Open Label Extension of ISIS 301012 (Mipomersen) to Treat... | NCT00477594

ID	Type	Description	Link
2007-001024-12	EudraCT Number

Name	Class
Ionis Pharmaceuticals, Inc.	INDUSTRY

Label	Type	Description	Intervention Names
Mipomersen 200 mg per week	Experimental	Participants received 200 mg mipomersen once a week by subcutaneous injection, for up to 3 years.	Drug: mipomersen sodium
Mipomersen 200 mg every other week	Experimental	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.	Drug: mipomersen sodium

Name	Type	Description	Arm Group Labels	Other Names
mipomersen sodium	Drug	200 mg/ml, in 1 ml solution for subcutaneous injection.	Mipomersen 200 mg every other week Mipomersen 200 mg per week

Measure	Description	Time Frame
Percent Change From Baseline in Low-density Lipoprotein Cholesterol (LDL-C)	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.	Baseline and Weeks 52 and 104
Low-density Lipoprotein Cholesterol (LDL-C) Over Time	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.	Baseline and Weeks 52 and 104.

Measure	Description	Time Frame
Percent Change From Baseline in Apolipoprotein B	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.

Measure	Description	Time Frame
Percent Change From Baseline in Triglycerides	Triglycerides were measured in mg/dL. Samples were taken following an overnight fast.	Baseline and Weeks 52 and 104.
Triglycerides Over Time	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.

Name	Affiliation	Role
Medical Monitor	Genzyme, a Sanofi Company	Study Director

Facility	Status	City	State	ZIP	Country	Contacts
		Chicago	Illinois	60654	United States

PubMed Identifier	Type	Citation	Retractions
20707601	Derived	Patel N, Hegele RA. Mipomersen as a potential adjunctive therapy for hypercholesterolemia. Expert Opin Pharmacother. 2010 Oct;11(15):2569-72. doi: 10.1517/14656566.2010.512006.

ID	Title	Description
FG000	Mipomersen 200 mg Per Week	Participants received 200 mg mipomersen once a week by subcutaneous injection, for up to 3 years.
FG001	Mipomersen 200 mg Every Other Week	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.

Type

Title

Description

Population Description

Reporting Status

Anticipated Posting Date

Parameter Type

Dispersion Type

Unit of Measure

Calculate Percentage

Time Frame

Units Analyzed

Denominator Units Selected

Arm/Group Information

Denominators

Classes

Analyses

Primary

Percent Change From Baseline in Low-density Lipoprotein Cholesterol (LDL-C)

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.

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.

Posted

Median

Inter-Quartile Range

percentage of baseline

Baseline and Weeks 52 and 104

ID	Title	Description
OG000	Mipomersen 200 mg Per Week	Participants received 200 mg mipomersen once a week by subcutaneous injection, for up to 3 years.
OG001	Mipomersen 200 mg Every Other Week	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.

Units	Counts
Participants	OG00016 OG0015

Title

Denominators

Categories

Week 52 [N=13, 4]

Title	Measurements
	OG000-16.2(-41.8 to -7.8) OG001-2.6(-10.2 to 14.8)

Week 104 [N=7, 1]

Title	Measurements
	OG000

Primary

Low-density Lipoprotein Cholesterol (LDL-C) Over Time

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.

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.

Posted

Median

Inter-Quartile Range

mg/dL

Baseline and Weeks 52 and 104.

ID	Title	Description
OG000	Mipomersen 200 mg Per Week	Participants received 200 mg mipomersen once a week by subcutaneous injection, for up to 3 years.
OG001	Mipomersen 200 mg Every Other Week	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.

Secondary

Percent Change From Baseline in Apolipoprotein B

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.

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.

Posted

Median

Inter-Quartile Range

percentage of baseline

Baseline and Weeks 52 and 104

ID	Title	Description
OG000	Mipomersen 200 mg Per Week	Participants received 200 mg mipomersen once a week by subcutaneous injection, for up to 3 years.
OG001	Mipomersen 200 mg Every Other Week	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.

Secondary

Apolipoprotein B Over Time

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.

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.

Posted

Median

Inter-Quartile Range

mg/dL

Baseline and Weeks 52 and 104.

ID	Title	Description
OG000	Mipomersen 200 mg Per Week	Participants received 200 mg mipomersen once a week by subcutaneous injection, for up to 3 years.
OG001	Mipomersen 200 mg Every Other Week	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.

