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| ID | Type | Description | Link |
|---|---|---|---|
| 2017-002297-39 | EudraCT Number | ||
| U1111-1200-2046 | Other Identifier | UTN |
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| Name | Class |
|---|---|
| Regeneron Pharmaceuticals | INDUSTRY |
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Primary Objective:
To evaluate the efficacy of alirocumab (75 or 150 milligrams [mg] depending on body weight [BW]), administered every 2 weeks (Q2W), on low-density lipoprotein cholesterol (LDL-C) levels at Week 12 of treatment in children and adolescents with homozygous familial hypercholesterolemia (hoFH) of 8 to 17 years of age on top of background treatments.
Secondary Objectives:
The study duration was up to 62 weeks, which included (if needed) a run-in period of up to 4 weeks, a screening period of up to 2 weeks, a treatment period of up to 48 weeks, and a follow-up of 8 weeks.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Alirocumab | Experimental | Participants with BW less than (<) 50 kilograms (kg) received subcutaneous (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 greater than or equal to [>=] 50 kg. Participants with BW >=50 kg received SC injection of alirocumab 150 mg Q2W for 48 weeks. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Alirocumab SAR236553 (REGN727) | Drug | Pharmaceutical form: solution for injection in pre-filled syringe, Route of administration: subcutaneous (SC) |
|
| Measure | Description | Time Frame |
|---|---|---|
| Percent Change From Baseline in Low-Density Lipoprotein Cholesterol (LDL-C) at Week 12: Intent-to-Treat (ITT) Analysis | 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). | Baseline to Week 12 |
| Measure | Description | Time Frame |
|---|---|---|
| Percent Change From Baseline in Low-Density Lipoprotein Cholesterol at Week 12: On-treatment Analysis | 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). | Baseline to Week 12 |
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Inclusion criteria :
Exclusion criteria:
The above information was not intended to contain all considerations relevant to a participants potential participation in a clinical trial.
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| Name | Affiliation | Role |
|---|---|---|
| Clinical Sciences & Operations | Sanofi | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Investigational Site Number 0760001 | São Paulo | 05403-000 | Brazil | |||
| Investigational Site Number 1240001 |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 36325897 | Derived | Bruckert E, Caprio S, Wiegman A, Charng MJ, Zarate-Morales CA, Baccara-Dinet MT, Manvelian G, Ourliac A, Scemama M, Daniels SR. Efficacy and Safety of Alirocumab in Children and Adolescents With Homozygous Familial Hypercholesterolemia: Phase 3, Multinational Open-Label Study. Arterioscler Thromb Vasc Biol. 2022 Dec;42(12):1447-1457. doi: 10.1161/ATVBAHA.122.317793. Epub 2022 Nov 3. |
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A total of 18 participants were enrolled and received treatment in this study.
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.
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| ID | Title | Description |
|---|---|---|
| FG000 | 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. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Sep 11, 2018 | Dec 2, 2020 |
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| Atorvastatin | Drug | Pharmaceutical form: tablet, Route of administration: oral |
|
| Simvastatin | Drug | Pharmaceutical form: tablet, Route of administration: oral |
|
| Fluvastatin | Drug | Pharmaceutical form: capsule, Route of administration: oral |
|
| Pravastatin | Drug | Pharmaceutical form: tablet, Route of administration: oral |
|
| Lovastatin | Drug | Pharmaceutical form: tablet, Route of administration: oral |
|
| Rosuvastatin | Drug | Pharmaceutical form: tablet, Route of administration: oral |
|
| Ezetimibe | Drug | Pharmaceutical form: tablet, Route of administration: oral |
|
| Cholestyramine | Drug | Pharmaceutical form: oral suspension, Route of administration: oral |
|
| Nicotinic acid | Drug | Pharmaceutical form: tablet, Route of administration: oral |
|
| Fenofibrate | Drug | Pharmaceutical form: tablet, Route of administration: oral |
|
| Omega-3 fatty acids | Drug | Pharmaceutical form: capsule, Route of administration: oral |
|
| Percent Change From Baseline in Low-Density Lipoprotein Cholesterol at Weeks 24 and 48: ITT Analysis/On-treatment Analysis |
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. |
| Baseline to Weeks 24 and 48 |
| Percent Change From Baseline in Apolipoprotein (Apo) B at Weeks 12, 24 and 48: ITT Analysis/On-treatment Analysis | 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. | Baseline to Weeks 12, 24 and 48 |
| Percent Change From Baseline in Non-High Density Lipoprotein Cholesterol (Non-HDL-C) at Weeks 12, 24 and 48 - ITT Analysis/On-treatment Analysis | 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. | Baseline to Weeks 12, 24 and 48 |
