Trial · NCT03399786
Efficacy and Safety of Evinacumab in Patients With Homozygous Familial Hypercholesterolemia
NCT03399786, Phase 3, Completed, Regeneron Pharmaceuticals; results first posted 2021-05-18
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Efficacy and Safety of Evinacumab in Patients With Homozygous Familial Hypercholesterolemia. Trials, record NCT03399786. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03399786
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Efficacy and Safety of Evinacumab in Patients With Homozygous Familial Hypercholesterolemia
- NCT number
- NCT03399786
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 65 participants, actual
- Lead sponsor
- Regeneron Pharmaceuticals
- Conditions
- Homozygous Familial Hypercholesterolemia
- Interventions
- evinacumab, Placebo
- First posted
- 2018-01-16
- Last updated
- 2021-05-18
- Other registries' IDs
- eudract:2017-001388-19
- Results first posted
- 2021-05-18
Source ClinicalTrials.gov
Registered outcomes17
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Percent Change in Calculated Low-Density Lipoprotein Cholesterol (LDL-C) From Baseline to Week 24 (Intent-to-Treat [ITT] Estimand) | Week 24 | Percent change was calculated as 100x(calculated LDL-C value at Week 24 - calculated LDL-C value at baseline)/calculated LDL-C value at baseline. The baseline LDL-C value was the last calculated LDL-C value obtained before the first dose of double-blind-study drug. The calculated LDL-C at week 24 was the LDL-C value obtained within the week 24 efficacy analysis window, regardless of adherence to treatment and subsequent therapies (intent-to-treat \[ITT\] estimand). The ITT population included all randomized participants who received at least one dose or part of a dose of double-blind study drug. Participants in the ITT population were analyzed according to the treatment group allocated by randomization (i.e., as randomized participant group). |
| Secondary | Percent Change in Apolipoprotein B (Apo B) From Baseline to Week 24 (ITT Estimand) | Week 24 | Percent change in Apo B from baseline at Week 24 in the DBTP was reported; value obtained regardless of adherence to study treatment and subsequent therapies (ITT estimand). ITT population included all randomized participants who received at least one dose or part of a dose of double-blind study drug. Participants in the ITT population were analyzed according to the treatment group allocated by randomization (i.e., as randomized participant group). |
| Secondary | Percent Change in Non-High-Density Lipoprotein Cholesterol (Non-HDL-C) From Baseline to Week 24 (ITT Estimand) | Week 24 | Percent change from baseline in non-HDL-C at Week 24 in the DBTP was reported; value obtained regardless of adherence to study treatment and subsequent therapies (ITT estimand). ITT population included all randomized participants who received at least one dose or part of a dose of double-blind study drug. Participants in the ITT population were analyzed according to the treatment group allocated by randomization (i.e., as randomized participant group). |
| Secondary | Percent Change in Total Cholesterol (TC) From Baseline to Week 24 (ITT Estimand) | Week 24 | Percent change in TC from baseline at Week 24 in the DBTP was reported; value obtained regardless of adherence to study treatment and subsequent therapies (ITT estimand). ITT population included all randomized participants who received at least one dose or part of a dose of double-blind study drug. Participants in the ITT population were analyzed according to the treatment group allocated by randomization (i.e., as randomized participant group). |
