Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu
Trial record 1 of 1 for:    EFC14660
Previous Study | Return to List | Next Study

An Efficacy and Safety Study of Alirocumab in Children and Adolescents With Homozygous Familial Hypercholesterolemia

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03510715
Recruitment Status : Completed
First Posted : April 27, 2018
Results First Posted : December 29, 2020
Last Update Posted : December 29, 2020
Sponsor:
Collaborator:
Regeneron Pharmaceuticals
Information provided by (Responsible Party):
Sanofi

Study Type Interventional
Study Design Allocation: N/A;   Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Hypercholesterolemia
Interventions Drug: Alirocumab SAR236553 (REGN727)
Drug: Atorvastatin
Drug: Simvastatin
Drug: Fluvastatin
Drug: Pravastatin
Drug: Lovastatin
Drug: Rosuvastatin
Drug: Ezetimibe
Drug: Cholestyramine
Drug: Nicotinic acid
Drug: Fenofibrate
Drug: Omega-3 fatty acids
Enrollment 18
Recruitment Details 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.
Pre-assignment Details A total of 18 participants were enrolled and received treatment in this study.
Arm/Group Title Alirocumab 75 mg Q2W/up to 150 mg Q2W Alirocumab 150 mg Q2W
Hide Arm/Group Description 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. Participants with BW >=50 kg received SC injection of alirocumab 150 mg Q2W for 48 weeks.
Period Title: Overall Study
Started 9 9
Completed 8 9
Not Completed 1 0
Reason Not Completed
Adverse Event             1             0
Arm/Group Title Alirocumab 75 mg Q2W/up to 150 mg Q2W Alirocumab 150 mg Q2W Total
Hide Arm/Group Description 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. Participants with BW >=50 kg received SC injection of alirocumab 150 mg Q2W for 48 weeks. Total of all reporting groups
Overall Number of Baseline Participants 9 9 18
Hide Baseline Analysis Population Description
Analysis was performed on all enrolled participants.
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 9 participants 9 participants 18 participants
10.4  (1.5) 14.3  (2.4) 12.4  (2.8)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 9 participants 9 participants 18 participants
Female
4
  44.4%
5
  55.6%
9
  50.0%
Male
5
  55.6%
4
  44.4%
9
  50.0%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 9 participants 9 participants 18 participants
American Indian or Alaska Native
2
  22.2%
1
  11.1%
3
  16.7%
Asian
1
  11.1%
2
  22.2%
3
  16.7%
Native Hawaiian or Other Pacific Islander
0
   0.0%
0
   0.0%
0
   0.0%
Black or African American
0
   0.0%
1
  11.1%
1
   5.6%
White
6
  66.7%
5
  55.6%
11
  61.1%
More than one race
0
   0.0%
0
   0.0%
0
   0.0%
Unknown or Not Reported
0
   0.0%
0
   0.0%
0
   0.0%
Low-Density Lipoprotein Cholesterol (LDL-C)  
Mean (Standard Deviation)
Unit of measure:  Milligrams per deciliter (mg/dL)
Number Analyzed 9 participants 9 participants 18 participants
437.7  (192.8) 308.3  (181.6) 373.0  (193.5)
1.Primary Outcome
Title Percent Change From Baseline in Low-Density Lipoprotein Cholesterol (LDL-C) at Week 12: Intent-to-Treat (ITT) Analysis
Hide Description 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).
Time Frame Baseline to Week 12
Hide Outcome Measure Data
Hide Analysis Population Description
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).
Arm/Group Title Alirocumab
Hide Arm/Group Description:
All participants who received either 75 mg (BW <50 kg) or 150 mg (BW >=50 kg) alirocumab subcutaneously Q2W for 48 weeks.
Overall Number of Participants Analyzed 18
Least Squares Mean (Standard Error)
Unit of Measure: percent change
-4.1  (9.0)
2.Secondary Outcome
Title Percent Change From Baseline in Low-Density Lipoprotein Cholesterol at Week 12: On-treatment Analysis
Hide Description 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).
