Trial Outcomes & Findings for Study to Evaluate Efficacy and Safety of Inclisiran in Adolescents With Homozygous Familial Hypercholesterolemia (NCT NCT04659863)
NCT ID: NCT04659863
Last Updated: 2026-01-13
Results Overview
Percentage change in low-density lipoprotein cholesterol (LDL-C) from baseline to Day 330 (Year 1)
Recruitment status
COMPLETED
Study phase
PHASE3
Target enrollment
13 participants
Primary outcome timeframe
Baseline and Day 330
Results posted on
2026-01-13
Participant Flow
Thirteen participants were randomized from 9 study centers in 8 countries. The breakdown of countries and study centers for the randomized participants was as follows: Canada (1), France (1), Greece (1), Lebanon (1), Malaysia (1), Netherlands (1), Turkey (2), and United States (1).
The study had an approximately 4-week screening/run-in period
Participant milestones
| Measure |
Part 1- Inclisiran
Inclisiran sodium 300 mg subcutaneous (sc) injection (given at Days 1, 90 and 270)
|
Part 1 - Placebo
Placebo sc injection (given at Day 1, 90 and 270)
|
Part 2 - Inclisiran (Total)
Inclisiran sodium 300 mg sc injection (given at Days 450 and 630). In addition, participants assigned to placebo in Part 1 received inclisiran sodium 300 mg sc injection on Day 360, while participants assigned to inclisiran in Part 1 received placebo sc injection on Day 360
|
|---|---|---|---|
|
Part 1 (Double-blind Period)
STARTED
|
9
|
4
|
0
|
|
Part 1 (Double-blind Period)
COMPLETED
|
9
|
4
|
0
|
|
Part 1 (Double-blind Period)
NOT COMPLETED
|
0
|
0
|
0
|
|
Part 2 (Open-label Period)
STARTED
|
0
|
0
|
13
|
|
Part 2 (Open-label Period)
COMPLETED
|
0
|
0
|
13
|
|
Part 2 (Open-label Period)
NOT COMPLETED
|
0
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Study to Evaluate Efficacy and Safety of Inclisiran in Adolescents With Homozygous Familial Hypercholesterolemia
Baseline characteristics by cohort
| Measure |
Part 1- Inclisiran
n=9 Participants
Inclisiran sodium 300 mg subcutaneous (sc) injection (given at Days 1, 90 and 270)
|
Part 1 - Placebo
n=4 Participants
Placebo sc injection (given at Day 1, 90 and 270)
|
Total
n=13 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Categorical
<=18 years
|
9 Participants
n=210 Participants
|
4 Participants
n=19 Participants
|
13 Participants
n=123 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
0 Participants
n=210 Participants
|
0 Participants
n=19 Participants
|
0 Participants
n=123 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=210 Participants
|
0 Participants
n=19 Participants
|
0 Participants
n=123 Participants
|
|
Age, Continuous
|
14.6 years
STANDARD_DEVIATION 1.54 • n=210 Participants
|
15.1 years
STANDARD_DEVIATION 2.66 • n=19 Participants
|
14.8 years
STANDARD_DEVIATION 1.85 • n=123 Participants
|
|
Sex: Female, Male
Female
|
7 Participants
n=210 Participants
|
2 Participants
n=19 Participants
|
9 Participants
n=123 Participants
|
|
Sex: Female, Male
Male
|
2 Participants
n=210 Participants
|
2 Participants
n=19 Participants
|
4 Participants
n=123 Participants
|
|
Race/Ethnicity, Customized
Asian
|
1 Participants
n=210 Participants
|
1 Participants
n=19 Participants
|
2 Participants
n=123 Participants
|
|
Race/Ethnicity, Customized
White
|
8 Participants
n=210 Participants
|
3 Participants
n=19 Participants
|
11 Participants
n=123 Participants
|
PRIMARY outcome
Timeframe: Baseline and Day 330Population: Full Analysis Set (FAS) comprised all participants to whom study treatment had been assigned by randomization.
Percentage change in low-density lipoprotein cholesterol (LDL-C) from baseline to Day 330 (Year 1)
Outcome measures
| Measure |
Part 1 - Placebo
n=4 Participants
Placebo sc injection (given at Day 1, 90 and 270)
|
Part 1- Inclisiran
n=9 Participants
Inclisiran sodium 300 mg subcutaneous (sc) injection (given at Days 1, 90 and 270)
|
Part 2 - Inclisiran (Total)
Inclisiran sodium 300 mg sc injection (given at Days 450 and 630). In addition, participants assigned to placebo in Part 1 received inclisiran sodium 300 mg sc injection on Day 360, while participants assigned to inclisiran in Part 1 received placebo sc injection on Day 360.
|
|---|---|---|---|
|
Percentage Change in LDL-C From Baseline to Day 330 (Part 1/Year 1)
|
11.7 percent change in LDL-C
Standard Deviation 30.52
|
-21.6 percent change in LDL-C
Standard Deviation 13.36
|
—
|
SECONDARY outcome
Timeframe: Baseline, after Day 90 up to Day 330Population: Full Analysis Set (FAS) comprised all participants to whom study treatment had been assigned by randomization
Time-adjusted percent change in LDL-C (after Day 90 and up to Day 330), calculated as the average of percent changes from baseline to Days 150, 270 and 330
Outcome measures
| Measure |
Part 1 - Placebo
n=4 Participants
Placebo sc injection (given at Day 1, 90 and 270)
|
Part 1- Inclisiran
n=9 Participants
Inclisiran sodium 300 mg subcutaneous (sc) injection (given at Days 1, 90 and 270)
|
Part 2 - Inclisiran (Total)
Inclisiran sodium 300 mg sc injection (given at Days 450 and 630). In addition, participants assigned to placebo in Part 1 received inclisiran sodium 300 mg sc injection on Day 360, while participants assigned to inclisiran in Part 1 received placebo sc injection on Day 360.
|
|---|---|---|---|
|
Time-adjusted Percent Change in LDL-C From Baseline After Day 90 and up to Day 330 (Part 1/Year 1)
|
13.0 Time-adjusted percent change in LDL-C
Standard Deviation 41.88
|
-21.0 Time-adjusted percent change in LDL-C
Standard Deviation 15.11
|
—
|
SECONDARY outcome
Timeframe: Baseline, up to Day 720Population: Full Analysis Set (FAS) comprised all participants to whom study treatment had been assigned by randomization
Percentage change in LDL-C from baseline to each assessment time up to Day 720.
Outcome measures
| Measure |
Part 1 - Placebo
n=4 Participants
Placebo sc injection (given at Day 1, 90 and 270)
|
Part 1- Inclisiran
n=9 Participants
Inclisiran sodium 300 mg subcutaneous (sc) injection (given at Days 1, 90 and 270)
|
Part 2 - Inclisiran (Total)
n=13 Participants
Inclisiran sodium 300 mg sc injection (given at Days 450 and 630). In addition, participants assigned to placebo in Part 1 received inclisiran sodium 300 mg sc injection on Day 360, while participants assigned to inclisiran in Part 1 received placebo sc injection on Day 360.
|
|---|---|---|---|
|
Percent Change in LDL-C From Baseline up to Day 720
Day 90
|
6.8 Percent change in LDL-C
Standard Deviation 16.83
|
-26.1 Percent change in LDL-C
Standard Deviation 13.53
|
—
|
|
Percent Change in LDL-C From Baseline up to Day 720
Day 150
|
17.0 Percent change in LDL-C
Standard Deviation 41.22
|
-24.1 Percent change in LDL-C
Standard Deviation 14.65
|
—
|
|
Percent Change in LDL-C From Baseline up to Day 720
Day 270
|
10.2 Percent change in LDL-C
Standard Deviation 54.00
|
-17.3 Percent change in LDL-C
Standard Deviation 21.64
|
—
|
|
Percent Change in LDL-C From Baseline up to Day 720
Day 330
|
11.7 Percent change in LDL-C
Standard Deviation 30.52
|
-21.6 Percent change in LDL-C
Standard Deviation 13.36
|
—
|
|
Percent Change in LDL-C From Baseline up to Day 720
Day 360
|
5.4 Percent change in LDL-C
Standard Deviation 28.24
|
-19.3 Percent change in LDL-C
Standard Deviation 14.75
|
—
|
|
Percent Change in LDL-C From Baseline up to Day 720
Day 450
|
—
|
—
|
-11.0 Percent change in LDL-C
Standard Deviation 25.28
|
|
Percent Change in LDL-C From Baseline up to Day 720
Day 510
|
—
|
—
|
-12.5 Percent change in LDL-C
Standard Deviation 22.52
|
|
Percent Change in LDL-C From Baseline up to Day 720
Day 630
|
—
|
—
|
-9.4 Percent change in LDL-C
Standard Deviation 26.16
|
|
Percent Change in LDL-C From Baseline up to Day 720
Day 720
|
—
|
—
|
-12.6 Percent change in LDL-C
Standard Deviation 28.45
|
SECONDARY outcome
Timeframe: Baseline, up to Day 720Population: Full Analysis Set (FAS) comprised all participants to whom study treatment had been assigned by randomization
Absolute change in LDL-C from baseline to each assessment time up to Day 720.