| Secondary | Percentage of Participants With ≥30% Reduction in Calculated Low-Density Lipoprotein Cholesterol (LDL-C) at Week 24 (ITT Estimand) | At Week 24 | Percentage of participants who achieved reduction in calculated LDL-C ≥30% at Week 24 in the DBTP was reported; value obtained regardless of adherence to study treatment and subsequent therapies (ITT estimand). ITT population included all randomized participants who received at least one dose or part of a dose of double-blind study drug. Participants in the ITT population were analyzed according to the treatment group allocated by randomization (i.e., as randomized participant group). |
| Secondary | Percentage of Participants With ≥50% Reduction in Calculated Low-Density Lipoprotein Cholesterol (LDL-C) at Week 24 (ITT Estimand) | At Week 24 | Percentage of participants who achieved reduction in calculated LDL-C ≥ 50% at Week 24 in the DBTP was reported; value obtained regardless of adherence to study treatment and subsequent therapies (ITT estimand). ITT population included all randomized participants who received at least one dose or part of a dose of double-blind study drug. Participants in the ITT population were analyzed according to the treatment group allocated by randomization (i.e., as randomized participant group). |
| Secondary | Absolute Change in Calculated Low-Density Lipoprotein Cholesterol (LDL-C) From Baseline to Week 24 (ITT Estimand) | Week 24 | Absolute change in calculated LDL-C from baseline at Week 24 in the DBTP was reported; value obtained regardless of adherence to study treatment and subsequent therapies (ITT estimand). ITT population included all randomized participants who received at least one dose or part of a dose of double-blind study drug. Participants in the ITT population were analyzed according to the treatment group allocated by randomization (i.e., as randomized participant group). |
| Secondary | Percentage of Participants Who Met United States (US) Apheresis Eligibility Criteria at Week 24 (ITT Estimand) | At Week 24 | US apheresis eligibility criteria included participants who had inadequate response to diet and LMTs after 6 months of treatment and with functional Homozygous familial hypercholesterolemia (HoFH) or Heterozygous familial hypercholesterolemia (HeFH) (with 0-1 risk factor) with LDL-C ≥ 300 mg/dL (7.77 mmol/L). Percentage of participants who met US apheresis eligibility criteria at Week 24 in the DBTP was reported; value obtained regardless of adherence to study treatment and subsequent therapies (ITT estimand). ITT population included all randomized participants who received at least one dose or part of a dose of double-blind study drug. Participants in the ITT population were analyzed according to the treatment group allocated by randomization (i.e., as randomized participant group). |
| Secondary | Percentage of Participants With Low-Density Lipoprotein Cholesterol (LDL-C) <100 Milligrams Per Deciliter (mg/dL) (2.59 Millimoles Per Liter [mmol/L]) at Week 24 (ITT Estimand) | At Week 24 | Percentage of participants with LDL-C value \<100 mg/dL (2.59 mmol/L) in the DBTP at Week 24 was reported; value obtained regardless of adherence to study treatment and subsequent therapies (ITT estimand). ITT population included all randomized participants who received at least one dose or part of a dose of double-blind study drug. Participants in the ITT population were analysed according to the treatment group allocated by randomization (i.e., as randomized participant group). |