Time Frame Baseline to Week 12
Hide Outcome Measure Data
Hide Analysis Population Description
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).
Arm/Group Title Alirocumab
Hide Arm/Group Description:
All participants who received either 75 mg (BW <50 kg) or 150 mg (BW >=50 kg) alirocumab subcutaneously Q2W for 48 weeks.
Overall Number of Participants Analyzed 18
Least Squares Mean (Standard Error)
Unit of Measure: percent change
-4.1  (9.0)
3.Secondary Outcome
Title Percent Change From Baseline in Low-Density Lipoprotein Cholesterol at Weeks 24 and 48: ITT Analysis/On-treatment Analysis
Hide Description 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.
Time Frame Baseline to Weeks 24 and 48
Hide Outcome Measure Data
Hide Analysis Population Description
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).
Arm/Group Title Alirocumab
Hide Arm/Group Description:
All participants who received either 75 mg (BW <50 kg) or 150 mg (BW >=50 kg) alirocumab subcutaneously Q2W for 48 weeks.
Overall Number of Participants Analyzed 18
Least Squares Mean (Standard Error)
Unit of Measure: percent change
Week 24 -10.1  (7.6)
Week 48 4.2  (12.8)
4.Secondary Outcome
Title Percent Change From Baseline in Apolipoprotein (Apo) B at Weeks 12, 24 and 48: ITT Analysis/On-treatment Analysis
Hide Description 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.
Time Frame Baseline to Weeks 12, 24 and 48
Hide Outcome Measure Data
Hide Analysis Population Description
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).
Arm/Group Title Alirocumab
Hide Arm/Group Description:
All participants who received either 75 mg (BW <50 kg) or 150 mg (BW >=50 kg) alirocumab subcutaneously Q2W for 48 weeks.
Overall Number of Participants Analyzed 18
Least Squares Mean (Standard Error)
Unit of Measure: percent change
Week 12 -4.2  (6.8)
Week 24 -11.8  (6.1)
Week 48 0.9  (10.4)
5.Secondary Outcome
Title Percent Change From Baseline in Non-High Density Lipoprotein Cholesterol (Non-HDL-C) at Weeks 12, 24 and 48 - ITT Analysis/On-treatment Analysis
Hide Description 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.
Time Frame Baseline to Weeks 12, 24 and 48
Hide Outcome Measure Data
Hide Analysis Population Description
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).
Arm/Group Title Alirocumab
Hide Arm/Group Description:
All participants who received either 75 mg (BW <50 kg) or 150 mg (BW >=50 kg) alirocumab subcutaneously Q2W for 48 weeks.
Overall Number of Participants Analyzed 18
Least Squares Mean (Standard Error)
Unit of Measure: percent change
Week 12 -3.9  (8.3)
Week 24 -9.2  (7.3)
Week 48 5.7  (13.1)
6.Secondary Outcome
Title Percent Change From Baseline in Total Cholesterol (Total-C) at Weeks 12, 24 and 48: ITT Analysis/On-treatment Analysis
Hide Description 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.
Time Frame Baseline to Weeks 12, 24 and 48
Hide Outcome Measure Data
Hide Analysis Population Description
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).
Arm/Group Title Alirocumab
Hide Arm/Group Description:
All participants who received either 75 mg (BW <50 kg) or 150 mg (BW >=50 kg) alirocumab subcutaneously Q2W for 48 weeks.
Overall Number of Participants Analyzed 18
Least Squares Mean (Standard Error)
Unit of Measure: percent change
Week 12 -1.9  (7.2)
Week 24 -6.3  (6.5)
Week 48 5.5  (10.7)
7.Secondary Outcome
Title Percent Change From Baseline in Lipoprotein a (Lp) (a) at Weeks 12, 24 and 48: ITT Analysis/On-treatment Analysis
Hide Description 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.
