Trial Outcomes & Findings for An Efficacy and Safety Study of Alirocumab in Children and Adolescents With Homozygous Familial Hypercholesterolemia (NCT NCT03510715)
NCT ID: NCT03510715
Last Updated: 2020-12-29
Results Overview
Adjusted least square (LS) means and standard errors were obtained from the mixed model analysis with repeated measures (MMRM) to account for missing data using all available post-baseline data from Week 4 to Week 48 regardless of status on- or off-treatment used in the model (ITT analysis).
Recruitment status
COMPLETED
Study phase
PHASE3
Target enrollment
18 participants
Primary outcome timeframe
Baseline to Week 12
Results posted on
2020-12-29
Participant Flow
The study was conducted at 10 active centers (which screened at least 1 participant) in 10 countries worldwide. Overall, 20 participants were screened between 31 August 2018 and 4 January 2019, of whom 2 were screen failures. Of the 10 centers which screened participants, 9 centers enrolled at least 1 participant.
A total of 18 participants were enrolled and received treatment in this study.
Participant milestones
| Measure |
Alirocumab 75 mg Q2W/up to 150 mg Q2W
Participants with body weight (BW) less than (\<) 50 kilograms (kg) received subcutaneous (SC) injection of alirocumab 75 milligrams (mg) every 2 weeks (Q2W) for 48 weeks. Alirocumab dose was up-titrated to 150 mg Q2W from Week 12 in case of increase in BW with BW greater than or equal to \[\>=\] 50 kg.
|
Alirocumab 150 mg Q2W
Participants with BW \>=50 kg received SC injection of alirocumab 150 mg Q2W for 48 weeks.
|
|---|---|---|
|
Overall Study
STARTED
|
9
|
9
|
|
Overall Study
COMPLETED
|
8
|
9
|
|
Overall Study
NOT COMPLETED
|
1
|
0
|
Reasons for withdrawal
| Measure |
Alirocumab 75 mg Q2W/up to 150 mg Q2W
Participants with body weight (BW) less than (\<) 50 kilograms (kg) received subcutaneous (SC) injection of alirocumab 75 milligrams (mg) every 2 weeks (Q2W) for 48 weeks. Alirocumab dose was up-titrated to 150 mg Q2W from Week 12 in case of increase in BW with BW greater than or equal to \[\>=\] 50 kg.
|
Alirocumab 150 mg Q2W
Participants with BW \>=50 kg received SC injection of alirocumab 150 mg Q2W for 48 weeks.
|
|---|---|---|
|
Overall Study
Adverse Event
|
1
|
0
|
Baseline Characteristics
An Efficacy and Safety Study of Alirocumab in Children and Adolescents With Homozygous Familial Hypercholesterolemia
Baseline characteristics by cohort
| Measure |
Alirocumab 75 mg Q2W/up to 150 mg Q2W
n=9 Participants
Participants with BW \<50 kg received SC injection of alirocumab 75 mg Q2W for 48 weeks. Alirocumab dose was up-titrated to 150 mg Q2W from Week 12 in case of increase in BW with BW \>=50 kg.
|
Alirocumab 150 mg Q2W
n=9 Participants
Participants with BW \>=50 kg received SC injection of alirocumab 150 mg Q2W for 48 weeks.
|
Total
n=18 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
10.4 years
STANDARD_DEVIATION 1.5 • n=5 Participants
|
14.3 years
STANDARD_DEVIATION 2.4 • n=7 Participants
|
12.4 years
STANDARD_DEVIATION 2.8 • n=5 Participants
|
|
Sex: Female, Male
Female
|
4 Participants
n=5 Participants
|
5 Participants
n=7 Participants
|
9 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
5 Participants
n=5 Participants
|
4 Participants
n=7 Participants
|
9 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
2 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
3 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
1 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
3 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
6 Participants
n=5 Participants
|
5 Participants
n=7 Participants
|
11 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
|
|
Low-Density Lipoprotein Cholesterol (LDL-C)
|
437.7 milligrams per deciliter (mg/dL)
STANDARD_DEVIATION 192.8 • n=5 Participants
|
308.3 milligrams per deciliter (mg/dL)
STANDARD_DEVIATION 181.6 • n=7 Participants
|
373.0 milligrams per deciliter (mg/dL)
STANDARD_DEVIATION 193.5 • n=5 Participants
|
PRIMARY outcome
Timeframe: Baseline to Week 12Population: Analysis was performed on ITT population which included all enrolled participants who had received at least one dose or partial dose of alirocumab. As pre-specified, efficacy analysis data were planned to be collected and analyzed for all doses combined (i.e. for combined population).
Adjusted least square (LS) means and standard errors were obtained from the mixed model analysis with repeated measures (MMRM) to account for missing data using all available post-baseline data from Week 4 to Week 48 regardless of status on- or off-treatment used in the model (ITT analysis).
Outcome measures
| Measure |
Alirocumab
n=18 Participants
All participants who received either 75 mg (BW \<50 kg) or 150 mg (BW \>=50 kg) alirocumab subcutaneously Q2W for 48 weeks.
|
Alirocumab 150 mg Q2W
Participants with BW \>=50 kg received SC injection of alirocumab 150 mg Q2W for 48 weeks.
|
|---|---|---|
|
Percent Change From Baseline in Low-Density Lipoprotein Cholesterol (LDL-C) at Week 12: Intent-to-Treat (ITT) Analysis
|
-4.1 percent change
Standard Error 9.0
|
—
|
SECONDARY outcome
Timeframe: Baseline to Week 12Population: Analysis was performed on ITT population. As pre-specified, efficacy analysis data were planned to be collected and analyzed for all doses combined (i.e. for combined population).
Adjusted LS means and standard errors were obtained from the MMRM model to account for missing data using all available post-baseline on-treatment data from Week 4 to Week 48 (on-treatment Analysis).
Outcome measures
| Measure |
Alirocumab
n=18 Participants
All participants who received either 75 mg (BW \<50 kg) or 150 mg (BW \>=50 kg) alirocumab subcutaneously Q2W for 48 weeks.
|
Alirocumab 150 mg Q2W
Participants with BW \>=50 kg received SC injection of alirocumab 150 mg Q2W for 48 weeks.
|
|---|---|---|
|
Percent Change From Baseline in Low-Density Lipoprotein Cholesterol at Week 12: On-treatment Analysis
|
-4.1 percent change
Standard Error 9.0
|
—
|
SECONDARY outcome
Timeframe: Baseline to Weeks 24 and 48Population: Analysis was performed on ITT population. As pre-specified, efficacy analysis data were planned to be collected and analyzed for all doses combined (i.e., for combined population).
Adjusted LS means and standard errors were obtained from the MMRM model to account for missing data using all available post-baseline data from Week 4 to Week 48 regardless of status on- or off-treatment used in the model (ITT analysis). Although separate analyses of all available data (ITT analysis) and only data collected within a defined time window (On-treatment analysis) were planned, if all values used in the ITT approach were within the on-treatment time window, the on-treatment analysis would be identical to the ITT analysis, thus the results would be identical and a single outcome measure presenting the results for both types of analysis would be provided.
