Trial · NCT04233918
Evaluate the Efficacy and Safety of Evinacumab in Pediatric Patients With Homozygous Familial Hypercholesterolemia
NCT04233918, Phase 3, Completed, Regeneron Pharmaceuticals; results first posted 2023-06-07
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Evaluate the Efficacy and Safety of Evinacumab in Pediatric Patients With Homozygous Familial Hypercholesterolemia. Trials, record NCT04233918. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04233918
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Evaluate the Efficacy and Safety of Evinacumab in Pediatric Patients With Homozygous Familial Hypercholesterolemia
- NCT number
- NCT04233918
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 20 participants, actual
- Lead sponsor
- Regeneron Pharmaceuticals
- Conditions
- Homozygous Familial Hypercholesterolemia
- Interventions
- Evinacumab
- First posted
- 2020-01-18
- Last updated
- 2024-07-16
- Other registries' IDs
- eudract:2019-001931-30
- Results first posted
- 2023-06-07
Source ClinicalTrials.gov
Registered outcomes17
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Part A: Maximum Observed Serum Concentration (Cmax) of Evinacumab | At day 12 | Cmax was obtained directly from the plasma concentration versus time curve. |
| Primary | Part A: Area Under the Serum Concentration-Time Curve From Time Zero to the Time of the Last Measurable Concentration (AUClast) of Evinacumab | Up to Week 12 | AUClast was defined as area under the serum concentration time-curve from zero to the last measured concentration. |
| Primary | Part A: Terminal Half-Life (t1/2) of Evinacumab | Up to week 12 | T1/2 was defined as the time required for the plasma concentration of drug to decrease 50 percent in the final stage of its elimination. |
| Primary | Part B: Percent Change in Calculated Low-Density Lipoprotein Cholesterol (LDL-C) From Baseline to Week 24 | Baseline to Week 24 | Percent change was calculated as 100 multiplied by (calculated LDL-C value at Week 24 minus calculated LDL-C value at baseline) divided by calculated LDL-C value at baseline. |
| Secondary | Part A and Part B: Number of Participants With Treatment-Emergent Adverse Events (TEAEs) | Part A: up to Week 24; Part B: up to Week 48 | Any untoward medical occurrence in a participant who received investigational medicinal product (IMP) was considered an AE without regard to possibility of causal relationship with this treatment. 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. TEAE was defined as AE starting/worsening after first intake of study drug. TEAE included participants with both serious and non-serious AEs. 1 participant experienced an AE during part B that was recorded after Part B database lock. This was not reflected in the reported endpoint number of participants of 10. |
| Secondary | Part B: Percent Change in Apolipoprotein B (Apo B) From Baseline to Week 24 | Baseline to Week 24 | Percent change in Apo B from baseline to Week 24 was reported. |
| Secondary | Part B: Percent Change in Non-High-Density Lipoprotein Cholesterol (Non-HDL-C) From Baseline to Week 24 | Baseline to Week 24 | Percent change in Non-HDL-C from baseline to Week 24 was reported. |
| Secondary | Part B: Percent Change in Total Cholesterol (TC) From Baseline to Week 24 | Baseline to Week 24 | Percent change in TC from baseline to Week 24 was reported. |
| Secondary | Part B: Percentage of Participants With ≥50 Percent (%) Reduction in Calculated Low-Density Lipoprotein Cholesterol (LDL-C) at Week 24 | Week 24 | Percentage of participants who achieved reduction in calculated LDL-C ≥ 50% at Week 24 was reported. |
| Secondary | Part B: Percent Change in Calculated Low-Density Lipoprotein Cholesterol (LDL-C) From Baseline to Week 24 in Participants Who Have Negative/Negative and Null/Null Mutations | Baseline to Week 24 | Participants with HoFH was classified based on the phenotype of the Low-density lipoprotein receptor (LDLR) mutation(s), ranging from defective mutations (where the LDLR retains some LDL-binding functionality) to null or negative mutations where no functioning LDLR was expressed. Participants who have LDLR activity \<15% are considered null and participants whose LDLR activity was impaired but \>15% are LDLR defective. Percent change in calculated LDL-C from baseline to Week 24 in participants who have negative/negative and null/null mutations was reported. |
