(s) of a sufficient severity to cause the patient severe discomfort, may cause cessation of treatment with the study drug). Serious AEs (SAEs) are those that resulted in death, were life-threatening, required or prolonged inpatient hospitalization, resulted in persistent or significant disability/incapacity, congenital anomaly, or resulted in an important medical event that may have jeopardized the patient or required medical or surgical intervention to prevent one of the outcomes listed above.
Time Frame 2 years
Hide Outcome Measure Data
Hide Analysis Population Description
Safety set
Arm/Group Title Mipomersen 200 mg Per Week Mipomersen 200 mg Every Other Week
Hide Arm/Group Description:
Participants received 200 mg mipomersen once a week by subcutaneous injection, for up to 3 years.
Participants received 200 mg mipomersen every other week by subcutaneous injection, for up to 3 years. Participants could receive mipomersen 200 mg once a week at the Investigator’s discretion after the first 52 weeks of the treatment period.
Overall Number of Participants Analyzed 16 5
Measure Type: Number
Unit of Measure: participants
Any adverse event 16 5
Treatment-related adverse event 16 5
Mild adverse event 5 2
Moderate adverse event 8 3
Severe adverse event 3 0
Serious adverse event 4 2
Adverse event leading to treatment discontinuation 4 2
10.Secondary Outcome
Title Percent Change From Baseline in Clinical Chemistry Parameters
Hide Description [Not Specified]
Time Frame Baseline and Week 104 or the Early Termination visit for participants who did not complete 2 years of treatment.
Hide Outcome Measure Data
Hide Analysis Population Description
Safety set.
Arm/Group Title Mipomersen 200 mg Per Week Mipomersen 200 mg Every Other Week
Hide Arm/Group Description:
Participants received 200 mg mipomersen once a week by subcutaneous injection, for up to 3 years.
Participants received 200 mg mipomersen every other week by subcutaneous injection, for up to 3 years. Participants could receive mipomersen 200 mg once a week at the Investigator’s discretion after the first 52 weeks of the treatment period.
Overall Number of Participants Analyzed 16 5
Mean (Standard Deviation)
Unit of Measure: percentage of baseline
Alanine aminotransferase (ALT) 63.73  (112.98) 29.96  (80.01)
Aspartate aminotransferase (AST) 48.73  (86.00) 3.53  (39.14)
Bilirubin, Indirect 13.81  (20.56) 12.50  (29.86)
Bilirubin, Total 34.79  (57.97) 0.48  (10.96)
Blood Urea Nitrogen (BUN) 6.08  (22.90) -11.06  (16.56)
Creatine Kinase -17.24  (36.85) 40.95  (149.95)
Creatinine -9.94  (12.54) -9.84  (14.05)
Glomerular Filtration Rate (GFR) 16.20  (24.56) 15.49  (21.58)
11.Secondary Outcome
Title Percent Change From Baseline in Hematology Parameters
Hide Description [Not Specified]
Time Frame Baseline and Week 104 or the Early Termination visit for participants who did not complete 2 years of treatment
Hide Outcome Measure Data
Hide Analysis Population Description
Safety set
Arm/Group Title Mipomersen 200 mg Per Week Mipomersen 200 mg Every Other Week
Hide Arm/Group Description:
Participants received 200 mg mipomersen once a week by subcutaneous injection, for up to 3 years.
Participants received 200 mg mipomersen every other week by subcutaneous injection, for up to 3 years. Participants could receive mipomersen 200 mg once a week at the Investigator’s discretion after the first 52 weeks of the treatment period.
Overall Number of Participants Analyzed 16 5
Mean (Standard Deviation)
Unit of Measure: percentage of baseline
Hematocrit -0.04  (8.69) -5.24  (3.69)
Hemoglobin 0.90  (7.61) -4.24  (4.44)
Leukocytes -0.80  (31.95) -5.99  (43.17)
Platelets -8.55  (11.88) -1.36  (12.31)
12.Secondary Outcome
Title Percent Change From Baseline in Blood Pressure
Hide Description [Not Specified]
Time Frame Baseline and Week 104 or the Early Termination visit for participants who did not complete 2 years of treatment.
Hide Outcome Measure Data
Hide Analysis Population Description
Safety set
Arm/Group Title Mipomersen 200 mg Per Week Mipomersen 200 mg Every Other Week
Hide Arm/Group Description:
Participants received 200 mg mipomersen once a week by subcutaneous injection, for up to 3 years.
Participants received 200 mg mipomersen every other week by subcutaneous injection, for up to 3 years. Participants could receive mipomersen 200 mg once a week at the Investigator’s discretion after the first 52 weeks of the treatment period.