Outcome measures
| Measure |
Alirocumab
n=18 Participants
All participants who received either 75 mg (BW \<50 kg) or 150 mg (BW \>=50 kg) alirocumab subcutaneously Q2W for 48 weeks.
|
Alirocumab 150 mg Q2W
Participants with BW \>=50 kg received SC injection of alirocumab 150 mg Q2W for 48 weeks.
|
|---|---|---|
|
Percent Change From Baseline in Low-Density Lipoprotein Cholesterol at Weeks 24 and 48: ITT Analysis/On-treatment Analysis
Week 24
|
-10.1 percent change
Standard Error 7.6
|
—
|
|
Percent Change From Baseline in Low-Density Lipoprotein Cholesterol at Weeks 24 and 48: ITT Analysis/On-treatment Analysis
Week 48
|
4.2 percent change
Standard Error 12.8
|
—
|
SECONDARY outcome
Timeframe: Baseline to Weeks 12, 24 and 48Population: Analysis was performed on ITT population. As pre-specified, efficacy analysis data were planned to be collected and analyzed for all doses combined (i.e., for combined population).
Adjusted LS means and standard errors were obtained from the MMRM model to account for missing data using all available post-baseline data from Week 4 to Week 48 regardless of status on- or off-treatment used in the model (ITT analysis). Although separate analyses of all available data (ITT analysis) and only data collected within a defined time window (On-treatment analysis) were planned, if all values used in the ITT approach were within the on-treatment time window, the on-treatment analysis would be identical to the ITT analysis, thus the results would be identical and a single outcome measure presenting the results for both types of analysis would be provided.
Outcome measures
| Measure |
Alirocumab
n=18 Participants
All participants who received either 75 mg (BW \<50 kg) or 150 mg (BW \>=50 kg) alirocumab subcutaneously Q2W for 48 weeks.
|
Alirocumab 150 mg Q2W
Participants with BW \>=50 kg received SC injection of alirocumab 150 mg Q2W for 48 weeks.
|
|---|---|---|
|
Percent Change From Baseline in Apolipoprotein (Apo) B at Weeks 12, 24 and 48: ITT Analysis/On-treatment Analysis
Week 12
|
-4.2 percent change
Standard Error 6.8
|
—
|
|
Percent Change From Baseline in Apolipoprotein (Apo) B at Weeks 12, 24 and 48: ITT Analysis/On-treatment Analysis
Week 24
|
-11.8 percent change
Standard Error 6.1
|
—
|
|
Percent Change From Baseline in Apolipoprotein (Apo) B at Weeks 12, 24 and 48: ITT Analysis/On-treatment Analysis
Week 48
|
0.9 percent change
Standard Error 10.4
|
—
|
SECONDARY outcome
Timeframe: Baseline to Weeks 12, 24 and 48Population: Analysis was performed on ITT population. As pre-specified, efficacy analysis data were planned to be collected and analyzed for all doses combined (i.e., for combined population).
Adjusted LS means and standard errors were obtained from the MMRM model to account for missing data using all available post-baseline data from Week 4 to Week 48 regardless of status on- or off-treatment used in the model (ITT analysis). Although separate analyses of all available data (ITT analysis) and only data collected within a defined time window (On-treatment analysis) were planned, if all values used in the ITT approach were within the on-treatment time window, the on-treatment analysis would be identical to the ITT analysis, thus the results would be identical and a single outcome measure presenting the results for both types of analysis would be provided.
Outcome measures
| Measure |
Alirocumab
n=18 Participants
All participants who received either 75 mg (BW \<50 kg) or 150 mg (BW \>=50 kg) alirocumab subcutaneously Q2W for 48 weeks.
|
Alirocumab 150 mg Q2W
Participants with BW \>=50 kg received SC injection of alirocumab 150 mg Q2W for 48 weeks.
|
|---|---|---|
|
Percent Change From Baseline in Non-High Density Lipoprotein Cholesterol (Non-HDL-C) at Weeks 12, 24 and 48 - ITT Analysis/On-treatment Analysis
Week 12
|
-3.9 percent change
Standard Error 8.3
|
—
|
|
Percent Change From Baseline in Non-High Density Lipoprotein Cholesterol (Non-HDL-C) at Weeks 12, 24 and 48 - ITT Analysis/On-treatment Analysis
Week 24
|
-9.2 percent change
Standard Error 7.3
|
—
|
|
Percent Change From Baseline in Non-High Density Lipoprotein Cholesterol (Non-HDL-C) at Weeks 12, 24 and 48 - ITT Analysis/On-treatment Analysis
Week 48
|
5.7 percent change
Standard Error 13.1
|
—
|
SECONDARY outcome
Timeframe: Baseline to Weeks 12, 24 and 48Population: Analysis was performed on ITT population. As pre-specified, efficacy analysis data were planned to be collected and analyzed for all doses combined (i.e., for combined population).
Adjusted LS means and standard errors were obtained from the MMRM model to account for missing data using all available post-baseline data from Weeks 4 to Week 48 regardless of status on- or off-treatment used in the model (ITT analysis). Although separate analyses of all available data (ITT analysis) and only data collected within a defined time window (On-treatment analysis) were planned, if all values used in the ITT approach were within the on-treatment time window, the on-treatment analysis would be identical to the ITT analysis, thus the results would be identical and a single outcome measure presenting the results for both types of analysis would be provided.
Outcome measures
| Measure |
Alirocumab
n=18 Participants
All participants who received either 75 mg (BW \<50 kg) or 150 mg (BW \>=50 kg) alirocumab subcutaneously Q2W for 48 weeks.
|
Alirocumab 150 mg Q2W
Participants with BW \>=50 kg received SC injection of alirocumab 150 mg Q2W for 48 weeks.
|
|---|---|---|
|
Percent Change From Baseline in Total Cholesterol (Total-C) at Weeks 12, 24 and 48: ITT Analysis/On-treatment Analysis
Week 12
|
-1.9 percent change
Standard Error 7.2
|
—
|
|
Percent Change From Baseline in Total Cholesterol (Total-C) at Weeks 12, 24 and 48: ITT Analysis/On-treatment Analysis
Week 24
|
-6.3 percent change
Standard Error 6.5
|
—
|
|
Percent Change From Baseline in Total Cholesterol (Total-C) at Weeks 12, 24 and 48: ITT Analysis/On-treatment Analysis
Week 48
|
5.5 percent change
Standard Error 10.7
|
—
|
SECONDARY outcome
Timeframe: Baseline to Weeks 12, 24 and 48Population: Analysis was performed on ITT population. As pre-specified, efficacy analysis data were planned to be collected and analyzed for all doses combined (i.e., for combined population).
Adjusted means and standard errors were obtained from a multiple imputation approach followed by a robust regression model including all available post-baseline data from Week 4 to Week 48 regardless of status on-or off-treatment used in the model (ITT analysis). Although separate analyses of all available data (ITT analysis) and only data collected within a defined time window (On-treatment analysis) were planned, if all values used in the ITT approach were within the on-treatment time window, the on-treatment analysis would be identical to the ITT analysis, thus the results would be identical and a single outcome measure presenting the results for both types of analysis would be provided.
