Trial Outcomes & Findings for Trial to Evaluate the Effect of Inclisiran Treatment on Low Density Lipoprotein Cholesterol (LDL-C) in Subjects With Heterozygous Familial Hypercholesterolemia (HeFH) (NCT NCT03397121)
NCT ID: NCT03397121
Last Updated: 2020-10-28
Results Overview
Recruitment status
COMPLETED
Study phase
PHASE3
Target enrollment
482 participants
Primary outcome timeframe
Baseline, Day 510
Results posted on
2020-10-28
Participant Flow
Participant milestones
| Measure |
Inclisiran
Inclisiran sodium 300 milligrams (mg) will be administered as a SC injection on Day 1, Day 90 then every 6 months.
Inclisiran: Inclisiran is a small interfering ribonucleic acid (RNA) that inhibits PCSK9 synthesis.
|
Placebo
Placebo will be administered as SC injections of saline solution on Day 1, Day 90 then every 6 months.
Placebo: Placebo will be supplied as sterile normal saline (0.9% sodium chloride in water for injection).
|
|---|---|---|
|
Overall Study
STARTED
|
242
|
240
|
|
Overall Study
COMPLETED
|
235
|
231
|
|
Overall Study
NOT COMPLETED
|
7
|
9
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Trial to Evaluate the Effect of Inclisiran Treatment on Low Density Lipoprotein Cholesterol (LDL-C) in Subjects With Heterozygous Familial Hypercholesterolemia (HeFH)
Baseline characteristics by cohort
| Measure |
Inclisiran
n=242 Participants
Inclisiran sodium 300 milligrams (mg) will be administered as a SC injection on Day 1, Day 90 then every 6 months.
Inclisiran: Inclisiran is a small interfering ribonucleic acid (RNA) that inhibits PCSK9 synthesis.
|
Placebo
n=240 Participants
Placebo will be administered as SC injections of saline solution on Day 1, Day 90 then every 6 months.
Placebo: Placebo will be supplied as sterile normal saline (0.9% sodium chloride in water for injection).
|
Total
n=482 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
189 Participants
n=5 Participants
|
185 Participants
n=7 Participants
|
374 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
53 Participants
n=5 Participants
|
55 Participants
n=7 Participants
|
108 Participants
n=5 Participants
|
|
Age, Continuous
|
54.4 years
STANDARD_DEVIATION 12.48 • n=5 Participants
|
55.0 years
STANDARD_DEVIATION 11.81 • n=7 Participants
|
54.7 years
STANDARD_DEVIATION 12.14 • n=5 Participants
|
|
Sex: Female, Male
Female
|
130 Participants
n=5 Participants
|
125 Participants
n=7 Participants
|
255 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
112 Participants
n=5 Participants
|
115 Participants
n=7 Participants
|
227 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
7 Participants
n=5 Participants
|
8 Participants
n=7 Participants
|
15 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
235 Participants
n=5 Participants
|
232 Participants
n=7 Participants
|
467 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 (NIH/OMB)
American Indian or Alaska Native
|
1 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
7 Participants
n=5 Participants
|
5 Participants
n=7 Participants
|
12 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
8 Participants
n=5 Participants
|
7 Participants
n=7 Participants
|
15 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
226 Participants
n=5 Participants
|
227 Participants
n=7 Participants
|
453 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Region of Enrollment
Canada
|
12 participants
n=5 Participants
|
11 participants
n=7 Participants
|
23 participants
n=5 Participants
|
|
Region of Enrollment
Netherlands
|
19 participants
n=5 Participants
|
19 participants
n=7 Participants
|
38 participants
n=5 Participants
|
|
Region of Enrollment
Sweden
|
18 participants
n=5 Participants
|
16 participants
n=7 Participants
|
34 participants
n=5 Participants
|
|
Region of Enrollment
United States
|
33 participants
n=5 Participants
|
32 participants
n=7 Participants
|
65 participants
n=5 Participants
|
|
Region of Enrollment
Czechia
|
7 participants
n=5 Participants
|
5 participants
n=7 Participants
|
12 participants
n=5 Participants
|
|
Region of Enrollment
Denmark
|
23 participants
n=5 Participants
|
26 participants
n=7 Participants
|
49 participants
n=5 Participants
|
|
Region of Enrollment
South Africa
|
88 participants
n=5 Participants
|
89 participants
n=7 Participants
|
177 participants
n=5 Participants
|
|
Region of Enrollment
Spain
|
42 participants
n=5 Participants
|
42 participants
n=7 Participants
|
84 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Baseline, Day 510Population: ITT (intent-to-treat) Population
Outcome measures
| Measure |
Inclisiran
n=242 Participants
Inclisiran sodium 300 milligrams (mg) (equivalent to 284 mg inclisiran) administered as a subcutaneous injection on Day 1, Day 90, and then every 6 months.
|
Placebo
n=240 Participants
Placebo administered as a subcutaneous injection of sterile saline solution (0.9% sodium chloride in water for injection) on Day 1, Day 90, and then every 6 months.
|
|---|---|---|
|
Percent Change in LDL-C From Baseline To Day 510
|
-41.15 percent change
Interval -44.52 to -37.77
|
8.37 percent change
Interval 3.96 to 12.77
|
PRIMARY outcome
Timeframe: Baseline, Day 90Population: ITT (intent-to-treat) Population
Assessments performed at Baseline, Day 90, Day 540, time-adjusted percent change at Day 90 reported
Outcome measures
| Measure |
Inclisiran
n=242 Participants
Inclisiran sodium 300 milligrams (mg) (equivalent to 284 mg inclisiran) administered as a subcutaneous injection on Day 1, Day 90, and then every 6 months.
|
Placebo
n=240 Participants
Placebo administered as a subcutaneous injection of sterile saline solution (0.9% sodium chloride in water for injection) on Day 1, Day 90, and then every 6 months.