Time Frame Baseline to Weeks 12, 24 and 48
Hide Outcome Measure Data
Hide Analysis Population Description
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).
Arm/Group Title Alirocumab
Hide Arm/Group Description:
All participants who received either 75 mg (BW <50 kg) or 150 mg (BW >=50 kg) alirocumab subcutaneously Q2W for 48 weeks.
Overall Number of Participants Analyzed 18
Mean (Standard Error)
Unit of Measure: percent change
Week 12 7.4  (7.6)
Week 24 -5.2  (8.1)
Week 48 -6.4  (12.2)
8.Secondary Outcome
Title Percent Change From Baseline in High Density Lipoprotein Cholesterol (HDL-C) at Weeks 12, 24 and 48: ITT Analysis/On-treatment Analysis
Hide Description 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.
Time Frame Baseline to Weeks 12, 24 and 48
Hide Outcome Measure Data
Hide Analysis Population Description
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).
Arm/Group Title Alirocumab
Hide Arm/Group Description:
All participants who received either 75 mg (BW <50 kg) or 150 mg (BW >=50 kg) alirocumab subcutaneously Q2W for 48 weeks.
Overall Number of Participants Analyzed 18
Least Squares Mean (Standard Error)
Unit of Measure: percent change
Week 12 13.0  (5.9)
Week 24 8.9  (4.4)
Week 48 10.1  (5.5)
9.Secondary Outcome
Title Percent Change From Baseline in Fasting Triglycerides (TG) at Weeks 12, 24 and 48: ITT Analysis/On-treatment Analysis
Hide Description 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.
Time Frame Baseline to Weeks 12, 24 and 48
Hide Outcome Measure Data
Hide Analysis Population Description
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).
Arm/Group Title Alirocumab
Hide Arm/Group Description:
All participants who received either 75 mg (BW <50 kg) or 150 mg (BW >=50 kg) alirocumab subcutaneously Q2W for 48 weeks.
Overall Number of Participants Analyzed 18
Mean (Standard Error)
Unit of Measure: percent change
Week 12 2.8  (8.0)
Week 24 5.2  (16.2)
Week 48 10.0  (8.2)
10.Secondary Outcome
Title Percent Change From Baseline in Apolipoprotein A1 (Apo A1) at Weeks 12, 24 and 48: ITT Analysis/On-treatment Analysis
Hide Description 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.
Time Frame Baseline to Weeks 12, 24 and 48
Hide Outcome Measure Data
Hide Analysis Population Description
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).
Arm/Group Title Alirocumab
Hide Arm/Group Description:
All participants who received either 75 mg (BW <50 kg) or 150 mg (BW >=50 kg) alirocumab subcutaneously Q2W for 48 weeks.
Overall Number of Participants Analyzed 18
Least Squares Mean (Standard Error)
Unit of Measure: percent change
Week 12 11.3  (6.9)
Week 24 14.6  (6.0)
Week 48 11.3  (5.8)
11.Secondary Outcome
Title Percentage of Participants Reporting >=15 Percent (%) Reduction in LDL-C Level at Weeks 12, 24 and 48: ITT Analysis/On-treatment Analysis
Hide Description 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.
Time Frame Baseline to Weeks 12, 24 and 48
Hide Outcome Measure Data
Hide Analysis Population Description
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).
Arm/Group Title Alirocumab
Hide Arm/Group Description:
All participants who received either 75 mg (BW <50 kg) or 150 mg (BW >=50 kg) alirocumab subcutaneously Q2W for 48 weeks.
Overall Number of Participants Analyzed 18
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: percentage of participants
Week 12
50.0
(26.2 to 73.8)
Week 24
50.0
(26.2 to 73.8)
Week 48
39.0
(15.8 to 62.2)
12.Secondary Outcome
Title Absolute Change From Baseline in LDL-C Level at Weeks 12, 24 and 48: ITT Analysis/On-treatment Analysis
Hide Description 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.