Outcome measures
| Measure |
Part 1 - Placebo
n=4 Participants
Placebo sc injection (given at Day 1, 90 and 270)
|
Part 1- Inclisiran
n=9 Participants
Inclisiran sodium 300 mg subcutaneous (sc) injection (given at Days 1, 90 and 270)
|
Part 2 - Inclisiran (Total)
n=13 Participants
Inclisiran sodium 300 mg sc injection (given at Days 450 and 630). In addition, participants assigned to placebo in Part 1 received inclisiran sodium 300 mg sc injection on Day 360, while participants assigned to inclisiran in Part 1 received placebo sc injection on Day 360.
|
|---|---|---|---|
|
Absolute Change in LDL-C From Baseline up to Day 720
Day 360
|
-4.3 mg/dL
Standard Deviation 69.84
|
-50.8 mg/dL
Standard Deviation 44.65
|
—
|
|
Absolute Change in LDL-C From Baseline up to Day 720
Day 450
|
—
|
—
|
-36.6 mg/dL
Standard Deviation 75.08
|
|
Absolute Change in LDL-C From Baseline up to Day 720
Day 510
|
—
|
—
|
-46.8 mg/dL
Standard Deviation 73.04
|
|
Absolute Change in LDL-C From Baseline up to Day 720
Day 630
|
—
|
—
|
-41.9 mg/dL
Standard Deviation 92.47
|
|
Absolute Change in LDL-C From Baseline up to Day 720
Day 720
|
—
|
—
|
-39.8 mg/dL
Standard Deviation 96.59
|
|
Absolute Change in LDL-C From Baseline up to Day 720
Day 90
|
16.5 mg/dL
Standard Deviation 44.96
|
-70.6 mg/dL
Standard Deviation 48.81
|
—
|
|
Absolute Change in LDL-C From Baseline up to Day 720
Day 150
|
20.3 mg/dL
Standard Deviation 91.34
|
-66.1 mg/dL
Standard Deviation 61.85
|
—
|
|
Absolute Change in LDL-C From Baseline up to Day 720
Day 270
|
-9.3 mg/dL
Standard Deviation 134.14
|
-47.1 mg/dL
Standard Deviation 83.15
|
—
|
|
Absolute Change in LDL-C From Baseline up to Day 720
Day 330
|
11.8 mg/dL
Standard Deviation 69.94
|
-62.9 mg/dL
Standard Deviation 52.35
|
—
|
SECONDARY outcome
Timeframe: Baseline, up to Day 720Population: Full Analysis Set (FAS) comprised all participants to whom study treatment had been assigned by randomization
Percentage change in apolipoprotein B (Apo B) from baseline to each assessment time up to Day 720.
Outcome measures
| Measure |
Part 1 - Placebo
n=4 Participants
Placebo sc injection (given at Day 1, 90 and 270)
|
Part 1- Inclisiran
n=9 Participants
Inclisiran sodium 300 mg subcutaneous (sc) injection (given at Days 1, 90 and 270)
|
Part 2 - Inclisiran (Total)
n=13 Participants
Inclisiran sodium 300 mg sc injection (given at Days 450 and 630). In addition, participants assigned to placebo in Part 1 received inclisiran sodium 300 mg sc injection on Day 360, while participants assigned to inclisiran in Part 1 received placebo sc injection on Day 360.
|
|---|---|---|---|
|
Percent Change in Apo B From Baseline up to Day 720
Day 150
|
10.0 Percent change in Apo B
Standard Deviation 27.05
|
-20.3 Percent change in Apo B
Standard Deviation 12.92
|
—
|
|
Percent Change in Apo B From Baseline up to Day 720
Day 330
|
4.5 Percent change in Apo B
Standard Deviation 17.91
|
-18.5 Percent change in Apo B
Standard Deviation 10.47
|
—
|
|
Percent Change in Apo B From Baseline up to Day 720
Day 360
|
10.6 Percent change in Apo B
Standard Deviation 21.33
|
-14.8 Percent change in Apo B
Standard Deviation 10.11
|
—
|
|
Percent Change in Apo B From Baseline up to Day 720
Day 510
|
—
|
—
|
-5.7 Percent change in Apo B
Standard Deviation 25.96
|
|
Percent Change in Apo B From Baseline up to Day 720
Day 720 (study completion)
|
—
|
—
|
-7.3 Percent change in Apo B
Standard Deviation 29.36
|
SECONDARY outcome
Timeframe: Baseline, up to Day 720Population: Full Analysis Set (FAS) comprised all participants to whom study treatment had been assigned by randomization
Absolute change in apolipoprotein B (Apo B) from baseline to each assessment time up to Day 720.
Outcome measures
| Measure |
Part 1 - Placebo
n=4 Participants
Placebo sc injection (given at Day 1, 90 and 270)
|
Part 1- Inclisiran
n=9 Participants
Inclisiran sodium 300 mg subcutaneous (sc) injection (given at Days 1, 90 and 270)
|
Part 2 - Inclisiran (Total)
n=13 Participants
Inclisiran sodium 300 mg sc injection (given at Days 450 and 630). In addition, participants assigned to placebo in Part 1 received inclisiran sodium 300 mg sc injection on Day 360, while participants assigned to inclisiran in Part 1 received placebo sc injection on Day 360.
|
|---|---|---|---|
|
Absolute Change in Apo B From Baseline up to Day 720
Day 150
|
7.5 mg/dL
Standard Deviation 48.72
|
-37.3 mg/dL
Standard Deviation 33.35
|
—
|
|
Absolute Change in Apo B From Baseline up to Day 720
Day 330
|
0.3 mg/dL
Standard Deviation 35.85
|
-35.8 mg/dL
Standard Deviation 30.33
|
—
|
|
Absolute Change in Apo B From Baseline up to Day 720
Day 360
|
12.0 mg/dL
Standard Deviation 32.89
|
-28.7 mg/dL
Standard Deviation 27.16
|
—
|
|
Absolute Change in Apo B From Baseline up to Day 720
Day 510
|
—
|
—
|
-18.8 mg/dL
Standard Deviation 45.81
|
|
Absolute Change in Apo B From Baseline up to Day 720
Day 720 (study completion)
|
—
|
—
|
-19.6 mg/dL
Standard Deviation 51.64
|
SECONDARY outcome
Timeframe: Baseline, up to Day 720Population: Full Analysis Set (FAS) comprised all participants to whom study treatment had been assigned by randomization
Percentage change in lipoprotein (a) \[Lp(a)\] from baseline to each assessment time up to Day 720.
Outcome measures
| Measure |
Part 1 - Placebo
n=4 Participants
Placebo sc injection (given at Day 1, 90 and 270)
|
Part 1- Inclisiran
n=9 Participants
Inclisiran sodium 300 mg subcutaneous (sc) injection (given at Days 1, 90 and 270)
|
Part 2 - Inclisiran (Total)
n=13 Participants
Inclisiran sodium 300 mg sc injection (given at Days 450 and 630). In addition, participants assigned to placebo in Part 1 received inclisiran sodium 300 mg sc injection on Day 360, while participants assigned to inclisiran in Part 1 received placebo sc injection on Day 360.
|
|---|---|---|---|
|
Percent Change in Lp(a) From Baseline up to Day 720
Day 150
|
-4.4 Percent change in Lp(a)
Standard Deviation 9.20
|
-0.7 Percent change in Lp(a)
Standard Deviation 24.81
|
—
|
|
Percent Change in Lp(a) From Baseline up to Day 720
Day 330
|
-16.5 Percent change in Lp(a)
Standard Deviation 27.29
|
-0.3 Percent change in Lp(a)
Standard Deviation 21.27
|
—
|
|
Percent Change in Lp(a) From Baseline up to Day 720
Day 360
|
-12.1 Percent change in Lp(a)
Standard Deviation 31.51
|
-1.7 Percent change in Lp(a)
Standard Deviation 16.57
|
—
|
|
Percent Change in Lp(a) From Baseline up to Day 720
Day 510
|
—
|
—
|
-7.2 Percent change in Lp(a)
Standard Deviation 31.39
|
|
Percent Change in Lp(a) From Baseline up to Day 720
Day 720 (study completion)
|
—
|
—
|
-8.8 Percent change in Lp(a)
Standard Deviation 22.03
|
SECONDARY outcome
Timeframe: Baseline, up to Day 720Population: Full Analysis Set (FAS) comprised all participants to whom study treatment had been assigned by randomization
Absolute change in lipoprotein (a) \[Lp(a)\] from baseline to each assessment time up to Day 720.
