Trial · NCT03510715
An Efficacy and Safety Study of Alirocumab in Children and Adolescents With Homozygous Familial Hypercholesterolemia
NCT03510715, Phase 3, Completed, Sanofi; results first posted 2020-12-29
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. An Efficacy and Safety Study of Alirocumab in Children and Adolescents With Homozygous Familial Hypercholesterolemia. Trials, record NCT03510715. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03510715
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- An Efficacy and Safety Study of Alirocumab in Children and Adolescents With Homozygous Familial Hypercholesterolemia
- NCT number
- NCT03510715
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 18 participants, actual
- Lead sponsor
- Sanofi
- Conditions
- Hypercholesterolemia
- Interventions
- Alirocumab SAR236553 (REGN727), Atorvastatin, Simvastatin, Fluvastatin, Pravastatin, Lovastatin, Rosuvastatin, Ezetimibe, Cholestyramine, Nicotinic acid, Fenofibrate, Omega-3 fatty acids
- First posted
- 2018-04-27
- Last updated
- 2020-12-29
- Other registries' IDs
- eudract:2017-002297-39
- Results first posted
- 2020-12-29
Source ClinicalTrials.gov
Registered outcomes13
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Percent Change From Baseline in Low-Density Lipoprotein Cholesterol (LDL-C) at Week 12: Intent-to-Treat (ITT) Analysis | Baseline to Week 12 | 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). |
| Secondary | Percent Change From Baseline in Low-Density Lipoprotein Cholesterol at Week 12: On-treatment Analysis | Baseline to Week 12 | 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). |
| Secondary | Percent Change From Baseline in Low-Density Lipoprotein Cholesterol at Weeks 24 and 48: ITT Analysis/On-treatment Analysis | Baseline to Weeks 24 and 48 | 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. |
| Secondary | Percent Change From Baseline in Apolipoprotein (Apo) B at Weeks 12, 24 and 48: ITT Analysis/On-treatment Analysis | Baseline to Weeks 12, 24 and 48 | 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. |
| 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 | Baseline to Weeks 12, 24 and 48 | 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. |
| Secondary | Percent Change From Baseline in Total Cholesterol (Total-C) at Weeks 12, 24 and 48: ITT Analysis/On-treatment Analysis | Baseline to Weeks 12, 24 and 48 | 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. |
| Secondary | Percent Change From Baseline in Lipoprotein a (Lp) (a) at Weeks 12, 24 and 48: ITT Analysis/On-treatment Analysis | Baseline to Weeks 12, 24 and 48 | 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. |
| Secondary | Percent Change From Baseline in High Density Lipoprotein Cholesterol (HDL-C) at Weeks 12, 24 and 48: ITT Analysis/On-treatment Analysis | Baseline to Weeks 12, 24 and 48 | 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. |
| Secondary | Percent Change From Baseline in Fasting Triglycerides (TG) at Weeks 12, 24 and 48: ITT Analysis/On-treatment Analysis | Baseline to Weeks 12, 24 and 48 | 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. |
| Secondary | Percent Change From Baseline in Apolipoprotein A1 (Apo A1) at Weeks 12, 24 and 48: ITT Analysis/On-treatment Analysis | Baseline to Weeks 12, 24 and 48 | 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. |
1 to 10 of 13Source ClinicalTrials.gov
See all 13 registered outcomesOutcomes13