Overall Number of Participants Analyzed 16 5
Mean (Standard Deviation)
Unit of Measure: percentage of baseline
Systolic Blood Pressure 6.99  (12.33) 2.35  (12.54)
Diastolic Blood Pressure -0.88  (11.31) 10.67  (15.35)
13.Secondary Outcome
Title Percent Change From Baseline in Pulse Rate
Hide Description [Not Specified]
Time Frame Baseline and Week 104 or the Early Termination visit for participants who did not complete 2 years of treatment.
Hide Outcome Measure Data
Hide Analysis Population Description
Safety set
Arm/Group Title Mipomersen 200 mg Per Week Mipomersen 200 mg Every Other Week
Hide Arm/Group Description:
Participants received 200 mg mipomersen once a week by subcutaneous injection, for up to 3 years.
Participants received 200 mg mipomersen every other week by subcutaneous injection, for up to 3 years. Participants could receive mipomersen 200 mg once a week at the Investigator’s discretion after the first 52 weeks of the treatment period.
Overall Number of Participants Analyzed 16 5
Mean (Standard Deviation)
Unit of Measure: percentage of baseline
4.89  (14.41) 6.28  (17.45)
14.Secondary Outcome
Title Percent Change From Baseline in Respiratory Rate
Hide Description [Not Specified]
Time Frame Baseline and Week 104 or the Early Termination visit for participants who did not complete 2 years of treatment.
Hide Outcome Measure Data
Hide Analysis Population Description
Safety set
Arm/Group Title Mipomersen 200 mg Per Week Mipomersen 200 mg Every Other Week
Hide Arm/Group Description:
Participants received 200 mg mipomersen once a week by subcutaneous injection, for up to 3 years.
Participants received 200 mg mipomersen every other week by subcutaneous injection, for up to 3 years. Participants could receive mipomersen 200 mg once a week at the Investigator’s discretion after the first 52 weeks of the treatment period.
Overall Number of Participants Analyzed 16 5
Mean (Standard Deviation)
Unit of Measure: percentage of baseline
0.87  (10.57) -4.44  (13.30)
15.Other Pre-specified Outcome
Title Percent Change From Baseline in Triglycerides
Hide Description Triglycerides were measured in mg/dL. Samples were taken following an overnight fast.
Time Frame Baseline and Weeks 52 and 104.
Hide Outcome Measure Data
Hide Analysis Population Description
The Safety Set included all enrolled patients who received at least 1 injection of study drug. "N" indicates the number of participants with available data at the specified time point.
Arm/Group Title Mipomersen 200 mg Per Week Mipomersen 200 mg Every Other Week
Hide Arm/Group Description:
Participants received 200 mg mipomersen once a week by subcutaneous injection, for up to 3 years.
Participants received 200 mg mipomersen every other week by subcutaneous injection, for up to 3 years. Participants could receive mipomersen 200 mg once a week at the Investigator’s discretion after the first 52 weeks of the treatment period.
Overall Number of Participants Analyzed 16 5
Median (Inter-Quartile Range)
Unit of Measure: percentage of baseline
Week 52 [N=13, 4]
-30.2
(-36.3 to -26.7)
-27.0
(-43.0 to -3.1)
Week 104 [N=7, 1]
-46.8
(-58.2 to -30.2)
-44.8
(-44.8 to -44.8)
Week 104/Early Termination [N=16, 5]
-34.3
(-42.9 to 2.2)
-34.7
(-36.1 to 9.0)
16.Other Pre-specified Outcome
Title Triglycerides Over Time
Hide Description For patients who were on placebo in the index study or who took their last dose of mipomersen ≥6 months prior to first dose in this study, Baseline is defined as the last measurement prior to first dose in this study. For participants who took their last dose of mipomersen less than 6 months before starting this study, Baseline is defined as the last measurement taken prior to receiving a first dose in the index study.
Time Frame Baseline and Weeks 52 and 104.
Hide Outcome Measure Data
Hide Analysis Population Description
The Safety Set included all enrolled patients who received at least 1 injection of study drug. "N" indicates the number of participants with available data at the specified time point.
Arm/Group Title Mipomersen 200 mg Per Week Mipomersen 200 mg Every Other Week
Hide Arm/Group Description:
Participants received 200 mg mipomersen once a week by subcutaneous injection, for up to 3 years.
Participants received 200 mg mipomersen every other week by subcutaneous injection, for up to 3 years. Participants could receive mipomersen 200 mg once a week at the Investigator’s discretion after the first 52 weeks of the treatment period.