Units	Counts

Secondary

Percent Change From Baseline in Total Cholesterol

Total cholesterol was measured in mg/dL. Samples were taken following an overnight fast.

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.

Posted

Median

Inter-Quartile Range

percentage of baseline

Baseline and Weeks 52 and 104.

ID	Title	Description
OG000	Mipomersen 200 mg Per Week	Participants received 200 mg mipomersen once a week by subcutaneous injection, for up to 3 years.
OG001	Mipomersen 200 mg Every Other Week	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.

Units	Counts
Participants	OG000

Secondary

Total Cholesterol Over Time

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.

Posted

Median

Inter-Quartile Range

mg/dL

Baseline and Weeks 52 and 104.

ID	Title	Description
OG000	Mipomersen 200 mg Per Week	Participants received 200 mg mipomersen once a week by subcutaneous injection, for up to 3 years.
OG001	Mipomersen 200 mg Every Other Week	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.

Units	Counts

Secondary

Percent Change From Baseline in Non-High-Density Lipoprotein Cholesterol

Non-high-density lipoprotein cholesterol was measured in mg/dL. Samples were taken following an overnight fast.

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.

Posted

Median

Inter-Quartile Range

percentage of baseline

Baseline and Weeks 52 and 104.

ID	Title	Description
OG000	Mipomersen 200 mg Per Week	Participants received 200 mg mipomersen once a week by subcutaneous injection, for up to 3 years.
OG001	Mipomersen 200 mg Every Other Week	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.

Units	Counts
Participants

Secondary

Non-High-Density Lipoprotein Cholesterol Over Time

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.

Posted

Median

Inter-Quartile Range

mg/dL

Baseline and Weeks 52 and 104.

ID	Title	Description
OG000	Mipomersen 200 mg Per Week	Participants received 200 mg mipomersen once a week by subcutaneous injection, for up to 3 years.
OG001	Mipomersen 200 mg Every Other Week	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.

Units	Counts

Secondary

Number of Participants With Treatment-Emergent Adverse Events (AEs)

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.

Safety set

Posted

Number

participants

2 years

ID	Title	Description
OG000	Mipomersen 200 mg Per Week	Participants received 200 mg mipomersen once a week by subcutaneous injection, for up to 3 years.
OG001	Mipomersen 200 mg Every Other Week	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.

Secondary

Percent Change From Baseline in Clinical Chemistry Parameters

Safety set.

Posted

Mean

Standard Deviation

percentage of baseline

Baseline and Week 104 or the Early Termination visit for participants who did not complete 2 years of treatment.

ID	Title	Description
OG000	Mipomersen 200 mg Per Week	Participants received 200 mg mipomersen once a week by subcutaneous injection, for up to 3 years.
OG001	Mipomersen 200 mg Every Other Week	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.

Units	Counts
Participants	OG000

Secondary

Percent Change From Baseline in Hematology Parameters

Safety set

Posted

Mean

Standard Deviation

percentage of baseline

Baseline and Week 104 or the Early Termination visit for participants who did not complete 2 years of treatment

ID	Title	Description
OG000	Mipomersen 200 mg Per Week	Participants received 200 mg mipomersen once a week by subcutaneous injection, for up to 3 years.
OG001	Mipomersen 200 mg Every Other Week	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.

Units	Counts
Participants	OG000

Secondary

Percent Change From Baseline in Blood Pressure

Safety set

Posted

Mean

Standard Deviation

percentage of baseline

Baseline and Week 104 or the Early Termination visit for participants who did not complete 2 years of treatment.

ID	Title	Description
OG000	Mipomersen 200 mg Per Week	Participants received 200 mg mipomersen once a week by subcutaneous injection, for up to 3 years.
OG001	Mipomersen 200 mg Every Other Week	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.

Units	Counts
Participants	OG000

Secondary

Percent Change From Baseline in Pulse Rate

Safety set

Posted

Mean

Standard Deviation

percentage of baseline

Baseline and Week 104 or the Early Termination visit for participants who did not complete 2 years of treatment.

ID	Title	Description
OG000	Mipomersen 200 mg Per Week	Participants received 200 mg mipomersen once a week by subcutaneous injection, for up to 3 years.
OG001	Mipomersen 200 mg Every Other Week	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.

Units	Counts
Participants	OG000

Secondary

Percent Change From Baseline in Respiratory Rate

Safety set

Posted

Mean

Standard Deviation

percentage of baseline

Baseline and Week 104 or the Early Termination visit for participants who did not complete 2 years of treatment.

