Trial Outcomes & Findings for Safety and Efficacy of Mipomersen (ISIS 301012) As Add-on Therapy in High Risk Hypercholesterolemic Patients (NCT NCT00770146)

Last Updated: 2016-09-09

Results Overview

LDL cholesterol was measured in mg/dL. Samples were taken following an overnight fast. LDL-C was obtained using Friedewald's calculation for patients with triglycerides ≤400 mg/dL and was directly measured by the central laboratory using ultracentrifugation for patients with triglycerides \>400 mg/dL. The primary efficacy time point was the post-baseline visit closest to 14 days after the last dose of study treatment.

Recruitment status

COMPLETED

Study phase

PHASE3

Target enrollment

158 participants

Primary outcome timeframe

Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET; Week 28 or the post-baseline visit closest to 14 days after the last dose).

Results posted on

2016-09-09

Participant Flow

Five hundred and seventy-seven patients were screened and 158 patients were randomized at 43 study centers in a 2:1 ratio to receive mipomersen or placebo once a week for 26 weeks. Participants who finished treatment or who discontinued prematurely from the study for any reason were assessed for safety for 24 weeks after the last study drug dose.

Participant milestones

Participant milestones
Measure	Placebo Participants received placebo subcutaneous injection once a week for 26 weeks.	Mipomersen Participants received mipomersen 200 mg as a subcutaneous injection once a week for 26 weeks.
Treatment Period STARTED	53	105
Treatment Period Treated	52	105
Treatment Period Full Analysis Set	50	101
Treatment Period COMPLETED	44	60
Treatment Period NOT COMPLETED	9	45
Follow-up Period STARTED	53	105
Follow-up Period COMPLETED	42	85
Follow-up Period NOT COMPLETED	11	20

Reasons for withdrawal

Reasons for withdrawal
Measure	Placebo Participants received placebo subcutaneous injection once a week for 26 weeks.	Mipomersen Participants received mipomersen 200 mg as a subcutaneous injection once a week for 26 weeks.
Treatment Period Withdrawal by Subject	6	13
Treatment Period Adverse Event	2	26
Treatment Period Other	1	5
Treatment Period Protocol non-compliance	0	1
Follow-up Period Withdrawal by Subject	7	13
Follow-up Period Adverse Event	0	2
Follow-up Period Other	4	5

Baseline Characteristics

Safety and Efficacy of Mipomersen (ISIS 301012) As Add-on Therapy in High Risk Hypercholesterolemic Patients