|
|---|---|---|
|
Time-adjusted Percent Change in LDL-C From Baseline After Day 90 and up to Day 540
|
-38.08 percent change
Interval -41.03 to -35.14
|
6.22 percent change
Interval 3.26 to 9.17
|
SECONDARY outcome
Timeframe: Baseline, Day 510Population: ITT (intent-to-treat) Population
Outcome measures
| Measure |
Inclisiran
n=242 Participants
Inclisiran sodium 300 milligrams (mg) (equivalent to 284 mg inclisiran) administered as a subcutaneous injection on Day 1, Day 90, and then every 6 months.
|
Placebo
n=240 Participants
Placebo administered as a subcutaneous injection of sterile saline solution (0.9% sodium chloride in water for injection) on Day 1, Day 90, and then every 6 months.
|
|---|---|---|
|
Absolute Change in LDL-C From Baseline to Day 510
|
-58.95 mg/dL
Interval -64.75 to -53.15
|
9.94 mg/dL
Interval 4.1 to 15.78
|
SECONDARY outcome
Timeframe: Baseline, Day 90Population: ITT (intent-to-treat) Population
Assessments performed at Baseline, Day 90, Day 540, absolute change at Day 90 reported
Outcome measures
| Measure |
Inclisiran
n=242 Participants
Inclisiran sodium 300 milligrams (mg) (equivalent to 284 mg inclisiran) administered as a subcutaneous injection on Day 1, Day 90, and then every 6 months.
|
Placebo
n=240 Participants
Placebo administered as a subcutaneous injection of sterile saline solution (0.9% sodium chloride in water for injection) on Day 1, Day 90, and then every 6 months.
|
|---|---|---|
|
Time-adjusted Absolute Change in LDL-C From Baseline After Day 90 and up to Day 540
|
-56.58 mg/dL
Interval -60.98 to -52.17
|
6.17 mg/dL
Interval 1.72 to 10.62
|
SECONDARY outcome
Timeframe: Baseline, Day 510Population: ITT (intent-to-treat) Population
Outcome measures
| Measure |
Inclisiran
n=242 Participants
Inclisiran sodium 300 milligrams (mg) (equivalent to 284 mg inclisiran) administered as a subcutaneous injection on Day 1, Day 90, and then every 6 months.
|
Placebo
n=240 Participants
Placebo administered as a subcutaneous injection of sterile saline solution (0.9% sodium chloride in water for injection) on Day 1, Day 90, and then every 6 months.
|
|---|---|---|
|
Percentage Change in Proprotein Convertase Subtilisin/Kexin Type 9 (PCSK9) From Baseline to Day 510
|
-60.68 percent change
Interval -64.4 to -56.96
|
17.66 percent change
Interval 13.91 to 21.42
|
SECONDARY outcome
Timeframe: Baseline, Day 510Population: ITT (intent-to-treat) Population
Outcome measures
| Measure |
Inclisiran
n=242 Participants
Inclisiran sodium 300 milligrams (mg) (equivalent to 284 mg inclisiran) administered as a subcutaneous injection on Day 1, Day 90, and then every 6 months.
|
Placebo
n=240 Participants
Placebo administered as a subcutaneous injection of sterile saline solution (0.9% sodium chloride in water for injection) on Day 1, Day 90, and then every 6 months.
|
|---|---|---|
|
Percentage Change in Total Cholesterol From Baseline to Day 510
|
-25.11 percent change
Interval -27.83 to -22.39
|
6.66 percent change
Interval 3.96 to 9.36
|
SECONDARY outcome
Timeframe: Baseline, Day 510Population: ITT (intent-to-treat) Population
Outcome measures
| Measure |
Inclisiran
n=242 Participants
Inclisiran sodium 300 milligrams (mg) (equivalent to 284 mg inclisiran) administered as a subcutaneous injection on Day 1, Day 90, and then every 6 months.
|
Placebo
n=240 Participants
Placebo administered as a subcutaneous injection of sterile saline solution (0.9% sodium chloride in water for injection) on Day 1, Day 90, and then every 6 months.
|
|---|---|---|
|
Percent Change in Apolipoprotein B (Apo-B) From Baseline To Day 510
|
-33.14 percent change
Interval -35.91 to -30.36
|
2.93 percent change
Interval 0.14 to 5.71
|
SECONDARY outcome
Timeframe: Baseline, Day 510Population: ITT (intent-to-treat) Population
Outcome measures
| Measure |
Inclisiran
n=242 Participants
Inclisiran sodium 300 milligrams (mg) (equivalent to 284 mg inclisiran) administered as a subcutaneous injection on Day 1, Day 90, and then every 6 months.
|
Placebo
n=240 Participants
Placebo administered as a subcutaneous injection of sterile saline solution (0.9% sodium chloride in water for injection) on Day 1, Day 90, and then every 6 months.
|
|---|---|---|
|
Percent Change in Non-high-density Lipoprotein (HDL)-C From Baseline To Day 510
|
-34.93 percent change
Interval -38.46 to -31.4
|
7.43 percent change
Interval 3.93 to 10.92
|
Adverse Events
Inclisiran
Serious events: 18 serious events
Other events: 96 other events
Deaths: 1 deaths
Placebo
Serious events: 33 serious events
Other events: 67 other events
Deaths: 1 deaths
Serious adverse events
| Measure |
Inclisiran
n=241 participants at risk
Inclisiran sodium 300 milligrams (mg) will be administered as a SC injection on Day 1, Day 90 then every 6 months.
Inclisiran: Inclisiran is a small interfering ribonucleic acid (RNA) that inhibits PCSK9 synthesis.
|
Placebo
n=240 participants at risk
Placebo will be administered as SC injections of saline solution on Day 1, Day 90 then every 6 months.