ID	Title	Description
OG000	Mipomersen 200 mg Per Week	Participants received 200 mg mipomersen once a week by subcutaneous injection, for up to 3 years.
OG001	Mipomersen 200 mg Every Other Week	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.

Units	Counts
Participants	OG000

Other Pre-specified

Percent Change From Baseline in Triglycerides

Triglycerides were measured in mg/dL. Samples were taken following an overnight fast.

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.

Posted

Median

Inter-Quartile Range

percentage of baseline

Baseline and Weeks 52 and 104.

ID	Title	Description
OG000	Mipomersen 200 mg Per Week	Participants received 200 mg mipomersen once a week by subcutaneous injection, for up to 3 years.
OG001	Mipomersen 200 mg Every Other Week	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.

Units	Counts
Participants	OG000

Other Pre-specified

Triglycerides Over Time

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.

Posted

Median

Inter-Quartile Range

mg/dL

Baseline and Weeks 52 and 104.

ID	Title	Description
OG000	Mipomersen 200 mg Per Week	Participants received 200 mg mipomersen once a week by subcutaneous injection, for up to 3 years.
OG001	Mipomersen 200 mg Every Other Week	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.

Units	Counts

Other Pre-specified

Percent Change From Baseline in Lipoprotein(a)

Lipoprotein(a) was measured in mg/dL. Samples were taken following an overnight fast.

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.

Posted

Median

Inter-Quartile Range

percentage of baseline

Baseline and Weeks 52 and 104.

ID	Title	Description
OG000	Mipomersen 200 mg Per Week	Participants received 200 mg mipomersen once a week by subcutaneous injection, for up to 3 years.
OG001	Mipomersen 200 mg Every Other Week	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.

Units	Counts
Participants	OG000

Other Pre-specified

Lipoprotein(a) Over Time

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.

Posted

Median

Inter-Quartile Range

mg/dL

Baseline and Weeks 52 and 104.

ID	Title	Description
OG000	Mipomersen 200 mg Per Week	Participants received 200 mg mipomersen once a week by subcutaneous injection, for up to 3 years.
OG001	Mipomersen 200 mg Every Other Week	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.

Units	Counts

Other Pre-specified

Percent Change From Baseline in Very-Low-Density Lipoprotein (VLDL) Cholesterol

Very-Low-Density Lipoprotein (VLDL) Cholesterol was measured in mg/dL. Samples were taken following an overnight fast.

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.

Posted

Median

Inter-Quartile Range

percentage of baseline

Baseline and Weeks 52 and 104.

ID	Title	Description
OG000	Mipomersen 200 mg Per Week	Participants received 200 mg mipomersen once a week by subcutaneous injection, for up to 3 years.
OG001	Mipomersen 200 mg Every Other Week	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.

Units	Counts
Participants

Other Pre-specified

Very-Low-Density Lipoprotein (VLDL) Cholesterol Over Time

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.

Posted

Median

Inter-Quartile Range

mg/dL

Baseline and Weeks 52 and 104.

ID	Title	Description
OG000	Mipomersen 200 mg Per Week	Participants received 200 mg mipomersen once a week by subcutaneous injection, for up to 3 years.
OG001	Mipomersen 200 mg Every Other Week	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.

Units

Other Pre-specified

Percent Change From Baseline in Ratio of Low-density Lipoprotein Cholesterol to High-density Lipoprotein Cholesterol

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.

Posted

Median

Inter-Quartile Range

percentage of baseline

Baseline and Weeks 52 and 104.

ID	Title	Description
OG000	Mipomersen 200 mg Per Week	Participants received 200 mg mipomersen once a week by subcutaneous injection, for up to 3 years.
OG001	Mipomersen 200 mg Every Other Week	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.

Units	Counts
Participants	OG000

Other Pre-specified

Ratio of Low-density Lipoprotein Cholesterol to High-density Lipoprotein Cholesterol Over Time

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.

Posted

Median

Inter-Quartile Range

ratio

Baseline and Weeks 52 and 104.

ID	Title	Description
OG000	Mipomersen 200 mg Per Week	Participants received 200 mg mipomersen once a week by subcutaneous injection, for up to 3 years.
OG001	Mipomersen 200 mg Every Other Week	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.

Other Pre-specified

Percent Change From Baseline in Apolipoprotein A1

Apolipoprotein A1 was measured in mg/dL. Samples were taken following an overnight fast.

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.

Posted

Median

Inter-Quartile Range

percentage of baseline

Baseline and Weeks 52 and 104.