Outcome measures
| Measure |
Alirocumab
n=18 Participants
All participants who received either 75 mg (BW \<50 kg) or 150 mg (BW \>=50 kg) alirocumab subcutaneously Q2W for 48 weeks.
|
Alirocumab 150 mg Q2W
Participants with BW \>=50 kg received SC injection of alirocumab 150 mg Q2W for 48 weeks.
|
|---|---|---|
|
Percent Change From Baseline in Lipoprotein a (Lp) (a) at Weeks 12, 24 and 48: ITT Analysis/On-treatment Analysis
Week 12
|
7.4 percent change
Standard Error 7.6
|
—
|
|
Percent Change From Baseline in Lipoprotein a (Lp) (a) at Weeks 12, 24 and 48: ITT Analysis/On-treatment Analysis
Week 24
|
-5.2 percent change
Standard Error 8.1
|
—
|
|
Percent Change From Baseline in Lipoprotein a (Lp) (a) at Weeks 12, 24 and 48: ITT Analysis/On-treatment Analysis
Week 48
|
-6.4 percent change
Standard Error 12.2
|
—
|
SECONDARY outcome
Timeframe: Baseline to Weeks 12, 24 and 48Population: Analysis was performed on ITT population. As pre-specified, efficacy analysis data were planned to be collected and analyzed for all doses combined (i.e., for combined population).
Adjusted LS means and standard errors were obtained from the MMRM model to account for missing data using all available post-baseline data from Weeks 4 to Week 48 regardless of status on- or off-treatment used in the model (ITT analysis). Although separate analyses of all available data (ITT analysis) and only data collected within a defined time window (On-treatment analysis) were planned, if all values used in the ITT approach were within the on-treatment time window, the on-treatment analysis would be identical to the ITT analysis, thus the results would be identical and a single outcome measure presenting the results for both types of analysis would be provided.
Outcome measures
| Measure |
Alirocumab
n=18 Participants
All participants who received either 75 mg (BW \<50 kg) or 150 mg (BW \>=50 kg) alirocumab subcutaneously Q2W for 48 weeks.
|
Alirocumab 150 mg Q2W
Participants with BW \>=50 kg received SC injection of alirocumab 150 mg Q2W for 48 weeks.
|
|---|---|---|
|
Percent Change From Baseline in High Density Lipoprotein Cholesterol (HDL-C) at Weeks 12, 24 and 48: ITT Analysis/On-treatment Analysis
Week 24
|
8.9 percent change
Standard Error 4.4
|
—
|
|
Percent Change From Baseline in High Density Lipoprotein Cholesterol (HDL-C) at Weeks 12, 24 and 48: ITT Analysis/On-treatment Analysis
Week 48
|
10.1 percent change
Standard Error 5.5
|
—
|
|
Percent Change From Baseline in High Density Lipoprotein Cholesterol (HDL-C) at Weeks 12, 24 and 48: ITT Analysis/On-treatment Analysis
Week 12
|
13.0 percent change
Standard Error 5.9
|
—
|
SECONDARY outcome
Timeframe: Baseline to Weeks 12, 24 and 48Population: Analysis was performed on ITT population. As pre-specified, efficacy analysis data were planned to be collected and analyzed for all doses combined (i.e., for combined population).
Adjusted means and standard errors were obtained from a multiple imputation approach followed by a robust regression model including all available post-baseline data from Week 4 to Week 48 regardless of status on- or off-treatment used in the model (ITT analysis). Although separate analyses of all available data (ITT analysis) and only data collected within a defined time window (On-treatment analysis) were planned, if all values used in the ITT approach were within the on-treatment time window, the on-treatment analysis would be identical to the ITT analysis, thus the results would be identical and a single outcome measure presenting the results for both types of analysis would be provided.
Outcome measures
| Measure |
Alirocumab
n=18 Participants
All participants who received either 75 mg (BW \<50 kg) or 150 mg (BW \>=50 kg) alirocumab subcutaneously Q2W for 48 weeks.
|
Alirocumab 150 mg Q2W
Participants with BW \>=50 kg received SC injection of alirocumab 150 mg Q2W for 48 weeks.
|
|---|---|---|
|
Percent Change From Baseline in Fasting Triglycerides (TG) at Weeks 12, 24 and 48: ITT Analysis/On-treatment Analysis
Week 12
|
2.8 percent change
Standard Error 8.0
|
—
|
|
Percent Change From Baseline in Fasting Triglycerides (TG) at Weeks 12, 24 and 48: ITT Analysis/On-treatment Analysis
Week 24
|
5.2 percent change
Standard Error 16.2
|
—
|
|
Percent Change From Baseline in Fasting Triglycerides (TG) at Weeks 12, 24 and 48: ITT Analysis/On-treatment Analysis
Week 48
|
10.0 percent change
Standard Error 8.2
|
—
|
SECONDARY outcome
Timeframe: Baseline to Weeks 12, 24 and 48Population: Analysis was performed on ITT population. As pre-specified, efficacy analysis data was planned to be collected and analyzed for all doses combined (i.e., for combined population).
Adjusted LS means and standard errors were obtained from the MMRM model to account for missing data using all available post-baseline data from Week 4 to 48 regardless of status on- or off-treatment used in the model (ITT analysis). Although separate analyses of all available data (ITT analysis) and only data collected within a defined time window (On-treatment analysis) were planned, if all values used in the ITT approach were within the on-treatment time window, the on-treatment analysis would be identical to the ITT analysis, thus the results would be identical and a single outcome measure presenting the results for both types of analysis would be provided.
Outcome measures
| Measure |
Alirocumab
n=18 Participants
All participants who received either 75 mg (BW \<50 kg) or 150 mg (BW \>=50 kg) alirocumab subcutaneously Q2W for 48 weeks.
|
Alirocumab 150 mg Q2W
Participants with BW \>=50 kg received SC injection of alirocumab 150 mg Q2W for 48 weeks.
|
|---|---|---|
|
Percent Change From Baseline in Apolipoprotein A1 (Apo A1) at Weeks 12, 24 and 48: ITT Analysis/On-treatment Analysis
Week 12
|
11.3 percent change
Standard Error 6.9
|
—
|
|
Percent Change From Baseline in Apolipoprotein A1 (Apo A1) at Weeks 12, 24 and 48: ITT Analysis/On-treatment Analysis
Week 24
|
14.6 percent change
Standard Error 6.0
|
—
|
|
Percent Change From Baseline in Apolipoprotein A1 (Apo A1) at Weeks 12, 24 and 48: ITT Analysis/On-treatment Analysis
Week 48
|
11.3 percent change
Standard Error 5.8
|
—
|
SECONDARY outcome
Timeframe: Baseline to Weeks 12, 24 and 48Population: Analysis was performed on ITT population. As pre-specified, efficacy analysis data were planned to be collected and analyzed for all doses combined (i.e., for combined population).
Adjusted Percentage were obtained from a multiple imputation approach for handling of missing data including all available post-baseline data from Week 4 to Week 48 regardless of status on- or off-treatment used in the model (ITT analysis). Although separate analyses of all available data (ITT analysis) and only data collected within a defined time window (On-treatment analysis) were planned, if all values used in the ITT approach were within the on-treatment time window, the on-treatment analysis would be identical to the ITT analysis, thus the results would be identical and a single outcome measure presenting the results for both types of analysis would be provided.