Outcome measures
| Measure |
Part 1 - Placebo
n=4 Participants
Placebo sc injection (given at Day 1, 90 and 270)
|
Part 1- Inclisiran
n=9 Participants
Inclisiran sodium 300 mg subcutaneous (sc) injection (given at Days 1, 90 and 270)
|
Part 2 - Inclisiran (Total)
n=13 Participants
Inclisiran sodium 300 mg sc injection (given at Days 450 and 630). In addition, participants assigned to placebo in Part 1 received inclisiran sodium 300 mg sc injection on Day 360, while participants assigned to inclisiran in Part 1 received placebo sc injection on Day 360.
|
|---|---|---|---|
|
Absolute Change in Lp(a) From Baseline up to Day 720
Day 150
|
-0.3 nmol/L
Standard Deviation 1.26
|
8.9 nmol/L
Standard Deviation 28.41
|
—
|
|
Absolute Change in Lp(a) From Baseline up to Day 720
Day 330
|
5.3 nmol/L
Standard Deviation 15.86
|
2.8 nmol/L
Standard Deviation 15.01
|
—
|
|
Absolute Change in Lp(a) From Baseline up to Day 720
Day 360
|
0.5 nmol/L
Standard Deviation 9.75
|
5.8 nmol/L
Standard Deviation 7.03
|
—
|
|
Absolute Change in Lp(a) From Baseline up to Day 720
Day 510
|
—
|
—
|
3.0 nmol/L
Standard Deviation 14.45
|
|
Absolute Change in Lp(a) From Baseline up to Day 720
Day 720 (study completion)
|
—
|
—
|
-0.3 nmol/L
Standard Deviation 9.12
|
SECONDARY outcome
Timeframe: Baseline, up to Day 720Population: Full Analysis Set (FAS) comprised all participants to whom study treatment had been assigned by randomization
Percentage change in non-high density lipoprotein cholesterol (non-HDL-C) from baseline to each assessment time up to Day 720.
Outcome measures
| Measure |
Part 1 - Placebo
n=4 Participants
Placebo sc injection (given at Day 1, 90 and 270)
|
Part 1- Inclisiran
n=9 Participants
Inclisiran sodium 300 mg subcutaneous (sc) injection (given at Days 1, 90 and 270)
|
Part 2 - Inclisiran (Total)
n=13 Participants
Inclisiran sodium 300 mg sc injection (given at Days 450 and 630). In addition, participants assigned to placebo in Part 1 received inclisiran sodium 300 mg sc injection on Day 360, while participants assigned to inclisiran in Part 1 received placebo sc injection on Day 360.
|
|---|---|---|---|
|
Percent Change in Non-HDL-C From Baseline up to Day 720
Day 150
|
19.0 Percent change in non-HDL-C
Standard Deviation 43.34
|
-24.0 Percent change in non-HDL-C
Standard Deviation 13.83
|
—
|
|
Percent Change in Non-HDL-C From Baseline up to Day 720
Day 330
|
9.4 Percent change in non-HDL-C
Standard Deviation 34.93
|
-23.3 Percent change in non-HDL-C
Standard Deviation 10.99
|
—
|
|
Percent Change in Non-HDL-C From Baseline up to Day 720
Day 360
|
7.1 Percent change in non-HDL-C
Standard Deviation 24.38
|
-20.1 Percent change in non-HDL-C
Standard Deviation 10.91
|
—
|
|
Percent Change in Non-HDL-C From Baseline up to Day 720
Day 510
|
—
|
—
|
-11.8 Percent change in non-HDL-C
Standard Deviation 25.33
|
|
Percent Change in Non-HDL-C From Baseline up to Day 720
Day 720 (study completion)
|
—
|
—
|
-13.0 Percent change in non-HDL-C
Standard Deviation 23.65
|
SECONDARY outcome
Timeframe: Baseline, up to Day 720Population: Full Analysis Set (FAS) comprised all participants to whom study treatment had been assigned by randomization
Absolute change in non-high density lipoprotein cholesterol (non-HDL-C) from baseline to each assessment time up to Day 720.
Outcome measures
| Measure |
Part 1 - Placebo
n=4 Participants
Placebo sc injection (given at Day 1, 90 and 270)
|
Part 1- Inclisiran
n=9 Participants
Inclisiran sodium 300 mg subcutaneous (sc) injection (given at Days 1, 90 and 270)
|
Part 2 - Inclisiran (Total)
n=13 Participants
Inclisiran sodium 300 mg sc injection (given at Days 450 and 630). In addition, participants assigned to placebo in Part 1 received inclisiran sodium 300 mg sc injection on Day 360, while participants assigned to inclisiran in Part 1 received placebo sc injection on Day 360.
|
|---|---|---|---|
|
Absolute Change in Non-HDL-C From Baseline up to Day 720
Day 150
|
28.8 mg/dL
Standard Deviation 94.01
|
-73.8 mg/dL
Standard Deviation 68.01
|
—
|
|
Absolute Change in Non-HDL-C From Baseline up to Day 720
Day 330
|
4.3 mg/dL
Standard Deviation 82.16
|
-74.4 mg/dL
Standard Deviation 57.63
|
—
|
|
Absolute Change in Non-HDL-C From Baseline up to Day 720
Day 360
|
6.0 mg/dL
Standard Deviation 56.11
|
-60.0 mg/dL
Standard Deviation 44.74
|
—
|
|
Absolute Change in Non-HDL-C From Baseline up to Day 720
Day 510
|
—
|
—
|
-51.4 mg/dL
Standard Deviation 81.53
|
|
Absolute Change in Non-HDL-C From Baseline up to Day 720
Day 720 (study completion)
|
—
|
—
|
-45.8 mg/dL
Standard Deviation 89.10
|
SECONDARY outcome
Timeframe: Baseline, up to Day 720Population: Full Analysis Set (FAS) comprised all participants to whom study treatment had been assigned by randomization
Percentage change in total cholesterol from baseline to each assessment time up to Day 720.
Outcome measures
| Measure |
Part 1 - Placebo
n=4 Participants
Placebo sc injection (given at Day 1, 90 and 270)
|
Part 1- Inclisiran
n=9 Participants
Inclisiran sodium 300 mg subcutaneous (sc) injection (given at Days 1, 90 and 270)
|
Part 2 - Inclisiran (Total)
n=13 Participants
Inclisiran sodium 300 mg sc injection (given at Days 450 and 630). In addition, participants assigned to placebo in Part 1 received inclisiran sodium 300 mg sc injection on Day 360, while participants assigned to inclisiran in Part 1 received placebo sc injection on Day 360.
|
|---|---|---|---|
|
Percent Change in Total Cholesterol From Baseline up to Day 720
Day 150
|
16.0 Percent change in total cholesterol
Standard Deviation 37.96
|
-19.0 Percent change in total cholesterol
Standard Deviation 12.49
|
—
|
|
Percent Change in Total Cholesterol From Baseline up to Day 720
Day 330
|
8.7 Percent change in total cholesterol
Standard Deviation 30.54
|
-19.1 Percent change in total cholesterol
Standard Deviation 10.30
|
—
|
|
Percent Change in Total Cholesterol From Baseline up to Day 720
Day 360
|
6.8 Percent change in total cholesterol
Standard Deviation 21.06
|
-16.1 Percent change in total cholesterol
Standard Deviation 9.53
|
—
|
|
Percent Change in Total Cholesterol From Baseline up to Day 720
Day 510
|
—
|
—
|
-9.3 Percent change in total cholesterol
Standard Deviation 20.73
|
|
Percent Change in Total Cholesterol From Baseline up to Day 720
Day 720 (study completion)
|
—
|
—
|
-9.6 Percent change in total cholesterol
Standard Deviation 20.86
|
SECONDARY outcome
Timeframe: Baseline, up to Day 720Population: Full Analysis Set (FAS) comprised all participants to whom study treatment had been assigned by randomization
Absolute change in total cholesterol from baseline to each assessment time up to Day 720.