Overall Number of Participants Analyzed 16 5
Median (Inter-Quartile Range)
Unit of Measure: mg/dL
Baseline [N=16, 5]
167
(96 to 254)
134
(92 to 199)
Week 52 [N=13, 4]
102
(67 to 150)
90
(80 to 123)
Week 104 [N=7, 1]
77
(67 to 113)
74
(74 to 74)
Week 104/Early Termination [N=16, 5]
98
(76 to 180)
122
(97 to 130)
17.Other Pre-specified Outcome
Title Percent Change From Baseline in Lipoprotein(a)
Hide Description Lipoprotein(a) was measured in mg/dL. Samples were taken following an overnight fast.
Time Frame Baseline and Weeks 52 and 104.
Hide Outcome Measure Data
Hide Analysis Population Description
The Safety Set included all enrolled patients who received at least 1 injection of study drug. "N" indicates the number of participants with available data at the specified time point.
Arm/Group Title Mipomersen 200 mg Per Week Mipomersen 200 mg Every Other Week
Hide Arm/Group Description:
Participants received 200 mg mipomersen once a week by subcutaneous injection, for up to 3 years.
Participants received 200 mg mipomersen every other week by subcutaneous injection, for up to 3 years. Participants could receive mipomersen 200 mg once a week at the Investigator’s discretion after the first 52 weeks of the treatment period.
Overall Number of Participants Analyzed 16 5
Median (Inter-Quartile Range)
Unit of Measure: percentage of baseline
Week 52 [N=13, 4]
-20.2
(-34.3 to -4.7)
0.0
(-27.3 to 0.0)
Week 104 [N=7, 1]
-30.7
(-32.7 to -24.1)
0.0
(0.0 to 0.0)
Week 104/Early Termination [N=16, 5]
-18.2
(-31.0 to 0.0)
-20.0
(-25.0 to 0.0)
18.Other Pre-specified Outcome
Title Lipoprotein(a) Over Time
Hide Description For patients who were on placebo in the index study or who took their last dose of mipomersen ≥6 months prior to first dose in this study, Baseline is defined as the last measurement prior to first dose in this study. For participants who took their last dose of mipomersen less than 6 months before starting this study, Baseline is defined as the last measurement taken prior to receiving a first dose in the index study.
Time Frame Baseline and Weeks 52 and 104.
Hide Outcome Measure Data
Hide Analysis Population Description
The Safety Set included all enrolled patients who received at least 1 injection of study drug. "N" indicates the number of participants with available data at the specified time point.
Arm/Group Title Mipomersen 200 mg Per Week Mipomersen 200 mg Every Other Week
Hide Arm/Group Description:
Participants received 200 mg mipomersen once a week by subcutaneous injection, for up to 3 years.
Participants received 200 mg mipomersen every other week by subcutaneous injection, for up to 3 years. Participants could receive mipomersen 200 mg once a week at the Investigator’s discretion after the first 52 weeks of the treatment period.
Overall Number of Participants Analyzed 16 5
Median (Inter-Quartile Range)
Unit of Measure: mg/dL
Baseline [N=16, 5]
50
(15 to 98)
5
(3 to 11)
Week 52 [N=13, 4]
54
(23 to 75)
4
(3 to 5)
Week 104 [N=7, 1]
49
(33 to 79)
3
(3 to 3)
Week 104/Early Termination [N=16, 5]
47
(24 to 71)
4
(3 to 4)
19.Other Pre-specified Outcome
Title Percent Change From Baseline in Very-Low-Density Lipoprotein (VLDL) Cholesterol
Hide Description Very-Low-Density Lipoprotein (VLDL) Cholesterol was measured in mg/dL. Samples were taken following an overnight fast.
Time Frame Baseline and Weeks 52 and 104.
Hide Outcome Measure Data
Hide Analysis Population Description
The Safety Set included all enrolled patients who received at least 1 injection of study drug. "N" indicates the number of participants with available data at the specified time point.
Arm/Group Title Mipomersen 200 mg Per Week Mipomersen 200 mg Every Other Week
Hide Arm/Group Description:
Participants received 200 mg mipomersen once a week by subcutaneous injection, for up to 3 years.
Participants received 200 mg mipomersen every other week by subcutaneous injection, for up to 3 years. Participants could receive mipomersen 200 mg once a week at the Investigator’s discretion after the first 52 weeks of the treatment period.
Overall Number of Participants Analyzed 16 5
Median (Inter-Quartile Range)
Unit of Measure: percentage of baseline
Week 52 [N=13, 4]
-31.6
(-37.1 to -25.0)
-25.9
(-43.6 to -2.8)
Week 104 [N=7, 1]
-48.6
(-58.3 to -31.6)
-44.4
(-44.4 to -44.4)
Week 104/Early Termination [N=16, 5]
-34.0
(-44.0 to 2.8)
-35.0
(-35.7 to 5.6)
20.Other Pre-specified Outcome
Title Very-Low-Density Lipoprotein (VLDL) Cholesterol Over Time
Hide Description For patients who were on placebo in the index study or who took their last dose of mipomersen ≥6 months prior to first dose in this study, Baseline is defined as the last measurement prior to first dose in this study. For participants who took their last dose of mipomersen less than 6 months before starting this study, Baseline is defined as the last measurement taken prior to receiving a first dose in the index study.
