Trial · NCT03156621
Study in Participants With Homozygous Familial Hypercholesterolemia (HoFH)
NCT03156621, Phase 3, Completed, Regeneron Pharmaceuticals; results first posted 2021-06-29
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Study in Participants With Homozygous Familial Hypercholesterolemia (HoFH). Trials, record NCT03156621. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03156621
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Study in Participants With Homozygous Familial Hypercholesterolemia (HoFH)
- NCT number
- NCT03156621
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 69 participants, actual
- Lead sponsor
- Regeneron Pharmaceuticals
- Conditions
- Homozygous Familial Hypercholesterolemia
- Interventions
- Alirocumab, Placebo
- First posted
- 2017-05-17
- Last updated
- 2021-06-29
- Other registries' IDs
- eudract:2017-000351-95
- Results first posted
- 2021-06-29
Source ClinicalTrials.gov
Registered outcomes23
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Percent Change in Low-density Lipoprotein Cholesterol (LDL-C) From Baseline to Week 12 (Intent-to-Treat [ITT] Estimand) | Baseline to Week 12 | The percent change in LDL-C from baseline to week 12 is defined as: 100x (LDL-C value at week 12 - LDL-C value at baseline) / LDL-C value at baseline. |
| Secondary | Percent Change in Apolipoprotein (Apo) B From Baseline to Week 12 (ITT Estimand) | Baseline to Week 12 | ITT estimand; The percent change in Apo B from baseline to week 12 is defined as: 100x (Apo B value at week 12 - Apo B value at baseline) / Apo B value at baseline. |
| Secondary | Percent Change in Non-high-density Lipoprotein Cholesterol (Non-HDL-C) From Baseline to Week 12 | Baseline to Week 12 | ITT estimand; The percent change in non-HDL-C from baseline to week 12 is defined as: 100x (non-HDL-C value at week 12 - non-HDL-C value at baseline) / non-HDL-C value at baseline. |
| Secondary | Percent Change in Total Cholesterol (TC) From Baseline to Week 12 | Baseline to Week 12 | ITT estimand; The percent change in TC from baseline to week 12 is defined as: 100x (TC value at week 12 - TC value at baseline) / TC value at baseline. |
| Secondary | Percentage of Participants With ≥15% Reduction in LDL-C at Week 12 | At Week 12 | ITT estimand |
| Secondary | Percentage of Participants With ≥30% Reduction in LDL-C at Week 12 | At Week 12 | ITT estimand |
| Secondary | Percent Change in Lipoprotein(a) [Lp(a)] From Baseline to Week 12 | Baseline to Week 12 | ITT estimand; The percent change in Lp(a) from baseline to week 12 is defined as: 100x (Lp(a) value at week 12 - Lp(a) value at baseline) / Lp(a) value at baseline. |
| Secondary | Percentage of Participants With ≥50% Reduction in LDL-C at Week 12 | At Week 12 | ITT estimand |
| Secondary | Percent Change in HDL-C From Baseline to Week 12 - ITT Analysis | Baseline to Week 12 | ITT estimand; The percent change in HDL-C from baseline to week 12 is defined as: 100x (HDL-C value at week 12 - HDL-C value at baseline) / HDL-C value at baseline. |
| Secondary | Percent Change in Fasting Triglycerides (TG) From Baseline to Week 12 | Baseline to Week 12 | ITT estimand; The percent change in TG from baseline to week 12 is defined as: 100x (TG value at week 12 - TG value at baseline) / TG value at baseline. |
1 to 10 of 23Source ClinicalTrials.gov
See all 23 registered outcomesOutcomes23