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 | Part A: Maximum Observed Serum Concentration (Cmax) of Evinacumab | At day 12 | Pharmacokinetic (PK) Analysis Set included all participants who received any study drug and who had at least 1 non-missing result for concentration of evinacumab following the first dose of study drug. | Milligrams per Liter (mg/L) | MEAN |
| PRIMARY | Part A: Area Under the Serum Concentration-Time Curve From Time Zero to the Time of the Last Measurable Concentration (AUClast) of Evinacumab | Up to Week 12 | PK Analysis Set included all participants who received any study drug and who had at least 1 non-missing result for concentration of evinacumab following the first dose of study drug. | Days*Milligrams per Liter (day*mg/L) | MEAN |
| PRIMARY | Part A: Terminal Half-Life (t1/2) of Evinacumab | Up to week 12 | PK Analysis Set included all participants who received any study drug and who had at least 1 non-missing result for concentration of evinacumab following the first dose of study drug. | Days | MEDIAN |
| PRIMARY | Part B: Percent Change in Calculated Low-Density Lipoprotein Cholesterol (LDL-C) From Baseline to Week 24 | Baseline to Week 24 | The intent-to-treat (ITT) population included all participants who received at least 1 dose or part of a dose of study drug in Part B. | Percentage of Change | MEAN |
| SECONDARY | Part A and Part B: Number of Participants With Treatment-Emergent Adverse Events (TEAEs) | Part A: up to Week 24; Part B: up to Week 48 | The safety analysis set (SAF) includes all patients in Part A or Part B who received at least 1 dose or part of a dose of study drug in the respective study treatment period. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Part B: Percent Change in Apolipoprotein B (Apo B) From Baseline to Week 24 | Baseline to Week 24 | ITT population included all participants who received at least 1 dose or part of a dose of study drug in Part B. | Percentage of Change | MEAN |
| SECONDARY | Part B: Percent Change in Non-High-Density Lipoprotein Cholesterol (Non-HDL-C) From Baseline to Week 24 | Baseline to Week 24 | ITT population included all participants who received at least 1 dose or part of a dose of study drug in Part B. | Percentage of Change | MEAN |
| SECONDARY | Part B: Percent Change in Total Cholesterol (TC) From Baseline to Week 24 | Baseline to Week 24 | ITT population included all participants who received at least 1 dose or part of a dose of study drug in Part B. | Percentage of Change | MEAN |
| SECONDARY | Part B: Percentage of Participants With ≥50 Percent (%) Reduction in Calculated Low-Density Lipoprotein Cholesterol (LDL-C) at Week 24 | Week 24 | ITT population included all participants who received at least 1 dose or part of a dose of study drug in Part B. | Percentage of Participants | NUMBER |
| SECONDARY | Part B: Percent Change in Calculated Low-Density Lipoprotein Cholesterol (LDL-C) From Baseline to Week 24 in Participants Who Have Negative/Negative and Null/Null Mutations | Baseline to Week 24 | ITT population included all participants who received at least 1 dose or part of a dose of study drug in Part B. Here, Overall Number of Participants Analyzed signifies those participants who were evaluable for this outcome measure and Number Analyzed signifies those participants who were evaluable at specified category. | Percentage of Change | MEAN |
1 to 10 of 17Source ClinicalTrials.gov
See all 17 outcomesResults by group30
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Part A: Maximum Observed Serum Concentration (Cmax) of Evinacumab | Part A: Evinacumab 15mg/Kg IV | 238 | Standard Deviation 90.8 | Milligrams per Liter (mg/L) | |
| Part A: Area Under the Serum Concentration-Time Curve From Time Zero to the Time of the Last Measurable Concentration (AUClast) of Evinacumab | Part A: Evinacumab 15mg/Kg IV | 4576 | Standard Deviation 1568 | Days*Milligrams per Liter (day*mg/L) | |
| Part A: Terminal Half-Life (t1/2) of Evinacumab | Part A: Evinacumab 15mg/Kg IV | 7.69 | Full Range 6.18 to 12.4 | Days | |
| Part B: Percent Change in Calculated Low-Density Lipoprotein Cholesterol (LDL-C) From Baseline to Week 24 | Part B: Evinacumab 15mg/Kg IV Q4W | -48.3 | 95% Confidence Interval -68.8 to -27.8 | Percentage of Change | |
