Trial Outcomes & Findings for Safety and Efficacy Study of ISIS 301012 (Mipomersen) Administration in High Risk Statin Intolerant Subjects (NCT NCT00707746)

Last Updated: 2016-09-09

Results Overview

LDL-C 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. 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

PHASE2

Target enrollment

34 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

42 patients were screened, 34 participants were randomized, and 33 participants were treated: 21 participants to mipomersen and 12 participants to placebo.

Participant milestones

Participant milestones
Measure	Placebo 1 mL placebo saline, weekly subcutaneous injections for 26 weeks	Mipomersen 200 mg (1 mL) of mipomersen, weekly subcutaneous injections for 26 weeks
Treatment Period STARTED	12	22
Treatment Period Full Analysis Set (FAS) and Safety Set	12	21
Treatment Period COMPLETED	10	17
Treatment Period NOT COMPLETED	2	5
Follow-up Period STARTED	12	21
Follow-up Period COMPLETED	12	21
Follow-up Period NOT COMPLETED	0	0

Reasons for withdrawal

Reasons for withdrawal
Measure	Placebo 1 mL placebo saline, weekly subcutaneous injections for 26 weeks	Mipomersen 200 mg (1 mL) of mipomersen, weekly subcutaneous injections for 26 weeks
Treatment Period Adverse Event	2	4
Treatment Period Ineligibility	0	1

Baseline Characteristics

Safety and Efficacy Study of ISIS 301012 (Mipomersen) Administration in High Risk Statin Intolerant Subjects

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Placebo n=12 Participants 1 mL placebo saline, weekly subcutaneous injections for 26 weeks	Mipomersen n=21 Participants 200 mg (1 mL) of mipomersen, weekly subcutaneous injections for 26 weeks	Total n=33 Participants Total of all reporting groups
Age, Continuous	51.7 years STANDARD_DEVIATION 8.7 • n=5 Participants	55.6 years STANDARD_DEVIATION 6.8 • n=7 Participants	54.2 years STANDARD_DEVIATION 7.6 • n=5 Participants
Sex: Female, Male Female	8 Participants n=5 Participants	10 Participants n=7 Participants	18 Participants n=5 Participants
Sex: Female, Male Male	4 Participants n=5 Participants	11 Participants n=7 Participants	15 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	12 Participants n=5 Participants	21 Participants n=7 Participants	33 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race/Ethnicity, Customized White	12 participants n=5 Participants	20 participants n=7 Participants	32 participants n=5 Participants
Race/Ethnicity, Customized Black	0 participants n=5 Participants	1 participants n=7 Participants	1 participants n=5 Participants
Body Mass Index (BMI)	26.06 kg/m^2 STANDARD_DEVIATION 2.49 • n=5 Participants	26.46 kg/m^2 STANDARD_DEVIATION 3.25 • n=7 Participants	26.31 kg/m^2 STANDARD_DEVIATION 2.96 • n=5 Participants
Waist/hip Ratio	0.92 ratio STANDARD_DEVIATION 0.09 • n=5 Participants	0.93 ratio STANDARD_DEVIATION 0.08 • n=7 Participants	0.93 ratio STANDARD_DEVIATION 0.08 • n=5 Participants
Metabolic syndrome No	4 participants n=5 Participants	12 participants n=7 Participants	16 participants n=5 Participants
Metabolic syndrome Yes	8 participants n=5 Participants	9 participants n=7 Participants	17 participants n=5 Participants
Tobacco Use Current	2 participants n=5 Participants	6 participants n=7 Participants	8 participants n=5 Participants
Tobacco Use Non-current	5 participants n=5 Participants	9 participants n=7 Participants	14 participants n=5 Participants
Tobacco Use Never	5 participants n=5 Participants	6 participants n=7 Participants	11 participants n=5 Participants
Alcohol use Current	8 participants n=5 Participants	16 participants n=7 Participants	24 participants n=5 Participants
Alcohol use Non-current	2 participants n=5 Participants	3 participants n=7 Participants	5 participants n=5 Participants
Alcohol use Never	2 participants n=5 Participants	2 participants n=7 Participants	4 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=12 Participants 1 mL placebo saline, weekly subcutaneous injections for 26 weeks	Mipomersen n=21 Participants 200 mg (1 mL) of mipomersen, weekly subcutaneous injections for 26 weeks
Percent Change From Baseline in Low-density Lipoprotein Cholesterol (LDL-C) at the Primary Efficacy Time Point	-2.0 percentage of baseline Standard Deviation 8.40	-47.3 percentage of baseline Standard Deviation 18.43