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Placebo n=52 Participants Participants received placebo subcutaneous injection once a week for 26 weeks.	Mipomersen n=105 Participants Participants received mipomersen 200 mg as a subcutaneous injection once a week for 26 weeks.	Total n=157 Participants Total of all reporting groups
Age, Continuous	59.3 years STANDARD_DEVIATION 9.5 • n=5 Participants	59.3 years STANDARD_DEVIATION 10.0 • n=7 Participants	59.3 years STANDARD_DEVIATION 9.8 • n=5 Participants
Sex: Female, Male Female	23 Participants n=5 Participants	53 Participants n=7 Participants	76 Participants n=5 Participants
Sex: Female, Male Male	29 Participants n=5 Participants	52 Participants n=7 Participants	81 Participants n=5 Participants
Race/Ethnicity, Customized White	40 participants n=5 Participants	83 participants n=7 Participants	123 participants n=5 Participants
Race/Ethnicity, Customized Black	11 participants n=5 Participants	20 participants n=7 Participants	31 participants n=5 Participants
Race/Ethnicity, Customized American Indian or Alaskan Native	1 participants n=5 Participants	1 participants n=7 Participants	2 participants n=5 Participants
Race/Ethnicity, Customized Other race	0 participants n=5 Participants	1 participants n=7 Participants	1 participants n=5 Participants
Race/Ethnicity, Customized Hispanic or Latino	9 participants n=5 Participants	16 participants n=7 Participants	25 participants n=5 Participants
Race/Ethnicity, Customized Not Hispanic or Latino	43 participants n=5 Participants	89 participants n=7 Participants	132 participants n=5 Participants
Body Mass Index (BMI)	30.0 kg/m^2 STANDARD_DEVIATION 4.42 • n=5 Participants	30.7 kg/m^2 STANDARD_DEVIATION 4.61 • n=7 Participants	30.4 kg/m^2 STANDARD_DEVIATION 4.55 • n=5 Participants
Waist/hip ratio	0.94 ratio STANDARD_DEVIATION 0.07 • n=5 Participants	0.94 ratio STANDARD_DEVIATION 0.06 • n=7 Participants	0.94 ratio STANDARD_DEVIATION 0.06 • n=5 Participants
Metabolic syndrome No	12 participants n=5 Participants	32 participants n=7 Participants	44 participants n=5 Participants
Metabolic syndrome Yes	40 participants n=5 Participants	73 participants n=7 Participants	113 participants n=5 Participants
Tobacco use Current	11 participants n=5 Participants	18 participants n=7 Participants	29 participants n=5 Participants
Tobacco use Non-current	19 participants n=5 Participants	31 participants n=7 Participants	50 participants n=5 Participants
Tobacco use Never	22 participants n=5 Participants	56 participants n=7 Participants	78 participants n=5 Participants
Alcohol use Current	20 participants n=5 Participants	50 participants n=7 Participants	70 participants n=5 Participants
Alcohol use Non-current	12 participants n=5 Participants	24 participants n=7 Participants	36 participants n=5 Participants
Alcohol use Never	20 participants n=5 Participants	31 participants n=7 Participants	51 participants n=5 Participants
Diabetic status at screening Yes	30 participants n=5 Participants	58 participants n=7 Participants	88 participants n=5 Participants
Diabetic status at screening No	22 participants n=5 Participants	47 participants n=7 Participants	69 participants n=5 Participants
Cardiovascular history Angina	5 participants n=5 Participants	9 participants n=7 Participants	14 participants n=5 Participants
Cardiovascular history Coronary heart disease (CHD)	21 participants n=5 Participants	52 participants n=7 Participants	73 participants n=5 Participants
Cardiovascular history Myocardial infarction	11 participants n=5 Participants	17 participants n=7 Participants	28 participants n=5 Participants
Cardiovascular history Coronary artery bypass graft surgery	4 participants n=5 Participants	14 participants n=7 Participants	18 participants n=5 Participants
Cardiovascular history Percutaneous coronary intervention	4 participants n=5 Participants	18 participants n=7 Participants	22 participants n=5 Participants
Cardiovascular history Coronary artery disease without event	6 participants n=5 Participants	14 participants n=7 Participants	20 participants n=5 Participants
Cardiovascular history Other clinical atherosclerotic disease	3 participants n=5 Participants	11 participants n=7 Participants	14 participants n=5 Participants
Cardiovascular history Peripheral artery disease	1 participants n=5 Participants	5 participants n=7 Participants	6 participants n=5 Participants
Cardiovascular history Abdominal aortic aneurysm	1 participants n=5 Participants	0 participants n=7 Participants	1 participants n=5 Participants
Cardiovascular history Carotid	1 participants n=5 Participants	8 participants n=7 Participants	9 participants n=5 Participants
Cardiovascular history CHD or other atherosclerotic disease	24 participants n=5 Participants	58 participants n=7 Participants	82 participants n=5 Participants

PRIMARY outcome

Timeframe: Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET; Week 28 or the post-baseline visit closest to 14 days after the last dose).

Population: The Full Analysis Set, which consisted of all randomized patients who received at least 1 injection of study drug (mipomersen or placebo) and with a valid baseline and at least 1 post-baseline LDL-C measurement.