Outcome measures
| Measure |
Part 1 - Placebo
n=4 Participants
Placebo sc injection (given at Day 1, 90 and 270)
|
Part 1- Inclisiran
n=9 Participants
Inclisiran sodium 300 mg subcutaneous (sc) injection (given at Days 1, 90 and 270)
|
Part 2 - Inclisiran (Total)
n=13 Participants
Inclisiran sodium 300 mg sc injection (given at Days 450 and 630). In addition, participants assigned to placebo in Part 1 received inclisiran sodium 300 mg sc injection on Day 360, while participants assigned to inclisiran in Part 1 received placebo sc injection on Day 360.
|
|---|---|---|---|
|
Absolute Change in Total Cholesterol From Baseline up to Day 720
Day 150
|
29.5 mg/dL
Standard Deviation 99.30
|
-69.7 mg/dL
Standard Deviation 67.42
|
—
|
|
Absolute Change in Total Cholesterol From Baseline up to Day 720
Day 330
|
8.8 mg/dL
Standard Deviation 84.68
|
-71.4 mg/dL
Standard Deviation 56.65
|
—
|
|
Absolute Change in Total Cholesterol From Baseline up to Day 720
Day 360
|
10.0 mg/dL
Standard Deviation 59.14
|
-57.3 mg/dL
Standard Deviation 43.82
|
—
|
|
Absolute Change in Total Cholesterol From Baseline up to Day 720
Day 510
|
—
|
—
|
-46.5 mg/dL
Standard Deviation 80.87
|
|
Absolute Change in Total Cholesterol From Baseline up to Day 720
Day 720 (study completion)
|
—
|
—
|
-40.4 mg/dL
Standard Deviation 88.44
|
SECONDARY outcome
Timeframe: Baseline, up to Day 720Population: Full Analysis Set (FAS) comprised all participants to whom study treatment had been assigned by randomization
Percentage change in triglycerides from baseline to each assessment time up to Day 720.
Outcome measures
| Measure |
Part 1 - Placebo
n=4 Participants
Placebo sc injection (given at Day 1, 90 and 270)
|
Part 1- Inclisiran
n=9 Participants
Inclisiran sodium 300 mg subcutaneous (sc) injection (given at Days 1, 90 and 270)
|
Part 2 - Inclisiran (Total)
n=13 Participants
Inclisiran sodium 300 mg sc injection (given at Days 450 and 630). In addition, participants assigned to placebo in Part 1 received inclisiran sodium 300 mg sc injection on Day 360, while participants assigned to inclisiran in Part 1 received placebo sc injection on Day 360.
|
|---|---|---|---|
|
Percent Change in Triglycerides From Baseline up to Day 720
Day 150
|
-15.7 Percent change in triglycerides
Standard Deviation 5.97
|
0.3 Percent change in triglycerides
Standard Deviation 31.92
|
—
|
|
Percent Change in Triglycerides From Baseline up to Day 720
Day 330
|
-5.8 Percent change in triglycerides
Standard Deviation 31.90
|
-9.6 Percent change in triglycerides
Standard Deviation 30.66
|
—
|
|
Percent Change in Triglycerides From Baseline up to Day 720
Day 360
|
14.1 Percent change in triglycerides
Standard Deviation 54.93
|
11.4 Percent change in triglycerides
Standard Deviation 24.72
|
—
|
|
Percent Change in Triglycerides From Baseline up to Day 720
Day 510
|
—
|
—
|
4.7 Percent change in triglycerides
Standard Deviation 37.21
|
|
Percent Change in Triglycerides From Baseline up to Day 720
Day 720 (study completion)
|
—
|
—
|
2.9 Percent change in triglycerides
Standard Deviation 26.02
|
SECONDARY outcome
Timeframe: Baseline, up to Day 720Population: Full Analysis Set (FAS) comprised all participants to whom study treatment had been assigned by randomization
Absolute change in triglycerides from baseline to each assessment time up to Day 720.
Outcome measures
| Measure |
Part 1 - Placebo
n=4 Participants
Placebo sc injection (given at Day 1, 90 and 270)
|
Part 1- Inclisiran
n=9 Participants
Inclisiran sodium 300 mg subcutaneous (sc) injection (given at Days 1, 90 and 270)
|
Part 2 - Inclisiran (Total)
n=13 Participants
Inclisiran sodium 300 mg sc injection (given at Days 450 and 630). In addition, participants assigned to placebo in Part 1 received inclisiran sodium 300 mg sc injection on Day 360, while participants assigned to inclisiran in Part 1 received placebo sc injection on Day 360.
|
|---|---|---|---|
|
Absolute Change in Triglycerides From Baseline up to Day 720
Day 150
|
-15.0 mg/dL
Standard Deviation 7.53
|
-3.4 mg/dL
Standard Deviation 23.80
|
—
|
|
Absolute Change in Triglycerides From Baseline up to Day 720
Day 330
|
-2.3 mg/dL
Standard Deviation 25.01
|
-9.4 mg/dL
Standard Deviation 21.98
|
—
|
|
Absolute Change in Triglycerides From Baseline up to Day 720
Day 360
|
28.0 mg/dL
Standard Deviation 72.23
|
8.7 mg/dL
Standard Deviation 24.15
|
—
|
|
Absolute Change in Triglycerides From Baseline up to Day 720
Day 510
|
—
|
—
|
3.3 mg/dL
Standard Deviation 34.32
|
|
Absolute Change in Triglycerides From Baseline up to Day 720
Day 720 (study completion)
|
—
|
—
|
3.9 mg/dL
Standard Deviation 27.40
|
SECONDARY outcome
Timeframe: Baseline, up to Day 720Population: Full Analysis Set (FAS) comprised all participants to whom study treatment had been assigned by randomization
Percentage change in high density lipoprotein cholesterol (HDL-C) from baseline to each assessment time up to Day 720.
Outcome measures
| Measure |
Part 1 - Placebo
n=4 Participants
Placebo sc injection (given at Day 1, 90 and 270)
|
Part 1- Inclisiran
n=9 Participants
Inclisiran sodium 300 mg subcutaneous (sc) injection (given at Days 1, 90 and 270)
|
Part 2 - Inclisiran (Total)
n=13 Participants
Inclisiran sodium 300 mg sc injection (given at Days 450 and 630). In addition, participants assigned to placebo in Part 1 received inclisiran sodium 300 mg sc injection on Day 360, while participants assigned to inclisiran in Part 1 received placebo sc injection on Day 360.
|
|---|---|---|---|
|
Percent Change in HDL-C From Baseline up to Day 720
Day 150
|
-0.1 Percent change in HDL-C
Standard Deviation 20.06
|
10.0 Percent change in HDL-C
Standard Deviation 13.22
|
—
|
|
Percent Change in HDL-C From Baseline up to Day 720
Day 330
|
10.9 Percent change in HDL-C
Standard Deviation 12.48
|
7.5 Percent change in HDL-C
Standard Deviation 12.93
|
—
|
|
Percent Change in HDL-C From Baseline up to Day 720
Day 360
|
9.2 Percent change in HDL-C
Standard Deviation 21.20
|
6.7 Percent change in HDL-C
Standard Deviation 20.60
|
—
|
|
Percent Change in HDL-C From Baseline up to Day 720
Day 510
|
—
|
—
|
12.3 Percent change in HDL-C
Standard Deviation 17.63
|
|
Percent Change in HDL-C From Baseline up to Day 720
Day 720 (study completion)
|
—
|
—
|
13.9 Percent change in HDL-C
Standard Deviation 18.81
|
SECONDARY outcome
Timeframe: Baseline, up to Day 720Population: Full Analysis Set (FAS) comprised all participants to whom study treatment had been assigned by randomization
Absolute change in high density lipoprotein cholesterol (HDL-C) from baseline to each assessment time up to Day 720.