Time Frame Baseline and Weeks 52 and 104.
Hide Outcome Measure Data
Hide Analysis Population Description
The Safety Set included all enrolled patients who received at least 1 injection of study drug. "N" indicates the number of participants with available data at the specified time point.
Arm/Group Title Mipomersen 200 mg Per Week Mipomersen 200 mg Every Other Week
Hide Arm/Group Description:
Participants received 200 mg mipomersen once a week by subcutaneous injection, for up to 3 years.
Participants received 200 mg mipomersen every other week by subcutaneous injection, for up to 3 years. Participants could receive mipomersen 200 mg once a week at the Investigator’s discretion after the first 52 weeks of the treatment period.
Overall Number of Participants Analyzed 16 5
Median (Inter-Quartile Range)
Unit of Measure: mg/dL
Baseline [N=16, 5]
34
(19 to 51)
27
(18 to 40)
Week 52 [N=13, 4]
20
(13 to 30)
18
(16 to 25)
Week 104 [N=7, 1]
15
(13 to 23)
15
(15 to 15)
Week 104/Early Termination [N=16, 5]
20
(15 to 36)
24
(19 to 26)
21.Other Pre-specified Outcome
Title Percent Change From Baseline in Ratio of Low-density Lipoprotein Cholesterol to High-density Lipoprotein Cholesterol
Hide Description [Not Specified]
Time Frame Baseline and Weeks 52 and 104.
Hide Outcome Measure Data
Hide Analysis Population Description
The Safety Set included all enrolled patients who received at least 1 injection of study drug. "N" indicates the number of participants with available data at the specified time point.
Arm/Group Title Mipomersen 200 mg Per Week Mipomersen 200 mg Every Other Week
Hide Arm/Group Description:
Participants received 200 mg mipomersen once a week by subcutaneous injection, for up to 3 years.
Participants received 200 mg mipomersen every other week by subcutaneous injection, for up to 3 years. Participants could receive mipomersen 200 mg once a week at the Investigator’s discretion after the first 52 weeks of the treatment period.
Overall Number of Participants Analyzed 16 5
Median (Inter-Quartile Range)
Unit of Measure: percentage of baseline
Week 52 [N=13, 4]
-33.5
(-43.6 to -25.8)
2.6
(-11.8 to 10.2)
Week 104 [N=7, 1]
-41.2
(-57.8 to -34.6)
230.1
(230.1 to 230.1)
Week 104/Early Termination [N=16, 5]
-24.9
(-38.2 to -18.5)
15.2
(3.1 to 35.7)
22.Other Pre-specified Outcome
Title Ratio of Low-density Lipoprotein Cholesterol to High-density Lipoprotein Cholesterol Over Time
Hide Description For patients who were on placebo in the index study or who took their last dose of mipomersen ≥6 months prior to first dose in this study, Baseline is defined as the last measurement prior to first dose in this study. For participants who took their last dose of mipomersen less than 6 months before starting this study, Baseline is defined as the last measurement taken prior to receiving a first dose in the index study.
Time Frame Baseline and Weeks 52 and 104.
Hide Outcome Measure Data
Hide Analysis Population Description
The Safety Set included all enrolled patients who received at least 1 injection of study drug. "N" indicates the number of participants with available data at the specified time point.
Arm/Group Title Mipomersen 200 mg Per Week Mipomersen 200 mg Every Other Week
Hide Arm/Group Description:
Participants received 200 mg mipomersen once a week by subcutaneous injection, for up to 3 years.
Participants received 200 mg mipomersen every other week by subcutaneous injection, for up to 3 years. Participants could receive mipomersen 200 mg once a week at the Investigator’s discretion after the first 52 weeks of the treatment period.
Overall Number of Participants Analyzed 16 5
Median (Inter-Quartile Range)
Unit of Measure: ratio
Baseline [N=16, 5]
4
(3 to 6)
3
(2 to 4)
Week 52 [N=13, 4]
3
(2 to 4)
3
(2 to 3)
Week 104 [N=7, 1]
3
(2 to 4)
9
(9 to 9)
Week 104/Early Termination [N=16, 5]
3
(2 to 4)
5
(2 to 5)
23.Other Pre-specified Outcome
Title Percent Change From Baseline in Apolipoprotein A1
Hide Description Apolipoprotein A1 was measured in mg/dL. Samples were taken following an overnight fast.