Outcome measures

Outcome measures
Measure	Placebo n=50 Participants Participants received placebo subcutaneous injection once a week for 26 weeks.	Mipomersen n=101 Participants Participants received mipomersen 200 mg as a subcutaneous injection once a week for 26 weeks.
Percent Change From Baseline in Low-density Lipoprotein Cholesterol (LDL-C) at the Primary Efficacy Time Point	-3.4 percentage of baseline Inter-Quartile Range 24.22 • Interval -14.8 to 11.8	-40.2 percentage of baseline Inter-Quartile Range 26.85 • Interval -56.4 to -19.1

PRIMARY outcome

Population: Full analysis set

The primary efficacy time point (PET) was the post-baseline visit closest to 14 days after the last dose of study treatment.

Outcome measures

Outcome measures
Measure	Placebo n=50 Participants Participants received placebo subcutaneous injection once a week for 26 weeks.	Mipomersen n=101 Participants Participants received mipomersen 200 mg as a subcutaneous injection once a week for 26 weeks.
Low-density Lipoprotein Cholesterol (LDL-C) at Baseline and at the Primary Efficacy Time Point Baseline	112 mg/dL Inter-Quartile Range 38.6 • Interval 101.0 to 134.0	116 mg/dL Inter-Quartile Range 31.7 • Interval 106.0 to 137.0
Low-density Lipoprotein Cholesterol (LDL-C) at Baseline and at the Primary Efficacy Time Point Primary efficacy time point	109 mg/dL Inter-Quartile Range 35.1 • Interval 90.0 to 128.0	69 mg/dL Inter-Quartile Range 32.4 • Interval 51.0 to 98.0

SECONDARY outcome

Population: Full analysis set

Apolipoprotein B was measured in mg/dL. Samples were taken following an overnight fast. The primary efficacy time point was the post-baseline visit closest to 14 days after the last dose of study treatment.

Outcome measures

Outcome measures
Measure	Placebo n=50 Participants Participants received placebo subcutaneous injection once a week for 26 weeks.	Mipomersen n=101 Participants Participants received mipomersen 200 mg as a subcutaneous injection once a week for 26 weeks.
Percent Change From Baseline in Apolipoprotein B at the Primary Efficacy Time Point	-1.7 percentage of baseline Inter-Quartile Range 18.09 • Interval -12.6 to 7.5	-40.6 percentage of baseline Inter-Quartile Range 23.59 • Interval -53.9 to -22.6

SECONDARY outcome

Population: Full analysis set

The primary efficacy time point (PET) was the post-baseline visit closest to 14 days after the last dose of study treatment.

Outcome measures

Outcome measures
Measure	Placebo n=50 Participants Participants received placebo subcutaneous injection once a week for 26 weeks.	Mipomersen n=101 Participants Participants received mipomersen 200 mg as a subcutaneous injection once a week for 26 weeks.
Apolipoprotein B at Baseline and at the Primary Efficacy Time Point Baseline	106 mg/dL Inter-Quartile Range 30.1 • Interval 98.0 to 132.0	114 mg/dL Inter-Quartile Range 25.2 • Interval 102.0 to 129.0
Apolipoprotein B at Baseline and at the Primary Efficacy Time Point Primary efficacy time point	108 mg/dL Inter-Quartile Range 27.2 • Interval 91.0 to 122.0	64 mg/dL Inter-Quartile Range 30.7 • Interval 52.0 to 95.0

SECONDARY outcome

Population: Full analysis set

Total cholesterol was measured in mg/dL. Samples were taken following an overnight fast. The primary efficacy time point was the post-baseline visit closest to 14 days after the last dose of study treatment.

Outcome measures

Outcome measures
Measure	Placebo n=50 Participants Participants received placebo subcutaneous injection once a week for 26 weeks.	Mipomersen n=101 Participants Participants received mipomersen 200 mg as a subcutaneous injection once a week for 26 weeks.
Percent Change From Baseline in Total Cholesterol at the Primary Efficacy Time Point	-2.7 percentage of baseline Standard Deviation 14.58	-26.4 percentage of baseline Standard Deviation 18.65

SECONDARY outcome

Population: Full analysis set

The primary efficacy time point (PET) was the post-baseline visit closest to 14 days after the last dose of study treatment.