Outcome measures
| Measure |
Alirocumab
n=18 Participants
All participants who received either 75 mg (BW \<50 kg) or 150 mg (BW \>=50 kg) alirocumab subcutaneously Q2W for 48 weeks.
|
Alirocumab 150 mg Q2W
Participants with BW \>=50 kg received SC injection of alirocumab 150 mg Q2W for 48 weeks.
|
|---|---|---|
|
Percentage of Participants Reporting >=15 Percent (%) Reduction in LDL-C Level at Weeks 12, 24 and 48: ITT Analysis/On-treatment Analysis
Week 12
|
50.0 percentage of participants
Interval 26.2 to 73.8
|
—
|
|
Percentage of Participants Reporting >=15 Percent (%) Reduction in LDL-C Level at Weeks 12, 24 and 48: ITT Analysis/On-treatment Analysis
Week 24
|
50.0 percentage of participants
Interval 26.2 to 73.8
|
—
|
|
Percentage of Participants Reporting >=15 Percent (%) Reduction in LDL-C Level at Weeks 12, 24 and 48: ITT Analysis/On-treatment Analysis
Week 48
|
39.0 percentage of participants
Interval 15.8 to 62.2
|
—
|
SECONDARY outcome
Timeframe: Baseline to Weeks 12, 24 and 48Population: Analysis was performed on ITT population. As pre-specified, efficacy analysis data were planned to be collected and analyzed for all doses combined (i.e., for combined population).
Adjusted LS means and standard errors were obtained from the MMRM model to account for missing data using all available post-baseline data from Week 4 to Week 48 regardless of status on- or off-treatment used in the model (ITT analysis). Although separate analyses of all available data (ITT analysis) and only data collected within a defined time window (On-treatment analysis) were planned, if all values used in the ITT approach were within the on-treatment time window, the on-treatment analysis would be identical to the ITT analysis, thus the results would be identical and a single outcome measure presenting the results for both types of analysis would be provided.
Outcome measures
| Measure |
Alirocumab
n=18 Participants
All participants who received either 75 mg (BW \<50 kg) or 150 mg (BW \>=50 kg) alirocumab subcutaneously Q2W for 48 weeks.
|
Alirocumab 150 mg Q2W
Participants with BW \>=50 kg received SC injection of alirocumab 150 mg Q2W for 48 weeks.
|
|---|---|---|
|
Absolute Change From Baseline in LDL-C Level at Weeks 12, 24 and 48: ITT Analysis/On-treatment Analysis
Week 12
|
-33.4 mg/dL
Standard Error 19.1
|
—
|
|
Absolute Change From Baseline in LDL-C Level at Weeks 12, 24 and 48: ITT Analysis/On-treatment Analysis
Week 24
|
-43.0 mg/dL
Standard Error 19.0
|
—
|
|
Absolute Change From Baseline in LDL-C Level at Weeks 12, 24 and 48: ITT Analysis/On-treatment Analysis
Week 48
|
-15.0 mg/dL
Standard Error 25.7
|
—
|
SECONDARY outcome
Timeframe: Baseline, Weeks 12, 24 and 48Population: Analysis was performed on safety population which included participants who had received at least one dose or partial dose of alirocumab. Here, 'number analyzed' = participants with available data for each specified category.
Tanner stage defines physical measurements of development in children and adolescent based on external primary and secondary sex characteristics. Participants were evaluated for pubic hair distribution, breast development (only females) and genital development (only males), and classified in 3 categories as: Prepubescent (defined as a person just before start of the development of adult sexual characteristics), Pubescent (defined as a person at or approaching the age of puberty), Postpubescent (sexually mature or a person who has completed puberty).
Outcome measures
| Measure |
Alirocumab
n=9 Participants
All participants who received either 75 mg (BW \<50 kg) or 150 mg (BW \>=50 kg) alirocumab subcutaneously Q2W for 48 weeks.
|
Alirocumab 150 mg Q2W
n=9 Participants
Participants with BW \>=50 kg received SC injection of alirocumab 150 mg Q2W for 48 weeks.
|
|---|---|---|
|
Number of Participants With Tanner Staging at Baseline, Weeks 12, 24 and 48
Week 48: Prepubescent
|
1 Participants
|
0 Participants
|
|
Number of Participants With Tanner Staging at Baseline, Weeks 12, 24 and 48
Week 48: Pubescent
|
7 Participants
|
7 Participants
|
|
Number of Participants With Tanner Staging at Baseline, Weeks 12, 24 and 48
Week 48: Post-pubescent
|
0 Participants
|
2 Participants
|
|
Number of Participants With Tanner Staging at Baseline, Weeks 12, 24 and 48
Baseline: Prepubescent
|
3 Participants
|
0 Participants
|
|
Number of Participants With Tanner Staging at Baseline, Weeks 12, 24 and 48
Baseline: Pubescent
|
6 Participants
|
9 Participants
|
|
Number of Participants With Tanner Staging at Baseline, Weeks 12, 24 and 48
Baseline: Post-pubescent
|
0 Participants
|
0 Participants
|
|
Number of Participants With Tanner Staging at Baseline, Weeks 12, 24 and 48
Week 12: Prepubescent
|
3 Participants
|
0 Participants
|
|
Number of Participants With Tanner Staging at Baseline, Weeks 12, 24 and 48
Week 12: Pubescent
|
6 Participants
|
8 Participants
|
|
Number of Participants With Tanner Staging at Baseline, Weeks 12, 24 and 48
Week 12: Post-pubescent
|
0 Participants
|
1 Participants
|
|
Number of Participants With Tanner Staging at Baseline, Weeks 12, 24 and 48
Week 24: Prepubescent
|
2 Participants
|
0 Participants
|
|
Number of Participants With Tanner Staging at Baseline, Weeks 12, 24 and 48
Week 24: Pubescent
|
6 Participants
|
8 Participants
|
|
Number of Participants With Tanner Staging at Baseline, Weeks 12, 24 and 48
Week 24: Post-pubescent
|
0 Participants
|
1 Participants
|
Adverse Events
Alirocumab 75 mg Q2W/up to 150 mg Q2W
Serious events: 1 serious events
Other events: 8 other events
Deaths: 1 deaths
Alirocumab 150 mg Q2W
Serious events: 0 serious events
Other events: 9 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Alirocumab 75 mg Q2W/up to 150 mg Q2W
n=9 participants at risk
Participants with BW \<50 kg received SC injection of alirocumab 75 mg Q2W for 48 weeks. Alirocumab dose was up-titrated to 150 mg Q2W from Week 12 in case of increase in BW with BW \>=50 kg.
|
Alirocumab 150 mg Q2W
n=9 participants at risk
Participants with BW \>=50 kg received SC injection of alirocumab 150 mg Q2W for 48 weeks.
|
|---|---|---|
|
Cardiac disorders
Cardiac Failure
|
11.1%
1/9 • Number of events 1 • All Adverse Events (AEs) were collected from signature of the informed consent form up to 56 weeks of the study regardless of seriousness or relationship to investigational product.
Reported AEs and death were treatment-emergent AEs (TEAE) that developed/worsened, and death that occurred during TEAE period (the time from the first dose of alirocumab up to the last dose of alirocumab + 70 days \[10 weeks\]). Analysis was performed on safety population.
|
0.00%
0/9 • All Adverse Events (AEs) were collected from signature of the informed consent form up to 56 weeks of the study regardless of seriousness or relationship to investigational product.