Placebo: Placebo will be supplied as sterile normal saline (0.9% sodium chloride in water for injection).
|
|---|---|---|
|
Cardiac disorders
Acute myocardial infarction
|
0.83%
2/241 • Day 0 - 510
The safety population for adverse event collection was 481 subjects which is different from the Intent-to-Treat (ITT) population used in efficacy analysis (482 subjects). One subject was randomized in the Inclisiran arm, but never received study drug, therefore this subject not included in the safety population.
|
0.42%
1/240 • Day 0 - 510
The safety population for adverse event collection was 481 subjects which is different from the Intent-to-Treat (ITT) population used in efficacy analysis (482 subjects). One subject was randomized in the Inclisiran arm, but never received study drug, therefore this subject not included in the safety population.
|
|
Cardiac disorders
Angina Pectoris
|
0.41%
1/241 • Day 0 - 510
The safety population for adverse event collection was 481 subjects which is different from the Intent-to-Treat (ITT) population used in efficacy analysis (482 subjects). One subject was randomized in the Inclisiran arm, but never received study drug, therefore this subject not included in the safety population.
|
0.00%
0/240 • Day 0 - 510
The safety population for adverse event collection was 481 subjects which is different from the Intent-to-Treat (ITT) population used in efficacy analysis (482 subjects). One subject was randomized in the Inclisiran arm, but never received study drug, therefore this subject not included in the safety population.
|
|
Cardiac disorders
Angina unstable
|
0.41%
1/241 • Day 0 - 510
The safety population for adverse event collection was 481 subjects which is different from the Intent-to-Treat (ITT) population used in efficacy analysis (482 subjects). One subject was randomized in the Inclisiran arm, but never received study drug, therefore this subject not included in the safety population.
|
1.7%
4/240 • Day 0 - 510
The safety population for adverse event collection was 481 subjects which is different from the Intent-to-Treat (ITT) population used in efficacy analysis (482 subjects). One subject was randomized in the Inclisiran arm, but never received study drug, therefore this subject not included in the safety population.
|
|
Cardiac disorders
Cardiac failure
|
0.41%
1/241 • Day 0 - 510
The safety population for adverse event collection was 481 subjects which is different from the Intent-to-Treat (ITT) population used in efficacy analysis (482 subjects). One subject was randomized in the Inclisiran arm, but never received study drug, therefore this subject not included in the safety population.
|
0.00%
0/240 • Day 0 - 510
The safety population for adverse event collection was 481 subjects which is different from the Intent-to-Treat (ITT) population used in efficacy analysis (482 subjects). One subject was randomized in the Inclisiran arm, but never received study drug, therefore this subject not included in the safety population.
|
|
Cardiac disorders
Aortic valve stenosis
|
0.83%
2/241 • Day 0 - 510
The safety population for adverse event collection was 481 subjects which is different from the Intent-to-Treat (ITT) population used in efficacy analysis (482 subjects). One subject was randomized in the Inclisiran arm, but never received study drug, therefore this subject not included in the safety population.
|
0.00%
0/240 • Day 0 - 510
The safety population for adverse event collection was 481 subjects which is different from the Intent-to-Treat (ITT) population used in efficacy analysis (482 subjects). One subject was randomized in the Inclisiran arm, but never received study drug, therefore this subject not included in the safety population.
|
|
Cardiac disorders
Arteriosclerosis coronary artery
|
0.00%
0/241 • Day 0 - 510
The safety population for adverse event collection was 481 subjects which is different from the Intent-to-Treat (ITT) population used in efficacy analysis (482 subjects). One subject was randomized in the Inclisiran arm, but never received study drug, therefore this subject not included in the safety population.
|
0.42%
1/240 • Day 0 - 510
The safety population for adverse event collection was 481 subjects which is different from the Intent-to-Treat (ITT) population used in efficacy analysis (482 subjects). One subject was randomized in the Inclisiran arm, but never received study drug, therefore this subject not included in the safety population.
|
|
Cardiac disorders
Atrial fibrillation
|
0.00%
0/241 • Day 0 - 510
The safety population for adverse event collection was 481 subjects which is different from the Intent-to-Treat (ITT) population used in efficacy analysis (482 subjects). One subject was randomized in the Inclisiran arm, but never received study drug, therefore this subject not included in the safety population.
|
0.42%
1/240 • Day 0 - 510
The safety population for adverse event collection was 481 subjects which is different from the Intent-to-Treat (ITT) population used in efficacy analysis (482 subjects). One subject was randomized in the Inclisiran arm, but never received study drug, therefore this subject not included in the safety population.
|
|
Cardiac disorders
Cardiac arrest
|
0.41%
1/241 • Day 0 - 510
The safety population for adverse event collection was 481 subjects which is different from the Intent-to-Treat (ITT) population used in efficacy analysis (482 subjects). One subject was randomized in the Inclisiran arm, but never received study drug, therefore this subject not included in the safety population.
|
0.00%
0/240 • Day 0 - 510
The safety population for adverse event collection was 481 subjects which is different from the Intent-to-Treat (ITT) population used in efficacy analysis (482 subjects). One subject was randomized in the Inclisiran arm, but never received study drug, therefore this subject not included in the safety population.
|
|
Cardiac disorders
Cardiac failure acute
|
0.41%
1/241 • Day 0 - 510
The safety population for adverse event collection was 481 subjects which is different from the Intent-to-Treat (ITT) population used in efficacy analysis (482 subjects). One subject was randomized in the Inclisiran arm, but never received study drug, therefore this subject not included in the safety population.
|
0.00%
0/240 • Day 0 - 510
The safety population for adverse event collection was 481 subjects which is different from the Intent-to-Treat (ITT) population used in efficacy analysis (482 subjects). One subject was randomized in the Inclisiran arm, but never received study drug, therefore this subject not included in the safety population.
|
|
Cardiac disorders
Coronary artery disease
|
0.41%
1/241 • Day 0 - 510
The safety population for adverse event collection was 481 subjects which is different from the Intent-to-Treat (ITT) population used in efficacy analysis (482 subjects). One subject was randomized in the Inclisiran arm, but never received study drug, therefore this subject not included in the safety population.