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=12 Participants 1 mL placebo saline, weekly subcutaneous injections for 26 weeks	Mipomersen n=21 Participants 200 mg (1 mL) of mipomersen, weekly subcutaneous injections for 26 weeks
Low-density Lipoprotein Cholesterol at Baseline and the Primary Efficacy Time Point Baseline	243.7 mg/dL Standard Deviation 65.6	241.9 mg/dL Standard Deviation 90.8
Low-density Lipoprotein Cholesterol at Baseline and the Primary Efficacy Time Point Primary efficacy time point	236.3 mg/dL Standard Deviation 52.8	128.3 mg/dL Standard Deviation 74.2

PRIMARY outcome

Timeframe: Pre-treatment (prior to first dose), On-treatment (Day 1 to week 28), Post-treatment (Week 28-52)

Population: Safety set of all randomized participants who received at least one injection of study drug.

The on-treatment time frame spanned the time during which the study drug was administered until the later of the primary efficacy time point and 14 days beyond the last study drug date. Adverse events (AEs) were considered related if assessed by the Investigator as possibly, probably or definitely related to study drug. Severity was assessed as: * Mild-symptom barely noticeable to the patient or do not make the patient uncomfortable; * Moderate-symptom makes the patient uncomfortable, affects performance of daily activities; * Severe-symptom causes 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.

Outcome measures

Outcome measures
Measure	Placebo n=12 Participants 1 mL placebo saline, weekly subcutaneous injections for 26 weeks	Mipomersen n=21 Participants 200 mg (1 mL) of mipomersen, weekly subcutaneous injections for 26 weeks
Summary of Participants With Adverse Events Pre-treatment adverse events (AEs)	3 participants	7 participants
Summary of Participants With Adverse Events Post-treatment AEs	9 participants	20 participants
Summary of Participants With Adverse Events On-treatment AEs	12 participants	21 participants
Summary of Participants With Adverse Events On-treatment AEs-related	12 participants	21 participants
Summary of Participants With Adverse Events On-treatment AEs-mild	6 participants	5 participants
Summary of Participants With Adverse Events On-treatment AEs-moderate	5 participants	16 participants
Summary of Participants With Adverse Events On-treatment AEs-severe	1 participants	0 participants
Summary of Participants With Adverse Events On-treatment AEs-leading to trt discontinuation	2 participants	4 participants
Summary of Participants With Adverse Events On-treatment Serious Adverse Events (SAEs)	1 participants	0 participants
Summary of Participants With Adverse Events On-treatment SAEs-related	1 participants	0 participants
Summary of Participants With Adverse Events On-treatment SAEs-mild	0 participants	0 participants
Summary of Participants With Adverse Events On-treatment SAEs-moderate	0 participants	0 participants
Summary of Participants With Adverse Events On-treatment SAEs-severe	1 participants	0 participants
Summary of Participants With Adverse Events On-treatment SAEs-leading to trt discontinuation	1 participants	0 participants
Summary of Participants With Adverse Events Deaths	0 participants	0 participants

SECONDARY outcome

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=12 Participants 1 mL placebo saline, weekly subcutaneous injections for 26 weeks	Mipomersen n=21 Participants 200 mg (1 mL) of mipomersen, weekly subcutaneous injections for 26 weeks
Percent Change From Baseline in Apolipoprotein B at the Primary Efficacy Time Point	-4.0 percentage of baseline Inter-Quartile Range 8.40 • Interval -8.8 to 0.5	-45.8 percentage of baseline Inter-Quartile Range 18.43 • Interval -57.5 to -34.4