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Percent Change in Low-density Lipoprotein Cholesterol (LDL-C) From Baseline to Week 12 (Intent-to-Treat [ITT] Estimand) | Baseline to Week 12 | ITT estimand (All randomized participants who had at least 1 measurement value for LDL-C before the first dose of double blind investigational study drug) | Percentage of change | LEAST_SQUARES_MEAN |
| SECONDARY | Percent Change in Apolipoprotein (Apo) B From Baseline to Week 12 (ITT Estimand) | Baseline to Week 12 | Percentage of change | LEAST_SQUARES_MEAN | |
| SECONDARY | Percent Change in Non-high-density Lipoprotein Cholesterol (Non-HDL-C) From Baseline to Week 12 | Baseline to Week 12 | Percentage of change | LEAST_SQUARES_MEAN | |
| SECONDARY | Percent Change in Total Cholesterol (TC) From Baseline to Week 12 | Baseline to Week 12 | Percentage of change | LEAST_SQUARES_MEAN | |
| SECONDARY | Percentage of Participants With ≥15% Reduction in LDL-C at Week 12 | At Week 12 | Percentage of participants | NUMBER | |
| SECONDARY | Percentage of Participants With ≥30% Reduction in LDL-C at Week 12 | At Week 12 | Percentage of participants | NUMBER | |
| SECONDARY | Percent Change in Lipoprotein(a) [Lp(a)] From Baseline to Week 12 | Baseline to Week 12 | Percentage of change | LEAST_SQUARES_MEAN | |
| SECONDARY | Percentage of Participants With ≥50% Reduction in LDL-C at Week 12 | At Week 12 | Percentage of participants | NUMBER | |
| SECONDARY | Percent Change in HDL-C From Baseline to Week 12 - ITT Analysis | Baseline to Week 12 | Percentage of change | LEAST_SQUARES_MEAN | |
| SECONDARY | Percent Change in Fasting Triglycerides (TG) From Baseline to Week 12 | Baseline to Week 12 | Percentage of change | LEAST_SQUARES_MEAN |
1 to 10 of 23Source ClinicalTrials.gov
See all 23 outcomesResults by group54
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Percent Change in Low-density Lipoprotein Cholesterol (LDL-C) From Baseline to Week 12 (Intent-to-Treat [ITT] Estimand) | Placebo in DBTP | 8.6 | Standard Error 6.3 | Percentage of change | |
| Percent Change in Low-density Lipoprotein Cholesterol (LDL-C) From Baseline to Week 12 (Intent-to-Treat [ITT] Estimand) | Alirocumab 150 mg SC Q2W | -26.9 | Standard Error 4.6 | Percentage of change | |
| Percent Change in Apolipoprotein (Apo) B From Baseline to Week 12 (ITT Estimand) | Placebo in DBTP | 7.2 | Standard Error 5.0 | Percentage of change | |
| Percent Change in Apolipoprotein (Apo) B From Baseline to Week 12 (ITT Estimand) | Alirocumab 150 mg SC Q2W | -22.5 | Standard Error 3.7 | Percentage of change | |
| Percent Change in Non-high-density Lipoprotein Cholesterol (Non-HDL-C) From Baseline to Week 12 | Placebo in DBTP | 8.0 | Standard Error 5.9 | Percentage of change | |
| Percent Change in Non-high-density Lipoprotein Cholesterol (Non-HDL-C) From Baseline to Week 12 | Alirocumab 150 mg SC Q2W | -24.8 | Standard Error 4.3 | Percentage of change | |
| Percent Change in Total Cholesterol (TC) From Baseline to Week 12 | Placebo in DBTP | 6.6 | Standard Error 5.0 | Percentage of change | |
| Percent Change in Total Cholesterol (TC) From Baseline to Week 12 | Alirocumab 150 mg SC Q2W | -19.8 | Standard Error 3.7 | Percentage of change | |
| Percentage of Participants With ≥15% Reduction in LDL-C at Week 12 | Placebo in DBTP | 12.5 | Percentage of participants | ||