| Percent Change From Baseline in Total Cholesterol (Total-C) at Weeks 12, 24 and 48: ITT Analysis/On-treatment Analysis | 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. | Baseline to Weeks 12, 24 and 48 |
| Percent Change From Baseline in Lipoprotein a (Lp) (a) at Weeks 12, 24 and 48: ITT Analysis/On-treatment Analysis | 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. | Baseline to Weeks 12, 24 and 48 |
| Percent Change From Baseline in High Density Lipoprotein Cholesterol (HDL-C) at Weeks 12, 24 and 48: ITT Analysis/On-treatment Analysis | 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. | Baseline to Weeks 12, 24 and 48 |
| Percent Change From Baseline in Fasting Triglycerides (TG) at Weeks 12, 24 and 48: ITT Analysis/On-treatment Analysis | 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. | Baseline to Weeks 12, 24 and 48 |
| Percent Change From Baseline in Apolipoprotein A1 (Apo A1) at Weeks 12, 24 and 48: ITT Analysis/On-treatment Analysis | 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. | Baseline to Weeks 12, 24 and 48 |
| Percentage of Participants Reporting >=15 Percent (%) Reduction in LDL-C Level at Weeks 12, 24 and 48: ITT Analysis/On-treatment Analysis | 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. | Baseline to Weeks 12, 24 and 48 |
| Absolute Change From Baseline in LDL-C Level at Weeks 12, 24 and 48: ITT Analysis/On-treatment Analysis | 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. | Baseline to Weeks 12, 24 and 48 |
| Number of Participants With Tanner Staging at Baseline, Weeks 12, 24 and 48 | 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). | Baseline, Weeks 12, 24 and 48 |
| Québec |
| G1V 4W2 |
| Canada |
| Investigational Site Number 2080001 | Viborg | 8800 | Denmark |
| Investigational Site Number 4840006 | Oaxaca City | 68000 | Mexico |
| Investigational Site Number 5280001 | Amsterdam | 1105AZ | Netherlands |
| Investigational Site Number 6430002 | Kemerovo | 650002 | Russia |
| Investigational Site Number 7050001 | Ljubljana | 1000 | Slovenia |
| Investigational Site Number 7240001 | A Coruña | 15001 | Spain |
| Investigational Site Number 1580001 | Taipei | 112 | Taiwan |
| Investigational Site Number 7920001 | Izmir | 35040 | Turkey (Türkiye) |
| FG001 |
| Alirocumab 150 mg Q2W |
Participants with BW >=50 kg received SC injection of alirocumab 150 mg Q2W for 48 weeks. |
| COMPLETED |
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| NOT COMPLETED |
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|
Analysis was performed on all enrolled participants.
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| ID | Title | Description |
|---|---|---|
| BG000 | Alirocumab 75 mg Q2W/up to 150 mg Q2W | 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. |
| BG001 | Alirocumab 150 mg Q2W | Participants with BW >=50 kg received SC injection of alirocumab 150 mg Q2W for 48 weeks. |
| BG002 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | years |
| |||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||
| Race (NIH/OMB) | Count of Participants | Participants |
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| Low-Density Lipoprotein Cholesterol (LDL-C) | Mean | Standard Deviation | milligrams per deciliter (mg/dL) |
|
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Percent Change From Baseline in Low-Density Lipoprotein Cholesterol (LDL-C) at Week 12: Intent-to-Treat (ITT) Analysis | 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). | 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). | Posted | Least Squares Mean | Standard Error | percent change | Baseline to Week 12 |
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| Secondary | Percent Change From Baseline in Low-Density Lipoprotein Cholesterol at Week 12: On-treatment Analysis | 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). | 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). | Posted | Least Squares Mean | Standard Error | percent change | Baseline to Week 12 |
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| Secondary | Percent Change From Baseline in Low-Density Lipoprotein Cholesterol at Weeks 24 and 48: ITT Analysis/On-treatment Analysis | 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. | 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). | Posted | Least Squares Mean | Standard Error | percent change | Baseline to Weeks 24 and 48 |
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| Secondary | Percent Change From Baseline in Apolipoprotein (Apo) B at Weeks 12, 24 and 48: ITT Analysis/On-treatment Analysis | 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. | 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). | Posted | Least Squares Mean | Standard Error | percent change | Baseline to Weeks 12, 24 and 48 |