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=12 Participants 1 mL placebo saline, weekly subcutaneous injections for 26 weeks	Mipomersen n=21 Participants 200 mg (1 mL) of mipomersen, weekly subcutaneous injections for 26 weeks
Apolipoprotein B at Baseline and at the Primary Efficacy Time Point Baseline	180 mg/dL Inter-Quartile Range 65.6 • Interval 145.0 to 196.0	177 mg/dL Inter-Quartile Range 90.8 • Interval 142.0 to 205.0
Apolipoprotein B at Baseline and at the Primary Efficacy Time Point Primary efficacy time point	173 mg/dL Inter-Quartile Range 52.8 • Interval 143.0 to 187.0	96 mg/dL Inter-Quartile Range 74.2 • Interval 66.0 to 110.0

SECONDARY outcome

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=12 Participants 1 mL placebo saline, weekly subcutaneous injections for 26 weeks	Mipomersen n=21 Participants 200 mg (1 mL) of mipomersen, weekly subcutaneous injections for 26 weeks
Percent Change From Baseline in Total Cholesterol at the Primary Efficacy Time Point	-1.7 percentage of baseline Standard Deviation 6.46 • Interval -8.8 to 0.5	-36.9 percentage of baseline Standard Deviation 14.66 • Interval -57.5 to -34.4

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=12 Participants 1 mL placebo saline, weekly subcutaneous injections for 26 weeks	Mipomersen n=21 Participants 200 mg (1 mL) of mipomersen, weekly subcutaneous injections for 26 weeks
Total Cholesterol at Baseline and at the Primary Efficacy Time Point Baseline	322.3 mg/dL Standard Deviation 66.2	318.9 mg/dL Standard Deviation 91.8
Total Cholesterol at Baseline and at the Primary Efficacy Time Point Primary efficacy time point	314.5 mg/dL Standard Deviation 53.0	200.1 mg/dL Standard Deviation 77.6

SECONDARY outcome

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=12 Participants 1 mL placebo saline, weekly subcutaneous injections for 26 weeks	Mipomersen n=21 Participants 200 mg (1 mL) of mipomersen, weekly subcutaneous injections for 26 weeks
Percent Change From Baseline in Non-High-Density Lipoprotein Cholesterol at the Primary Efficacy Time Point	-1.9 percentage of baseline Standard Deviation 7.06 • Interval -8.8 to 0.5	-45.6 percentage of baseline Standard Deviation 18.22 • Interval -57.5 to -34.4

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=12 Participants 1 mL placebo saline, weekly subcutaneous injections for 26 weeks	Mipomersen n=21 Participants 200 mg (1 mL) of mipomersen, weekly subcutaneous injections for 26 weeks
Non-High-Density Lipoprotein Cholesterol at Baseline and at the Primary Efficacy Time Point Baseline	273.5 mg/dL Standard Deviation 65.3	270.1 mg/dL Standard Deviation 92.9
Non-High-Density Lipoprotein Cholesterol at Baseline and at the Primary Efficacy Time Point Primary efficacy time point	266.0 mg/dL Standard Deviation 53.0	147.7 mg/dL Standard Deviation 81.1

OTHER_PRE_SPECIFIED outcome

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=12 Participants 1 mL placebo saline, weekly subcutaneous injections for 26 weeks	Mipomersen n=21 Participants 200 mg (1 mL) of mipomersen, weekly subcutaneous injections for 26 weeks
Percent Change From Baseline in Triglycerides at the Primary Efficacy Time Point	5.0 percentage of baseline Standard Deviation 26.81	-27.0 percentage of baseline Standard Deviation 30.21