|
0.00%
0/240 • Day 0 - 510
The safety population for adverse event collection was 481 subjects which is different from the Intent-to-Treat (ITT) population used in efficacy analysis (482 subjects). One subject was randomized in the Inclisiran arm, but never received study drug, therefore this subject not included in the safety population.
|
|
Cardiac disorders
Myocardial infarction
|
0.41%
1/241 • Day 0 - 510
The safety population for adverse event collection was 481 subjects which is different from the Intent-to-Treat (ITT) population used in efficacy analysis (482 subjects). One subject was randomized in the Inclisiran arm, but never received study drug, therefore this subject not included in the safety population.
|
0.00%
0/240 • Day 0 - 510
The safety population for adverse event collection was 481 subjects which is different from the Intent-to-Treat (ITT) population used in efficacy analysis (482 subjects). One subject was randomized in the Inclisiran arm, but never received study drug, therefore this subject not included in the safety population.
|
|
Cardiac disorders
Myocardial ischaemia
|
0.41%
1/241 • Day 0 - 510
The safety population for adverse event collection was 481 subjects which is different from the Intent-to-Treat (ITT) population used in efficacy analysis (482 subjects). One subject was randomized in the Inclisiran arm, but never received study drug, therefore this subject not included in the safety population.
|
1.2%
3/240 • Day 0 - 510
The safety population for adverse event collection was 481 subjects which is different from the Intent-to-Treat (ITT) population used in efficacy analysis (482 subjects). One subject was randomized in the Inclisiran arm, but never received study drug, therefore this subject not included in the safety population.
|
|
Cardiac disorders
Supraventricular tachycardia
|
0.00%
0/241 • Day 0 - 510
The safety population for adverse event collection was 481 subjects which is different from the Intent-to-Treat (ITT) population used in efficacy analysis (482 subjects). One subject was randomized in the Inclisiran arm, but never received study drug, therefore this subject not included in the safety population.
|
0.42%
1/240 • Day 0 - 510
The safety population for adverse event collection was 481 subjects which is different from the Intent-to-Treat (ITT) population used in efficacy analysis (482 subjects). One subject was randomized in the Inclisiran arm, but never received study drug, therefore this subject not included in the safety population.
|
|
Eye disorders
Optic ischaemic neuropathy
|
0.00%
0/241 • Day 0 - 510
The safety population for adverse event collection was 481 subjects which is different from the Intent-to-Treat (ITT) population used in efficacy analysis (482 subjects). One subject was randomized in the Inclisiran arm, but never received study drug, therefore this subject not included in the safety population.
|
0.83%
2/240 • Day 0 - 510
The safety population for adverse event collection was 481 subjects which is different from the Intent-to-Treat (ITT) population used in efficacy analysis (482 subjects). One subject was randomized in the Inclisiran arm, but never received study drug, therefore this subject not included in the safety population.
|
|
Gastrointestinal disorders
Gastritis erosive
|
0.00%
0/241 • Day 0 - 510
The safety population for adverse event collection was 481 subjects which is different from the Intent-to-Treat (ITT) population used in efficacy analysis (482 subjects). One subject was randomized in the Inclisiran arm, but never received study drug, therefore this subject not included in the safety population.
|
0.42%
1/240 • Day 0 - 510
The safety population for adverse event collection was 481 subjects which is different from the Intent-to-Treat (ITT) population used in efficacy analysis (482 subjects). One subject was randomized in the Inclisiran arm, but never received study drug, therefore this subject not included in the safety population.
|
|
Gastrointestinal disorders
Pancreatitis acute
|
0.00%
0/241 • Day 0 - 510
The safety population for adverse event collection was 481 subjects which is different from the Intent-to-Treat (ITT) population used in efficacy analysis (482 subjects). One subject was randomized in the Inclisiran arm, but never received study drug, therefore this subject not included in the safety population.
|
0.42%
1/240 • Day 0 - 510
The safety population for adverse event collection was 481 subjects which is different from the Intent-to-Treat (ITT) population used in efficacy analysis (482 subjects). One subject was randomized in the Inclisiran arm, but never received study drug, therefore this subject not included in the safety population.
|
|
General disorders
Hernia
|
0.00%
0/241 • Day 0 - 510
The safety population for adverse event collection was 481 subjects which is different from the Intent-to-Treat (ITT) population used in efficacy analysis (482 subjects). One subject was randomized in the Inclisiran arm, but never received study drug, therefore this subject not included in the safety population.
|
0.42%
1/240 • Day 0 - 510
The safety population for adverse event collection was 481 subjects which is different from the Intent-to-Treat (ITT) population used in efficacy analysis (482 subjects). One subject was randomized in the Inclisiran arm, but never received study drug, therefore this subject not included in the safety population.
|
|
General disorders
Non-cardiac chest pain
|
0.00%
0/241 • Day 0 - 510
The safety population for adverse event collection was 481 subjects which is different from the Intent-to-Treat (ITT) population used in efficacy analysis (482 subjects). One subject was randomized in the Inclisiran arm, but never received study drug, therefore this subject not included in the safety population.
|
0.42%
1/240 • Day 0 - 510
The safety population for adverse event collection was 481 subjects which is different from the Intent-to-Treat (ITT) population used in efficacy analysis (482 subjects). One subject was randomized in the Inclisiran arm, but never received study drug, therefore this subject not included in the safety population.
|
|
Hepatobiliary disorders
Biliary colic
|
0.00%
0/241 • Day 0 - 510
The safety population for adverse event collection was 481 subjects which is different from the Intent-to-Treat (ITT) population used in efficacy analysis (482 subjects). One subject was randomized in the Inclisiran arm, but never received study drug, therefore this subject not included in the safety population.
|
0.42%
1/240 • Day 0 - 510
The safety population for adverse event collection was 481 subjects which is different from the Intent-to-Treat (ITT) population used in efficacy analysis (482 subjects). One subject was randomized in the Inclisiran arm, but never received study drug, therefore this subject not included in the safety population.