Outcome measures
| Measure |
Part 1 - Placebo
n=4 Participants
Placebo sc injection (given at Day 1, 90 and 270)
|
Part 1- Inclisiran
n=9 Participants
Inclisiran sodium 300 mg subcutaneous (sc) injection (given at Days 1, 90 and 270)
|
Part 2 - Inclisiran (Total)
n=13 Participants
Inclisiran sodium 300 mg sc injection (given at Days 450 and 630). In addition, participants assigned to placebo in Part 1 received inclisiran sodium 300 mg sc injection on Day 360, while participants assigned to inclisiran in Part 1 received placebo sc injection on Day 360.
|
|---|---|---|---|
|
Absolute Change in HDL-C From Baseline up to Day 720
Day 150
|
0.8 mg/dL
Standard Deviation 7.50
|
4.1 mg/dL
Standard Deviation 5.18
|
—
|
|
Absolute Change in HDL-C From Baseline up to Day 720
Day 330
|
4.5 mg/dL
Standard Deviation 5.07
|
3.0 mg/dL
Standard Deviation 5.05
|
—
|
|
Absolute Change in HDL-C From Baseline up to Day 720
Day 360
|
4.0 mg/dL
Standard Deviation 8.76
|
2.7 mg/dL
Standard Deviation 7.75
|
—
|
|
Absolute Change in HDL-C From Baseline up to Day 720
Day 510
|
—
|
—
|
4.9 mg/dL
Standard Deviation 7.94
|
|
Absolute Change in HDL-C From Baseline up to Day 720
Day 720 (study completion)
|
—
|
—
|
5.4 mg/dL
Standard Deviation 7.81
|
SECONDARY outcome
Timeframe: Baseline, up to Day 720Population: Full Analysis Set (FAS) comprised all participants to whom study treatment had been assigned by randomization
Percentage change in very low density lipoprotein cholesterol (VLDL-C) from baseline to each assessment time up to Day 720.
Outcome measures
| Measure |
Part 1 - Placebo
n=4 Participants
Placebo sc injection (given at Day 1, 90 and 270)
|
Part 1- Inclisiran
n=9 Participants
Inclisiran sodium 300 mg subcutaneous (sc) injection (given at Days 1, 90 and 270)
|
Part 2 - Inclisiran (Total)
n=13 Participants
Inclisiran sodium 300 mg sc injection (given at Days 450 and 630). In addition, participants assigned to placebo in Part 1 received inclisiran sodium 300 mg sc injection on Day 360, while participants assigned to inclisiran in Part 1 received placebo sc injection on Day 360.
|
|---|---|---|---|
|
Percent Change in VLDL-C From Baseline up to Day 720
Day 150
|
74.5 Percent change in VLDL-C
Standard Deviation 108.97
|
-7.0 Percent change in VLDL-C
Standard Deviation 70.31
|
—
|
|
Percent Change in VLDL-C From Baseline up to Day 720
Day 330
|
2.0 Percent change in VLDL-C
Standard Deviation 113.44
|
-29.0 Percent change in VLDL-C
Standard Deviation 49.96
|
—
|
|
Percent Change in VLDL-C From Baseline up to Day 720
Day 360
|
34.6 Percent change in VLDL-C
Standard Deviation 56.37
|
-17.0 Percent change in VLDL-C
Standard Deviation 60.36
|
—
|
|
Percent Change in VLDL-C From Baseline up to Day 720
Day 510
|
—
|
—
|
9.5 Percent change in VLDL-C
Standard Deviation 98.44
|
|
Percent Change in VLDL-C From Baseline up to Day 720
Day 720 (study completion)
|
—
|
—
|
-1.4 Percent change in VLDL-C
Standard Deviation 69.03
|
SECONDARY outcome
Timeframe: Baseline, up to Day 720Population: Full Analysis Set (FAS) comprised all participants to whom study treatment had been assigned by randomization
Absolute change in very low density lipoprotein cholesterol (VLDL-C) from baseline to each assessment time up to Day 720.
Outcome measures
| Measure |
Part 1 - Placebo
n=4 Participants
Placebo sc injection (given at Day 1, 90 and 270)
|
Part 1- Inclisiran
n=9 Participants
Inclisiran sodium 300 mg subcutaneous (sc) injection (given at Days 1, 90 and 270)
|
Part 2 - Inclisiran (Total)
n=13 Participants
Inclisiran sodium 300 mg sc injection (given at Days 450 and 630). In addition, participants assigned to placebo in Part 1 received inclisiran sodium 300 mg sc injection on Day 360, while participants assigned to inclisiran in Part 1 received placebo sc injection on Day 360.
|
|---|---|---|---|
|
Absolut Change in VLDL-C From Baseline up to Day 720
Day 150
|
8.5 mg/dL
Standard Deviation 20.37
|
-7.7 mg/dL
Standard Deviation 11.39
|
—
|
|
Absolut Change in VLDL-C From Baseline up to Day 720
Day 330
|
-7.5 mg/dL
Standard Deviation 18.21
|
-11.6 mg/dL
Standard Deviation 11.71
|
—
|
|
Absolut Change in VLDL-C From Baseline up to Day 720
Day 360
|
10.3 mg/dL
Standard Deviation 21.00
|
-9.2 mg/dL
Standard Deviation 17.89
|
—
|
|
Absolut Change in VLDL-C From Baseline up to Day 720
Day 510
|
—
|
—
|
-4.6 mg/dL
Standard Deviation 18.41
|
|
Absolut Change in VLDL-C From Baseline up to Day 720
Day 720 (study completion)
|
—
|
—
|
-6.0 mg/dL
Standard Deviation 18.81
|
SECONDARY outcome
Timeframe: Baseline, up to Day 720Population: Full Analysis Set (FAS) comprised all participants to whom study treatment had been assigned by randomization
Percentage change in apolipoprotein A1 (Apo A1) from baseline to each assessment time up to Day 720.
Outcome measures
| Measure |
Part 1 - Placebo
n=4 Participants
Placebo sc injection (given at Day 1, 90 and 270)
|
Part 1- Inclisiran
n=9 Participants
Inclisiran sodium 300 mg subcutaneous (sc) injection (given at Days 1, 90 and 270)
|
Part 2 - Inclisiran (Total)
n=13 Participants
Inclisiran sodium 300 mg sc injection (given at Days 450 and 630). In addition, participants assigned to placebo in Part 1 received inclisiran sodium 300 mg sc injection on Day 360, while participants assigned to inclisiran in Part 1 received placebo sc injection on Day 360.
|
|---|---|---|---|
|
Percent Change in Apo A1 From Baseline up to Day 720
Day 150
|
-1.3 Percent change in Apo A1
Standard Deviation 2.27
|
-0.9 Percent change in Apo A1
Standard Deviation 10.72
|
—
|
|
Percent Change in Apo A1 From Baseline up to Day 720
Day 330
|
9.0 Percent change in Apo A1
Standard Deviation 7.02
|
-2.1 Percent change in Apo A1
Standard Deviation 10.87
|
—
|
|
Percent Change in Apo A1 From Baseline up to Day 720
Day 360
|
13.7 Percent change in Apo A1
Standard Deviation 12.99
|
3.1 Percent change in Apo A1
Standard Deviation 16.49
|
—
|
|
Percent Change in Apo A1 From Baseline up to Day 720
Day 510
|
—
|
—
|
12.8 Percent change in Apo A1
Standard Deviation 12.08
|
|
Percent Change in Apo A1 From Baseline up to Day 720
Day 720 (study completion)
|
—
|
—
|
6.5 Percent change in Apo A1
Standard Deviation 10.10
|
SECONDARY outcome
Timeframe: Baseline, up to Day 720Population: Full Analysis Set (FAS) comprised all participants to whom study treatment had been assigned by randomization
Absolute change in apolipoprotein A1 (Apo A1) from baseline to each assessment time up to Day 720.