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| Secondary | Percent Change From Baseline in Non-High Density Lipoprotein Cholesterol (Non-HDL-C) at Weeks 12, 24 and 48 - ITT Analysis/On-treatment Analysis | 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. | 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). | Posted | Least Squares Mean | Standard Error | percent change | Baseline to Weeks 12, 24 and 48 |
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| Secondary | Percent Change From Baseline in Total Cholesterol (Total-C) at Weeks 12, 24 and 48: ITT Analysis/On-treatment Analysis | 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. | 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). | Posted | Least Squares Mean | Standard Error | percent change | Baseline to Weeks 12, 24 and 48 |
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| Secondary | Percent Change From Baseline in Lipoprotein a (Lp) (a) at Weeks 12, 24 and 48: ITT Analysis/On-treatment Analysis | 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. | 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). | Posted | Mean | Standard Error | percent change | Baseline to Weeks 12, 24 and 48 |
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| Secondary | Percent Change From Baseline in High Density Lipoprotein Cholesterol (HDL-C) at Weeks 12, 24 and 48: ITT Analysis/On-treatment Analysis | 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. | 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). | Posted | Least Squares Mean | Standard Error | percent change | Baseline to Weeks 12, 24 and 48 |
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| Secondary | Percent Change From Baseline in Fasting Triglycerides (TG) at Weeks 12, 24 and 48: ITT Analysis/On-treatment Analysis | 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. | 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). | Posted | Mean | Standard Error | percent change | Baseline to Weeks 12, 24 and 48 |
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| Secondary | Percent Change From Baseline in Apolipoprotein A1 (Apo A1) at Weeks 12, 24 and 48: ITT Analysis/On-treatment Analysis | 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. | 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). | Posted | Least Squares Mean | Standard Error | percent change | Baseline to Weeks 12, 24 and 48 |
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| Secondary | Percentage of Participants Reporting >=15 Percent (%) Reduction in LDL-C Level at Weeks 12, 24 and 48: ITT Analysis/On-treatment Analysis | 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. | 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). | Posted | Number | 95% Confidence Interval | percentage of participants | Baseline to Weeks 12, 24 and 48 |
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| Secondary | Absolute Change From Baseline in LDL-C Level at Weeks 12, 24 and 48: ITT Analysis/On-treatment Analysis | 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. | 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). | Posted | Least Squares Mean | Standard Error | mg/dL | Baseline to Weeks 12, 24 and 48 |
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| Secondary | Number of Participants With Tanner Staging at Baseline, Weeks 12, 24 and 48 | 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). | 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. | Posted | Count of Participants | Participants | Baseline, Weeks 12, 24 and 48 |
|
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.
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Alirocumab 75 mg Q2W/up to 150 mg Q2W | 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. | 1 | 9 | 1 | 9 | 8 | 9 |
| EG001 | Alirocumab 150 mg Q2W | Participants with BW >=50 kg received SC injection of alirocumab 150 mg Q2W for 48 weeks. | 0 | 9 | 0 | 9 | 9 | 9 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Cardiac Failure | Cardiac disorders | MedDRA 22.1 | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Splenomegaly | Blood and lymphatic system disorders | MedDRA 22.1 | Systematic Assessment |
| |
| Aortic Valve Incompetence | Cardiac disorders | MedDRA 22.1 | Systematic Assessment |
| |
| Bicuspid Aortic Valve | Congenital, familial and genetic disorders | MedDRA 22.1 | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA 22.1 | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA 22.1 | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA 22.1 | Systematic Assessment |
| |
| Fatigue | General disorders | MedDRA 22.1 | Systematic Assessment |
| |
| Influenza Like Illness | General disorders | MedDRA 22.1 | Systematic Assessment |
| |
| Injection Site Pain | General disorders | MedDRA 22.1 | Systematic Assessment |
| |
| Injection Site Reaction | General disorders | MedDRA 22.1 | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA 22.1 | Systematic Assessment |
| |
| Hepatomegaly | Hepatobiliary disorders | MedDRA 22.1 | Systematic Assessment |