ID	Title	Description
OG000	Mipomersen 200 mg Per Week	Participants received 200 mg mipomersen once a week by subcutaneous injection, for up to 3 years.
OG001	Mipomersen 200 mg Every Other Week	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.

Units	Counts
Participants	OG000

Other Pre-specified

Apolipoprotein A1 Over Time

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.

Posted

Median

Inter-Quartile Range

mg/dL

Baseline and Weeks 52 and 104.

ID	Title	Description
OG000	Mipomersen 200 mg Per Week	Participants received 200 mg mipomersen once a week by subcutaneous injection, for up to 3 years.
OG001	Mipomersen 200 mg Every Other Week	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.

Units	Counts

Other Pre-specified

Percent Change From Baseline in High-Density Lipoprotein Cholesterol

High-Density Lipoprotein (HDL) Cholesterol was measured in mg/dL. Samples were taken following an overnight fast.

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.

Posted

Median

Inter-Quartile Range

percentage of baseline

Baseline and Weeks 52 and 104.

ID	Title	Description
OG000	Mipomersen 200 mg Per Week	Participants received 200 mg mipomersen once a week by subcutaneous injection, for up to 3 years.
OG001	Mipomersen 200 mg Every Other Week	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.

Units	Counts
Participants

Other Pre-specified

High-Density Lipoprotein Cholesterol Over Time

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.

Posted

Median

Inter-Quartile Range

mg/dL

Baseline and Weeks 52 and 104.

ID	Title	Description
OG000	Mipomersen 200 mg Per Week	Participants received 200 mg mipomersen once a week by subcutaneous injection, for up to 3 years.
OG001	Mipomersen 200 mg Every Other Week	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.