Outcome measures
| Measure |
Part 1 - Placebo
n=4 Participants
Placebo sc injection (given at Day 1, 90 and 270)
|
Part 1- Inclisiran
n=9 Participants
Inclisiran sodium 300 mg subcutaneous (sc) injection (given at Days 1, 90 and 270)
|
Part 2 - Inclisiran (Total)
n=13 Participants
Inclisiran sodium 300 mg sc injection (given at Days 450 and 630). In addition, participants assigned to placebo in Part 1 received inclisiran sodium 300 mg sc injection on Day 360, while participants assigned to inclisiran in Part 1 received placebo sc injection on Day 360.
|
|---|---|---|---|
|
Absolute Change in Apo A1 From Baseline up to Day 720
Day 150
|
-1.3 mg/dL
Standard Deviation 2.36
|
-1.2 mg/dL
Standard Deviation 14.10
|
—
|
|
Absolute Change in Apo A1 From Baseline up to Day 720
Day 330
|
10.3 mg/dL
Standard Deviation 7.41
|
-2.7 mg/dL
Standard Deviation 13.47
|
—
|
|
Absolute Change in Apo A1 From Baseline up to Day 720
Day 360
|
16.0 mg/dL
Standard Deviation 14.79
|
4.2 mg/dL
Standard Deviation 18.82
|
—
|
|
Absolute Change in Apo A1 From Baseline up to Day 720
Day 510
|
—
|
—
|
14.9 mg/dL
Standard Deviation 13.25
|
|
Absolute Change in Apo A1 From Baseline up to Day 720
Day 720 (study completion)
|
—
|
—
|
7.4 mg/dL
Standard Deviation 12.40
|
SECONDARY outcome
Timeframe: Baseline, up to Day 720Population: Full Analysis Set (FAS) comprised all participants to whom study treatment had been assigned by randomization
Percentage change in proprotein convertase subtilisin/kexin type 9 (PCSK9) from baseline to each assessment time up to Day 720.
Outcome measures
| Measure |
Part 1 - Placebo
n=4 Participants
Placebo sc injection (given at Day 1, 90 and 270)
|
Part 1- Inclisiran
n=9 Participants
Inclisiran sodium 300 mg subcutaneous (sc) injection (given at Days 1, 90 and 270)
|
Part 2 - Inclisiran (Total)
n=13 Participants
Inclisiran sodium 300 mg sc injection (given at Days 450 and 630). In addition, participants assigned to placebo in Part 1 received inclisiran sodium 300 mg sc injection on Day 360, while participants assigned to inclisiran in Part 1 received placebo sc injection on Day 360.
|
|---|---|---|---|
|
Percent Change in PCSK9 From Baseline up to Day 720
Day 90
|
-17.3 Percent change in PCSK9
Standard Deviation 14.77
|
-56.8 Percent change in PCSK9
Standard Deviation 13.73
|
—
|
|
Percent Change in PCSK9 From Baseline up to Day 720
Day 150
|
-1.7 Percent change in PCSK9
Standard Deviation 11.11
|
-60.4 Percent change in PCSK9
Standard Deviation 12.62
|
—
|
|
Percent Change in PCSK9 From Baseline up to Day 720
Day 330
|
-5.1 Percent change in PCSK9
Standard Deviation 19.60
|
-65.3 Percent change in PCSK9
Standard Deviation 12.51
|
—
|
|
Percent Change in PCSK9 From Baseline up to Day 720
Day 360
|
-2.8 Percent change in PCSK9
Standard Deviation 22.65
|
-59.7 Percent change in PCSK9
Standard Deviation 9.05
|
—
|
|
Percent Change in PCSK9 From Baseline up to Day 720
Day 510
|
—
|
—
|
-68.1 Percent change in PCSK9
Standard Deviation 10.57
|
|
Percent Change in PCSK9 From Baseline up to Day 720
Day 720 (study completion)
|
—
|
—
|
-64.1 Percent change in PCSK9
Standard Deviation 12.12
|
SECONDARY outcome
Timeframe: Baseline, up to Day 720Population: Full Analysis Set (FAS) comprised all participants to whom study treatment had been assigned by randomization
Absolute change in proprotein convertase subtilisin/kexin type 9 (PCSK9) from baseline to each assessment time up to Day 720.
Outcome measures
| Measure |
Part 1 - Placebo
n=4 Participants
Placebo sc injection (given at Day 1, 90 and 270)
|
Part 1- Inclisiran
n=9 Participants
Inclisiran sodium 300 mg subcutaneous (sc) injection (given at Days 1, 90 and 270)
|
Part 2 - Inclisiran (Total)
n=13 Participants
Inclisiran sodium 300 mg sc injection (given at Days 450 and 630). In addition, participants assigned to placebo in Part 1 received inclisiran sodium 300 mg sc injection on Day 360, while participants assigned to inclisiran in Part 1 received placebo sc injection on Day 360.
|
|---|---|---|---|
|
Absolut Change in PCSK9 From Baseline up to Day 720
Day 90
|
-98.5 ng/mL
Standard Deviation 87.46
|
-278.7 ng/mL
Standard Deviation 113.31
|
—
|
|
Absolut Change in PCSK9 From Baseline up to Day 720
Day 150
|
-6.1 ng/mL
Standard Deviation 64.82
|
-296.3 ng/mL
Standard Deviation 118.18
|
—
|
|
Absolut Change in PCSK9 From Baseline up to Day 720
Day 330
|
-35.3 ng/mL
Standard Deviation 100.97
|
-323.1 ng/mL
Standard Deviation 129.32
|
—
|
|
Absolut Change in PCSK9 From Baseline up to Day 720
Day 360
|
-14.0 ng/mL
Standard Deviation 127.42
|
-292.6 ng/mL
Standard Deviation 100.44
|
—
|
|
Absolut Change in PCSK9 From Baseline up to Day 720
Day 510
|
—
|
—
|
-347.3 ng/mL
Standard Deviation 106.3
|
|
Absolut Change in PCSK9 From Baseline up to Day 720
Day 720 (study completion)
|
—
|
—
|
-327.7 ng/mL
Standard Deviation 110.4
|
Adverse Events
Part 1 - Inclisiran
Serious events: 0 serious events
Other events: 7 other events
Deaths: 0 deaths
Part 1 - Placebo
Serious events: 0 serious events
Other events: 1 other events
Deaths: 0 deaths
Part 2 - Inclisiran (Total)
Serious events: 0 serious events
Other events: 8 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Part 1 - Inclisiran
n=9 participants at risk
Inclisiran sodium 300 mg subcutaneous injection (given at Days 1, 90 and 270)
|
Part 1 - Placebo
n=4 participants at risk
Placebo subcutaneous injection (given at Day 1, 90 and 270)
|
Part 2 - Inclisiran (Total)
n=13 participants at risk
Inclisiran sodium 300 mg subcutaneous injection (given at Days 450 and 630). In addition, participants assigned to placebo in Part 1 received inclisiran sodium 300 mg sc injection on Day 360, while participants assigned to inclisiran in Part 1 received placebo sc injection on Day 360
|
|---|---|---|---|
|
Gastrointestinal disorders
Abdominal pain upper
|
22.2%
2/9 • Adverse events were reported from first dose of study treatment until end of study treatment plus 90 days post treatment or 30 days after last study visit, whichever was longer, up to a maximum duration of approximately 2 years.
|
0.00%
0/4 • Adverse events were reported from first dose of study treatment until end of study treatment plus 90 days post treatment or 30 days after last study visit, whichever was longer, up to a maximum duration of approximately 2 years.
|
7.7%
1/13 • Adverse events were reported from first dose of study treatment until end of study treatment plus 90 days post treatment or 30 days after last study visit, whichever was longer, up to a maximum duration of approximately 2 years.
|
|
Gastrointestinal disorders
Toothache
|
11.1%
1/9 • Adverse events were reported from first dose of study treatment until end of study treatment plus 90 days post treatment or 30 days after last study visit, whichever was longer, up to a maximum duration of approximately 2 years.
|
0.00%
0/4 • Adverse events were reported from first dose of study treatment until end of study treatment plus 90 days post treatment or 30 days after last study visit, whichever was longer, up to a maximum duration of approximately 2 years.
|
0.00%
0/13 • Adverse events were reported from first dose of study treatment until end of study treatment plus 90 days post treatment or 30 days after last study visit, whichever was longer, up to a maximum duration of approximately 2 years.
|
|
Gastrointestinal disorders
Vomiting
|
11.1%
1/9 • Adverse events were reported from first dose of study treatment until end of study treatment plus 90 days post treatment or 30 days after last study visit, whichever was longer, up to a maximum duration of approximately 2 years.
|
0.00%
0/4 • Adverse events were reported from first dose of study treatment until end of study treatment plus 90 days post treatment or 30 days after last study visit, whichever was longer, up to a maximum duration of approximately 2 years.
|
0.00%
0/13 • Adverse events were reported from first dose of study treatment until end of study treatment plus 90 days post treatment or 30 days after last study visit, whichever was longer, up to a maximum duration of approximately 2 years.