|
|
Infections and infestations
Cellulitis
|
0.41%
1/241 • Day 0 - 510
The safety population for adverse event collection was 481 subjects which is different from the Intent-to-Treat (ITT) population used in efficacy analysis (482 subjects). One subject was randomized in the Inclisiran arm, but never received study drug, therefore this subject not included in the safety population.
|
0.00%
0/240 • Day 0 - 510
The safety population for adverse event collection was 481 subjects which is different from the Intent-to-Treat (ITT) population used in efficacy analysis (482 subjects). One subject was randomized in the Inclisiran arm, but never received study drug, therefore this subject not included in the safety population.
|
|
Infections and infestations
Diverticulitis
|
0.00%
0/241 • Day 0 - 510
The safety population for adverse event collection was 481 subjects which is different from the Intent-to-Treat (ITT) population used in efficacy analysis (482 subjects). One subject was randomized in the Inclisiran arm, but never received study drug, therefore this subject not included in the safety population.
|
0.42%
1/240 • Day 0 - 510
The safety population for adverse event collection was 481 subjects which is different from the Intent-to-Treat (ITT) population used in efficacy analysis (482 subjects). One subject was randomized in the Inclisiran arm, but never received study drug, therefore this subject not included in the safety population.
|
|
Infections and infestations
Infective tenosynovitis
|
0.41%
1/241 • Day 0 - 510
The safety population for adverse event collection was 481 subjects which is different from the Intent-to-Treat (ITT) population used in efficacy analysis (482 subjects). One subject was randomized in the Inclisiran arm, but never received study drug, therefore this subject not included in the safety population.
|
0.00%
0/240 • Day 0 - 510
The safety population for adverse event collection was 481 subjects which is different from the Intent-to-Treat (ITT) population used in efficacy analysis (482 subjects). One subject was randomized in the Inclisiran arm, but never received study drug, therefore this subject not included in the safety population.
|
|
Infections and infestations
Influenza
|
0.00%
0/241 • Day 0 - 510
The safety population for adverse event collection was 481 subjects which is different from the Intent-to-Treat (ITT) population used in efficacy analysis (482 subjects). One subject was randomized in the Inclisiran arm, but never received study drug, therefore this subject not included in the safety population.
|
0.42%
1/240 • Day 0 - 510
The safety population for adverse event collection was 481 subjects which is different from the Intent-to-Treat (ITT) population used in efficacy analysis (482 subjects). One subject was randomized in the Inclisiran arm, but never received study drug, therefore this subject not included in the safety population.
|
|
Infections and infestations
Pneumonia
|
0.41%
1/241 • Day 0 - 510
The safety population for adverse event collection was 481 subjects which is different from the Intent-to-Treat (ITT) population used in efficacy analysis (482 subjects). One subject was randomized in the Inclisiran arm, but never received study drug, therefore this subject not included in the safety population.
|
0.42%
1/240 • Day 0 - 510
The safety population for adverse event collection was 481 subjects which is different from the Intent-to-Treat (ITT) population used in efficacy analysis (482 subjects). One subject was randomized in the Inclisiran arm, but never received study drug, therefore this subject not included in the safety population.
|
|
Infections and infestations
Post procedural infection
|
0.00%
0/241 • Day 0 - 510
The safety population for adverse event collection was 481 subjects which is different from the Intent-to-Treat (ITT) population used in efficacy analysis (482 subjects). One subject was randomized in the Inclisiran arm, but never received study drug, therefore this subject not included in the safety population.
|
0.42%
1/240 • Day 0 - 510
The safety population for adverse event collection was 481 subjects which is different from the Intent-to-Treat (ITT) population used in efficacy analysis (482 subjects). One subject was randomized in the Inclisiran arm, but never received study drug, therefore this subject not included in the safety population.
|
|
Infections and infestations
Pyelitis
|
0.00%
0/241 • Day 0 - 510
The safety population for adverse event collection was 481 subjects which is different from the Intent-to-Treat (ITT) population used in efficacy analysis (482 subjects). One subject was randomized in the Inclisiran arm, but never received study drug, therefore this subject not included in the safety population.
|
0.42%
1/240 • Day 0 - 510
The safety population for adverse event collection was 481 subjects which is different from the Intent-to-Treat (ITT) population used in efficacy analysis (482 subjects). One subject was randomized in the Inclisiran arm, but never received study drug, therefore this subject not included in the safety population.
|
|
Infections and infestations
Pyelonephritis
|
0.00%
0/241 • Day 0 - 510
The safety population for adverse event collection was 481 subjects which is different from the Intent-to-Treat (ITT) population used in efficacy analysis (482 subjects). One subject was randomized in the Inclisiran arm, but never received study drug, therefore this subject not included in the safety population.
|
0.42%
1/240 • Day 0 - 510
The safety population for adverse event collection was 481 subjects which is different from the Intent-to-Treat (ITT) population used in efficacy analysis (482 subjects). One subject was randomized in the Inclisiran arm, but never received study drug, therefore this subject not included in the safety population.
|
|
Infections and infestations
Tick-borne fever
|
0.00%
0/241 • Day 0 - 510
The safety population for adverse event collection was 481 subjects which is different from the Intent-to-Treat (ITT) population used in efficacy analysis (482 subjects). One subject was randomized in the Inclisiran arm, but never received study drug, therefore this subject not included in the safety population.
|
0.42%
1/240 • Day 0 - 510
The safety population for adverse event collection was 481 subjects which is different from the Intent-to-Treat (ITT) population used in efficacy analysis (482 subjects). One subject was randomized in the Inclisiran arm, but never received study drug, therefore this subject not included in the safety population.
|
|
Infections and infestations
Viral upper respiratory tract infection
|
0.00%
0/241 • Day 0 - 510
The safety population for adverse event collection was 481 subjects which is different from the Intent-to-Treat (ITT) population used in efficacy analysis (482 subjects). One subject was randomized in the Inclisiran arm, but never received study drug, therefore this subject not included in the safety population.