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=12 Participants 1 mL placebo saline, weekly subcutaneous injections for 26 weeks	Mipomersen n=21 Participants 200 mg (1 mL) of mipomersen, weekly subcutaneous injections for 26 weeks
Triglycerides at Baseline and at the Primary Efficacy Time Point Baseline	149.0 mg/dL Standard Deviation 63.9	141.4 mg/dL Standard Deviation 50.9
Triglycerides at Baseline and at the Primary Efficacy Time Point Primary efficacy time point	149.3 mg/dL Standard Deviation 62.4	97.3 mg/dL Standard Deviation 47.3

OTHER_PRE_SPECIFIED outcome

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=12 Participants 1 mL placebo saline, weekly subcutaneous injections for 26 weeks	Mipomersen n=21 Participants 200 mg (1 mL) of mipomersen, weekly subcutaneous injections for 26 weeks
Percent Change From Baseline in Lipoprotein (a) at the Primary Efficacy Time Point	0.0 percentage of baseline Standard Deviation 8.65	-27.1 percentage of baseline Standard Deviation 31.19

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=12 Participants 1 mL placebo saline, weekly subcutaneous injections for 26 weeks	Mipomersen n=21 Participants 200 mg (1 mL) of mipomersen, weekly subcutaneous injections for 26 weeks
Lipoprotein (a) at Baseline and at the Primary Efficacy Time Point Baseline	37.3 mg/dL Standard Deviation 76.3	53.5 mg/dL Standard Deviation 45.8
Lipoprotein (a) at Baseline and at the Primary Efficacy Time Point Primary efficacy time point	41.3 mg/dL Standard Deviation 90.1	42.3 mg/dL Standard Deviation 48.2

OTHER_PRE_SPECIFIED outcome

Very low 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=12 Participants 1 mL placebo saline, weekly subcutaneous injections for 26 weeks	Mipomersen n=21 Participants 200 mg (1 mL) of mipomersen, weekly subcutaneous injections for 26 weeks
Percent Change From Baseline in Very Low Density Lipoprotein Cholesterol at the Primary Efficacy Time Point	4.6 percentage of baseline Standard Deviation 26.53	-27.1 percentage of baseline Standard Deviation 30.79

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=12 Participants 1 mL placebo saline, weekly subcutaneous injections for 26 weeks	Mipomersen n=21 Participants 200 mg (1 mL) of mipomersen, weekly subcutaneous injections for 26 weeks
Very Low Density Lipoprotein Cholesterol at Baseline and at the Primary Efficacy Time Point Baseline	29.8 mg/dL Standard Deviation 12.7	28.3 mg/dL Standard Deviation 10.2
Very Low Density Lipoprotein Cholesterol at Baseline and at the Primary Efficacy Time Point Primary efficacy time point	29.8 mg/dL Standard Deviation 12.5	19.4 mg/dL Standard Deviation 9.5

OTHER_PRE_SPECIFIED outcome

The ratio of low-density lipoprotein (LDL) cholesterol to high-density lipoprotein (HDL) 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=12 Participants 1 mL placebo saline, weekly subcutaneous injections for 26 weeks	Mipomersen n=21 Participants 200 mg (1 mL) of mipomersen, weekly subcutaneous injections for 26 weeks
Percent Change From Baseline in the Ratio of Low-Density Lipoprotein Cholesterol to High-Density Lipoprotein Cholesterol at the Primary Efficacy Time Point	1.4 percentage of baseline Standard Deviation 12.84	-49.2 percentage of baseline Standard Deviation 22.16

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=12 Participants 1 mL placebo saline, weekly subcutaneous injections for 26 weeks	Mipomersen n=21 Participants 200 mg (1 mL) of mipomersen, weekly subcutaneous injections for 26 weeks
Ratio of Low-Density Lipoprotein Cholesterol to High-Density Lipoprotein Cholesterol at Baseline and at the Primary Efficacy Time Point Baseline	5.3 ratio Standard Deviation 2.18	5.2 ratio Standard Deviation 2.38
Ratio of Low-Density Lipoprotein Cholesterol to High-Density Lipoprotein Cholesterol at Baseline and at the Primary Efficacy Time Point Primary efficacy time point	5.4 ratio Standard Deviation 2.27	2.7 ratio Standard Deviation 2.21

