Trial · NCT01709500
Study of Alirocumab (REGN727/SAR236553) in Patients With heFH (Heterozygous Familial Hypercholesterolemia) Who Are Not Adequately Controlled With Their LMT (Lipid-Modifying Therapy)
NCT01709500, Phase 3, Completed, Regeneron Pharmaceuticals; results first posted 2015-10-28
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Study of Alirocumab (REGN727/SAR236553) in Patients With heFH (Heterozygous Familial Hypercholesterolemia) Who Are Not Adequately Controlled With Their LMT (Lipid-Modifying Therapy). Trials, record NCT01709500. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01709500
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Study of Alirocumab (REGN727/SAR236553) in Patients With heFH (Heterozygous Familial Hypercholesterolemia) Who Are Not Adequately Controlled With Their LMT (Lipid-Modifying Therapy)
- NCT number
- NCT01709500
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 249 participants, actual
- Lead sponsor
- Regeneron Pharmaceuticals
- Conditions
- Heterozygous Familial Hypercholesterolemia
- Interventions
- LMT (atorvastatin, simvastatin, or rosuvastatin), alirocumab, Placebo
- First posted
- 2012-10-18
- Last updated
- 2015-10-28
- Results first posted
- 2015-10-28
Source ClinicalTrials.gov
Registered outcomes25
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Percent Change From Baseline in Calculated LDL-C at Week 24 - Intent--to--Treat (ITT) Analysis | From Baseline to Week 52 | Calculated LDL-C values were obtained using the Friedewald formula. Adjusted Least- squares (LS) means and standard errors at Week 24 were obtained from a mixed -effect model with repeated measures (MMRM) to account for missing data. All available post -baseline data from Week 4 to Week 52 regardless of status on- or off-treatment were used in the model. |
| Secondary | Percent Change From Baseline in Calculated LDL-C at Week 24 - On-Treatment Analysis | From Baseline to Week 52 | Calculated LDL-C values were obtained using the Friedewald formula. Adjusted LS means and standard errors at Week 24 were obtained from MMRM model including available post baseline on-treatment data from Week 4 to Week 52 (i.e. up to 21 days after last injection) (on-treatment analysis). |
| Secondary | Percent Change From Baseline in Calculated LDL-C at Week 12 - ITT Analysis | From Baseline to Week 52 | Adjusted LS means and standard errors at Week 12 from MMRM model including all available post-baseline data from Week 4 to Week 52 regardless of status on- or off-treatment (ITT analysis). |
| Secondary | Percent Change From Baseline in Calculated LDL-C at Week 12 - On- Treatment Analysis | From Baseline to Week 52 | Calculated LDL-C values were obtained using the Friedewald formula. Adjusted LS means and standard errors at Week 12 were obtained from MMRM model including available post-baseline on-treatment data from Week 4 to Week 52 (i.e. up to 21 days after last injection) (on-treatment analysis). |
| Secondary | Percent Change From Baseline in Apolipoprotein (Apo) B at Week 24 - ITT Analysis | From Baseline to Week 52 | Adjusted LS means and standard errors at Week 24 from MMRM model including all available post-baseline data from Week 4 to Week 52 regardless of status on- or off-treatment. |
| Secondary | Percent Change From Baseline in Apo B at Week 24 - On-Treatment Analysis | From Baseline to Week 52 | Adjusted LS means and standard errors at Week 24 were obtained from MMRM model including available post-baseline on-treatment data from Week 4 to Week 52 (i.e. up to 21 days after last injection). |
| Secondary | Percent Change From Baseline in Non-High -Density Lipoprotein Cholesterol (Non-HDL-C) at Week 24 - ITT Analysis | From Baseline to Week 52 | Adjusted LS means and standard errors at Week 24 from MMRM model including all available post-baseline data from Week 4 to Week 52 regardless of status on- or off-treatment. |