|
0.42%
1/240 • Day 0 - 510
The safety population for adverse event collection was 481 subjects which is different from the Intent-to-Treat (ITT) population used in efficacy analysis (482 subjects). One subject was randomized in the Inclisiran arm, but never received study drug, therefore this subject not included in the safety population.
|
|
Infections and infestations
Wound sepsis
|
0.00%
0/241 • Day 0 - 510
The safety population for adverse event collection was 481 subjects which is different from the Intent-to-Treat (ITT) population used in efficacy analysis (482 subjects). One subject was randomized in the Inclisiran arm, but never received study drug, therefore this subject not included in the safety population.
|
0.42%
1/240 • Day 0 - 510
The safety population for adverse event collection was 481 subjects which is different from the Intent-to-Treat (ITT) population used in efficacy analysis (482 subjects). One subject was randomized in the Inclisiran arm, but never received study drug, therefore this subject not included in the safety population.
|
|
Injury, poisoning and procedural complications
Limb injury
|
0.00%
0/241 • Day 0 - 510
The safety population for adverse event collection was 481 subjects which is different from the Intent-to-Treat (ITT) population used in efficacy analysis (482 subjects). One subject was randomized in the Inclisiran arm, but never received study drug, therefore this subject not included in the safety population.
|
0.42%
1/240 • Day 0 - 510
The safety population for adverse event collection was 481 subjects which is different from the Intent-to-Treat (ITT) population used in efficacy analysis (482 subjects). One subject was randomized in the Inclisiran arm, but never received study drug, therefore this subject not included in the safety population.
|
|
Injury, poisoning and procedural complications
Post procedural haematuria
|
0.00%
0/241 • Day 0 - 510
The safety population for adverse event collection was 481 subjects which is different from the Intent-to-Treat (ITT) population used in efficacy analysis (482 subjects). One subject was randomized in the Inclisiran arm, but never received study drug, therefore this subject not included in the safety population.
|
0.42%
1/240 • Day 0 - 510
The safety population for adverse event collection was 481 subjects which is different from the Intent-to-Treat (ITT) population used in efficacy analysis (482 subjects). One subject was randomized in the Inclisiran arm, but never received study drug, therefore this subject not included in the safety population.
|
|
Injury, poisoning and procedural complications
Road traffic accident
|
0.00%
0/241 • Day 0 - 510
The safety population for adverse event collection was 481 subjects which is different from the Intent-to-Treat (ITT) population used in efficacy analysis (482 subjects). One subject was randomized in the Inclisiran arm, but never received study drug, therefore this subject not included in the safety population.
|
0.42%
1/240 • Day 0 - 510
The safety population for adverse event collection was 481 subjects which is different from the Intent-to-Treat (ITT) population used in efficacy analysis (482 subjects). One subject was randomized in the Inclisiran arm, but never received study drug, therefore this subject not included in the safety population.
|
|
Injury, poisoning and procedural complications
Soft tissue injury
|
0.00%
0/241 • Day 0 - 510
The safety population for adverse event collection was 481 subjects which is different from the Intent-to-Treat (ITT) population used in efficacy analysis (482 subjects). One subject was randomized in the Inclisiran arm, but never received study drug, therefore this subject not included in the safety population.
|
0.42%
1/240 • Day 0 - 510
The safety population for adverse event collection was 481 subjects which is different from the Intent-to-Treat (ITT) population used in efficacy analysis (482 subjects). One subject was randomized in the Inclisiran arm, but never received study drug, therefore this subject not included in the safety population.
|
|
Injury, poisoning and procedural complications
Spinal compression fracture
|
0.41%
1/241 • Day 0 - 510
The safety population for adverse event collection was 481 subjects which is different from the Intent-to-Treat (ITT) population used in efficacy analysis (482 subjects). One subject was randomized in the Inclisiran arm, but never received study drug, therefore this subject not included in the safety population.
|
0.00%
0/240 • Day 0 - 510
The safety population for adverse event collection was 481 subjects which is different from the Intent-to-Treat (ITT) population used in efficacy analysis (482 subjects). One subject was randomized in the Inclisiran arm, but never received study drug, therefore this subject not included in the safety population.
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
0.00%
0/241 • Day 0 - 510
The safety population for adverse event collection was 481 subjects which is different from the Intent-to-Treat (ITT) population used in efficacy analysis (482 subjects). One subject was randomized in the Inclisiran arm, but never received study drug, therefore this subject not included in the safety population.
|
0.83%
2/240 • Day 0 - 510
The safety population for adverse event collection was 481 subjects which is different from the Intent-to-Treat (ITT) population used in efficacy analysis (482 subjects). One subject was randomized in the Inclisiran arm, but never received study drug, therefore this subject not included in the safety population.
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
0.00%
0/241 • Day 0 - 510
The safety population for adverse event collection was 481 subjects which is different from the Intent-to-Treat (ITT) population used in efficacy analysis (482 subjects). One subject was randomized in the Inclisiran arm, but never received study drug, therefore this subject not included in the safety population.
|
0.42%
1/240 • Day 0 - 510
The safety population for adverse event collection was 481 subjects which is different from the Intent-to-Treat (ITT) population used in efficacy analysis (482 subjects). One subject was randomized in the Inclisiran arm, but never received study drug, therefore this subject not included in the safety population.
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Invasive ductal breast carcinoma
|
0.00%
0/241 • Day 0 - 510
The safety population for adverse event collection was 481 subjects which is different from the Intent-to-Treat (ITT) population used in efficacy analysis (482 subjects). One subject was randomized in the Inclisiran arm, but never received study drug, therefore this subject not included in the safety population.
|
0.42%
1/240 • Day 0 - 510
The safety population for adverse event collection was 481 subjects which is different from the Intent-to-Treat (ITT) population used in efficacy analysis (482 subjects). One subject was randomized in the Inclisiran arm, but never received study drug, therefore this subject not included in the safety population.