Time Frame Baseline to Weeks 12, 24 and 48
Hide Outcome Measure Data
Hide Analysis Population Description
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).
Arm/Group Title Alirocumab
Hide Arm/Group Description:
All participants who received either 75 mg (BW <50 kg) or 150 mg (BW >=50 kg) alirocumab subcutaneously Q2W for 48 weeks.
Overall Number of Participants Analyzed 18
Least Squares Mean (Standard Error)
Unit of Measure: mg/dL
Week 12 -33.4  (19.1)
Week 24 -43.0  (19.0)
Week 48 -15.0  (25.7)
13.Secondary Outcome
Title Number of Participants With Tanner Staging at Baseline, Weeks 12, 24 and 48
Hide Description 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).
Time Frame Baseline, Weeks 12, 24 and 48
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Alirocumab 75 mg Q2W/up to 150 mg Q2W Alirocumab 150 mg Q2W
Hide Arm/Group Description:
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.
Participants with BW >=50 kg received SC injection of alirocumab 150 mg Q2W for 48 weeks.
Overall Number of Participants Analyzed 9 9
Measure Type: Count of Participants
Unit of Measure: Participants
Baseline: Prepubescent Number Analyzed 9 participants 9 participants
3
  33.3%
0
   0.0%
Baseline: Pubescent Number Analyzed 9 participants 9 participants
6
  66.7%
9
 100.0%
Baseline: Post-pubescent Number Analyzed 9 participants 9 participants
0
   0.0%
0
   0.0%
Week 12: Prepubescent Number Analyzed 9 participants 9 participants
3
  33.3%
0
   0.0%
Week 12: Pubescent Number Analyzed 9 participants 9 participants
6
  66.7%
8
  88.9%
Week 12: Post-pubescent Number Analyzed 9 participants 9 participants
0
   0.0%
1
  11.1%
Week 24: Prepubescent Number Analyzed 8 participants 9 participants
2
  25.0%
0
   0.0%
Week 24: Pubescent Number Analyzed 8 participants 9 participants
6
  75.0%
8
  88.9%
Week 24: Post-pubescent Number Analyzed 8 participants 9 participants
0
   0.0%
1
  11.1%
Week 48: Prepubescent Number Analyzed 8 participants 9 participants
1
  12.5%
0
   0.0%
Week 48: Pubescent Number Analyzed 8 participants 9 participants
7
  87.5%
7
  77.8%
Week 48: Post-pubescent Number Analyzed 8 participants 9 participants
0
   0.0%
2
  22.2%
Time Frame 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.
Adverse Event Reporting Description 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.
 
Arm/Group Title Alirocumab 75 mg Q2W/up to 150 mg Q2W Alirocumab 150 mg Q2W
Hide Arm/Group Description 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. Participants with BW >=50 kg received SC injection of alirocumab 150 mg Q2W for 48 weeks.