Time Frame Baseline and Weeks 52 and 104.
Hide Outcome Measure Data
Hide Analysis Population Description
The Safety Set included all enrolled patients who received at least 1 injection of study drug. "N" indicates the number of participants with available data at the specified time point.
Arm/Group Title Mipomersen 200 mg Per Week Mipomersen 200 mg Every Other Week
Hide Arm/Group Description:
Participants received 200 mg mipomersen once a week by subcutaneous injection, for up to 3 years.
Participants received 200 mg mipomersen every other week by subcutaneous injection, for up to 3 years. Participants could receive mipomersen 200 mg once a week at the Investigator’s discretion after the first 52 weeks of the treatment period.
Overall Number of Participants Analyzed 16 5
Median (Inter-Quartile Range)
Unit of Measure: percentage of baseline
Week 52 [N=13, 4]
4.6
(-4.3 to 11.2)
7.4
(-5.3 to 17.0)
Week 104 [N=7, 1]
5.9
(-2.7 to 7.6)
-55.1
(-55.1 to -55.1)
Week 104/Early Termination [N=16, 5]
6.3
(-0.7 to 9.5)
-7.9
(-11.8 to -3.4)
24.Other Pre-specified Outcome
Title Apolipoprotein A1 Over Time
Hide Description For patients who were on placebo in the index study or who took their last dose of mipomersen ≥6 months prior to first dose in this study, Baseline is defined as the last measurement prior to first dose in this study. For participants who took their last dose of mipomersen less than 6 months before starting this study, Baseline is defined as the last measurement taken prior to receiving a first dose in the index study.
Time Frame Baseline and Weeks 52 and 104.
Hide Outcome Measure Data
Hide Analysis Population Description
The Safety Set included all enrolled patients who received at least 1 injection of study drug. "N" indicates the number of participants with available data at the specified time point.
Arm/Group Title Mipomersen 200 mg Per Week Mipomersen 200 mg Every Other Week
Hide Arm/Group Description:
Participants received 200 mg mipomersen once a week by subcutaneous injection, for up to 3 years.
Participants received 200 mg mipomersen every other week by subcutaneous injection, for up to 3 years. Participants could receive mipomersen 200 mg once a week at the Investigator’s discretion after the first 52 weeks of the treatment period.
Overall Number of Participants Analyzed 16 5
Median (Inter-Quartile Range)
Unit of Measure: mg/dL
Baseline [N=16, 5]
132
(120 to 149)
149
(147 to 178)
Week 52 [N=13, 4]
139
(123 to 160)
159
(144 to 178)
Week 104 [N=7, 1]
142
(113 to 155)
66
(66 to 66)
Week 104/Early Termination [N=16, 5]
141
(129 to 153)
144
(126 to 164)
25.Other Pre-specified Outcome
Title Percent Change From Baseline in High-Density Lipoprotein Cholesterol
Hide Description High-Density Lipoprotein (HDL) Cholesterol was measured in mg/dL. Samples were taken following an overnight fast.
Time Frame Baseline and Weeks 52 and 104.
Hide Outcome Measure Data
Hide Analysis Population Description
The Safety Set included all enrolled patients who received at least 1 injection of study drug. "N" indicates the number of participants with available data at the specified time point.
Arm/Group Title Mipomersen 200 mg Per Week Mipomersen 200 mg Every Other Week
Hide Arm/Group Description:
Participants received 200 mg mipomersen once a week by subcutaneous injection, for up to 3 years.
Participants received 200 mg mipomersen every other week by subcutaneous injection, for up to 3 years. Participants could receive mipomersen 200 mg once a week at the Investigator’s discretion after the first 52 weeks of the treatment period.
Overall Number of Participants Analyzed 16 5
Median (Inter-Quartile Range)
Unit of Measure: percentage of baseline
Week 52 [N=13, 4]
12.8
(6.4 to 32.4)
6.8
(-4.9 to 12.0)
Week 104 [N=7, 1]
14.9
(6.1 to 43.8)
-60.4
(-60.4 to -60.4)
Week 104/Early Termination [N=16, 5]
15.3
(4.9 to 26.9)
-17.9
(-19.6 to 6.3)
26.Other Pre-specified Outcome
Title High-Density Lipoprotein Cholesterol Over Time
Hide Description For patients who were on placebo in the index study or who took their last dose of mipomersen ≥6 months prior to first dose in this study, Baseline is defined as the last measurement prior to first dose in this study. For participants who took their last dose of mipomersen less than 6 months before starting this study, Baseline is defined as the last measurement taken prior to receiving a first dose in the index study.