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Malignant melanoma in situ
|
0.00%
0/241 • Day 0 - 510
The safety population for adverse event collection was 481 subjects which is different from the Intent-to-Treat (ITT) population used in efficacy analysis (482 subjects). One subject was randomized in the Inclisiran arm, but never received study drug, therefore this subject not included in the safety population.
|
0.42%
1/240 • Day 0 - 510
The safety population for adverse event collection was 481 subjects which is different from the Intent-to-Treat (ITT) population used in efficacy analysis (482 subjects). One subject was randomized in the Inclisiran arm, but never received study drug, therefore this subject not included in the safety population.
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Prostate cancer
|
0.41%
1/241 • Day 0 - 510
The safety population for adverse event collection was 481 subjects which is different from the Intent-to-Treat (ITT) population used in efficacy analysis (482 subjects). One subject was randomized in the Inclisiran arm, but never received study drug, therefore this subject not included in the safety population.
|
0.00%
0/240 • Day 0 - 510
The safety population for adverse event collection was 481 subjects which is different from the Intent-to-Treat (ITT) population used in efficacy analysis (482 subjects). One subject was randomized in the Inclisiran arm, but never received study drug, therefore this subject not included in the safety population.
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Squamous cell carcinoma of skin
|
0.41%
1/241 • Day 0 - 510
The safety population for adverse event collection was 481 subjects which is different from the Intent-to-Treat (ITT) population used in efficacy analysis (482 subjects). One subject was randomized in the Inclisiran arm, but never received study drug, therefore this subject not included in the safety population.
|
0.42%
1/240 • Day 0 - 510
The safety population for adverse event collection was 481 subjects which is different from the Intent-to-Treat (ITT) population used in efficacy analysis (482 subjects). One subject was randomized in the Inclisiran arm, but never received study drug, therefore this subject not included in the safety population.
|
|
Nervous system disorders
Sensory disturbance
|
0.00%
0/241 • Day 0 - 510
The safety population for adverse event collection was 481 subjects which is different from the Intent-to-Treat (ITT) population used in efficacy analysis (482 subjects). One subject was randomized in the Inclisiran arm, but never received study drug, therefore this subject not included in the safety population.
|
0.42%
1/240 • Day 0 - 510
The safety population for adverse event collection was 481 subjects which is different from the Intent-to-Treat (ITT) population used in efficacy analysis (482 subjects). One subject was randomized in the Inclisiran arm, but never received study drug, therefore this subject not included in the safety population.
|
|
Nervous system disorders
Trigeminal neuralgia
|
0.00%
0/241 • Day 0 - 510
The safety population for adverse event collection was 481 subjects which is different from the Intent-to-Treat (ITT) population used in efficacy analysis (482 subjects). One subject was randomized in the Inclisiran arm, but never received study drug, therefore this subject not included in the safety population.
|
0.42%
1/240 • Day 0 - 510
The safety population for adverse event collection was 481 subjects which is different from the Intent-to-Treat (ITT) population used in efficacy analysis (482 subjects). One subject was randomized in the Inclisiran arm, but never received study drug, therefore this subject not included in the safety population.
|
|
Product Issues
Device loosening
|
0.41%
1/241 • Day 0 - 510
The safety population for adverse event collection was 481 subjects which is different from the Intent-to-Treat (ITT) population used in efficacy analysis (482 subjects). One subject was randomized in the Inclisiran arm, but never received study drug, therefore this subject not included in the safety population.
|
0.00%
0/240 • Day 0 - 510
The safety population for adverse event collection was 481 subjects which is different from the Intent-to-Treat (ITT) population used in efficacy analysis (482 subjects). One subject was randomized in the Inclisiran arm, but never received study drug, therefore this subject not included in the safety population.
|
|
Psychiatric disorders
Anxiety
|
0.41%
1/241 • Day 0 - 510
The safety population for adverse event collection was 481 subjects which is different from the Intent-to-Treat (ITT) population used in efficacy analysis (482 subjects). One subject was randomized in the Inclisiran arm, but never received study drug, therefore this subject not included in the safety population.
|
0.00%
0/240 • Day 0 - 510
The safety population for adverse event collection was 481 subjects which is different from the Intent-to-Treat (ITT) population used in efficacy analysis (482 subjects). One subject was randomized in the Inclisiran arm, but never received study drug, therefore this subject not included in the safety population.
|
|
Psychiatric disorders
Depression
|
0.00%
0/241 • Day 0 - 510
The safety population for adverse event collection was 481 subjects which is different from the Intent-to-Treat (ITT) population used in efficacy analysis (482 subjects). One subject was randomized in the Inclisiran arm, but never received study drug, therefore this subject not included in the safety population.
|
0.42%
1/240 • Day 0 - 510
The safety population for adverse event collection was 481 subjects which is different from the Intent-to-Treat (ITT) population used in efficacy analysis (482 subjects). One subject was randomized in the Inclisiran arm, but never received study drug, therefore this subject not included in the safety population.
|
|
Psychiatric disorders
Major depression
|
0.00%
0/241 • Day 0 - 510
The safety population for adverse event collection was 481 subjects which is different from the Intent-to-Treat (ITT) population used in efficacy analysis (482 subjects). One subject was randomized in the Inclisiran arm, but never received study drug, therefore this subject not included in the safety population.
|
0.42%
1/240 • Day 0 - 510
The safety population for adverse event collection was 481 subjects which is different from the Intent-to-Treat (ITT) population used in efficacy analysis (482 subjects). One subject was randomized in the Inclisiran arm, but never received study drug, therefore this subject not included in the safety population.
|
|
Renal and urinary disorders
Hydronephrosis
|
0.00%
0/241 • Day 0 - 510
The safety population for adverse event collection was 481 subjects which is different from the Intent-to-Treat (ITT) population used in efficacy analysis (482 subjects). One subject was randomized in the Inclisiran arm, but never received study drug, therefore this subject not included in the safety population.