| Secondary | Percent Change From Baseline in Non-HDL-C at Week 24 - On-Treatment Analysis | From Baseline to Week 52 | Adjusted LS means and standard errors at Week 24 were obtained from MMRM model including available post-baseline on-treatment data from Week 4 to Week 52 (i.e. up to 21 days after last injection). |
| Secondary | Percent Change From Baseline in Total Cholesterol (Total-C) at Week 24 - ITT Analysis | From Baseline to Week 52 | Adjusted LS means and standard errors at Week 24 from MMRM model including all available post-baseline data from Week 4 to Week 52 regardless of status on- or off-treatment. |
| Secondary | Percent Change From Baseline in Apo B at Week 12 - ITT Analysis | From Baseline to Week 52 | Adjusted LS means and standard errors at Week 12 from MMRM model including all available post-baseline data from Week 4 to Week 52 regardless of status on- or off-treatment. |
1 to 10 of 25Source ClinicalTrials.gov
See all 25 registered outcomesOutcomes25
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 Calculated LDL-C at Week 24 - Intent--to--Treat (ITT) Analysis | From Baseline to Week 52 | ITT population: all randomized participants with one baseline and at least one post-baseline calculated LDL-C value on- or off-treatment. | percent change | LEAST_SQUARES_MEAN |
| SECONDARY | Percent Change From Baseline in Calculated LDL-C at Week 24 - On-Treatment Analysis | From Baseline to Week 52 | Modified ITT (mITT) population: all randomized and treated participants with one baseline and at least one post-baseline calculated LDL-C value on-treatment. | percent change | LEAST_SQUARES_MEAN |
| SECONDARY | Percent Change From Baseline in Calculated LDL-C at Week 12 - ITT Analysis | From Baseline to Week 52 | ITT population. | percent change | LEAST_SQUARES_MEAN |
| SECONDARY | Percent Change From Baseline in Calculated LDL-C at Week 12 - On- Treatment Analysis | From Baseline to Week 52 | mITT population. | percent change | LEAST_SQUARES_MEAN |
| SECONDARY | Percent Change From Baseline in Apolipoprotein (Apo) B at Week 24 - ITT Analysis | From Baseline to Week 52 | Participants analyzed: participants of the ITT population with one baseline and at least one post-baseline Apo B value on- or off-treatment. | percent change | LEAST_SQUARES_MEAN |
| SECONDARY | Percent Change From Baseline in Apo B at Week 24 - On-Treatment Analysis | From Baseline to Week 52 | mITT population | percent change | LEAST_SQUARES_MEAN |
| SECONDARY | Percent Change From Baseline in Non-High -Density Lipoprotein Cholesterol (Non-HDL-C) at Week 24 - ITT Analysis | From Baseline to Week 52 | Participants analyzed: participants of the ITT population with one baseline and at least one post-baseline non-HDL-C value on- or off-treatment. | percent change | LEAST_SQUARES_MEAN |
| SECONDARY | Percent Change From Baseline in Non-HDL-C at Week 24 - On-Treatment Analysis | From Baseline to Week 52 | mITT population | percent change | LEAST_SQUARES_MEAN |
| SECONDARY | Percent Change From Baseline in Total Cholesterol (Total-C) at Week 24 - ITT Analysis | From Baseline to Week 52 | Participants analyzed: participants of the ITT population with one baseline and at least one post-baseline total-C value on- or off-treatment. | percent change | LEAST_SQUARES_MEAN |
| SECONDARY | Percent Change From Baseline in Apo B at Week 12 - ITT Analysis | From Baseline to Week 52 | Apo B ITT population. | percent change | LEAST_SQUARES_MEAN |
1 to 10 of 25Source ClinicalTrials.gov
See all 25 outcomesResults by group50
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 Calculated LDL-C at Week 24 - Intent--to--Treat (ITT) Analysis | Alirocumab 75 mg/up to 150 mg | -48.7 | Standard Error 1.9 | percent change | |