Outcome measures

Outcome measures
Measure	Placebo n=50 Participants Participants received placebo subcutaneous injection once a week for 26 weeks.	Mipomersen n=101 Participants Participants received mipomersen 200 mg as a subcutaneous injection once a week for 26 weeks.
Total Cholesterol at Baseline and at the Primary Efficacy Time Point Baseline	200.0 mg/dL Standard Deviation 42.1	202.6 mg/dL Standard Deviation 36.8
Total Cholesterol at Baseline and at the Primary Efficacy Time Point Primary efficacy time point	192.2 mg/dL Standard Deviation 38.3	147.4 mg/dL Standard Deviation 39.9

SECONDARY outcome

Population: Full analysis set

Non-high-density lipoprotein cholesterol was measured in mg/dL. Samples were taken following an overnight fast. The primary efficacy time point was the post-baseline visit closest to 14 days after the last dose of study treatment.

Outcome measures

Outcome measures
Measure	Placebo n=50 Participants Participants received placebo subcutaneous injection once a week for 26 weeks.	Mipomersen n=101 Participants Participants received mipomersen 200 mg as a subcutaneous injection once a week for 26 weeks.
Percent Change From Baseline in Non-High-Density Lipoprotein Cholesterol at the Primary Efficacy Time Point	-1.2 percentage of baseline Inter-Quartile Range 20.43 • Interval -13.6 to 11.5	-38.7 percentage of baseline Inter-Quartile Range 23.75 • Interval -54.0 to -24.2

SECONDARY outcome

Population: Full analysis set

The primary efficacy time point (PET) was the post-baseline visit closest to 14 days after the last dose of study treatment.

Outcome measures

Outcome measures
Measure	Placebo n=50 Participants Participants received placebo subcutaneous injection once a week for 26 weeks.	Mipomersen n=101 Participants Participants received mipomersen 200 mg as a subcutaneous injection once a week for 26 weeks.
Non-High-Density Lipoprotein Cholesterol at Baseline and at the Primary Efficacy Time Point Baseline	144 mg/dL Inter-Quartile Range 44.4 • Interval 125.0 to 175.0	144 mg/dL Inter-Quartile Range 35.1 • Interval 132.0 to 171.0
Non-High-Density Lipoprotein Cholesterol at Baseline and at the Primary Efficacy Time Point Primary efficacy time point	140 mg/dL Inter-Quartile Range 38.7 • Interval 115.0 to 165.0	90 mg/dL Inter-Quartile Range 38.3 • Interval 67.0 to 116.0

OTHER_PRE_SPECIFIED outcome

Population: Full analysis set

Triglycerides were measured in mg/dL. Samples were taken following an overnight fast. The primary efficacy time point was the post-baseline visit closest to 14 days after the last dose of study treatment.

Outcome measures

Outcome measures
Measure	Placebo n=50 Participants Participants received placebo subcutaneous injection once a week for 26 weeks.	Mipomersen n=101 Participants Participants received mipomersen 200 mg as a subcutaneous injection once a week for 26 weeks.
Percent Change From Baseline in Triglycerides at the Primary Efficacy Time Point	2.7 percentage of baseline Inter-Quartile Range 53.26 • Interval -24.0 to 24.2	-26.2 percentage of baseline Inter-Quartile Range 29.21 • Interval -48.1 to -8.8

OTHER_PRE_SPECIFIED outcome

Population: Full analysis set

The primary efficacy time point (PET) was the post-baseline visit closest to 14 days after the last dose of study treatment.