| Part A and Part B: Number of Participants With Treatment-Emergent Adverse Events (TEAEs) | Part A: Evinacumab 15mg/Kg IV | 5 | Participants | ||
| Part A and Part B: Number of Participants With Treatment-Emergent Adverse Events (TEAEs) | Part B: Evinacumab 15mg/Kg IV Q4W | 10 | Participants | ||
| Part B: Percent Change in Apolipoprotein B (Apo B) From Baseline to Week 24 | Part B: Evinacumab 15mg/Kg IV Q4W | -41.3 | 95% Confidence Interval -58.9 to -23.8 | Percentage of Change | |
| Part B: Percent Change in Non-High-Density Lipoprotein Cholesterol (Non-HDL-C) From Baseline to Week 24 | Part B: Evinacumab 15mg/Kg IV Q4W | -48.9 | 95% Confidence Interval -68.1 to -29.7 | Percentage of Change | |
| Part B: Percent Change in Total Cholesterol (TC) From Baseline to Week 24 | Part B: Evinacumab 15mg/Kg IV Q4W | -49.1 | 95% Confidence Interval -64.9 to -33.2 | Percentage of Change | |
| Part B: Percentage of Participants With ≥50 Percent (%) Reduction in Calculated Low-Density Lipoprotein Cholesterol (LDL-C) at Week 24 | Part B: Evinacumab 15mg/Kg IV Q4W | 78.6 | Percentage of Participants |
1 to 10 of 30Source ClinicalTrials.gov
See all 30 results by groupParticipant flow24
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Period 1: Part A and B | STARTED | Part A: Evinacumab 15mg/Kg IV | 6 | |
| Period 1: Part A and B | STARTED | Part B: Evinacumab 15mg/Kg IV Q4W | 14 | |
| Period 1: Part A and B | STARTED | Part A to C | 0 | |
| Period 1: Part A and B | STARTED | Part B to C | 0 | |
| Period 1: Part A and B | COMPLETED | Part A: Evinacumab 15mg/Kg IV | 6 | |
| Period 1: Part A and B | COMPLETED | Part B: Evinacumab 15mg/Kg IV Q4W | 14 | |
| Period 1: Part A and B | COMPLETED | Part A to C | 0 | |
| Period 1: Part A and B | COMPLETED | Part B to C | 0 | |
| Period 1: Part A and B | NOT COMPLETED | Part A: Evinacumab 15mg/Kg IV | 0 | |
| Period 1: Part A and B | NOT COMPLETED | Part B: Evinacumab 15mg/Kg IV Q4W | 0 |
1 to 10 of 24Source ClinicalTrials.gov
See all 24 participant flowAdverse events268
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 | Abdominal pain upper | Gastrointestinal disorders | Part A Evinacumab 15mg | 0 | 6 | 0 |
| other | Abdominal pain upper | Gastrointestinal disorders | Part B Evinacumab 15mg | 2 | 14 | 3 |
| other | Abdominal pain upper | Gastrointestinal disorders | Part A-C Evinacumab 15mg | 1 | 6 | 2 |
| other | Abdominal pain upper | Gastrointestinal disorders | Part B-C Evinacumab 15mg | 1 | 14 | 1 |
| other | Diarrhoea | Gastrointestinal disorders | Part A Evinacumab 15mg | 0 | 6 | 0 |
| other | Diarrhoea | Gastrointestinal disorders | Part B Evinacumab 15mg | 2 | 14 | 2 |
| other | Diarrhoea | Gastrointestinal disorders | Part A-C Evinacumab 15mg | 0 | 6 | 0 |
| other | Diarrhoea | Gastrointestinal disorders | Part B-C Evinacumab 15mg | 2 | 14 | 3 |
| other | Nausea | Gastrointestinal disorders | Part A Evinacumab 15mg | 0 | 6 | 0 |
| other | Nausea | Gastrointestinal disorders | Part B Evinacumab 15mg | 2 | 14 | 2 |
1 to 10 of 268Source ClinicalTrials.gov
See all 268 adverse eventsAdverse event totals12
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Part A Evinacumab 15mg | 0 | 6 |
| Total, all-cause mortality | Part B Evinacumab 15mg | 0 | 14 |
| Total, all-cause mortality | Part A-C Evinacumab 15mg | 0 | 6 |
| Total, all-cause mortality | Part B-C Evinacumab 15mg | 0 | 14 |
| Total, other adverse events | Part A Evinacumab 15mg | 5 | 6 |
| Total, other adverse events | Part B Evinacumab 15mg | 11 | 14 |
| Total, other adverse events | Part A-C Evinacumab 15mg | 6 | 6 |
| Total, other adverse events | Part B-C Evinacumab 15mg | 11 | 14 |
| Total, serious adverse events | Part A Evinacumab 15mg | 0 | 6 |
| Total, serious adverse events | Part B Evinacumab 15mg | 1 | 14 |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 adverse event totalsPapers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 37860863 | 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 |
|---|
| Part A: Evinacumab 15mg/Kg IV vs Part B: Evinacumab 15mg/Kg IV Q4W: Part A and Part B: Number of Participants With Treatment-Emergent Adverse Events (TEAEs) |
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/NCT04233918
GET https://conductscience.org/api/trials/v1/NCT04233918/results?page=2