Trial · NCT01576484
Open-Label Extension of Study R727-CL-1003 (NCT01266876) to Evaluate the Long-Term Safety and Efficacy of Alirocumab (REGN727) in Participants With Heterozygous Familial Hypercholesterolemia (HeFH)
NCT01576484, Phase 2, Completed, Regeneron Pharmaceuticals; results first posted 2020-08-05
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Open-Label Extension of Study R727-CL-1003 (NCT01266876) to Evaluate the Long-Term Safety and Efficacy of Alirocumab (REGN727) in Participants With Heterozygous Familial Hypercholesterolemia (HeFH). Trials, record NCT01576484. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01576484
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Open-Label Extension of Study R727-CL-1003 (NCT01266876) to Evaluate the Long-Term Safety and Efficacy of Alirocumab (REGN727) in Participants With Heterozygous Familial Hypercholesterolemia (HeFH)
- NCT number
- NCT01576484
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 58 participants, actual
- Lead sponsor
- Regeneron Pharmaceuticals
- Conditions
- Hypercholesterolemia, Heterozygous Familial Hypercholesterolemia
- Interventions
- Alirocumab, Placebo Matched to Alirocumab
- First posted
- 2012-04-12
- Last updated
- 2020-08-05
- Results first posted
- 2020-08-05
Source ClinicalTrials.gov
Registered outcomes20
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Number of Participants Who Experienced Treatment Emergent Adverse Events (TEAEs), Serious TEAEs and TEAEs Leading to Death | Baseline (Day 1 of current study) to end of study (Week 218) | An AE was defined as any untoward medical occurrence in a participant administered a study drug which may or may not have a causal relationship with the study drug. Treatment- emergent adverse events (TEAEs) were defined as AEs that developed or worsened or became serious during on- treatment period (time from the first dose of study drug to the last dose of study drug plus 70 days). A serious adverse event (SAE) was defined as any untoward medical occurrence that resulted in any of the following outcomes: death, life- threatening, required initial or prolonged in-patient hospitalization, persistent or significant disability/incapacity, congenital anomaly/birth defect, or considered as medically important event. Any TEAE included participants with both serious and non-serious AEs. |
| Secondary | Percent Change in Low-density Lipoprotein Cholesterol (LDL-C) From Baseline in the Current Study to Week 24 | Baseline (current study) to Week 24 | Percent change for serum LDL-C (Low-density lipoprotein cholesterol) from baseline to Week 24 during the efficacy treatment period, which is defined as the time from the first study drug injection up to 21 days after the last study drug injection in the current study. |
| Secondary | Percent Change in Low Density Lipoprotein Cholesterol (LDL-C) From Baseline in Current Study to Week 12 | Baseline (current study) up to Week 12 | Percent change for serum LDL-C (Low-density lipoprotein cholesterol) from baseline to Week 12 during the efficacy treatment period, which is defined as the time from the first study drug injection up to 21 days after the last study drug injection in the current study. |
| Secondary | Percent Change in Apolipoprotein (Apo) B, Non-High Density Lipoprotein Cholesterol (HDL-C), and Total Cholesterol From Baseline in Current Study to Week 24 | Baseline(current study) up to Week 24 | Percent change for Apo B, Non-HDL-C and Total Cholesterol from baseline to Week 24 during the efficacy treatment period, which is defined as the time from the first study drug injection up to 21 days after the last study drug injection in the current study. |
| Secondary | Percent Change in Apolipoprotein (Apo) B, Non-High Density Lipoprotein Cholesterol (HDL-C), and Total Cholesterol From Baseline in Current Study to Week 12 | Baseline (current study) up to Week 12 | Percent change for serum Apo B, Non-HDL-C, and Total Cholesterol from baseline to Week 12 during the efficacy treatment period, which is defined as the time from the first study drug injection up to 21 days after the last study drug injection in the current study. |
| Secondary | Percent Change in Low Density Lipoprotein Cholesterol (LDL-C) From Baseline in Current Study to Week 52 | Baseline (current study) up to Week 52 | Percent change for serum LDL-C from baseline to Week 52 during the efficacy treatment period, which is defined as the time from the first study drug injection up to 21 days after the last study drug injection in the current study. |
| Secondary | Percentage of Participants With Low Density Lipoprotein Cholesterol (LDL-C) Less Than (<) 70 Milligrams Per Deciliter (mg/dL) for Prior Myocardial Infarction (MI)/Stroke, or <100 mg/dL [2.59 mmol/L] for Participants Without Prior MI/Stroke at Week 24 | At Week 24 | Percentage of participants reaching LDL-C goal (ie, LDL-C \<70 mg/dL (1.81 millimoles per liter \[mmol/L\]) in case of prior MI/stroke, or \<100 mg/dL \[2.59 mmol/L\] for participants without prior MI/stroke) at week 24 during the efficacy treatment period, which is defined as the time from the first study drug injection up to 21 days after the last study drug injection in the current study, were reported. |