| Percentage of Participants With ≥15% Reduction in LDL-C at Week 12 | Alirocumab 150 mg SC Q2W | 61.9 | Percentage of participants |
1 to 10 of 54Source ClinicalTrials.gov
See all 54 results by groupParticipant flow12
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Double-Blind Treatment Period (DBTP) | STARTED | Placebo in DBTP | 24 | |
| Double-Blind Treatment Period (DBTP) | STARTED | Alirocumab 150 mg SC Q2W | 45 | |
| Double-Blind Treatment Period (DBTP) | COMPLETED | Placebo in DBTP | 24 | |
| Double-Blind Treatment Period (DBTP) | COMPLETED | Alirocumab 150 mg SC Q2W | 45 | |
| Double-Blind Treatment Period (DBTP) | NOT COMPLETED | Placebo in DBTP | 0 | |
| Double-Blind Treatment Period (DBTP) | NOT COMPLETED | Alirocumab 150 mg SC Q2W | 0 | |
| Open-Label Treatment Period (OLTP) | STARTED | Placebo in DBTP | 24 | |
| Open-Label Treatment Period (OLTP) | STARTED | Alirocumab 150 mg SC Q2W | 45 | |
| Open-Label Treatment Period (OLTP) | COMPLETED | Placebo in DBTP | 24 | |
| Open-Label Treatment Period (OLTP) | COMPLETED | Alirocumab 150 mg SC Q2W | 42 |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 participant flowAdverse events20
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 | DB Placebo (DBTP) | 0 | 24 | 0 |
| other | Diarrhoea | Gastrointestinal disorders | DB Alirocumab 150 Q2W (DBTP) | 3 | 45 | 3 |
| other | Diarrhoea | Gastrointestinal disorders | DB Placebo (OLTP) | 0 | 24 | 0 |
| other | Diarrhoea | Gastrointestinal disorders | DB Alirocumab 150 Q2W (OLTP) | 1 | 45 | 1 |
| other | Nasopharyngitis | Infections and infestations | DB Placebo (DBTP) | 0 | 24 | 0 |
| other | Nasopharyngitis | Infections and infestations | DB Alirocumab 150 Q2W (DBTP) | 2 | 45 | 3 |
| other | Nasopharyngitis | Infections and infestations | DB Placebo (OLTP) | 1 | 24 | 1 |
| other | Nasopharyngitis | Infections and infestations | DB Alirocumab 150 Q2W (OLTP) | 3 | 45 | 4 |
| other | Upper respiratory tract infection | Infections and infestations | DB Placebo (DBTP) | 2 | 24 | 3 |
| other | Upper respiratory tract infection | Infections and infestations | DB Alirocumab 150 Q2W (DBTP) | 2 | 45 | 2 |
1 to 10 of 20Source ClinicalTrials.gov
See all 20 adverse eventsAdverse event totals12
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | DB Placebo (DBTP) | 0 | 24 |
| Total, all-cause mortality | DB Alirocumab 150 Q2W (DBTP) | 0 | 45 |
| Total, all-cause mortality | DB Placebo (OLTP) | 0 | 24 |
| Total, all-cause mortality | DB Alirocumab 150 Q2W (OLTP) | 0 | 45 |
| Total, other adverse events | DB Placebo (DBTP) | 4 | 24 |
| Total, other adverse events | DB Alirocumab 150 Q2W (DBTP) | 8 | 45 |
| Total, other adverse events | DB Placebo (OLTP) | 2 | 24 |
| Total, other adverse events | DB Alirocumab 150 Q2W (OLTP) | 4 | 45 |
| Total, serious adverse events | DB Placebo (DBTP) | 0 | 24 |
| Total, serious adverse events | DB Alirocumab 150 Q2W (DBTP) | 0 | 45 |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 adverse event totalsPapers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 32646561 | 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 comparisons1
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison |
|---|
| Alirocumab 150 mg SC Q2W vs Placebo in DBTP: Number of Participants With Anti-Drug Antibodies (ADA) to REGN727 Over Time |
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/NCT03156621
GET https://conductscience.org/api/trials/v1/NCT03156621/registered?page=2