Reported AEs and death were treatment-emergent AEs (TEAE) that developed/worsened, and death that occurred during TEAE period (the time from the first dose of alirocumab up to the last dose of alirocumab + 70 days \[10 weeks\]). Analysis was performed on safety population.
|
Other adverse events
| Measure |
Alirocumab 75 mg Q2W/up to 150 mg Q2W
n=9 participants at risk
Participants with BW \<50 kg received SC injection of alirocumab 75 mg Q2W for 48 weeks. Alirocumab dose was up-titrated to 150 mg Q2W from Week 12 in case of increase in BW with BW \>=50 kg.
|
Alirocumab 150 mg Q2W
n=9 participants at risk
Participants with BW \>=50 kg received SC injection of alirocumab 150 mg Q2W for 48 weeks.
|
|---|---|---|
|
Blood and lymphatic system disorders
Splenomegaly
|
11.1%
1/9 • Number of events 1 • All Adverse Events (AEs) were collected from signature of the informed consent form up to 56 weeks of the study regardless of seriousness or relationship to investigational product.
Reported AEs and death were treatment-emergent AEs (TEAE) that developed/worsened, and death that occurred during TEAE period (the time from the first dose of alirocumab up to the last dose of alirocumab + 70 days \[10 weeks\]). Analysis was performed on safety population.
|
0.00%
0/9 • All Adverse Events (AEs) were collected from signature of the informed consent form up to 56 weeks of the study regardless of seriousness or relationship to investigational product.
Reported AEs and death were treatment-emergent AEs (TEAE) that developed/worsened, and death that occurred during TEAE period (the time from the first dose of alirocumab up to the last dose of alirocumab + 70 days \[10 weeks\]). Analysis was performed on safety population.
|
|
Cardiac disorders
Aortic Valve Incompetence
|
22.2%
2/9 • Number of events 2 • All Adverse Events (AEs) were collected from signature of the informed consent form up to 56 weeks of the study regardless of seriousness or relationship to investigational product.
Reported AEs and death were treatment-emergent AEs (TEAE) that developed/worsened, and death that occurred during TEAE period (the time from the first dose of alirocumab up to the last dose of alirocumab + 70 days \[10 weeks\]). Analysis was performed on safety population.
|
11.1%
1/9 • Number of events 1 • All Adverse Events (AEs) were collected from signature of the informed consent form up to 56 weeks of the study regardless of seriousness or relationship to investigational product.
Reported AEs and death were treatment-emergent AEs (TEAE) that developed/worsened, and death that occurred during TEAE period (the time from the first dose of alirocumab up to the last dose of alirocumab + 70 days \[10 weeks\]). Analysis was performed on safety population.
|
|
Infections and infestations
Nasopharyngitis
|
11.1%
1/9 • Number of events 1 • All Adverse Events (AEs) were collected from signature of the informed consent form up to 56 weeks of the study regardless of seriousness or relationship to investigational product.
Reported AEs and death were treatment-emergent AEs (TEAE) that developed/worsened, and death that occurred during TEAE period (the time from the first dose of alirocumab up to the last dose of alirocumab + 70 days \[10 weeks\]). Analysis was performed on safety population.
|
22.2%
2/9 • Number of events 2 • All Adverse Events (AEs) were collected from signature of the informed consent form up to 56 weeks of the study regardless of seriousness or relationship to investigational product.
Reported AEs and death were treatment-emergent AEs (TEAE) that developed/worsened, and death that occurred during TEAE period (the time from the first dose of alirocumab up to the last dose of alirocumab + 70 days \[10 weeks\]). Analysis was performed on safety population.
|
|
Infections and infestations
Parvovirus B19 Infection
|
11.1%
1/9 • Number of events 1 • All Adverse Events (AEs) were collected from signature of the informed consent form up to 56 weeks of the study regardless of seriousness or relationship to investigational product.
Reported AEs and death were treatment-emergent AEs (TEAE) that developed/worsened, and death that occurred during TEAE period (the time from the first dose of alirocumab up to the last dose of alirocumab + 70 days \[10 weeks\]). Analysis was performed on safety population.
|
0.00%
0/9 • All Adverse Events (AEs) were collected from signature of the informed consent form up to 56 weeks of the study regardless of seriousness or relationship to investigational product.
Reported AEs and death were treatment-emergent AEs (TEAE) that developed/worsened, and death that occurred during TEAE period (the time from the first dose of alirocumab up to the last dose of alirocumab + 70 days \[10 weeks\]). Analysis was performed on safety population.
|
|
Infections and infestations
Pharyngotonsillitis
|
11.1%
1/9 • Number of events 1 • All Adverse Events (AEs) were collected from signature of the informed consent form up to 56 weeks of the study regardless of seriousness or relationship to investigational product.
Reported AEs and death were treatment-emergent AEs (TEAE) that developed/worsened, and death that occurred during TEAE period (the time from the first dose of alirocumab up to the last dose of alirocumab + 70 days \[10 weeks\]). Analysis was performed on safety population.
|
0.00%
0/9 • All Adverse Events (AEs) were collected from signature of the informed consent form up to 56 weeks of the study regardless of seriousness or relationship to investigational product.
Reported AEs and death were treatment-emergent AEs (TEAE) that developed/worsened, and death that occurred during TEAE period (the time from the first dose of alirocumab up to the last dose of alirocumab + 70 days \[10 weeks\]). Analysis was performed on safety population.
|
|
Congenital, familial and genetic disorders
Bicuspid Aortic Valve
|
0.00%
0/9 • All Adverse Events (AEs) were collected from signature of the informed consent form up to 56 weeks of the study regardless of seriousness or relationship to investigational product.
Reported AEs and death were treatment-emergent AEs (TEAE) that developed/worsened, and death that occurred during TEAE period (the time from the first dose of alirocumab up to the last dose of alirocumab + 70 days \[10 weeks\]). Analysis was performed on safety population.
|
11.1%
1/9 • Number of events 1 • All Adverse Events (AEs) were collected from signature of the informed consent form up to 56 weeks of the study regardless of seriousness or relationship to investigational product.
Reported AEs and death were treatment-emergent AEs (TEAE) that developed/worsened, and death that occurred during TEAE period (the time from the first dose of alirocumab up to the last dose of alirocumab + 70 days \[10 weeks\]). Analysis was performed on safety population.
|
|
Gastrointestinal disorders
Diarrhoea
|
0.00%
0/9 • All Adverse Events (AEs) were collected from signature of the informed consent form up to 56 weeks of the study regardless of seriousness or relationship to investigational product.
Reported AEs and death were treatment-emergent AEs (TEAE) that developed/worsened, and death that occurred during TEAE period (the time from the first dose of alirocumab up to the last dose of alirocumab + 70 days \[10 weeks\]). Analysis was performed on safety population.
|
11.1%
1/9 • Number of events 1 • All Adverse Events (AEs) were collected from signature of the informed consent form up to 56 weeks of the study regardless of seriousness or relationship to investigational product.
Reported AEs and death were treatment-emergent AEs (TEAE) that developed/worsened, and death that occurred during TEAE period (the time from the first dose of alirocumab up to the last dose of alirocumab + 70 days \[10 weeks\]). Analysis was performed on safety population.
|
|
Gastrointestinal disorders
Nausea
|
11.1%
1/9 • Number of events 1 • All Adverse Events (AEs) were collected from signature of the informed consent form up to 56 weeks of the study regardless of seriousness or relationship to investigational product.
Reported AEs and death were treatment-emergent AEs (TEAE) that developed/worsened, and death that occurred during TEAE period (the time from the first dose of alirocumab up to the last dose of alirocumab + 70 days \[10 weeks\]). Analysis was performed on safety population.
|
0.00%
0/9 • All Adverse Events (AEs) were collected from signature of the informed consent form up to 56 weeks of the study regardless of seriousness or relationship to investigational product.