| Secondary | Percentage of Participants Who Met European Union (EU) Apheresis Eligibility Criteria at Week 24 (ITT Estimand) | At Week 24 | EU apheresis eligibility criteria included participants who had inadequate response to diet and Lipid modifying therapies (LMTs) after 3 months of treatment, Primary prevention: Participants with Familial hypercholesterolemia (FH) with LDL-C \>160 mg/dL (4.2 mmol/L) and Cardiovascular (CV) events in close relatives. Secondary prevention: Participants with progressive CV events with LDL-C \> 120 to 130 mg/dL (3.1-3.4 mmol/L). Percentage of participants who met EU apheresis eligibility criteria at Week 24 in the DBTP was reported; value obtained regardless of adherence to study treatment and subsequent therapies (ITT estimand). ITT population included all randomized participants who received at least one dose or part of a dose of double-blind study drug. Participants in the ITT population were analyzed according to the treatment group allocated by randomization (i.e., as randomized participant group). |
1 to 10 of 17Source ClinicalTrials.gov
See all 17 registered outcomesOutcomes17
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Percent Change in Calculated Low-Density Lipoprotein Cholesterol (LDL-C) From Baseline to Week 24 (Intent-to-Treat [ITT] Estimand) | Week 24 | Percent Change | LEAST_SQUARES_MEAN | |
| SECONDARY | Percent Change in Apolipoprotein B (Apo B) From Baseline to Week 24 (ITT Estimand) | Week 24 | Percent Change | LEAST_SQUARES_MEAN | |
| SECONDARY | Percent Change in Non-High-Density Lipoprotein Cholesterol (Non-HDL-C) From Baseline to Week 24 (ITT Estimand) | Week 24 | Percent Change | LEAST_SQUARES_MEAN | |
| SECONDARY | Percent Change in Total Cholesterol (TC) From Baseline to Week 24 (ITT Estimand) | Week 24 | Percent Change | LEAST_SQUARES_MEAN | |
| SECONDARY | Percentage of Participants With ≥30% Reduction in Calculated Low-Density Lipoprotein Cholesterol (LDL-C) at Week 24 (ITT Estimand) | At Week 24 | Percentage of Participants | NUMBER | |
| SECONDARY | Percentage of Participants With ≥50% Reduction in Calculated Low-Density Lipoprotein Cholesterol (LDL-C) at Week 24 (ITT Estimand) | At Week 24 | Percentage of Participants | NUMBER | |
| SECONDARY | Absolute Change in Calculated Low-Density Lipoprotein Cholesterol (LDL-C) From Baseline to Week 24 (ITT Estimand) | Week 24 | Milligrams per Deciliter (mg/dL) | LEAST_SQUARES_MEAN | |
| SECONDARY | Percentage of Participants Who Met United States (US) Apheresis Eligibility Criteria at Week 24 (ITT Estimand) | At Week 24 | Percentage of Participants | NUMBER | |
| SECONDARY | Percentage of Participants With Low-Density Lipoprotein Cholesterol (LDL-C) <100 Milligrams Per Deciliter (mg/dL) (2.59 Millimoles Per Liter [mmol/L]) at Week 24 (ITT Estimand) | At Week 24 | Percentage of Participants | NUMBER | |
| SECONDARY | Percentage of Participants Who Met European Union (EU) Apheresis Eligibility Criteria at Week 24 (ITT Estimand) | At Week 24 | Percentage of Participants | NUMBER |
1 to 10 of 17Source ClinicalTrials.gov
See all 17 outcomesResults by group34
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Percent Change in Calculated Low-Density Lipoprotein Cholesterol (LDL-C) From Baseline to Week 24 (Intent-to-Treat [ITT] Estimand) | Placebo IV Q4W | 1.9 | Standard Error 6.5 | Percent Change | |
| Percent Change in Calculated Low-Density Lipoprotein Cholesterol (LDL-C) From Baseline to Week 24 (Intent-to-Treat [ITT] Estimand) | Evinacumab 15 mg/kg IV Q4W | -47.1 | Standard Error 4.6 | Percent Change | |
| Percent Change in Apolipoprotein B (Apo B) From Baseline to Week 24 (ITT Estimand) | Placebo IV Q4W | -4.5 | Standard Error 4.8 | Percent Change | |
| Percent Change in Apolipoprotein B (Apo B) From Baseline to Week 24 (ITT Estimand) | Evinacumab 15 mg/kg IV Q4W | -41.4 | Standard Error 3.3 | Percent Change | |
| Percent Change in Non-High-Density Lipoprotein Cholesterol (Non-HDL-C) From Baseline to Week 24 (ITT Estimand) | Placebo IV Q4W | 2.0 | Standard Error 5.4 | Percent Change | |
| Percent Change in Non-High-Density Lipoprotein Cholesterol (Non-HDL-C) From Baseline to Week 24 (ITT Estimand) | Evinacumab 15 mg/kg IV Q4W | -49.7 | Standard Error 3.8 | Percent Change | |
| Percent Change in Total Cholesterol (TC) From Baseline to Week 24 (ITT Estimand) | Placebo IV Q4W | 1.0 | Standard Error 4.2 | Percent Change | |