All-Cause Mortality
Alirocumab 75 mg Q2W/up to 150 mg Q2W Alirocumab 150 mg Q2W
Affected / at Risk (%) Affected / at Risk (%)
Total   1/9 (11.11%)      0/9 (0.00%)    
Hide Serious Adverse Events
Alirocumab 75 mg Q2W/up to 150 mg Q2W Alirocumab 150 mg Q2W
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   1/9 (11.11%)      0/9 (0.00%)    
Cardiac disorders     
Cardiac Failure  1  1/9 (11.11%)  1 0/9 (0.00%)  0
1
Term from vocabulary, MedDRA 22.1
Indicates events were collected by systematic assessment
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Alirocumab 75 mg Q2W/up to 150 mg Q2W Alirocumab 150 mg Q2W
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   8/9 (88.89%)      9/9 (100.00%)    
Blood and lymphatic system disorders     
Splenomegaly  1  1/9 (11.11%)  1 0/9 (0.00%)  0
Cardiac disorders     
Aortic Valve Incompetence  1  2/9 (22.22%)  2 1/9 (11.11%)  1
Congenital, familial and genetic disorders     
Bicuspid Aortic Valve  1  0/9 (0.00%)  0 1/9 (11.11%)  1
Gastrointestinal disorders     
Diarrhoea  1  0/9 (0.00%)  0 1/9 (11.11%)  1
Nausea  1  1/9 (11.11%)  1 0/9 (0.00%)  0
Vomiting  1  1/9 (11.11%)  1 0/9 (0.00%)  0
General disorders     
Fatigue  1  1/9 (11.11%)  1 0/9 (0.00%)  0
Influenza Like Illness  1  1/9 (11.11%)  1 0/9 (0.00%)  0
Injection Site Pain  1  1/9 (11.11%)  1 0/9 (0.00%)  0
Injection Site Reaction  1  0/9 (0.00%)  0 1/9 (11.11%)  1
Pyrexia  1  0/9 (0.00%)  0 1/9 (11.11%)  1
Hepatobiliary disorders     
Hepatomegaly  1  1/9 (11.11%)  1 0/9 (0.00%)  0
Infections and infestations     
Gastroenteritis  1  1/9 (11.11%)  1 0/9 (0.00%)  0
Influenza  1  2/9 (22.22%)  2 0/9 (0.00%)  0
Nasopharyngitis  1  1/9 (11.11%)  1 2/9 (22.22%)  2
Parvovirus B19 Infection  1  1/9 (11.11%)  1 0/9 (0.00%)  0
Pharyngotonsillitis  1  1/9 (11.11%)  1 0/9 (0.00%)  0
Upper Respiratory Tract Infection  1  1/9 (11.11%)  1 0/9 (0.00%)  0
Urinary Tract Infection  1  1/9 (11.11%)  1 0/9 (0.00%)  0
Injury, poisoning and procedural complications     
Thermal Burn  1  0/9 (0.00%)  0 1/9 (11.11%)  1
Investigations     
Alanine Aminotransferase Increased  1  0/9 (0.00%)  0 1/9 (11.11%)  1
Metabolism and nutrition disorders     
Hypertriglyceridaemia  1  1/9 (11.11%)  1 0/9 (0.00%)  0
Musculoskeletal and connective tissue disorders     
Pain In Extremity  1  0/9 (0.00%)  0 1/9 (11.11%)  1
Tendon Pain  1  0/9 (0.00%)  0 1/9 (11.11%)  1
Tendonitis  1  0/9 (0.00%)  0 1/9 (11.11%)  1
Nervous system disorders     
Headache  1  2/9 (22.22%)  4 1/9 (11.11%)  2
Respiratory, thoracic and mediastinal disorders     
Cough  1  1/9 (11.11%)  2 1/9 (11.11%)  1
Dyspnoea  1  0/9 (0.00%)  0 1/9 (11.11%)  1
Dyspnoea Paroxysmal Nocturnal  1  1/9 (11.11%)  1 0/9 (0.00%)  0
Epistaxis  1  0/9 (0.00%)  0 1/9 (11.11%)  1
Skin and subcutaneous tissue disorders     
Dry Skin  1  0/9 (0.00%)  0 1/9 (11.11%)  1
Rash  1  0/9 (0.00%)  0 1/9 (11.11%)  1
1
Term from vocabulary, MedDRA 22.1
Indicates events were collected by systematic assessment
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
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.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Trial Transparency Team
Organization: Sanofi
Phone: 800-633-1610 ext 1#
EMail: Contact-US@sanofi.com
Layout table for additonal information
Responsible Party: Sanofi
ClinicalTrials.gov Identifier: NCT03510715    
Other Study ID Numbers: EFC14660
2017-002297-39 ( EudraCT Number )
U1111-1200-2046 ( Other Identifier: UTN )
First Submitted: April 18, 2018
First Posted: April 27, 2018
Results First Submitted: December 2, 2020
Results First Posted: December 29, 2020
Last Update Posted: December 29, 2020