Reported AEs and death were treatment-emergent AEs (TEAE) that developed/worsened, and death that occurred during TEAE period (the time from the first dose of alirocumab up to the last dose of alirocumab + 70 days \[10 weeks\]). Analysis was performed on safety population.
|
|
Gastrointestinal disorders
Vomiting
|
11.1%
1/9 • Number of events 1 • All Adverse Events (AEs) were collected from signature of the informed consent form up to 56 weeks of the study regardless of seriousness or relationship to investigational product.
Reported AEs and death were treatment-emergent AEs (TEAE) that developed/worsened, and death that occurred during TEAE period (the time from the first dose of alirocumab up to the last dose of alirocumab + 70 days \[10 weeks\]). Analysis was performed on safety population.
|
0.00%
0/9 • All Adverse Events (AEs) were collected from signature of the informed consent form up to 56 weeks of the study regardless of seriousness or relationship to investigational product.
Reported AEs and death were treatment-emergent AEs (TEAE) that developed/worsened, and death that occurred during TEAE period (the time from the first dose of alirocumab up to the last dose of alirocumab + 70 days \[10 weeks\]). Analysis was performed on safety population.
|
|
General disorders
Fatigue
|
11.1%
1/9 • Number of events 1 • All Adverse Events (AEs) were collected from signature of the informed consent form up to 56 weeks of the study regardless of seriousness or relationship to investigational product.
Reported AEs and death were treatment-emergent AEs (TEAE) that developed/worsened, and death that occurred during TEAE period (the time from the first dose of alirocumab up to the last dose of alirocumab + 70 days \[10 weeks\]). Analysis was performed on safety population.
|
0.00%
0/9 • All Adverse Events (AEs) were collected from signature of the informed consent form up to 56 weeks of the study regardless of seriousness or relationship to investigational product.
Reported AEs and death were treatment-emergent AEs (TEAE) that developed/worsened, and death that occurred during TEAE period (the time from the first dose of alirocumab up to the last dose of alirocumab + 70 days \[10 weeks\]). Analysis was performed on safety population.
|
|
General disorders
Influenza Like Illness
|
11.1%
1/9 • Number of events 1 • All Adverse Events (AEs) were collected from signature of the informed consent form up to 56 weeks of the study regardless of seriousness or relationship to investigational product.
Reported AEs and death were treatment-emergent AEs (TEAE) that developed/worsened, and death that occurred during TEAE period (the time from the first dose of alirocumab up to the last dose of alirocumab + 70 days \[10 weeks\]). Analysis was performed on safety population.
|
0.00%
0/9 • All Adverse Events (AEs) were collected from signature of the informed consent form up to 56 weeks of the study regardless of seriousness or relationship to investigational product.
Reported AEs and death were treatment-emergent AEs (TEAE) that developed/worsened, and death that occurred during TEAE period (the time from the first dose of alirocumab up to the last dose of alirocumab + 70 days \[10 weeks\]). Analysis was performed on safety population.
|
|
General disorders
Injection Site Pain
|
11.1%
1/9 • Number of events 1 • All Adverse Events (AEs) were collected from signature of the informed consent form up to 56 weeks of the study regardless of seriousness or relationship to investigational product.
Reported AEs and death were treatment-emergent AEs (TEAE) that developed/worsened, and death that occurred during TEAE period (the time from the first dose of alirocumab up to the last dose of alirocumab + 70 days \[10 weeks\]). Analysis was performed on safety population.
|
0.00%
0/9 • All Adverse Events (AEs) were collected from signature of the informed consent form up to 56 weeks of the study regardless of seriousness or relationship to investigational product.
Reported AEs and death were treatment-emergent AEs (TEAE) that developed/worsened, and death that occurred during TEAE period (the time from the first dose of alirocumab up to the last dose of alirocumab + 70 days \[10 weeks\]). Analysis was performed on safety population.
|
|
General disorders
Injection Site Reaction
|
0.00%
0/9 • All Adverse Events (AEs) were collected from signature of the informed consent form up to 56 weeks of the study regardless of seriousness or relationship to investigational product.
Reported AEs and death were treatment-emergent AEs (TEAE) that developed/worsened, and death that occurred during TEAE period (the time from the first dose of alirocumab up to the last dose of alirocumab + 70 days \[10 weeks\]). Analysis was performed on safety population.
|
11.1%
1/9 • Number of events 1 • All Adverse Events (AEs) were collected from signature of the informed consent form up to 56 weeks of the study regardless of seriousness or relationship to investigational product.
Reported AEs and death were treatment-emergent AEs (TEAE) that developed/worsened, and death that occurred during TEAE period (the time from the first dose of alirocumab up to the last dose of alirocumab + 70 days \[10 weeks\]). Analysis was performed on safety population.
|
|
General disorders
Pyrexia
|
0.00%
0/9 • All Adverse Events (AEs) were collected from signature of the informed consent form up to 56 weeks of the study regardless of seriousness or relationship to investigational product.
Reported AEs and death were treatment-emergent AEs (TEAE) that developed/worsened, and death that occurred during TEAE period (the time from the first dose of alirocumab up to the last dose of alirocumab + 70 days \[10 weeks\]). Analysis was performed on safety population.
|
11.1%
1/9 • Number of events 1 • All Adverse Events (AEs) were collected from signature of the informed consent form up to 56 weeks of the study regardless of seriousness or relationship to investigational product.
Reported AEs and death were treatment-emergent AEs (TEAE) that developed/worsened, and death that occurred during TEAE period (the time from the first dose of alirocumab up to the last dose of alirocumab + 70 days \[10 weeks\]). Analysis was performed on safety population.
|
|
Hepatobiliary disorders
Hepatomegaly
|
11.1%
1/9 • Number of events 1 • All Adverse Events (AEs) were collected from signature of the informed consent form up to 56 weeks of the study regardless of seriousness or relationship to investigational product.
Reported AEs and death were treatment-emergent AEs (TEAE) that developed/worsened, and death that occurred during TEAE period (the time from the first dose of alirocumab up to the last dose of alirocumab + 70 days \[10 weeks\]). Analysis was performed on safety population.
|
0.00%
0/9 • All Adverse Events (AEs) were collected from signature of the informed consent form up to 56 weeks of the study regardless of seriousness or relationship to investigational product.
Reported AEs and death were treatment-emergent AEs (TEAE) that developed/worsened, and death that occurred during TEAE period (the time from the first dose of alirocumab up to the last dose of alirocumab + 70 days \[10 weeks\]). Analysis was performed on safety population.
|
|
Infections and infestations
Gastroenteritis
|
11.1%
1/9 • Number of events 1 • All Adverse Events (AEs) were collected from signature of the informed consent form up to 56 weeks of the study regardless of seriousness or relationship to investigational product.
Reported AEs and death were treatment-emergent AEs (TEAE) that developed/worsened, and death that occurred during TEAE period (the time from the first dose of alirocumab up to the last dose of alirocumab + 70 days \[10 weeks\]). Analysis was performed on safety population.
|
0.00%
0/9 • All Adverse Events (AEs) were collected from signature of the informed consent form up to 56 weeks of the study regardless of seriousness or relationship to investigational product.