Units

Other Adverse Events

Term	Organ System	Source Vocabulary	Assessment Type	Statistical Information
Lymphadenopathy	Blood and lymphatic system disorders	MedDRA 13.1	Systematic Assessment	EG0002 affected16 at risk EG0010 affected5 at risk
Splenomegaly	Blood and lymphatic system disorders	MedDRA 13.1	Systematic Assessment	EG0001 affected16 at risk EG0010 affected5 at risk
Angina pectoris	Cardiac disorders	MedDRA 13.1	Systematic Assessment	EG0000 affected16 at risk EG0012 affected5 at risk
Cardiomegaly	Cardiac disorders	MedDRA 13.1	Systematic Assessment	EG0001 affected16 at risk EG0010 affected5 at risk
Extrasystoles	Cardiac disorders	MedDRA 13.1	Systematic Assessment	EG0001 affected16 at risk EG0010 affected5 at risk
Arteriovenous malformation	Congenital, familial and genetic disorders	MedDRA 13.1	Systematic Assessment	EG0000 affected16 at risk EG0011 affected5 at risk
Ear discomfort	Ear and labyrinth disorders	MedDRA 13.1	Systematic Assessment	EG0001 affected16 at risk EG0010 affected5 at risk
Thyroid cyst	Endocrine disorders	MedDRA 13.1	Systematic Assessment	EG0001 affected16 at risk EG0010 affected5 at risk
Arcus lipoides	Eye disorders	MedDRA 13.1	Systematic Assessment	EG0001 affected16 at risk EG0010 affected5 at risk
Conjunctival haemorrhage	Eye disorders	MedDRA 13.1	Systematic Assessment	EG0001 affected16 at risk EG0010 affected5 at risk
Eye irritation	Eye disorders	MedDRA 13.1	Systematic Assessment	EG0001 affected16 at risk EG0010 affected5 at risk
Abdominal pain	Gastrointestinal disorders	MedDRA 13.1	Systematic Assessment	EG0001 affected16 at risk EG0011 affected5 at risk
Abdominal pain upper	Gastrointestinal disorders	MedDRA 13.1	Systematic Assessment	EG0001 affected16 at risk EG0010 affected5 at risk
Dental caries	Gastrointestinal disorders	MedDRA 13.1	Systematic Assessment	EG0001 affected16 at risk EG0010 affected5 at risk
Diarrhoea	Gastrointestinal disorders	MedDRA 13.1	Systematic Assessment	EG0002 affected16 at risk EG0010 affected5 at risk
Diverticulum intestinal	Gastrointestinal disorders	MedDRA 13.1	Systematic Assessment	EG0001 affected16 at risk EG0010 affected5 at risk
Gastrooesophageal reflux disease	Gastrointestinal disorders	MedDRA 13.1	Systematic Assessment	EG0000 affected16 at risk EG0011 affected5 at risk
Haemorrhoids	Gastrointestinal disorders	MedDRA 13.1	Systematic Assessment	EG0001 affected16 at risk EG0010 affected5 at risk
Hypoaesthesia oral	Gastrointestinal disorders	MedDRA 13.1	Systematic Assessment	EG0001 affected16 at risk EG0010 affected5 at risk
Lip swelling	Gastrointestinal disorders	MedDRA 13.1	Systematic Assessment	EG0001 affected16 at risk EG0010 affected5 at risk
Nausea	Gastrointestinal disorders	MedDRA 13.1	Systematic Assessment	EG0003 affected16 at risk EG0012 affected5 at risk
Toothache	Gastrointestinal disorders	MedDRA 13.1	Systematic Assessment	EG0001 affected16 at risk EG0010 affected5 at risk
Vomiting	Gastrointestinal disorders	MedDRA 13.1	Systematic Assessment	EG0002 affected16 at risk EG0010 affected5 at risk
Adverse drug reaction	General disorders	MedDRA 13.1	Systematic Assessment	EG0001 affected16 at risk EG0010 affected5 at risk
Chest pain	General disorders	MedDRA 13.1	Systematic Assessment	EG0001 affected16 at risk EG0010 affected5 at risk
Chills	General disorders	MedDRA 13.1	Systematic Assessment	EG0001 affected16 at risk EG0010 affected5 at risk
Fatigue	General disorders	MedDRA 13.1	Systematic Assessment	EG0002 affected16 at risk EG0011 affected5 at risk
Feeling jittery	General disorders	MedDRA 13.1	Systematic Assessment	EG0000 affected16 at risk EG0011 affected5 at risk
Influenza like illness	General disorders	MedDRA 13.1	Systematic Assessment	EG0002 affected16 at risk EG0013 affected5 at risk
Injection site anaesthesia	General disorders	MedDRA 13.1	Systematic Assessment	EG0001 affected16 at risk EG0010 affected5 at risk
Injection site coldness	General disorders	MedDRA 13.1	Systematic Assessment	EG0001 affected16 at risk EG0010 affected5 at risk
Injection site discolouration	General disorders	MedDRA 13.1	Systematic Assessment	EG0003 affected16 at risk EG0011 affected5 at risk
Injection site erythema	General disorders	MedDRA 13.1	Systematic Assessment	EG00010 affected16 at risk EG0012 affected5 at risk
Injection site haematoma	General disorders	MedDRA 13.1	Systematic Assessment	EG0001 affected16 at risk EG0013 affected5 at risk
Injection site haemorrhage	General disorders	MedDRA 13.1	Systematic Assessment	EG0002 affected16 at risk EG0010 affected5 at risk
Injection site hypertrophy	General disorders	MedDRA 13.1	Systematic Assessment	EG0002 affected16 at risk EG0010 affected5 at risk
Injection site pain	General disorders	MedDRA 13.1	Systematic Assessment	EG0003 affected16 at risk EG0012 affected5 at risk
Injection site pruritus	General disorders	MedDRA 13.1	Systematic Assessment	EG0002 affected16 at risk EG0012 affected5 at risk
Injection site rash	General disorders	MedDRA 13.1	Systematic Assessment	EG0001 affected16 at risk EG0010 affected5 at risk
Injection site reaction	General disorders	MedDRA 13.1	Systematic Assessment	EG0007 affected16 at risk EG0011 affected5 at risk
Injection site swelling	General disorders	MedDRA 13.1	Systematic Assessment	EG0001 affected16 at risk EG0012 affected5 at risk
Injection site urticaria	General disorders	MedDRA 13.1	Systematic Assessment	EG0001 affected16 at risk EG0011 affected5 at risk