Outcome measures

Outcome measures
Measure	Placebo n=50 Participants Participants received placebo subcutaneous injection once a week for 26 weeks.	Mipomersen n=101 Participants Participants received mipomersen 200 mg as a subcutaneous injection once a week for 26 weeks.
Triglycerides at Baseline and at the Primary Efficacy Time Point Baseline	139 mg/dL Inter-Quartile Range 66.1 • Interval 98.0 to 176.0	143 mg/dL Inter-Quartile Range 65.3 • Interval 105.0 to 175.0
Triglycerides at Baseline and at the Primary Efficacy Time Point Primary efficacy time point	135 mg/dL Inter-Quartile Range 97.5 • Interval 96.0 to 177.0	88 mg/dL Inter-Quartile Range 55.7 • Interval 67.0 to 128.0

OTHER_PRE_SPECIFIED outcome

Population: Full analysis set

High-density lipoprotein cholesterol (HDL-C) was measured in mg/dL. Samples were taken following an overnight fast. The primary efficacy time point was the post-baseline visit closest to 14 days after the last dose of study treatment.

Outcome measures

Outcome measures
Measure	Placebo n=50 Participants Participants received placebo subcutaneous injection once a week for 26 weeks.	Mipomersen n=101 Participants Participants received mipomersen 200 mg as a subcutaneous injection once a week for 26 weeks.
Percent Change From Baseline in High Density Lipoprotein Cholesterol (HDL-C) at the Primary Efficacy Time Point	2.2 percentage of baseline Standard Deviation 16.44	2.2 percentage of baseline Standard Deviation 17.99

OTHER_PRE_SPECIFIED outcome

Population: Full analysis set

The primary efficacy time point (PET) was the post-baseline visit closest to 14 days after the last dose of study treatment.

Outcome measures

Outcome measures
Measure	Placebo n=50 Participants Participants received placebo subcutaneous injection once a week for 26 weeks.	Mipomersen n=101 Participants Participants received mipomersen 200 mg as a subcutaneous injection once a week for 26 weeks.
High-Density Lipoprotein Cholesterol at Baseline and at the Primary Efficacy Time Point Baseline	48.4 mg/dL Standard Deviation 15.9	50.8 mg/dL Standard Deviation 12.0
High-Density Lipoprotein Cholesterol at Baseline and at the Primary Efficacy Time Point Primary efficacy time point	48.9 mg/dL Standard Deviation 16.1	51.1 mg/dL Standard Deviation 12.3

OTHER_PRE_SPECIFIED outcome

Population: Full analysis set

Lipoprotein (a) was measured in mg/dL. Samples were taken following an overnight fast. The primary efficacy time point was the post-baseline visit closest to 14 days after the last dose of study treatment.

Outcome measures

Outcome measures
Measure	Placebo n=50 Participants Participants received placebo subcutaneous injection once a week for 26 weeks.	Mipomersen n=101 Participants Participants received mipomersen 200 mg as a subcutaneous injection once a week for 26 weeks.
Percent Change From Baseline in Lipoprotein (a) at the Primary Efficacy Time Point	2.3 percentage of baseline Standard Deviation 28.09	-24.0 percentage of baseline Standard Deviation 24.47

OTHER_PRE_SPECIFIED outcome

Population: Full analysis set

The primary efficacy time point (PET) was the post-baseline visit closest to 14 days after the last dose of study treatment.

Outcome measures

Outcome measures
Measure	Placebo n=50 Participants Participants received placebo subcutaneous injection once a week for 26 weeks.	Mipomersen n=101 Participants Participants received mipomersen 200 mg as a subcutaneous injection once a week for 26 weeks.
Lipoprotein (a) at Baseline and at the Primary Efficacy Time Point Baseline	51.1 mg/dL Standard Deviation 48.6	54.3 mg/dL Standard Deviation 57.0
Lipoprotein (a) at Baseline and at the Primary Efficacy Time Point Primary efficacy time point	49.5 mg/dL Standard Deviation 47.3	39.6 mg/dL Standard Deviation 47.0

OTHER_PRE_SPECIFIED outcome

Population: Full analysis set

Very low density lipoprotein (VLDL) cholesterol was measured in mg/dL. Samples were taken following an overnight fast. The primary efficacy time point was the post-baseline visit closest to 14 days after the last dose of study treatment.