| |
| Gastroenteritis | Infections and infestations | MedDRA 22.1 | Systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA 22.1 | Systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA 22.1 | Systematic Assessment |
| |
| Parvovirus B19 Infection | Infections and infestations | MedDRA 22.1 | Systematic Assessment |
| |
| Pharyngotonsillitis | Infections and infestations | MedDRA 22.1 | Systematic Assessment |
| |
| Upper Respiratory Tract Infection | Infections and infestations | MedDRA 22.1 | Systematic Assessment |
| |
| Urinary Tract Infection | Infections and infestations | MedDRA 22.1 | Systematic Assessment |
| |
| Thermal Burn | Injury, poisoning and procedural complications | MedDRA 22.1 | Systematic Assessment |
| |
| Alanine Aminotransferase Increased | Investigations | MedDRA 22.1 | Systematic Assessment |
| |
| Hypertriglyceridaemia | Metabolism and nutrition disorders | MedDRA 22.1 | Systematic Assessment |
| |
| Pain In Extremity | Musculoskeletal and connective tissue disorders | MedDRA 22.1 | Systematic Assessment |
| |
| Tendon Pain | Musculoskeletal and connective tissue disorders | MedDRA 22.1 | Systematic Assessment |
| |
| Tendonitis | Musculoskeletal and connective tissue disorders | MedDRA 22.1 | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA 22.1 | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 22.1 | Systematic Assessment |
| |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA 22.1 | Systematic Assessment |
| |
| Dyspnoea Paroxysmal Nocturnal | Respiratory, thoracic and mediastinal disorders | MedDRA 22.1 | Systematic Assessment |
| |
| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA 22.1 | Systematic Assessment |
| |
| Dry Skin | Skin and subcutaneous tissue disorders | MedDRA 22.1 | Systematic Assessment |
| |
| Rash | Skin and subcutaneous tissue disorders | MedDRA 22.1 | Systematic Assessment |
|
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.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Trial Transparency Team | Sanofi | 800-633-1610 | 1# | Contact-US@sanofi.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Jan 27, 2020 | Dec 2, 2020 | SAP_001.pdf |
| ID | Term |
|---|---|
| D006937 | Hypercholesterolemia |
| D000090542 | Homozygous Familial Hypercholesterolemia |
| ID | Term |
|---|---|
| D006949 | Hyperlipidemias |
| D050171 | Dyslipidemias |
| D052439 | Lipid Metabolism Disorders |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
| D006938 | Hyperlipoproteinemia Type II |
| D008052 | Lipid Metabolism, Inborn Errors |
| D008661 | Metabolism, Inborn Errors |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D006951 | Hyperlipoproteinemias |
Not provided
Not provided
| ID | Term |
|---|---|
| C571059 | alirocumab |
| D000069059 | Atorvastatin |
| D019821 | Simvastatin |
| D000077340 | Fluvastatin |
| D017035 | Pravastatin |
| D008148 | Lovastatin |
| D000068718 | Rosuvastatin Calcium |
| D000069438 | Ezetimibe |
| D002792 | Cholestyramine Resin |
| D009525 | Niacin |
| D011345 | Fenofibrate |
| D015525 | Fatty Acids, Omega-3 |
| ID | Term |
|---|---|
| D011758 | Pyrroles |
| D001393 | Azoles |
| D006573 | Heterocyclic Compounds, 1-Ring |
| D006571 | Heterocyclic Compounds |
| D006538 | Heptanoic Acids |
| D005227 | Fatty Acids |
| D008055 | Lipids |
| D009281 | Naphthalenes |
| D011084 | Polycyclic Aromatic Hydrocarbons |
| D006841 | Hydrocarbons, Aromatic |
| D006844 | Hydrocarbons, Cyclic |
| D006838 | Hydrocarbons |
| D009930 | Organic Chemicals |
| D011083 | Polycyclic Compounds |
| D007211 | Indoles |
| D006574 | Heterocyclic Compounds, 2-Ring |
| D000072471 | Heterocyclic Compounds, Fused-Ring |
| D013449 | Sulfonamides |
| D000577 | Amides |
| D005464 | Fluorobenzenes |
| D006845 | Hydrocarbons, Fluorinated |
| D006846 | Hydrocarbons, Halogenated |
| D013450 | Sulfones |
| D013457 | Sulfur Compounds |
| D011743 | Pyrimidines |
| D001384 | Azetidines |
| D001385 | Azetines |
| D011137 | Polystyrenes |
| D010969 | Plastics |
| D011108 | Polymers |
| D046911 | Macromolecular Substances |
| D001697 | Biomedical and Dental Materials |
| D008420 | Manufactured Materials |
| D013676 | Technology, Industry, and Agriculture |
| D009539 | Nicotinic Acids |
| D000147 | Acids, Heterocyclic |
| D011725 | Pyridines |
| D058607 | Fibric Acids |
| D058610 | Isobutyrates |
| D002087 | Butyrates |
| D000144 | Acids, Acyclic |
| D002264 | Carboxylic Acids |
| D010647 | Phenyl Ethers |
| D004987 | Ethers |
| D001577 | Benzophenones |
| D001555 | Benzene Derivatives |
| D010636 | Phenols |
| D007659 | Ketones |
| D004042 | Dietary Fats, Unsaturated |
| D004041 | Dietary Fats |
| D005223 | Fats |
| D005231 | Fatty Acids, Unsaturated |
| D005395 | Fish Oils |
| D009821 | Oils |
Not provided
Not provided
| Male |
|
| Asian |
|
| Native Hawaiian or Other Pacific Islander |
|
| Black or African American |
|
| White |
|
| More than one race |
|
| Unknown or Not Reported |
|
|
|
|
|
|
|
|
|
|
|
|
| Units | Counts |
|---|---|
| Participants |
|
|