| Percent Change in Total Cholesterol (TC) From Baseline to Week 24 (ITT Estimand) | Evinacumab 15 mg/kg IV Q4W | -47.4 | Standard Error 3.0 | Percent Change | |
| Percentage of Participants With ≥30% Reduction in Calculated Low-Density Lipoprotein Cholesterol (LDL-C) at Week 24 (ITT Estimand) | Placebo IV Q4W | 18.2 | Percentage of Participants | ||
| Percentage of Participants With ≥30% Reduction in Calculated Low-Density Lipoprotein Cholesterol (LDL-C) at Week 24 (ITT Estimand) | Evinacumab 15 mg/kg IV Q4W | 83.7 | Percentage of Participants |
1 to 10 of 34Source ClinicalTrials.gov
See all 34 results by groupParticipant flow24
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Double-blind Treatment Period (DBTP) | STARTED | Placebo IV Q4W (DBTP) | 22 | |
| Double-blind Treatment Period (DBTP) | STARTED | Evinacumab 15 mg/kg (DBTP) | 43 | |
| Double-blind Treatment Period (DBTP) | STARTED | Placebo IV Q4W (OLTP) | 0 | |
| Double-blind Treatment Period (DBTP) | STARTED | Evinacumab 15 mg/kg (OLTP) | 0 | |
| Double-blind Treatment Period (DBTP) | COMPLETED | Placebo IV Q4W (DBTP) | 21 | |
| Double-blind Treatment Period (DBTP) | COMPLETED | Evinacumab 15 mg/kg (DBTP) | 43 | |
| Double-blind Treatment Period (DBTP) | COMPLETED | Placebo IV Q4W (OLTP) | 0 | |
| Double-blind Treatment Period (DBTP) | COMPLETED | Evinacumab 15 mg/kg (OLTP) | 0 | |
| Double-blind Treatment Period (DBTP) | NOT COMPLETED | Placebo IV Q4W (DBTP) | 1 | |
| Double-blind Treatment Period (DBTP) | NOT COMPLETED | Evinacumab 15 mg/kg (DBTP) | 0 |
1 to 10 of 24Source ClinicalTrials.gov
See all 24 participant flowAdverse events160
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 | Nasopharyngitis | Infections and infestations | Placebo IV Q4W (DBTP) | 5 | 21 | 6 |
| other | Nasopharyngitis | Infections and infestations | Evinacumab 15 mg/kg (DBTP) | 7 | 44 | 7 |
| other | Nasopharyngitis | Infections and infestations | Placebo IV Q4W (OLTP) | 1 | 20 | 2 |
| other | Nasopharyngitis | Infections and infestations | Evinacumab 15 mg/kg (OLTP) | 5 | 44 | 5 |
| other | Urinary tract infection | Infections and infestations | Placebo IV Q4W (DBTP) | 2 | 21 | 3 |
| other | Urinary tract infection | Infections and infestations | Evinacumab 15 mg/kg (DBTP) | 0 | 44 | 0 |
| other | Urinary tract infection | Infections and infestations | Placebo IV Q4W (OLTP) | 0 | 20 | 0 |
| other | Urinary tract infection | Infections and infestations | Evinacumab 15 mg/kg (OLTP) | 1 | 44 | 1 |
| other | Gastroenteritis viral | Infections and infestations | Placebo IV Q4W (DBTP) | 0 | 21 | 0 |
| other | Gastroenteritis viral | Infections and infestations | Evinacumab 15 mg/kg (DBTP) | 1 | 44 | 1 |
1 to 10 of 160Source ClinicalTrials.gov
See all 160 adverse eventsAdverse event totals12
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Placebo IV Q4W (DBTP) | 0 | 21 |
| Total, all-cause mortality | Evinacumab 15 mg/kg (DBTP) | 0 | 44 |
| Total, all-cause mortality | Placebo IV Q4W (OLTP) | 0 | 20 |
| Total, all-cause mortality | Evinacumab 15 mg/kg (OLTP) | 0 | 44 |
| Total, other adverse events | Placebo IV Q4W (DBTP) | 13 | 21 |
| Total, other adverse events | Evinacumab 15 mg/kg (DBTP) | 20 | 44 |
| Total, other adverse events | Placebo IV Q4W (OLTP) | 12 | 20 |
| Total, other adverse events | Evinacumab 15 mg/kg (OLTP) | 19 | 44 |
| Total, serious adverse events | Placebo IV Q4W (DBTP) | 0 | 21 |
| Total, serious adverse events | Evinacumab 15 mg/kg (DBTP) | 2 | 44 |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 adverse event totalsPapers2
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 comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
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/NCT03399786
GET https://conductscience.org/api/trials/v1/NCT03399786/results?page=2