Outcome measures

Outcome measures
Measure	Placebo n=50 Participants Participants received placebo subcutaneous injection once a week for 26 weeks.	Mipomersen n=101 Participants Participants received mipomersen 200 mg as a subcutaneous injection once a week for 26 weeks.
Percent Change From Baseline in Very Low Density Lipoprotein Cholesterol at the Primary Efficacy Time Point	1.9 percentage of baseline Inter-Quartile Range 53.83 • Interval -23.1 to 26.3	-26.7 percentage of baseline Inter-Quartile Range 28.75 • Interval -46.8 to -9.1

OTHER_PRE_SPECIFIED outcome

Population: Full analysis set

The primary efficacy time point (PET) was the post-baseline visit closest to 14 days after the last dose of study treatment.

Outcome measures

Outcome measures
Measure	Placebo n=50 Participants Participants received placebo subcutaneous injection once a week for 26 weeks.	Mipomersen n=101 Participants Participants received mipomersen 200 mg as a subcutaneous injection once a week for 26 weeks.
Very Low Density Lipoprotein Cholesterol at Baseline and at the Primary Efficacy Time Point Baseline	28 mg/dL Inter-Quartile Range 13.2 • Interval 20.0 to 35.0	29 mg/dL Inter-Quartile Range 12.1 • Interval 21.0 to 35.0
Very Low Density Lipoprotein Cholesterol at Baseline and at the Primary Efficacy Time Point Primary efficacy time point	27 mg/dL Inter-Quartile Range 16.3 • Interval 19.0 to 35.0	18 mg/dL Inter-Quartile Range 11.1 • Interval 13.0 to 26.0

OTHER_PRE_SPECIFIED outcome

Population: Full analysis set

The ratio of low-density lipoprotein (LDL) cholesterol to high-density lipoprotein (HDL) cholesterol was measured at Baseline and the post-baseline visit closest to 14 days after the last dose of study treatment.

Outcome measures

Outcome measures
Measure	Placebo n=50 Participants Participants received placebo subcutaneous injection once a week for 26 weeks.	Mipomersen n=101 Participants Participants received mipomersen 200 mg as a subcutaneous injection once a week for 26 weeks.
Percent Change From Baseline in the Ratio of LDL Cholesterol to HDL Cholesterol at the Primary Efficacy Time Point	-5.3 percentage of baseline Standard Deviation 25.31	-37.4 percentage of baseline Standard Deviation 27.24

OTHER_PRE_SPECIFIED outcome

Population: Full analysis set

The primary efficacy time point (PET) was the post-baseline visit closest to 14 days after the last dose of study treatment.

Outcome measures

Outcome measures
Measure	Placebo n=50 Participants Participants received placebo subcutaneous injection once a week for 26 weeks.	Mipomersen n=101 Participants Participants received mipomersen 200 mg as a subcutaneous injection once a week for 26 weeks.
Ratio of LDL Cholesterol to HDL Cholesterol at Baseline and at the Primary Efficacy Time Point Baseline	2.82 ratio Standard Deviation 1.383	2.54 ratio Standard Deviation 0.854
Ratio of LDL Cholesterol to HDL Cholesterol at Baseline and at the Primary Efficacy Time Point Primary efficacy time point	2.54 ratio Standard Deviation 1.147	1.54 ratio Standard Deviation 0.761

OTHER_PRE_SPECIFIED outcome

Population: Full analysis set

Apolipoprotein A1 was measured in mg/dL. Samples were taken following an overnight fast. The primary efficacy time point was the post-baseline visit closest to 14 days after the last dose of study treatment.

Outcome measures

Outcome measures
Measure	Placebo n=50 Participants Participants received placebo subcutaneous injection once a week for 26 weeks.	Mipomersen n=101 Participants Participants received mipomersen 200 mg as a subcutaneous injection once a week for 26 weeks.
Percent Change From Baseline in Apolipoprotein A1 at the Primary Efficacy Time Point	-1.0 percentage of baseline Standard Deviation 11.16	-5.6 percentage of baseline Standard Deviation 12.56

OTHER_PRE_SPECIFIED outcome

Population: Full analysis set

The primary efficacy time point (PET) was the post-baseline visit closest to 14 days after the last dose of study treatment.