| Secondary | Percent Change in Lipoprotein a (Lp[a]) at Week 24 | At Week 24 | Percent change in serum lipoprotein a at week 24 during the efficacy treatment period, which is defined as the time from the first study drug injection up to 21 days after the last study drug injection in the current study, was reported. |
| Secondary | Percent Change in High Density Lipoprotein Cholesterol (HDL-C) at Week 24 and Week 12 | At Week 24 and 12 | Percent change for serum High Density Lipoprotein Cholesterol (HDL-C) in the current study at weeks 24 and week 12, during the efficacy treatment period, which is defined as the time from the first study drug injection up to 21 days after the last study drug injection in the current study, was reported. |
| Secondary | Percent Change in Lipoprotein a at Week 12 | At Week 12 | Percent change for serum Lipoprotein a at Week 12 during the efficacy treatment period, which is defined as the time from the first study drug injection up to 21 days after the last study drug injection in the current study, was reported. |
1 to 10 of 20Source ClinicalTrials.gov
See all 20 registered outcomesOutcomes20
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Number of Participants Who Experienced Treatment Emergent Adverse Events (TEAEs), Serious TEAEs and TEAEs Leading to Death | Baseline (Day 1 of current study) to end of study (Week 218) | Safety Analysis Set (SAF) included all participants who received at least 1 dose or part of a dose of alirocumab. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Percent Change in Low-density Lipoprotein Cholesterol (LDL-C) From Baseline in the Current Study to Week 24 | Baseline (current study) to Week 24 | SAF included all participants who received at least 1 dose or part of a dose of alirocumab. Here, overall number of participants analyzed signifies those participants who were evaluable for this outcome measure. | percent change | MEAN |
| SECONDARY | Percent Change in Low Density Lipoprotein Cholesterol (LDL-C) From Baseline in Current Study to Week 12 | Baseline (current study) up to Week 12 | SAF included all participants who received at least 1 dose or part of a dose of alirocumab. Here, overall number of participants analyzed signifies those participants who were evaluable for this outcome measure. | percent change | MEAN |
| SECONDARY | Percent Change in Apolipoprotein (Apo) B, Non-High Density Lipoprotein Cholesterol (HDL-C), and Total Cholesterol From Baseline in Current Study to Week 24 | Baseline(current study) up to Week 24 | SAF included all participants who received at least 1 dose or part of a dose of alirocumab. Here Number analyzed signifies those participants who were evaluable for this outcome measure at specified category. | percent change | MEAN |
| SECONDARY | Percent Change in Apolipoprotein (Apo) B, Non-High Density Lipoprotein Cholesterol (HDL-C), and Total Cholesterol From Baseline in Current Study to Week 12 | Baseline (current study) up to Week 12 | SAF included all participants who received at least 1 dose or part of a dose of alirocumab. Here Number analyzed signifies those participants who were evaluable for this outcome measure at specified category. | percent change | MEAN |
| SECONDARY | Percent Change in Low Density Lipoprotein Cholesterol (LDL-C) From Baseline in Current Study to Week 52 | Baseline (current study) up to Week 52 | SAF included all participants who received at least 1 dose or part of a dose of alirocumab. Here, overall number of participants analyzed signifies those participants who were evaluable for this outcome measure. | percent change | MEAN |
| SECONDARY | Percentage of Participants With Low Density Lipoprotein Cholesterol (LDL-C) Less Than (<) 70 Milligrams Per Deciliter (mg/dL) for Prior Myocardial Infarction (MI)/Stroke, or <100 mg/dL [2.59 mmol/L] for Participants Without Prior MI/Stroke at Week 24 | At Week 24 | SAF included all participants who received at least 1 dose or part of a dose of alirocumab. Here, overall number of participants analyzed signifies those participants who were evaluable for this outcome measure. | Percentage of Participants | NUMBER |
| SECONDARY | Percent Change in Lipoprotein a (Lp[a]) at Week 24 | At Week 24 | SAF included all participants who received at least 1 dose or part of a dose of alirocumab. Here, overall number of participants analyzed signifies those participants who were evaluable for this outcome measure. | percent change | MEAN |
| SECONDARY | Percent Change in High Density Lipoprotein Cholesterol (HDL-C) at Week 24 and Week 12 | At Week 24 and 12 | SAF included all participants who received at least 1 dose or part of a dose of alirocumab. Here Number analyzed signifies those participants who were evaluable for this outcome measure at specified category. | percent change | MEAN |
| SECONDARY | Percent Change in Lipoprotein a at Week 12 | At Week 12 | SAF included all participants who received at least 1 dose or part of a dose of alirocumab. Here, overall number of participants analyzed signifies those participants who were evaluable for this outcome measure. | percent change | MEAN |
1 to 10 of 20Source ClinicalTrials.gov
See all 20 outcomesResults by group177