Malaise	General disorders	MedDRA 13.1	Systematic Assessment	EG0001 affected16 at risk EG0010 affected5 at risk
Pain	General disorders	MedDRA 13.1	Systematic Assessment	EG0000 affected16 at risk EG0011 affected5 at risk
Pyrexia	General disorders	MedDRA 13.1	Systematic Assessment	EG0001 affected16 at risk EG0010 affected5 at risk
Cholelithiasis	Hepatobiliary disorders	MedDRA 13.1	Systematic Assessment	EG0000 affected16 at risk EG0011 affected5 at risk
Hepatic steatosis	Hepatobiliary disorders	MedDRA 13.1	Systematic Assessment	EG0002 affected16 at risk EG0010 affected5 at risk
Hypersensitivity	Immune system disorders	MedDRA 13.1	Systematic Assessment	EG0001 affected16 at risk EG0010 affected5 at risk
Acarodermatitis	Infections and infestations	MedDRA 13.1	Systematic Assessment	EG0001 affected16 at risk EG0010 affected5 at risk
Cellulitis	Infections and infestations	MedDRA 13.1	Systematic Assessment	EG0001 affected16 at risk EG0010 affected5 at risk
Cystitis	Infections and infestations	MedDRA 13.1	Systematic Assessment	EG0001 affected16 at risk EG0010 affected5 at risk
Ear infection	Infections and infestations	MedDRA 13.1	Systematic Assessment	EG0001 affected16 at risk EG0010 affected5 at risk
Influenza	Infections and infestations	MedDRA 13.1	Systematic Assessment	EG0003 affected16 at risk EG0010 affected5 at risk
Nasopharyngitis	Infections and infestations	MedDRA 13.1	Systematic Assessment	EG0002 affected16 at risk EG0011 affected5 at risk
Sinusitis	Infections and infestations	MedDRA 13.1	Systematic Assessment	EG0001 affected16 at risk EG0010 affected5 at risk
Tooth infection	Infections and infestations	MedDRA 13.1	Systematic Assessment	EG0001 affected16 at risk EG0010 affected5 at risk
Upper respiratory tract infection	Infections and infestations	MedDRA 13.1	Systematic Assessment	EG0002 affected16 at risk EG0010 affected5 at risk
Back injury	Injury, poisoning and procedural complications	MedDRA 13.1	Systematic Assessment	EG0001 affected16 at risk EG0010 affected5 at risk
Contusion	Injury, poisoning and procedural complications	MedDRA 13.1	Systematic Assessment	EG0001 affected16 at risk EG0011 affected5 at risk
Excoriation	Injury, poisoning and procedural complications	MedDRA 13.1	Systematic Assessment	EG0001 affected16 at risk EG0010 affected5 at risk
Foot fracture	Injury, poisoning and procedural complications	MedDRA 13.1	Systematic Assessment	EG0001 affected16 at risk EG0010 affected5 at risk
Joint injury	Injury, poisoning and procedural complications	MedDRA 13.1	Systematic Assessment	EG0001 affected16 at risk EG0010 affected5 at risk
Joint sprain	Injury, poisoning and procedural complications	MedDRA 13.1	Systematic Assessment	EG0001 affected16 at risk EG0010 affected5 at risk
Limb injury	Injury, poisoning and procedural complications	MedDRA 13.1	Systematic Assessment	EG0001 affected16 at risk EG0011 affected5 at risk
Muscle strain	Injury, poisoning and procedural complications	MedDRA 13.1	Systematic Assessment	EG0002 affected16 at risk EG0010 affected5 at risk
Post procedural discomfort	Injury, poisoning and procedural complications	MedDRA 13.1	Systematic Assessment	EG0001 affected16 at risk EG0010 affected5 at risk
Procedural pain	Injury, poisoning and procedural complications	MedDRA 13.1	Systematic Assessment	EG0001 affected16 at risk EG0010 affected5 at risk
Skin laceration	Injury, poisoning and procedural complications	MedDRA 13.1	Systematic Assessment	EG0002 affected16 at risk EG0010 affected5 at risk
Splinter	Injury, poisoning and procedural complications	MedDRA 13.1	Systematic Assessment	EG0000 affected16 at risk EG0011 affected5 at risk
Wrong technique in drug usage process	Injury, poisoning and procedural complications	MedDRA 13.1	Systematic Assessment	EG0001 affected16 at risk EG0010 affected5 at risk
Alanine aminotransferase increased	Investigations	MedDRA 13.1	Systematic Assessment	EG0004 affected16 at risk EG0011 affected5 at risk
Aspartate aminotransferase increased	Investigations	MedDRA 13.1	Systematic Assessment	EG0003 affected16 at risk EG0010 affected5 at risk
Blood glucose increased	Investigations	MedDRA 13.1	Systematic Assessment	EG0001 affected16 at risk EG0010 affected5 at risk
Blood urine present	Investigations	MedDRA 13.1	Systematic Assessment	EG0001 affected16 at risk EG0010 affected5 at risk
Hepatic enzyme increased	Investigations	MedDRA 13.1	Systematic Assessment	EG0005 affected16 at risk EG0010 affected5 at risk
Platelet count decreased	Investigations	MedDRA 13.1	Systematic Assessment	EG0001 affected16 at risk EG0010 affected5 at risk
Protein urine present	Investigations	MedDRA 13.1	Systematic Assessment	EG0002 affected16 at risk EG0010 affected5 at risk
Urine analysis abnormal	Investigations	MedDRA 13.1	Systematic Assessment	EG0001 affected16 at risk EG0010 affected5 at risk
Decreased appetite	Metabolism and nutrition disorders	MedDRA 13.1	Systematic Assessment	EG0001 affected16 at risk EG0010 affected5 at risk
Increased appetite	Metabolism and nutrition disorders	MedDRA 13.1	Systematic Assessment	EG0000 affected16 at risk EG0011 affected5 at risk
Arthralgia	Musculoskeletal and connective tissue disorders	MedDRA 13.1	Systematic Assessment	EG0004 affected16 at risk EG0011 affected5 at risk
Back pain	Musculoskeletal and connective tissue disorders	MedDRA 13.1	Systematic Assessment	EG0001 affected16 at risk EG0010 affected5 at risk