Outcome measures

Outcome measures
Measure	Placebo n=50 Participants Participants received placebo subcutaneous injection once a week for 26 weeks.	Mipomersen n=101 Participants Participants received mipomersen 200 mg as a subcutaneous injection once a week for 26 weeks.
Apolipoprotein A1 at Baseline and at the Primary Efficacy Time Point Baseline	150.8 mg/dL Standard Deviation 30.5	156.8 mg/dL Standard Deviation 25.4
Apolipoprotein A1 at Baseline and at the Primary Efficacy Time Point Primary efficacy time point	147.8 mg/dL Standard Deviation 27.3	146.8 mg/dL Standard Deviation 24.5

Adverse Events

Placebo

Serious events: 4 serious events

Other events: 42 other events

Deaths: 0 deaths

Mipomersen

Serious events: 7 serious events

Other events: 97 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Placebo n=52 participants at risk Participants received placebo subcutaneous injection once a week for 26 weeks.	Mipomersen n=105 participants at risk Participants received mipomersen 200 mg as a subcutaneous injection once a week for 26 weeks.
Cardiac disorders Acute coronary syndrome	1.9% 1/52 • Up to Week 28 (2 weeks after last dose) 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.	0.00% 0/105 • Up to Week 28 (2 weeks after last dose) 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.
Cardiac disorders Acute myocardial infarction	1.9% 1/52 • Up to Week 28 (2 weeks after last dose) 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.	0.00% 0/105 • Up to Week 28 (2 weeks after last dose) 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.
Cardiac disorders Angina pectoris	0.00% 0/52 • Up to Week 28 (2 weeks after last dose) 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.	0.95% 1/105 • Up to Week 28 (2 weeks after last dose) 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.
Cardiac disorders Angina unstable	0.00% 0/52 • Up to Week 28 (2 weeks after last dose) 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.	0.95% 1/105 • Up to Week 28 (2 weeks after last dose) 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.
Cardiac disorders Cardiogenic shock	1.9% 1/52 • Up to Week 28 (2 weeks after last dose) 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.	0.00% 0/105 • Up to Week 28 (2 weeks after last dose) 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.
Cardiac disorders Coronary artery disease	0.00% 0/52 • Up to Week 28 (2 weeks after last dose) 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.	0.95% 1/105 • Up to Week 28 (2 weeks after last dose) 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.
General disorders Non-cardiac chest pain	1.9% 1/52 • Up to Week 28 (2 weeks after last dose) 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.	0.95% 1/105 • Up to Week 28 (2 weeks after last dose) 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.
Investigations Electrocardiogram abnormal	1.9% 1/52 • Up to Week 28 (2 weeks after last dose) 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.	0.00% 0/105 • Up to Week 28 (2 weeks after last dose) 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.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Non-small cell lung cancer	0.00% 0/52 • Up to Week 28 (2 weeks after last dose) 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.	0.95% 1/105 • Up to Week 28 (2 weeks after last dose) 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.
Nervous system disorders Hypoaesthesia	0.00% 0/52 • Up to Week 28 (2 weeks after last dose) 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.	0.95% 1/105 • Up to Week 28 (2 weeks after last dose) 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.
Respiratory, thoracic and mediastinal disorders Dyspnoea exertional	1.9% 1/52 • Up to Week 28 (2 weeks after last dose) 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.	0.00% 0/105 • Up to Week 28 (2 weeks after last dose) 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.
Vascular disorders Hypertension	0.00% 0/52 • Up to Week 28 (2 weeks after last dose) 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.	0.95% 1/105 • Up to Week 28 (2 weeks after last dose) 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.

Other adverse events

Additional Information

Genzyme Medical Information

Genzyme Corporation

Phone: 617-252-7832

Results disclosure agreements

Principal investigator is a sponsor employee 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.
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Restriction type: OTHER