Time Frame Baseline and Weeks 52 and 104.
Hide Outcome Measure Data
Hide Analysis Population Description
The Safety Set included all enrolled patients who received at least 1 injection of study drug. "N" indicates the number of participants with available data at the specified time point.
Arm/Group Title Mipomersen 200 mg Per Week Mipomersen 200 mg Every Other Week
Hide Arm/Group Description:
Participants received 200 mg mipomersen once a week by subcutaneous injection, for up to 3 years.
Participants received 200 mg mipomersen every other week by subcutaneous injection, for up to 3 years. Participants could receive mipomersen 200 mg once a week at the Investigator’s discretion after the first 52 weeks of the treatment period.
Overall Number of Participants Analyzed 16 5
Median (Inter-Quartile Range)
Unit of Measure: mg/dL
Baseline [N=16, 5]
40
(33 to 46)
52
(48 to 56)
Week 52 [N=13, 4]
46
(41 to 50)
54
(43 to 66)
Week 104 [N=7, 1]
47
(35 to 48)
19
(19 to 19)
Week 104/Early Termination [N=16, 5]
47
(40 to 50)
45
(34 to 58)
Time Frame Up to 3 years.
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 Mipomersen 200 mg Per Week Mipomersen 200 mg Every Other Week
Hide Arm/Group Description Participants received 200 mg mipomersen once a week by subcutaneous injection, for up to 3 years. Participants received 200 mg mipomersen every other week by subcutaneous injection, for up to 3 years. Participants could receive mipomersen 200 mg once a week at the Investigator’s discretion after the first 52 weeks of the treatment period.
All-Cause Mortality
Mipomersen 200 mg Per Week Mipomersen 200 mg Every Other Week
Affected / at Risk (%) Affected / at Risk (%)
Total   --/--   --/-- 
Show Serious Adverse Events Hide Serious Adverse Events
Mipomersen 200 mg Per Week Mipomersen 200 mg Every Other Week
Affected / at Risk (%) Affected / at Risk (%)
Total   4/16 (25.00%)   3/5 (60.00%) 
Cardiac disorders     
Angina pectoris  1  0/16 (0.00%)  1/5 (20.00%) 
Coronary artery disease  1  0/16 (0.00%)  2/5 (40.00%) 
Gastrointestinal disorders     
Inguinal hernia  1  1/16 (6.25%)  0/5 (0.00%) 
General disorders     
Non-cardiac chest pain  1  1/16 (6.25%)  0/5 (0.00%) 
Investigations     
Alanine aminotransferase increased  1  1/16 (6.25%)  0/5 (0.00%) 
Neoplasms benign, malignant and unspecified (incl cysts and polyps)     
Malignant melanoma in situ  1  1/16 (6.25%)  0/5 (0.00%) 
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA 13.1
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Mipomersen 200 mg Per Week Mipomersen 200 mg Every Other Week
Affected / at Risk (%) Affected / at Risk (%)
Total   16/16 (100.00%)   5/5 (100.00%) 
Blood and lymphatic system disorders     
Lymphadenopathy  1  2/16 (12.50%)  0/5 (0.00%) 
Splenomegaly  1  1/16 (6.25%)  0/5 (0.00%) 
Cardiac disorders     
Angina pectoris  1  0/16 (0.00%)  2/5 (40.00%) 
Cardiomegaly  1  1/16 (6.25%)  0/5 (0.00%) 
Extrasystoles  1  1/16 (6.25%)  0/5 (0.00%) 
Congenital, familial and genetic disorders     
Arteriovenous malformation  1  0/16 (0.00%)  1/5 (20.00%) 
Ear and labyrinth disorders     
Ear discomfort  1  1/16 (6.25%)  0/5 (0.00%) 
Endocrine disorders     
Thyroid cyst  1  1/16 (6.25%)  0/5 (0.00%) 
Eye disorders     