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Percent Change From Baseline in Low-Density Lipoprotein Cholesterol (LDL-C) at Week 12: Intent-to-Treat (ITT) Analysis | Baseline to Week 12 | 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). | percent change | LEAST_SQUARES_MEAN |
| SECONDARY | Percent Change From Baseline in Low-Density Lipoprotein Cholesterol at Week 12: On-treatment Analysis | Baseline to Week 12 | 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). | percent change | LEAST_SQUARES_MEAN |
| SECONDARY | Percent Change From Baseline in Low-Density Lipoprotein Cholesterol at Weeks 24 and 48: ITT Analysis/On-treatment Analysis | Baseline to Weeks 24 and 48 | 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). | percent change | LEAST_SQUARES_MEAN |
| SECONDARY | Percent Change From Baseline in Apolipoprotein (Apo) B at Weeks 12, 24 and 48: ITT Analysis/On-treatment Analysis | Baseline to Weeks 12, 24 and 48 | 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). | percent change | LEAST_SQUARES_MEAN |
| 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 | Baseline to Weeks 12, 24 and 48 | 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). | percent change | LEAST_SQUARES_MEAN |
| SECONDARY | Percent Change From Baseline in Total Cholesterol (Total-C) at Weeks 12, 24 and 48: ITT Analysis/On-treatment Analysis | Baseline to Weeks 12, 24 and 48 | 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). | percent change | LEAST_SQUARES_MEAN |
| SECONDARY | Percent Change From Baseline in Lipoprotein a (Lp) (a) at Weeks 12, 24 and 48: ITT Analysis/On-treatment Analysis | Baseline to Weeks 12, 24 and 48 | 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). | percent change | MEAN |
| SECONDARY | Percent Change From Baseline in High Density Lipoprotein Cholesterol (HDL-C) at Weeks 12, 24 and 48: ITT Analysis/On-treatment Analysis | Baseline to Weeks 12, 24 and 48 | 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). | percent change | LEAST_SQUARES_MEAN |
| SECONDARY | Percent Change From Baseline in Fasting Triglycerides (TG) at Weeks 12, 24 and 48: ITT Analysis/On-treatment Analysis | Baseline to Weeks 12, 24 and 48 | 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). | percent change | MEAN |
| SECONDARY | Percent Change From Baseline in Apolipoprotein A1 (Apo A1) at Weeks 12, 24 and 48: ITT Analysis/On-treatment Analysis | Baseline to Weeks 12, 24 and 48 | 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). | percent change | LEAST_SQUARES_MEAN |
1 to 10 of 13Source ClinicalTrials.gov
See all 13 outcomesResults by group55
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Percent Change From Baseline in Low-Density Lipoprotein Cholesterol (LDL-C) at Week 12: Intent-to-Treat (ITT) Analysis | Alirocumab | -4.1 | Standard Error 9.0 | percent change | |
| Percent Change From Baseline in Low-Density Lipoprotein Cholesterol at Week 12: On-treatment Analysis | Alirocumab | -4.1 | Standard Error 9.0 | percent change | |
| Percent Change From Baseline in Low-Density Lipoprotein Cholesterol at Weeks 24 and 48: ITT Analysis/On-treatment Analysis | Alirocumab | Week 24 | -10.1 | Standard Error 7.6 | percent change |
| Percent Change From Baseline in Low-Density Lipoprotein Cholesterol at Weeks 24 and 48: ITT Analysis/On-treatment Analysis | Alirocumab | Week 48 | 4.2 | Standard Error 12.8 | percent change |
| Percent Change From Baseline in Apolipoprotein (Apo) B at Weeks 12, 24 and 48: ITT Analysis/On-treatment Analysis | Alirocumab | Week 12 | -4.2 | Standard Error 6.8 | percent change |
| Percent Change From Baseline in Apolipoprotein (Apo) B at Weeks 12, 24 and 48: ITT Analysis/On-treatment Analysis | Alirocumab | Week 24 | -11.8 | Standard Error 6.1 | percent change |