|
|
General disorders
Influenza like illness
|
11.1%
1/9 • Adverse events were reported from first dose of study treatment until end of study treatment plus 90 days post treatment or 30 days after last study visit, whichever was longer, up to a maximum duration of approximately 2 years.
|
0.00%
0/4 • Adverse events were reported from first dose of study treatment until end of study treatment plus 90 days post treatment or 30 days after last study visit, whichever was longer, up to a maximum duration of approximately 2 years.
|
7.7%
1/13 • Adverse events were reported from first dose of study treatment until end of study treatment plus 90 days post treatment or 30 days after last study visit, whichever was longer, up to a maximum duration of approximately 2 years.
|
|
General disorders
Injection site erythema
|
11.1%
1/9 • Adverse events were reported from first dose of study treatment until end of study treatment plus 90 days post treatment or 30 days after last study visit, whichever was longer, up to a maximum duration of approximately 2 years.
|
0.00%
0/4 • Adverse events were reported from first dose of study treatment until end of study treatment plus 90 days post treatment or 30 days after last study visit, whichever was longer, up to a maximum duration of approximately 2 years.
|
7.7%
1/13 • Adverse events were reported from first dose of study treatment until end of study treatment plus 90 days post treatment or 30 days after last study visit, whichever was longer, up to a maximum duration of approximately 2 years.
|
|
General disorders
Injection site reaction
|
22.2%
2/9 • Adverse events were reported from first dose of study treatment until end of study treatment plus 90 days post treatment or 30 days after last study visit, whichever was longer, up to a maximum duration of approximately 2 years.
|
0.00%
0/4 • Adverse events were reported from first dose of study treatment until end of study treatment plus 90 days post treatment or 30 days after last study visit, whichever was longer, up to a maximum duration of approximately 2 years.
|
0.00%
0/13 • Adverse events were reported from first dose of study treatment until end of study treatment plus 90 days post treatment or 30 days after last study visit, whichever was longer, up to a maximum duration of approximately 2 years.
|
|
General disorders
Pyrexia
|
22.2%
2/9 • Adverse events were reported from first dose of study treatment until end of study treatment plus 90 days post treatment or 30 days after last study visit, whichever was longer, up to a maximum duration of approximately 2 years.
|
0.00%
0/4 • Adverse events were reported from first dose of study treatment until end of study treatment plus 90 days post treatment or 30 days after last study visit, whichever was longer, up to a maximum duration of approximately 2 years.
|
0.00%
0/13 • Adverse events were reported from first dose of study treatment until end of study treatment plus 90 days post treatment or 30 days after last study visit, whichever was longer, up to a maximum duration of approximately 2 years.
|
|
Infections and infestations
COVID-19
|
44.4%
4/9 • Adverse events were reported from first dose of study treatment until end of study treatment plus 90 days post treatment or 30 days after last study visit, whichever was longer, up to a maximum duration of approximately 2 years.
|
25.0%
1/4 • Adverse events were reported from first dose of study treatment until end of study treatment plus 90 days post treatment or 30 days after last study visit, whichever was longer, up to a maximum duration of approximately 2 years.
|
7.7%
1/13 • Adverse events were reported from first dose of study treatment until end of study treatment plus 90 days post treatment or 30 days after last study visit, whichever was longer, up to a maximum duration of approximately 2 years.
|
|
Infections and infestations
Conjunctivitis
|
11.1%
1/9 • Adverse events were reported from first dose of study treatment until end of study treatment plus 90 days post treatment or 30 days after last study visit, whichever was longer, up to a maximum duration of approximately 2 years.
|
0.00%
0/4 • Adverse events were reported from first dose of study treatment until end of study treatment plus 90 days post treatment or 30 days after last study visit, whichever was longer, up to a maximum duration of approximately 2 years.
|
0.00%
0/13 • Adverse events were reported from first dose of study treatment until end of study treatment plus 90 days post treatment or 30 days after last study visit, whichever was longer, up to a maximum duration of approximately 2 years.
|
|
Infections and infestations
Gastroenteritis
|
11.1%
1/9 • Adverse events were reported from first dose of study treatment until end of study treatment plus 90 days post treatment or 30 days after last study visit, whichever was longer, up to a maximum duration of approximately 2 years.
|
25.0%
1/4 • Adverse events were reported from first dose of study treatment until end of study treatment plus 90 days post treatment or 30 days after last study visit, whichever was longer, up to a maximum duration of approximately 2 years.
|
0.00%
0/13 • Adverse events were reported from first dose of study treatment until end of study treatment plus 90 days post treatment or 30 days after last study visit, whichever was longer, up to a maximum duration of approximately 2 years.
|
|
Infections and infestations
Gingivitis
|
0.00%
0/9 • Adverse events were reported from first dose of study treatment until end of study treatment plus 90 days post treatment or 30 days after last study visit, whichever was longer, up to a maximum duration of approximately 2 years.
|
25.0%
1/4 • Adverse events were reported from first dose of study treatment until end of study treatment plus 90 days post treatment or 30 days after last study visit, whichever was longer, up to a maximum duration of approximately 2 years.
|
0.00%
0/13 • Adverse events were reported from first dose of study treatment until end of study treatment plus 90 days post treatment or 30 days after last study visit, whichever was longer, up to a maximum duration of approximately 2 years.
|
|
Infections and infestations
Influenza
|
0.00%
0/9 • Adverse events were reported from first dose of study treatment until end of study treatment plus 90 days post treatment or 30 days after last study visit, whichever was longer, up to a maximum duration of approximately 2 years.
|
0.00%
0/4 • Adverse events were reported from first dose of study treatment until end of study treatment plus 90 days post treatment or 30 days after last study visit, whichever was longer, up to a maximum duration of approximately 2 years.
|
15.4%
2/13 • Adverse events were reported from first dose of study treatment until end of study treatment plus 90 days post treatment or 30 days after last study visit, whichever was longer, up to a maximum duration of approximately 2 years.
|
|
Infections and infestations
Oral herpes
|
0.00%
0/9 • Adverse events were reported from first dose of study treatment until end of study treatment plus 90 days post treatment or 30 days after last study visit, whichever was longer, up to a maximum duration of approximately 2 years.
|
25.0%
1/4 • Adverse events were reported from first dose of study treatment until end of study treatment plus 90 days post treatment or 30 days after last study visit, whichever was longer, up to a maximum duration of approximately 2 years.
|
0.00%
0/13 • Adverse events were reported from first dose of study treatment until end of study treatment plus 90 days post treatment or 30 days after last study visit, whichever was longer, up to a maximum duration of approximately 2 years.
|
|
Infections and infestations
Upper respiratory tract infection
|
0.00%
0/9 • Adverse events were reported from first dose of study treatment until end of study treatment plus 90 days post treatment or 30 days after last study visit, whichever was longer, up to a maximum duration of approximately 2 years.
|
0.00%
0/4 • Adverse events were reported from first dose of study treatment until end of study treatment plus 90 days post treatment or 30 days after last study visit, whichever was longer, up to a maximum duration of approximately 2 years.
|
7.7%
1/13 • Adverse events were reported from first dose of study treatment until end of study treatment plus 90 days post treatment or 30 days after last study visit, whichever was longer, up to a maximum duration of approximately 2 years.
|
|
Infections and infestations
Urinary tract infection
|
0.00%
0/9 • Adverse events were reported from first dose of study treatment until end of study treatment plus 90 days post treatment or 30 days after last study visit, whichever was longer, up to a maximum duration of approximately 2 years.
|
0.00%
0/4 • Adverse events were reported from first dose of study treatment until end of study treatment plus 90 days post treatment or 30 days after last study visit, whichever was longer, up to a maximum duration of approximately 2 years.
|
7.7%
1/13 • Adverse events were reported from first dose of study treatment until end of study treatment plus 90 days post treatment or 30 days after last study visit, whichever was longer, up to a maximum duration of approximately 2 years.
|
|
Infections and infestations
Viral infection
|
11.1%
1/9 • Adverse events were reported from first dose of study treatment until end of study treatment plus 90 days post treatment or 30 days after last study visit, whichever was longer, up to a maximum duration of approximately 2 years.
|
0.00%
0/4 • Adverse events were reported from first dose of study treatment until end of study treatment plus 90 days post treatment or 30 days after last study visit, whichever was longer, up to a maximum duration of approximately 2 years.
|
0.00%
0/13 • Adverse events were reported from first dose of study treatment until end of study treatment plus 90 days post treatment or 30 days after last study visit, whichever was longer, up to a maximum duration of approximately 2 years.