| Percent Change From Baseline in Calculated LDL-C at Week 24 - Intent--to--Treat (ITT) Analysis | Placebo | 2.8 | Standard Error 2.8 | percent change | |
| Percent Change From Baseline in Calculated LDL-C at Week 24 - On-Treatment Analysis | Alirocumab 75 mg/up to 150 mg | -49.4 | Standard Error 1.9 | percent change | |
| Percent Change From Baseline in Calculated LDL-C at Week 24 - On-Treatment Analysis | Placebo | 2.7 | Standard Error 2.7 | percent change | |
| Percent Change From Baseline in Calculated LDL-C at Week 12 - ITT Analysis | Alirocumab 75 mg/up to 150 mg | -43.8 | Standard Error 1.8 | percent change | |
| Percent Change From Baseline in Calculated LDL-C at Week 12 - ITT Analysis | Placebo | 4.6 | Standard Error 2.6 | percent change | |
| Percent Change From Baseline in Calculated LDL-C at Week 12 - On- Treatment Analysis | Alirocumab 75 mg/up to 150 mg | -44.2 | Standard Error 1.8 | percent change | |
| Percent Change From Baseline in Calculated LDL-C at Week 12 - On- Treatment Analysis | Placebo | 4.6 | Standard Error 2.6 | percent change | |
| Percent Change From Baseline in Apolipoprotein (Apo) B at Week 24 - ITT Analysis | Alirocumab 75 mg/up to 150 mg | -42.8 | Standard Error 1.4 | percent change | |
| Percent Change From Baseline in Apolipoprotein (Apo) B at Week 24 - ITT Analysis | Placebo | -3.5 | Standard Error 2 | percent change |
1 to 10 of 50Source ClinicalTrials.gov
See all 50 results by groupParticipant flow8
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Alirocumab 75 mg/up to 150 mg | 167 | |
| Overall Study | STARTED | Placebo | 82 | |
| Overall Study | Treated | Alirocumab 75 mg/up to 150 mg | 167 | |
| Overall Study | Treated | Placebo | 81 | |
| Overall Study | COMPLETED | Alirocumab 75 mg/up to 150 mg | 0 | |
| Overall Study | COMPLETED | Placebo | 0 | |
| Overall Study | NOT COMPLETED | Alirocumab 75 mg/up to 150 mg | 167 | |
| Overall Study | NOT COMPLETED | Placebo | 82 |
Source ClinicalTrials.gov
Adverse events54
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 | Diarrhoea | Gastrointestinal disorders | Alirocumab 75 mg/up to 150 mg | 9 | 167 | |
| other | Diarrhoea | Gastrointestinal disorders | Placebo | 1 | 81 | |
| other | Injection site reaction | General disorders | Alirocumab 75 mg/up to 150 mg | 18 | 167 | |
| other | Injection site reaction | General disorders | Placebo | 6 | 81 | |
| other | Influenza | Infections and infestations | Alirocumab 75 mg/up to 150 mg | 24 | 167 | |
| other | Influenza | Infections and infestations | Placebo | 6 | 81 | |
| other | Nasopharyngitis | Infections and infestations | Alirocumab 75 mg/up to 150 mg | 18 | 167 | |
| other | Nasopharyngitis | Infections and infestations | Placebo | 16 | 81 | |
| other | Myalgia | Musculoskeletal and connective tissue disorders | Alirocumab 75 mg/up to 150 mg | 10 | 167 | |
| other | Myalgia | Musculoskeletal and connective tissue disorders | Placebo | 5 | 81 |
1 to 10 of 54Source ClinicalTrials.gov
See all 54 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Alirocumab 75 mg/up to 150 mg | ||
| Total, all-cause mortality | Placebo | ||
| Total, other adverse events | Alirocumab 75 mg/up to 150 mg | 65 | 167 |
| Total, other adverse events | Placebo | 35 | 81 |
| Total, serious adverse events | Alirocumab 75 mg/up to 150 mg | 10 | 167 |
| Total, serious adverse events | Placebo | 7 | 81 |
Source ClinicalTrials.gov
Papers7
The papers the trial lists on ClinicalTrials.gov.
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 comparisons1
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison |
|---|
| Alirocumab 75 mg/up to 150 mg vs Placebo: serious adverse events |
Source 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.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT01709500
GET https://conductscience.org/api/trials/v1/NCT01709500/registered?page=2