Reported AEs and death were treatment-emergent AEs (TEAE) that developed/worsened, and death that occurred during TEAE period (the time from the first dose of alirocumab up to the last dose of alirocumab + 70 days \[10 weeks\]). Analysis was performed on safety population.
|
|
Infections and infestations
Influenza
|
22.2%
2/9 • Number of events 2 • All Adverse Events (AEs) were collected from signature of the informed consent form up to 56 weeks of the study regardless of seriousness or relationship to investigational product.
Reported AEs and death were treatment-emergent AEs (TEAE) that developed/worsened, and death that occurred during TEAE period (the time from the first dose of alirocumab up to the last dose of alirocumab + 70 days \[10 weeks\]). Analysis was performed on safety population.
|
0.00%
0/9 • All Adverse Events (AEs) were collected from signature of the informed consent form up to 56 weeks of the study regardless of seriousness or relationship to investigational product.
Reported AEs and death were treatment-emergent AEs (TEAE) that developed/worsened, and death that occurred during TEAE period (the time from the first dose of alirocumab up to the last dose of alirocumab + 70 days \[10 weeks\]). Analysis was performed on safety population.
|
|
Infections and infestations
Upper Respiratory Tract Infection
|
11.1%
1/9 • Number of events 1 • All Adverse Events (AEs) were collected from signature of the informed consent form up to 56 weeks of the study regardless of seriousness or relationship to investigational product.
Reported AEs and death were treatment-emergent AEs (TEAE) that developed/worsened, and death that occurred during TEAE period (the time from the first dose of alirocumab up to the last dose of alirocumab + 70 days \[10 weeks\]). Analysis was performed on safety population.
|
0.00%
0/9 • All Adverse Events (AEs) were collected from signature of the informed consent form up to 56 weeks of the study regardless of seriousness or relationship to investigational product.
Reported AEs and death were treatment-emergent AEs (TEAE) that developed/worsened, and death that occurred during TEAE period (the time from the first dose of alirocumab up to the last dose of alirocumab + 70 days \[10 weeks\]). Analysis was performed on safety population.
|
|
Infections and infestations
Urinary Tract Infection
|
11.1%
1/9 • Number of events 1 • All Adverse Events (AEs) were collected from signature of the informed consent form up to 56 weeks of the study regardless of seriousness or relationship to investigational product.
Reported AEs and death were treatment-emergent AEs (TEAE) that developed/worsened, and death that occurred during TEAE period (the time from the first dose of alirocumab up to the last dose of alirocumab + 70 days \[10 weeks\]). Analysis was performed on safety population.
|
0.00%
0/9 • All Adverse Events (AEs) were collected from signature of the informed consent form up to 56 weeks of the study regardless of seriousness or relationship to investigational product.
Reported AEs and death were treatment-emergent AEs (TEAE) that developed/worsened, and death that occurred during TEAE period (the time from the first dose of alirocumab up to the last dose of alirocumab + 70 days \[10 weeks\]). Analysis was performed on safety population.
|
|
Injury, poisoning and procedural complications
Thermal Burn
|
0.00%
0/9 • All Adverse Events (AEs) were collected from signature of the informed consent form up to 56 weeks of the study regardless of seriousness or relationship to investigational product.
Reported AEs and death were treatment-emergent AEs (TEAE) that developed/worsened, and death that occurred during TEAE period (the time from the first dose of alirocumab up to the last dose of alirocumab + 70 days \[10 weeks\]). Analysis was performed on safety population.
|
11.1%
1/9 • Number of events 1 • All Adverse Events (AEs) were collected from signature of the informed consent form up to 56 weeks of the study regardless of seriousness or relationship to investigational product.
Reported AEs and death were treatment-emergent AEs (TEAE) that developed/worsened, and death that occurred during TEAE period (the time from the first dose of alirocumab up to the last dose of alirocumab + 70 days \[10 weeks\]). Analysis was performed on safety population.
|
|
Investigations
Alanine Aminotransferase Increased
|
0.00%
0/9 • All Adverse Events (AEs) were collected from signature of the informed consent form up to 56 weeks of the study regardless of seriousness or relationship to investigational product.
Reported AEs and death were treatment-emergent AEs (TEAE) that developed/worsened, and death that occurred during TEAE period (the time from the first dose of alirocumab up to the last dose of alirocumab + 70 days \[10 weeks\]). Analysis was performed on safety population.
|
11.1%
1/9 • Number of events 1 • All Adverse Events (AEs) were collected from signature of the informed consent form up to 56 weeks of the study regardless of seriousness or relationship to investigational product.
Reported AEs and death were treatment-emergent AEs (TEAE) that developed/worsened, and death that occurred during TEAE period (the time from the first dose of alirocumab up to the last dose of alirocumab + 70 days \[10 weeks\]). Analysis was performed on safety population.
|
|
Metabolism and nutrition disorders
Hypertriglyceridaemia
|
11.1%
1/9 • Number of events 1 • All Adverse Events (AEs) were collected from signature of the informed consent form up to 56 weeks of the study regardless of seriousness or relationship to investigational product.
Reported AEs and death were treatment-emergent AEs (TEAE) that developed/worsened, and death that occurred during TEAE period (the time from the first dose of alirocumab up to the last dose of alirocumab + 70 days \[10 weeks\]). Analysis was performed on safety population.
|
0.00%
0/9 • All Adverse Events (AEs) were collected from signature of the informed consent form up to 56 weeks of the study regardless of seriousness or relationship to investigational product.
Reported AEs and death were treatment-emergent AEs (TEAE) that developed/worsened, and death that occurred during TEAE period (the time from the first dose of alirocumab up to the last dose of alirocumab + 70 days \[10 weeks\]). Analysis was performed on safety population.
|
|
Musculoskeletal and connective tissue disorders
Pain In Extremity
|
0.00%
0/9 • All Adverse Events (AEs) were collected from signature of the informed consent form up to 56 weeks of the study regardless of seriousness or relationship to investigational product.
Reported AEs and death were treatment-emergent AEs (TEAE) that developed/worsened, and death that occurred during TEAE period (the time from the first dose of alirocumab up to the last dose of alirocumab + 70 days \[10 weeks\]). Analysis was performed on safety population.
|
11.1%
1/9 • Number of events 1 • All Adverse Events (AEs) were collected from signature of the informed consent form up to 56 weeks of the study regardless of seriousness or relationship to investigational product.
Reported AEs and death were treatment-emergent AEs (TEAE) that developed/worsened, and death that occurred during TEAE period (the time from the first dose of alirocumab up to the last dose of alirocumab + 70 days \[10 weeks\]). Analysis was performed on safety population.
|
|
Musculoskeletal and connective tissue disorders
Tendon Pain
|
0.00%
0/9 • All Adverse Events (AEs) were collected from signature of the informed consent form up to 56 weeks of the study regardless of seriousness or relationship to investigational product.
Reported AEs and death were treatment-emergent AEs (TEAE) that developed/worsened, and death that occurred during TEAE period (the time from the first dose of alirocumab up to the last dose of alirocumab + 70 days \[10 weeks\]). Analysis was performed on safety population.
|
11.1%
1/9 • Number of events 1 • All Adverse Events (AEs) were collected from signature of the informed consent form up to 56 weeks of the study regardless of seriousness or relationship to investigational product.
Reported AEs and death were treatment-emergent AEs (TEAE) that developed/worsened, and death that occurred during TEAE period (the time from the first dose of alirocumab up to the last dose of alirocumab + 70 days \[10 weeks\]). Analysis was performed on safety population.
|
|
Musculoskeletal and connective tissue disorders
Tendonitis
|
0.00%
0/9 • All Adverse Events (AEs) were collected from signature of the informed consent form up to 56 weeks of the study regardless of seriousness or relationship to investigational product.