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Number of Participants Who Experienced Treatment Emergent Adverse Events (TEAEs), Serious TEAEs and TEAEs Leading to Death | Placebo Participants in Parent Study | Adverse Events | 12 | Participants | |
| Number of Participants Who Experienced Treatment Emergent Adverse Events (TEAEs), Serious TEAEs and TEAEs Leading to Death | Participants Previously Exposed to Alirocumab in Parent Study | Adverse Events | 42 | Participants | |
| Number of Participants Who Experienced Treatment Emergent Adverse Events (TEAEs), Serious TEAEs and TEAEs Leading to Death | All Participants | Adverse Events | 54 | Participants | |
| Number of Participants Who Experienced Treatment Emergent Adverse Events (TEAEs), Serious TEAEs and TEAEs Leading to Death | Placebo Participants in Parent Study | Serious Adverse Events | 4 | Participants | |
| Number of Participants Who Experienced Treatment Emergent Adverse Events (TEAEs), Serious TEAEs and TEAEs Leading to Death | Participants Previously Exposed to Alirocumab in Parent Study | Serious Adverse Events | 8 | Participants | |
| Number of Participants Who Experienced Treatment Emergent Adverse Events (TEAEs), Serious TEAEs and TEAEs Leading to Death | All Participants | Serious Adverse Events | 12 | Participants | |
| Number of Participants Who Experienced Treatment Emergent Adverse Events (TEAEs), Serious TEAEs and TEAEs Leading to Death | Placebo Participants in Parent Study | Adverse Events Leading to Death | 0 | Participants | |
| Number of Participants Who Experienced Treatment Emergent Adverse Events (TEAEs), Serious TEAEs and TEAEs Leading to Death | Participants Previously Exposed to Alirocumab in Parent Study | Adverse Events Leading to Death | 0 | Participants | |
| Number of Participants Who Experienced Treatment Emergent Adverse Events (TEAEs), Serious TEAEs and TEAEs Leading to Death | All Participants | Adverse Events Leading to Death | 0 | Participants | |
| Percent Change in Low-density Lipoprotein Cholesterol (LDL-C) From Baseline in the Current Study to Week 24 | Placebo Participants in Parent Study | -73.15 | Standard Deviation 15.01 | percent change |
1 to 10 of 177Source ClinicalTrials.gov
See all 177 results by groupParticipant flow6
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Placebo Participants in Parent Study | 12 | |
| Overall Study | STARTED | Participants Previously Exposed to Alirocumab in Parent Study | 46 | |
| Overall Study | COMPLETED | Placebo Participants in Parent Study | 7 | |
| Overall Study | COMPLETED | Participants Previously Exposed to Alirocumab in Parent Study | 27 | |
| Overall Study | NOT COMPLETED | Placebo Participants in Parent Study | 5 | |
| Overall Study | NOT COMPLETED | Participants Previously Exposed to Alirocumab in Parent Study | 19 |
Source ClinicalTrials.gov
Adverse events242
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 | Anaemia | Blood and lymphatic system disorders | Placebo Participants in Parent Study | 1 | 12 | 1 |
| other | Anaemia | Blood and lymphatic system disorders | Participants Previously Exposed to Alirocumab in Parent Study | 2 | 46 | 2 |
| other | Angina pectoris | Cardiac disorders | Placebo Participants in Parent Study | 1 | 12 | 1 |
| other | Angina pectoris | Cardiac disorders | Participants Previously Exposed to Alirocumab in Parent Study | 0 | 46 | 0 |
| other | Aortic valve stenosis | Cardiac disorders | Placebo Participants in Parent Study | 1 | 12 | 1 |
| other | Aortic valve stenosis | Cardiac disorders | Participants Previously Exposed to Alirocumab in Parent Study | 0 | 46 | 0 |
| other | Ischaemic cardiomyopathy | Cardiac disorders | Placebo Participants in Parent Study | 1 | 12 | 1 |
| other | Ischaemic cardiomyopathy | Cardiac disorders | Participants Previously Exposed to Alirocumab in Parent Study | 0 | 46 | 0 |
| other | Cerumen impaction | Ear and labyrinth disorders | Placebo Participants in Parent Study | 1 | 12 | 1 |
| other | Cerumen impaction | Ear and labyrinth disorders | Participants Previously Exposed to Alirocumab in Parent Study | 0 | 46 | 0 |
1 to 10 of 242Source ClinicalTrials.gov
See all 242 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Placebo Participants in Parent Study | 0 | 12 |
| Total, all-cause mortality | Participants Previously Exposed to Alirocumab in Parent Study | 0 | 46 |
| Total, other adverse events | Placebo Participants in Parent Study | 12 | 12 |
| Total, other adverse events | Participants Previously Exposed to Alirocumab in Parent Study | 38 | 46 |
| Total, serious adverse events | Placebo Participants in Parent Study | 4 | 12 |
| Total, serious adverse events | Participants Previously Exposed to Alirocumab in Parent Study | 8 | 46 |
Source ClinicalTrials.gov
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 27886619 | 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 |
|---|---|
| Participants Previously Exposed to Alirocumab in Parent Study vs Placebo Participants in Parent Study: deaths from any cause | Trial comparisons |
| Participants Previously Exposed to Alirocumab in Parent Study vs Placebo Participants in Parent Study: 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.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT01576484
GET https://conductscience.org/api/trials/v1/NCT01576484/results?page=2