| Percent Change From Baseline in Apolipoprotein (Apo) B at Weeks 12, 24 and 48: ITT Analysis/On-treatment Analysis | Alirocumab | Week 48 | 0.9 | Standard Error 10.4 | percent change |
| Percent Change From Baseline in Non-High Density Lipoprotein Cholesterol (Non-HDL-C) at Weeks 12, 24 and 48 - ITT Analysis/On-treatment Analysis | Alirocumab | Week 12 | -3.9 | Standard Error 8.3 | percent change |
| Percent Change From Baseline in Non-High Density Lipoprotein Cholesterol (Non-HDL-C) at Weeks 12, 24 and 48 - ITT Analysis/On-treatment Analysis | Alirocumab | Week 24 | -9.2 | Standard Error 7.3 | percent change |
| Percent Change From Baseline in Non-High Density Lipoprotein Cholesterol (Non-HDL-C) at Weeks 12, 24 and 48 - ITT Analysis/On-treatment Analysis | Alirocumab | Week 48 | 5.7 | Standard Error 13.1 | percent change |
1 to 10 of 55Source ClinicalTrials.gov
See all 55 results by groupParticipant flow6
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Alirocumab 75 mg Q2W/up to 150 mg Q2W | 9 | |
| Overall Study | STARTED | Alirocumab 150 mg Q2W | 9 | |
| Overall Study | COMPLETED | Alirocumab 75 mg Q2W/up to 150 mg Q2W | 8 | |
| Overall Study | COMPLETED | Alirocumab 150 mg Q2W | 9 | |
| Overall Study | NOT COMPLETED | Alirocumab 75 mg Q2W/up to 150 mg Q2W | 1 | |
| Overall Study | NOT COMPLETED | Alirocumab 150 mg Q2W | 0 |
Source ClinicalTrials.gov
Adverse events66
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Splenomegaly | Blood and lymphatic system disorders | Alirocumab 75 mg Q2W/up to 150 mg Q2W | 1 | 9 | 1 |
| other | Splenomegaly | Blood and lymphatic system disorders | Alirocumab 150 mg Q2W | 0 | 9 | 0 |
| other | Aortic Valve Incompetence | Cardiac disorders | Alirocumab 75 mg Q2W/up to 150 mg Q2W | 2 | 9 | 2 |
| other | Aortic Valve Incompetence | Cardiac disorders | Alirocumab 150 mg Q2W | 1 | 9 | 1 |
| other | Bicuspid Aortic Valve | Congenital, familial and genetic disorders | Alirocumab 75 mg Q2W/up to 150 mg Q2W | 0 | 9 | 0 |
| other | Bicuspid Aortic Valve | Congenital, familial and genetic disorders | Alirocumab 150 mg Q2W | 1 | 9 | 1 |
| other | Diarrhoea | Gastrointestinal disorders | Alirocumab 75 mg Q2W/up to 150 mg Q2W | 0 | 9 | 0 |
| other | Diarrhoea | Gastrointestinal disorders | Alirocumab 150 mg Q2W | 1 | 9 | 1 |
| other | Nausea | Gastrointestinal disorders | Alirocumab 75 mg Q2W/up to 150 mg Q2W | 1 | 9 | 1 |
| other | Nausea | Gastrointestinal disorders | Alirocumab 150 mg Q2W | 0 | 9 | 0 |
1 to 10 of 66Source ClinicalTrials.gov
See all 66 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Alirocumab 75 mg Q2W/up to 150 mg Q2W | 1 | 9 |
| Total, all-cause mortality | Alirocumab 150 mg Q2W | 0 | 9 |
| Total, other adverse events | Alirocumab 75 mg Q2W/up to 150 mg Q2W | 8 | 9 |
| Total, other adverse events | Alirocumab 150 mg Q2W | 9 | 9 |
| Total, serious adverse events | Alirocumab 75 mg Q2W/up to 150 mg Q2W | 1 | 9 |
| Total, serious adverse events | Alirocumab 150 mg Q2W | 0 | 9 |
Source ClinicalTrials.gov
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 36325897 | DERIVED |
Source PubMed
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons2
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| Alirocumab 75 mg Q2W/up to 150 mg Q2W vs Alirocumab 150 mg Q2W: deaths from any cause | Trial comparisons |
| Alirocumab 75 mg Q2W/up to 150 mg Q2W vs Alirocumab 150 mg Q2W: serious adverse events | Trial comparisons |
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
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