Exostosis	Musculoskeletal and connective tissue disorders	MedDRA 13.1	Systematic Assessment	EG0001 affected16 at risk EG0010 affected5 at risk
Myalgia	Musculoskeletal and connective tissue disorders	MedDRA 13.1	Systematic Assessment	EG0002 affected16 at risk EG0010 affected5 at risk
Pain in extremity	Musculoskeletal and connective tissue disorders	MedDRA 13.1	Systematic Assessment	EG0001 affected16 at risk EG0011 affected5 at risk
Rotator cuff syndrome	Musculoskeletal and connective tissue disorders	MedDRA 13.1	Systematic Assessment	EG0001 affected16 at risk EG0010 affected5 at risk
Sensation of heaviness	Musculoskeletal and connective tissue disorders	MedDRA 13.1	Systematic Assessment	EG0000 affected16 at risk EG0011 affected5 at risk
Angiomyolipoma	Neoplasms benign, malignant and unspecified (incl cysts and polyps)	MedDRA 13.1	Systematic Assessment	EG0001 affected16 at risk EG0010 affected5 at risk
Dizziness	Nervous system disorders	MedDRA 13.1	Systematic Assessment	EG0000 affected16 at risk EG0012 affected5 at risk
Headache	Nervous system disorders	MedDRA 13.1	Systematic Assessment	EG0003 affected16 at risk EG0010 affected5 at risk
Hypoaesthesia	Nervous system disorders	MedDRA 13.1	Systematic Assessment	EG0001 affected16 at risk EG0010 affected5 at risk
Nerve compression	Nervous system disorders	MedDRA 13.1	Systematic Assessment	EG0001 affected16 at risk EG0010 affected5 at risk
Sciatica	Nervous system disorders	MedDRA 13.1	Systematic Assessment	EG0000 affected16 at risk EG0011 affected5 at risk
Tremor	Nervous system disorders	MedDRA 13.1	Systematic Assessment	EG0001 affected16 at risk EG0010 affected5 at risk
Anxiety	Psychiatric disorders	MedDRA 13.1	Systematic Assessment	EG0001 affected16 at risk EG0010 affected5 at risk
Grief reaction	Psychiatric disorders	MedDRA 13.1	Systematic Assessment	EG0001 affected16 at risk EG0010 affected5 at risk
Insomnia	Psychiatric disorders	MedDRA 13.1	Systematic Assessment	EG0000 affected16 at risk EG0011 affected5 at risk
Mood altered	Psychiatric disorders	MedDRA 13.1	Systematic Assessment	EG0000 affected16 at risk EG0011 affected5 at risk
Chromaturia	Renal and urinary disorders	MedDRA 13.1	Systematic Assessment	EG0000 affected16 at risk EG0011 affected5 at risk
Hypertonic bladder	Renal and urinary disorders	MedDRA 13.1	Systematic Assessment	EG0001 affected16 at risk EG0010 affected5 at risk
Proteinuria	Renal and urinary disorders	MedDRA 13.1	Systematic Assessment	EG0001 affected16 at risk EG0010 affected5 at risk
Renal cyst	Renal and urinary disorders	MedDRA 13.1	Systematic Assessment	EG0001 affected16 at risk EG0010 affected5 at risk
Breast cyst	Reproductive system and breast disorders	MedDRA 13.1	Systematic Assessment	EG0001 affected16 at risk EG0010 affected5 at risk
Breast mass	Reproductive system and breast disorders	MedDRA 13.1	Systematic Assessment	EG0000 affected16 at risk EG0011 affected5 at risk
Asthma	Respiratory, thoracic and mediastinal disorders	MedDRA 13.1	Systematic Assessment	EG0001 affected16 at risk EG0010 affected5 at risk
Cough	Respiratory, thoracic and mediastinal disorders	MedDRA 13.1	Systematic Assessment	EG0000 affected16 at risk EG0011 affected5 at risk
Dyspnoea	Respiratory, thoracic and mediastinal disorders	MedDRA 13.1	Systematic Assessment	EG0000 affected16 at risk EG0011 affected5 at risk
Emphysema	Respiratory, thoracic and mediastinal disorders	MedDRA 13.1	Systematic Assessment	EG0001 affected16 at risk EG0010 affected5 at risk
Increased upper airway secretion	Respiratory, thoracic and mediastinal disorders	MedDRA 13.1	Systematic Assessment	EG0001 affected16 at risk EG0010 affected5 at risk
Respiratory tract congestion	Respiratory, thoracic and mediastinal disorders	MedDRA 13.1	Systematic Assessment	EG0001 affected16 at risk EG0010 affected5 at risk
Rhinorrhoea	Respiratory, thoracic and mediastinal disorders	MedDRA 13.1	Systematic Assessment	EG0000 affected16 at risk EG0011 affected5 at risk
Sinus congestion	Respiratory, thoracic and mediastinal disorders	MedDRA 13.1	Systematic Assessment	EG0001 affected16 at risk EG0010 affected5 at risk
Throat irritation	Respiratory, thoracic and mediastinal disorders	MedDRA 13.1	Systematic Assessment	EG0000 affected16 at risk EG0011 affected5 at risk
Pruritus	Skin and subcutaneous tissue disorders	MedDRA 13.1	Systematic Assessment	EG0001 affected16 at risk EG0010 affected5 at risk
Rash	Skin and subcutaneous tissue disorders	MedDRA 13.1	Systematic Assessment	EG0001 affected16 at risk EG0010 affected5 at risk
Rash erythematous	Skin and subcutaneous tissue disorders	MedDRA 13.1	Systematic Assessment	EG0001 affected16 at risk EG0010 affected5 at risk
Rash generalised	Skin and subcutaneous tissue disorders	MedDRA 13.1	Systematic Assessment	EG0001 affected16 at risk EG0010 affected5 at risk
Skin irritation	Skin and subcutaneous tissue disorders	MedDRA 13.1	Systematic Assessment	EG0000 affected16 at risk EG0011 affected5 at risk
Urticaria	Skin and subcutaneous tissue disorders	MedDRA 13.1	Systematic Assessment	EG0001 affected16 at risk EG0010 affected5 at risk
Haematoma	Vascular disorders	MedDRA 13.1	Systematic Assessment	EG0001 affected16 at risk EG0010 affected5 at risk
Hypertension	Vascular disorders	MedDRA 13.1	Systematic Assessment	EG0001 affected16 at risk EG0010 affected5 at risk