Arcus lipoides  1  1/16 (6.25%)  0/5 (0.00%) 
Conjunctival haemorrhage  1  1/16 (6.25%)  0/5 (0.00%) 
Eye irritation  1  1/16 (6.25%)  0/5 (0.00%) 
Gastrointestinal disorders     
Abdominal pain  1  1/16 (6.25%)  1/5 (20.00%) 
Abdominal pain upper  1  1/16 (6.25%)  0/5 (0.00%) 
Dental caries  1  1/16 (6.25%)  0/5 (0.00%) 
Diarrhoea  1  2/16 (12.50%)  0/5 (0.00%) 
Diverticulum intestinal  1  1/16 (6.25%)  0/5 (0.00%) 
Gastrooesophageal reflux disease  1  0/16 (0.00%)  1/5 (20.00%) 
Haemorrhoids  1  1/16 (6.25%)  0/5 (0.00%) 
Hypoaesthesia oral  1  1/16 (6.25%)  0/5 (0.00%) 
Lip swelling  1  1/16 (6.25%)  0/5 (0.00%) 
Nausea  1  3/16 (18.75%)  2/5 (40.00%) 
Toothache  1  1/16 (6.25%)  0/5 (0.00%) 
Vomiting  1  2/16 (12.50%)  0/5 (0.00%) 
General disorders     
Adverse drug reaction  1  1/16 (6.25%)  0/5 (0.00%) 
Chest pain  1  1/16 (6.25%)  0/5 (0.00%) 
Chills  1  1/16 (6.25%)  0/5 (0.00%) 
Fatigue  1  2/16 (12.50%)  1/5 (20.00%) 
Feeling jittery  1  0/16 (0.00%)  1/5 (20.00%) 
Influenza like illness  1  2/16 (12.50%)  3/5 (60.00%) 
Injection site anaesthesia  1  1/16 (6.25%)  0/5 (0.00%) 
Injection site coldness  1  1/16 (6.25%)  0/5 (0.00%) 
Injection site discolouration  1  3/16 (18.75%)  1/5 (20.00%) 
Injection site erythema  1  10/16 (62.50%)  2/5 (40.00%) 
Injection site haematoma  1  1/16 (6.25%)  3/5 (60.00%) 
Injection site haemorrhage  1  2/16 (12.50%)  0/5 (0.00%) 
Injection site hypertrophy  1  2/16 (12.50%)  0/5 (0.00%) 
Injection site pain  1  3/16 (18.75%)  2/5 (40.00%) 
Injection site pruritus  1  2/16 (12.50%)  2/5 (40.00%) 
Injection site rash  1  1/16 (6.25%)  0/5 (0.00%) 
Injection site reaction  1  7/16 (43.75%)  1/5 (20.00%) 
Injection site swelling  1  1/16 (6.25%)  2/5 (40.00%) 
Injection site urticaria  1  1/16 (6.25%)  1/5 (20.00%) 
Malaise  1  1/16 (6.25%)  0/5 (0.00%) 
Pain  1  0/16 (0.00%)  1/5 (20.00%) 
Pyrexia  1  1/16 (6.25%)  0/5 (0.00%) 
Hepatobiliary disorders     
Cholelithiasis  1  0/16 (0.00%)  1/5 (20.00%) 
Hepatic steatosis  1  2/16 (12.50%)  0/5 (0.00%) 
Immune system disorders     
Hypersensitivity  1  1/16 (6.25%)  0/5 (0.00%) 
Infections and infestations     
Acarodermatitis  1  1/16 (6.25%)  0/5 (0.00%) 
Cellulitis  1  1/16 (6.25%)  0/5 (0.00%) 
Cystitis  1  1/16 (6.25%)  0/5 (0.00%) 
Ear infection  1  1/16 (6.25%)  0/5 (0.00%) 
Influenza  1  3/16 (18.75%)  0/5 (0.00%) 
Nasopharyngitis  1  2/16 (12.50%)  1/5 (20.00%) 
Sinusitis  1  1/16 (6.25%)  0/5 (0.00%) 
Tooth infection  1  1/16 (6.25%)  0/5 (0.00%) 
Upper respiratory tract infection  1  2/16 (12.50%)  0/5 (0.00%) 
Injury, poisoning and procedural complications     
Back injury  1  1/16 (6.25%)  0/5 (0.00%) 
Contusion  1  1/16 (6.25%)  1/5 (20.00%) 
Excoriation  1  1/16 (6.25%)  0/5 (0.00%) 
Foot fracture  1  1/16 (6.25%)  0/5 (0.00%) 
Joint injury  1  1/16 (6.25%)  0/5 (0.00%) 
Joint sprain  1  1/16 (6.25%)  0/5 (0.00%) 
Limb injury  1  1/16 (6.25%)  1/5 (20.00%) 
Muscle strain  1  2/16 (12.50%)  0/5 (0.00%) 
Post procedural discomfort  1  1/16 (6.25%)  0/5 (0.00%) 
Procedural pain  1  1/16 (6.25%)  0/5 (0.00%) 
Skin laceration  1  2/16 (12.50%)  0/5 (0.00%) 
Splinter  1  0/16 (0.00%)  1/5 (20.00%) 
Wrong technique in drug usage process  1  1/16 (6.25%)  0/5 (0.00%) 