Reported AEs and death were treatment-emergent AEs (TEAE) that developed/worsened, and death that occurred during TEAE period (the time from the first dose of alirocumab up to the last dose of alirocumab + 70 days \[10 weeks\]). Analysis was performed on safety population.
|
11.1%
1/9 • Number of events 1 • All Adverse Events (AEs) were collected from signature of the informed consent form up to 56 weeks of the study regardless of seriousness or relationship to investigational product.
Reported AEs and death were treatment-emergent AEs (TEAE) that developed/worsened, and death that occurred during TEAE period (the time from the first dose of alirocumab up to the last dose of alirocumab + 70 days \[10 weeks\]). Analysis was performed on safety population.
|
|
Nervous system disorders
Headache
|
22.2%
2/9 • Number of events 4 • All Adverse Events (AEs) were collected from signature of the informed consent form up to 56 weeks of the study regardless of seriousness or relationship to investigational product.
Reported AEs and death were treatment-emergent AEs (TEAE) that developed/worsened, and death that occurred during TEAE period (the time from the first dose of alirocumab up to the last dose of alirocumab + 70 days \[10 weeks\]). Analysis was performed on safety population.
|
11.1%
1/9 • Number of events 2 • All Adverse Events (AEs) were collected from signature of the informed consent form up to 56 weeks of the study regardless of seriousness or relationship to investigational product.
Reported AEs and death were treatment-emergent AEs (TEAE) that developed/worsened, and death that occurred during TEAE period (the time from the first dose of alirocumab up to the last dose of alirocumab + 70 days \[10 weeks\]). Analysis was performed on safety population.
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
11.1%
1/9 • Number of events 2 • All Adverse Events (AEs) were collected from signature of the informed consent form up to 56 weeks of the study regardless of seriousness or relationship to investigational product.
Reported AEs and death were treatment-emergent AEs (TEAE) that developed/worsened, and death that occurred during TEAE period (the time from the first dose of alirocumab up to the last dose of alirocumab + 70 days \[10 weeks\]). Analysis was performed on safety population.
|
11.1%
1/9 • Number of events 1 • All Adverse Events (AEs) were collected from signature of the informed consent form up to 56 weeks of the study regardless of seriousness or relationship to investigational product.
Reported AEs and death were treatment-emergent AEs (TEAE) that developed/worsened, and death that occurred during TEAE period (the time from the first dose of alirocumab up to the last dose of alirocumab + 70 days \[10 weeks\]). Analysis was performed on safety population.
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea
|
0.00%
0/9 • All Adverse Events (AEs) were collected from signature of the informed consent form up to 56 weeks of the study regardless of seriousness or relationship to investigational product.
Reported AEs and death were treatment-emergent AEs (TEAE) that developed/worsened, and death that occurred during TEAE period (the time from the first dose of alirocumab up to the last dose of alirocumab + 70 days \[10 weeks\]). Analysis was performed on safety population.
|
11.1%
1/9 • Number of events 1 • All Adverse Events (AEs) were collected from signature of the informed consent form up to 56 weeks of the study regardless of seriousness or relationship to investigational product.
Reported AEs and death were treatment-emergent AEs (TEAE) that developed/worsened, and death that occurred during TEAE period (the time from the first dose of alirocumab up to the last dose of alirocumab + 70 days \[10 weeks\]). Analysis was performed on safety population.
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea Paroxysmal Nocturnal
|
11.1%
1/9 • Number of events 1 • All Adverse Events (AEs) were collected from signature of the informed consent form up to 56 weeks of the study regardless of seriousness or relationship to investigational product.
Reported AEs and death were treatment-emergent AEs (TEAE) that developed/worsened, and death that occurred during TEAE period (the time from the first dose of alirocumab up to the last dose of alirocumab + 70 days \[10 weeks\]). Analysis was performed on safety population.
|
0.00%
0/9 • All Adverse Events (AEs) were collected from signature of the informed consent form up to 56 weeks of the study regardless of seriousness or relationship to investigational product.
Reported AEs and death were treatment-emergent AEs (TEAE) that developed/worsened, and death that occurred during TEAE period (the time from the first dose of alirocumab up to the last dose of alirocumab + 70 days \[10 weeks\]). Analysis was performed on safety population.
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
0.00%
0/9 • All Adverse Events (AEs) were collected from signature of the informed consent form up to 56 weeks of the study regardless of seriousness or relationship to investigational product.
Reported AEs and death were treatment-emergent AEs (TEAE) that developed/worsened, and death that occurred during TEAE period (the time from the first dose of alirocumab up to the last dose of alirocumab + 70 days \[10 weeks\]). Analysis was performed on safety population.
|
11.1%
1/9 • Number of events 1 • All Adverse Events (AEs) were collected from signature of the informed consent form up to 56 weeks of the study regardless of seriousness or relationship to investigational product.
Reported AEs and death were treatment-emergent AEs (TEAE) that developed/worsened, and death that occurred during TEAE period (the time from the first dose of alirocumab up to the last dose of alirocumab + 70 days \[10 weeks\]). Analysis was performed on safety population.
|
|
Skin and subcutaneous tissue disorders
Dry Skin
|
0.00%
0/9 • All Adverse Events (AEs) were collected from signature of the informed consent form up to 56 weeks of the study regardless of seriousness or relationship to investigational product.
Reported AEs and death were treatment-emergent AEs (TEAE) that developed/worsened, and death that occurred during TEAE period (the time from the first dose of alirocumab up to the last dose of alirocumab + 70 days \[10 weeks\]). Analysis was performed on safety population.
|
11.1%
1/9 • Number of events 1 • All Adverse Events (AEs) were collected from signature of the informed consent form up to 56 weeks of the study regardless of seriousness or relationship to investigational product.
Reported AEs and death were treatment-emergent AEs (TEAE) that developed/worsened, and death that occurred during TEAE period (the time from the first dose of alirocumab up to the last dose of alirocumab + 70 days \[10 weeks\]). Analysis was performed on safety population.
|
|
Skin and subcutaneous tissue disorders
Rash
|
0.00%
0/9 • All Adverse Events (AEs) were collected from signature of the informed consent form up to 56 weeks of the study regardless of seriousness or relationship to investigational product.
Reported AEs and death were treatment-emergent AEs (TEAE) that developed/worsened, and death that occurred during TEAE period (the time from the first dose of alirocumab up to the last dose of alirocumab + 70 days \[10 weeks\]). Analysis was performed on safety population.
|
11.1%
1/9 • Number of events 1 • All Adverse Events (AEs) were collected from signature of the informed consent form up to 56 weeks of the study regardless of seriousness or relationship to investigational product.
Reported AEs and death were treatment-emergent AEs (TEAE) that developed/worsened, and death that occurred during TEAE period (the time from the first dose of alirocumab up to the last dose of alirocumab + 70 days \[10 weeks\]). Analysis was performed on safety population.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee The Sponsor supports publication of clinical trial results but may request that investigators temporarily delay or alter publications in order to protect proprietary information. The Sponsor may also require that the results of multicenter studies be published only in their entirety and not as individual site data.
- Publication restrictions are in place
Restriction type: OTHER