|
0.42%
1/240 • Day 0 - 510
The safety population for adverse event collection was 481 subjects which is different from the Intent-to-Treat (ITT) population used in efficacy analysis (482 subjects). One subject was randomized in the Inclisiran arm, but never received study drug, therefore this subject not included in the safety population.
|
|
Renal and urinary disorders
Nephrolithiasis
|
0.00%
0/241 • Day 0 - 510
The safety population for adverse event collection was 481 subjects which is different from the Intent-to-Treat (ITT) population used in efficacy analysis (482 subjects). One subject was randomized in the Inclisiran arm, but never received study drug, therefore this subject not included in the safety population.
|
0.42%
1/240 • Day 0 - 510
The safety population for adverse event collection was 481 subjects which is different from the Intent-to-Treat (ITT) population used in efficacy analysis (482 subjects). One subject was randomized in the Inclisiran arm, but never received study drug, therefore this subject not included in the safety population.
|
|
Respiratory, thoracic and mediastinal disorders
Asthma
|
0.00%
0/241 • Day 0 - 510
The safety population for adverse event collection was 481 subjects which is different from the Intent-to-Treat (ITT) population used in efficacy analysis (482 subjects). One subject was randomized in the Inclisiran arm, but never received study drug, therefore this subject not included in the safety population.
|
0.42%
1/240 • Day 0 - 510
The safety population for adverse event collection was 481 subjects which is different from the Intent-to-Treat (ITT) population used in efficacy analysis (482 subjects). One subject was randomized in the Inclisiran arm, but never received study drug, therefore this subject not included in the safety population.
|
|
Respiratory, thoracic and mediastinal disorders
Chronic obstructive pulmonary disease
|
0.00%
0/241 • Day 0 - 510
The safety population for adverse event collection was 481 subjects which is different from the Intent-to-Treat (ITT) population used in efficacy analysis (482 subjects). One subject was randomized in the Inclisiran arm, but never received study drug, therefore this subject not included in the safety population.
|
0.42%
1/240 • Day 0 - 510
The safety population for adverse event collection was 481 subjects which is different from the Intent-to-Treat (ITT) population used in efficacy analysis (482 subjects). One subject was randomized in the Inclisiran arm, but never received study drug, therefore this subject not included in the safety population.
|
Other adverse events
| Measure |
Inclisiran
n=241 participants at risk
Inclisiran sodium 300 milligrams (mg) will be administered as a SC injection on Day 1, Day 90 then every 6 months.
Inclisiran: Inclisiran is a small interfering ribonucleic acid (RNA) that inhibits PCSK9 synthesis.
|
Placebo
n=240 participants at risk
Placebo will be administered as SC injections of saline solution on Day 1, Day 90 then every 6 months.
Placebo: Placebo will be supplied as sterile normal saline (0.9% sodium chloride in water for injection).
|
|---|---|---|
|
General disorders
Injection site reaction
|
9.1%
22/241 • Day 0 - 510
The safety population for adverse event collection was 481 subjects which is different from the Intent-to-Treat (ITT) population used in efficacy analysis (482 subjects). One subject was randomized in the Inclisiran arm, but never received study drug, therefore this subject not included in the safety population.
|
0.00%
0/240 • Day 0 - 510
The safety population for adverse event collection was 481 subjects which is different from the Intent-to-Treat (ITT) population used in efficacy analysis (482 subjects). One subject was randomized in the Inclisiran arm, but never received study drug, therefore this subject not included in the safety population.
|
|
Infections and infestations
Influenza
|
5.4%
13/241 • Day 0 - 510
The safety population for adverse event collection was 481 subjects which is different from the Intent-to-Treat (ITT) population used in efficacy analysis (482 subjects). One subject was randomized in the Inclisiran arm, but never received study drug, therefore this subject not included in the safety population.
|
8.8%
21/240 • Day 0 - 510
The safety population for adverse event collection was 481 subjects which is different from the Intent-to-Treat (ITT) population used in efficacy analysis (482 subjects). One subject was randomized in the Inclisiran arm, but never received study drug, therefore this subject not included in the safety population.
|
|
Infections and infestations
Nasopharyngitis
|
11.6%
28/241 • Day 0 - 510
The safety population for adverse event collection was 481 subjects which is different from the Intent-to-Treat (ITT) population used in efficacy analysis (482 subjects). One subject was randomized in the Inclisiran arm, but never received study drug, therefore this subject not included in the safety population.
|
8.3%
20/240 • Day 0 - 510
The safety population for adverse event collection was 481 subjects which is different from the Intent-to-Treat (ITT) population used in efficacy analysis (482 subjects). One subject was randomized in the Inclisiran arm, but never received study drug, therefore this subject not included in the safety population.
|
|
Infections and infestations
Upper respiratory tract infection
|
6.6%
16/241 • Day 0 - 510
The safety population for adverse event collection was 481 subjects which is different from the Intent-to-Treat (ITT) population used in efficacy analysis (482 subjects). One subject was randomized in the Inclisiran arm, but never received study drug, therefore this subject not included in the safety population.
|
6.7%
16/240 • Day 0 - 510
The safety population for adverse event collection was 481 subjects which is different from the Intent-to-Treat (ITT) population used in efficacy analysis (482 subjects). One subject was randomized in the Inclisiran arm, but never received study drug, therefore this subject not included in the safety population.
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
7.1%
17/241 • Day 0 - 510
The safety population for adverse event collection was 481 subjects which is different from the Intent-to-Treat (ITT) population used in efficacy analysis (482 subjects). One subject was randomized in the Inclisiran arm, but never received study drug, therefore this subject not included in the safety population.
|
4.2%
10/240 • Day 0 - 510
The safety population for adverse event collection was 481 subjects which is different from the Intent-to-Treat (ITT) population used in efficacy analysis (482 subjects). One subject was randomized in the Inclisiran arm, but never received study drug, therefore this subject not included in the safety population.
|
Additional Information
Vice President - Regulatory Operations
The Medicines Company
Phone: 973-985-0597
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place