OTHER_PRE_SPECIFIED outcome

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=12 Participants 1 mL placebo saline, weekly subcutaneous injections for 26 weeks	Mipomersen n=21 Participants 200 mg (1 mL) of mipomersen, weekly subcutaneous injections for 26 weeks
Percent Change From Baseline in Apolipoprotein A1 at the Primary Efficacy Time Point	-1.2 percentage of baseline Standard Deviation 11.11	-0.0 percentage of baseline Standard Deviation 12.45

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=12 Participants 1 mL placebo saline, weekly subcutaneous injections for 26 weeks	Mipomersen n=21 Participants 200 mg (1 mL) of mipomersen, weekly subcutaneous injections for 26 weeks
Apolipoprotein A1 at Baseline and at the Primary Efficacy Time Point Baseline	150.0 mg/dL Standard Deviation 24.8	150.3 mg/dL Standard Deviation 25.6
Apolipoprotein A1 at Baseline and at the Primary Efficacy Time Point Primary efficacy time point	148.9 mg/dL Standard Deviation 33.2	149.1 mg/dL Standard Deviation 24.0

OTHER_PRE_SPECIFIED outcome

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=12 Participants 1 mL placebo saline, weekly subcutaneous injections for 26 weeks	Mipomersen n=21 Participants 200 mg (1 mL) of mipomersen, weekly subcutaneous injections for 26 weeks
Percent Change From Baseline in High-Density Lipoprotein Cholesterol at the Primary Efficacy Time Point	-2.3 percentage of baseline Standard Deviation 12.72	8.1 percentage of baseline Standard Deviation 17.15

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=12 Participants 1 mL placebo saline, weekly subcutaneous injections for 26 weeks	Mipomersen n=21 Participants 200 mg (1 mL) of mipomersen, weekly subcutaneous injections for 26 weeks
High-Density Lipoprotein Cholesterol at Baseline and at the Primary Efficacy Time Point Baseline	48.8 mg/dL Standard Deviation 12.4	48.8 mg/dL Standard Deviation 9.9
High-Density Lipoprotein Cholesterol at Baseline and at the Primary Efficacy Time Point Primary efficacy time point	48.5 mg/dL Standard Deviation 16.9	52.4 mg/dL Standard Deviation 12.4

Adverse Events

Placebo

Serious events: 1 serious events

Other events: 12 other events

Deaths: 0 deaths

Mipomersen

Serious events: 0 serious events

Other events: 21 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Placebo n=12 participants at risk 1 mL placebo saline, weekly subcutaneous injections for 26 weeks	Mipomersen n=21 participants at risk 200 mg (1 mL) of mipomersen, weekly subcutaneous injections for 26 weeks
Cardiac disorders Acute myocardial infarction	8.3% 1/12 • Day 1 to week 28. On-treatment AEs started on/after the first study drug dose and on/before the end of the treatment period. The treatment period was the time study drug was administered until the later of the PET or 14 days after last study drug dose. The Safety Set includes all randomized patients who receive at least 1 injection of the study treatment. 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% 0/21 • Day 1 to week 28. On-treatment AEs started on/after the first study drug dose and on/before the end of the treatment period. The treatment period was the time study drug was administered until the later of the PET or 14 days after last study drug dose. The Safety Set includes all randomized patients who receive at least 1 injection of the study treatment. 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 Institution will submit for review a proposed publication or presentation at least 90 days prior to submission date. Sponsor has the right to delay publication or presentation for not more than 6 months to address patent applications. Sponsor also has the right to demand in writing the deletion of confidential information. Investigator will not make public raw study data, detailed subject cases, or information identifying any subject.
Publication restrictions are in place

Restriction type: OTHER