Investigations     
Alanine aminotransferase increased  1  4/16 (25.00%)  1/5 (20.00%) 
Aspartate aminotransferase increased  1  3/16 (18.75%)  0/5 (0.00%) 
Blood glucose increased  1  1/16 (6.25%)  0/5 (0.00%) 
Blood urine present  1  1/16 (6.25%)  0/5 (0.00%) 
Hepatic enzyme increased  1  5/16 (31.25%)  0/5 (0.00%) 
Platelet count decreased  1  1/16 (6.25%)  0/5 (0.00%) 
Protein urine present  1  2/16 (12.50%)  0/5 (0.00%) 
Urine analysis abnormal  1  1/16 (6.25%)  0/5 (0.00%) 
Metabolism and nutrition disorders     
Decreased appetite  1  1/16 (6.25%)  0/5 (0.00%) 
Increased appetite  1  0/16 (0.00%)  1/5 (20.00%) 
Musculoskeletal and connective tissue disorders     
Arthralgia  1  4/16 (25.00%)  1/5 (20.00%) 
Back pain  1  1/16 (6.25%)  0/5 (0.00%) 
Exostosis  1  1/16 (6.25%)  0/5 (0.00%) 
Myalgia  1  2/16 (12.50%)  0/5 (0.00%) 
Pain in extremity  1  1/16 (6.25%)  1/5 (20.00%) 
Rotator cuff syndrome  1  1/16 (6.25%)  0/5 (0.00%) 
Sensation of heaviness  1  0/16 (0.00%)  1/5 (20.00%) 
Neoplasms benign, malignant and unspecified (incl cysts and polyps)     
Angiomyolipoma  1  1/16 (6.25%)  0/5 (0.00%) 
Nervous system disorders     
Dizziness  1  0/16 (0.00%)  2/5 (40.00%) 
Headache  1  3/16 (18.75%)  0/5 (0.00%) 
Hypoaesthesia  1  1/16 (6.25%)  0/5 (0.00%) 
Nerve compression  1  1/16 (6.25%)  0/5 (0.00%) 
Sciatica  1  0/16 (0.00%)  1/5 (20.00%) 
Tremor  1  1/16 (6.25%)  0/5 (0.00%) 
Psychiatric disorders     
Anxiety  1  1/16 (6.25%)  0/5 (0.00%) 
Grief reaction  1  1/16 (6.25%)  0/5 (0.00%) 
Insomnia  1  0/16 (0.00%)  1/5 (20.00%) 
Mood altered  1  0/16 (0.00%)  1/5 (20.00%) 
Renal and urinary disorders     
Chromaturia  1  0/16 (0.00%)  1/5 (20.00%) 
Hypertonic bladder  1  1/16 (6.25%)  0/5 (0.00%) 
Proteinuria  1  1/16 (6.25%)  0/5 (0.00%) 
Renal cyst  1  1/16 (6.25%)  0/5 (0.00%) 
Reproductive system and breast disorders     
Breast cyst  1  1/16 (6.25%)  0/5 (0.00%) 
Breast mass  1  0/16 (0.00%)  1/5 (20.00%) 
Respiratory, thoracic and mediastinal disorders     
Asthma  1  1/16 (6.25%)  0/5 (0.00%) 
Cough  1  0/16 (0.00%)  1/5 (20.00%) 
Dyspnoea  1  0/16 (0.00%)  1/5 (20.00%) 
Emphysema  1  1/16 (6.25%)  0/5 (0.00%) 
Increased upper airway secretion  1  1/16 (6.25%)  0/5 (0.00%) 
Respiratory tract congestion  1  1/16 (6.25%)  0/5 (0.00%) 
Rhinorrhoea  1  0/16 (0.00%)  1/5 (20.00%) 
Sinus congestion  1  1/16 (6.25%)  0/5 (0.00%) 
Throat irritation  1  0/16 (0.00%)  1/5 (20.00%) 
Skin and subcutaneous tissue disorders     
Pruritus  1  1/16 (6.25%)  0/5 (0.00%) 
Rash  1  1/16 (6.25%)  0/5 (0.00%) 
Rash erythematous  1  1/16 (6.25%)  0/5 (0.00%) 
Rash generalised  1  1/16 (6.25%)  0/5 (0.00%) 
Skin irritation  1  0/16 (0.00%)  1/5 (20.00%) 
Urticaria  1  1/16 (6.25%)  0/5 (0.00%) 
Vascular disorders     
Haematoma  1  1/16 (6.25%)  0/5 (0.00%) 
Hypertension  1  1/16 (6.25%)  0/5 (0.00%) 
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA 13.1
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: NCT00477594     History of Changes
Other Study ID Numbers: 301012-CS17
2007-001024-12 ( EudraCT Number )
First Submitted: May 22, 2007
First Posted: May 24, 2007
Results First Submitted: February 25, 2013
Results First Posted: April 5, 2013
Last Update Posted: September 9, 2016