|
|
Injury, poisoning and procedural complications
Concussion
|
11.1%
1/9 • Adverse events were reported from first dose of study treatment until end of study treatment plus 90 days post treatment or 30 days after last study visit, whichever was longer, up to a maximum duration of approximately 2 years.
|
0.00%
0/4 • Adverse events were reported from first dose of study treatment until end of study treatment plus 90 days post treatment or 30 days after last study visit, whichever was longer, up to a maximum duration of approximately 2 years.
|
0.00%
0/13 • Adverse events were reported from first dose of study treatment until end of study treatment plus 90 days post treatment or 30 days after last study visit, whichever was longer, up to a maximum duration of approximately 2 years.
|
|
Injury, poisoning and procedural complications
Head injury
|
11.1%
1/9 • Adverse events were reported from first dose of study treatment until end of study treatment plus 90 days post treatment or 30 days after last study visit, whichever was longer, up to a maximum duration of approximately 2 years.
|
0.00%
0/4 • Adverse events were reported from first dose of study treatment until end of study treatment plus 90 days post treatment or 30 days after last study visit, whichever was longer, up to a maximum duration of approximately 2 years.
|
0.00%
0/13 • Adverse events were reported from first dose of study treatment until end of study treatment plus 90 days post treatment or 30 days after last study visit, whichever was longer, up to a maximum duration of approximately 2 years.
|
|
Injury, poisoning and procedural complications
Ligament sprain
|
0.00%
0/9 • Adverse events were reported from first dose of study treatment until end of study treatment plus 90 days post treatment or 30 days after last study visit, whichever was longer, up to a maximum duration of approximately 2 years.
|
0.00%
0/4 • Adverse events were reported from first dose of study treatment until end of study treatment plus 90 days post treatment or 30 days after last study visit, whichever was longer, up to a maximum duration of approximately 2 years.
|
7.7%
1/13 • Adverse events were reported from first dose of study treatment until end of study treatment plus 90 days post treatment or 30 days after last study visit, whichever was longer, up to a maximum duration of approximately 2 years.
|
|
Investigations
Carotid intima-media thickness increased
|
0.00%
0/9 • Adverse events were reported from first dose of study treatment until end of study treatment plus 90 days post treatment or 30 days after last study visit, whichever was longer, up to a maximum duration of approximately 2 years.
|
0.00%
0/4 • Adverse events were reported from first dose of study treatment until end of study treatment plus 90 days post treatment or 30 days after last study visit, whichever was longer, up to a maximum duration of approximately 2 years.
|
7.7%
1/13 • Adverse events were reported from first dose of study treatment until end of study treatment plus 90 days post treatment or 30 days after last study visit, whichever was longer, up to a maximum duration of approximately 2 years.
|
|
Metabolism and nutrition disorders
Vitamin D deficiency
|
11.1%
1/9 • Adverse events were reported from first dose of study treatment until end of study treatment plus 90 days post treatment or 30 days after last study visit, whichever was longer, up to a maximum duration of approximately 2 years.
|
0.00%
0/4 • Adverse events were reported from first dose of study treatment until end of study treatment plus 90 days post treatment or 30 days after last study visit, whichever was longer, up to a maximum duration of approximately 2 years.
|
0.00%
0/13 • Adverse events were reported from first dose of study treatment until end of study treatment plus 90 days post treatment or 30 days after last study visit, whichever was longer, up to a maximum duration of approximately 2 years.
|
|
Musculoskeletal and connective tissue disorders
Costochondritis
|
0.00%
0/9 • Adverse events were reported from first dose of study treatment until end of study treatment plus 90 days post treatment or 30 days after last study visit, whichever was longer, up to a maximum duration of approximately 2 years.
|
0.00%
0/4 • Adverse events were reported from first dose of study treatment until end of study treatment plus 90 days post treatment or 30 days after last study visit, whichever was longer, up to a maximum duration of approximately 2 years.
|
7.7%
1/13 • Adverse events were reported from first dose of study treatment until end of study treatment plus 90 days post treatment or 30 days after last study visit, whichever was longer, up to a maximum duration of approximately 2 years.
|
|
Nervous system disorders
Headache
|
0.00%
0/9 • Adverse events were reported from first dose of study treatment until end of study treatment plus 90 days post treatment or 30 days after last study visit, whichever was longer, up to a maximum duration of approximately 2 years.
|
0.00%
0/4 • Adverse events were reported from first dose of study treatment until end of study treatment plus 90 days post treatment or 30 days after last study visit, whichever was longer, up to a maximum duration of approximately 2 years.
|
15.4%
2/13 • Adverse events were reported from first dose of study treatment until end of study treatment plus 90 days post treatment or 30 days after last study visit, whichever was longer, up to a maximum duration of approximately 2 years.
|
|
Reproductive system and breast disorders
Vaginal discharge
|
0.00%
0/9 • Adverse events were reported from first dose of study treatment until end of study treatment plus 90 days post treatment or 30 days after last study visit, whichever was longer, up to a maximum duration of approximately 2 years.
|
25.0%
1/4 • Adverse events were reported from first dose of study treatment until end of study treatment plus 90 days post treatment or 30 days after last study visit, whichever was longer, up to a maximum duration of approximately 2 years.
|
0.00%
0/13 • Adverse events were reported from first dose of study treatment until end of study treatment plus 90 days post treatment or 30 days after last study visit, whichever was longer, up to a maximum duration of approximately 2 years.
|
|
Skin and subcutaneous tissue disorders
Angioedema
|
0.00%
0/9 • Adverse events were reported from first dose of study treatment until end of study treatment plus 90 days post treatment or 30 days after last study visit, whichever was longer, up to a maximum duration of approximately 2 years.
|
25.0%
1/4 • Adverse events were reported from first dose of study treatment until end of study treatment plus 90 days post treatment or 30 days after last study visit, whichever was longer, up to a maximum duration of approximately 2 years.
|
0.00%
0/13 • Adverse events were reported from first dose of study treatment until end of study treatment plus 90 days post treatment or 30 days after last study visit, whichever was longer, up to a maximum duration of approximately 2 years.
|
|
Skin and subcutaneous tissue disorders
Rash macular
|
11.1%
1/9 • Adverse events were reported from first dose of study treatment until end of study treatment plus 90 days post treatment or 30 days after last study visit, whichever was longer, up to a maximum duration of approximately 2 years.
|
0.00%
0/4 • Adverse events were reported from first dose of study treatment until end of study treatment plus 90 days post treatment or 30 days after last study visit, whichever was longer, up to a maximum duration of approximately 2 years.
|
0.00%
0/13 • Adverse events were reported from first dose of study treatment until end of study treatment plus 90 days post treatment or 30 days after last study visit, whichever was longer, up to a maximum duration of approximately 2 years.
|
|
Skin and subcutaneous tissue disorders
Urticaria
|
0.00%
0/9 • Adverse events were reported from first dose of study treatment until end of study treatment plus 90 days post treatment or 30 days after last study visit, whichever was longer, up to a maximum duration of approximately 2 years.
|
25.0%
1/4 • Adverse events were reported from first dose of study treatment until end of study treatment plus 90 days post treatment or 30 days after last study visit, whichever was longer, up to a maximum duration of approximately 2 years.
|
0.00%
0/13 • Adverse events were reported from first dose of study treatment until end of study treatment plus 90 days post treatment or 30 days after last study visit, whichever was longer, up to a maximum duration of approximately 2 years.
|
|
Skin and subcutaneous tissue disorders
Xanthoma
|
0.00%
0/9 • Adverse events were reported from first dose of study treatment until end of study treatment plus 90 days post treatment or 30 days after last study visit, whichever was longer, up to a maximum duration of approximately 2 years.
|
0.00%
0/4 • Adverse events were reported from first dose of study treatment until end of study treatment plus 90 days post treatment or 30 days after last study visit, whichever was longer, up to a maximum duration of approximately 2 years.
|
7.7%
1/13 • Adverse events were reported from first dose of study treatment until end of study treatment plus 90 days post treatment or 30 days after last study visit, whichever was longer, up to a maximum duration of approximately 2 years.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee The terms and conditions of Novartis' agreements with its investigators may vary. However, Novartis does not prohibit any investigator from publishing. Any publications from a single-site are postponed until the publication of the pooled data (i.e., data from all sites) in the clinical trial.
- Publication restrictions are in place
Restriction type: OTHER