ID	Term
D008052	Lipid Metabolism, Inborn Errors
D006938	Hyperlipoproteinemia Type II
D006949	Hyperlipidemias
D008659	Metabolic Diseases
D008661	Metabolism, Inborn Errors
D030342	Genetic Diseases, Inborn
D007232	Infant, Newborn, Diseases
D000013	Congenital Abnormalities
D006937	Hypercholesterolemia
D006951	Hyperlipoproteinemias
D050171	Dyslipidemias
D052439	Lipid Metabolism Disorders
D000090542	Homozygous Familial Hypercholesterolemia

ID	Term
D009358	Congenital, Hereditary, and Neonatal Diseases and Abnormalities
D009750	Nutritional and Metabolic Diseases

ID	Title	Description
BG000	Mipomersen 200 mg Per Week	Participants received 200 mg mipomersen once a week by subcutaneous injection, for up to 3 years.
BG001	Mipomersen 200 mg Every Other Week	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.
BG002	Total	Total of all reporting groups

ID	Title	Description	Deaths (Affected)	Deaths (At Risk)	Serious Events (Affected)	Serious Events (At Risk)	Other Events (Affected)	Other Events (At Risk)
EG000	Mipomersen 200 mg Per Week	Participants received 200 mg mipomersen once a week by subcutaneous injection, for up to 3 years.			4	16	16	16
EG001	Mipomersen 200 mg Every Other Week	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.			3	5	5	5