Trial · NCT02392559
Trial Assessing Efficacy, Safety and Tolerability of Proprotein Convertase Subtilisin/Kexin Type 9 (PCSK9) Inhibition in Paediatric Subjects With Genetic Low-Density Lipoprotein (LDL) Disorders
NCT02392559, Phase 3, Completed, Amgen; results first posted 2020-07-15
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Trial Assessing Efficacy, Safety and Tolerability of Proprotein Convertase Subtilisin/Kexin Type 9 (PCSK9) Inhibition in Paediatric Subjects With Genetic Low-Density Lipoprotein (LDL) Disorders. Trials, record NCT02392559. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02392559
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Trial Assessing Efficacy, Safety and Tolerability of Proprotein Convertase Subtilisin/Kexin Type 9 (PCSK9) Inhibition in Paediatric Subjects With Genetic Low-Density Lipoprotein (LDL) Disorders
- NCT number
- NCT02392559
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 158 participants, actual
- Lead sponsor
- Amgen
- Conditions
- Heterozygous Familial Hypercholesterolemia
- Interventions
- Evolocumab, Placebo
- First posted
- 2015-03-19
- Last updated
- 2022-11-08
- Other registries' IDs
- eudract:2014-002277-11
- Results first posted
- 2020-07-15
Source ClinicalTrials.gov
Registered outcomes14
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Percent Change From Baseline to Week 24 in LDL-C | Baseline, Week 24 | Least squares mean is from the repeated measures model which includes treatment group, stratification factors of age and screening LDL-C (from interactive voice response system \[IVRS\]), scheduled visit and the interaction of treatment with scheduled visit as covariates. The model uses an unstructured covariance. |
| Secondary | Mean Percent Change From Baseline to Mean of Weeks 22 and 24 in LDL-C | Baseline, Week 22, Week 24 | Least squares mean is from the repeated measures model which includes treatment group, stratification factors of age and screening LDL-C (from IVRS), scheduled visit and the interaction of treatment with scheduled visit as covariates. |
| Secondary | Change From Baseline to Week 24 in LDL-C | Baseline, Week 24 | Least squares mean is from the repeated measures model which includes treatment group, stratification factors of age and screening LDL-C (from IVRS), scheduled visit and the interaction of treatment with scheduled visit as covariates. |
| Secondary | Percent Change From Baseline to Week 24 in Non-HDL-C | Baseline, Week 24 | Least squares mean is from the repeated measures model which includes treatment group, stratification factors of age and screening LDL-C (from IVRS), scheduled visit and the interaction of treatment with scheduled visit as covariates |
| Secondary | Percent Change From Baseline to Week 24 in Apoliprotein-B (ApoB) | Baseline, Week 24 | Least squares mean is from the repeated measures model which includes treatment group, stratification factors of age and screening LDL-C (from IVRS), scheduled visit and the interaction of treatment with scheduled visit as covariates. |
| Secondary | Percent Change From Baseline to Week 24 in Total Cholesterol/HDL-C Ratio | Baseline, Week 24 | Least squares mean is from the repeated measures model which includes treatment group, stratification factors of age and screening LDL-C (from IVRS), scheduled visit and the interaction of treatment with scheduled visit as covariates. |
| Secondary | Percent Change From Baseline to Week 24 in ApoB:ApoA1 Ratio | Baseline, Week 24 | Least squares mean is from the repeated measures model which includes treatment group, stratification factors of age and screening LDL-C (from IVRS), scheduled visit and the interaction of treatment with scheduled visit as covariates. |
| Secondary | Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Serious TEAEs, TEAEs Leading to Discontinuation (DC), Fatal TEAEs, and Device-Related TEAEs | From first dose of study drug up to and including 30 days after the last dose or end of study date (Week 24), whichever was earlier. Mean (SD) duration on study was 5.664 (0.278) and 5.608 (0.137) months for Placebo and EvoMab arms, respectively. | An adverse event (AE) is defined as any untoward medical occurrence that does not necessarily have a causal relationship with study treatment. An SAE is defined as an adverse event that: is fatal; is a life threatening; requires in-patient hospitalization or prolongation of existing hospitalization; results in persistent or significant disability/incapacity; is a congenital anomaly/birth defect; or other medically important serious event. Events were graded by the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) grading scale (1=mild; 2=moderate; 3=severe; 4=life-threatening; 5=death). Events were defined as treatment emergent if they occurred after the first dose of study drug and up to and including 30 days after the last dose or the end of study date, whichever is earlier. |
| Secondary | Number of Participants With Maximum Post-Baseline Laboratory Toxicities of Grade ≥ 3 | Week 24 | Laboratory toxicity grading was based on NCI CTCAE grading. Grade 3 indicates severe toxicity and Grade 4 indicates life-threatening toxicity. Values representing a worsening from baseline are shown. |
| Secondary | Change From Baseline Over Time in Systolic Blood Pressure | Baseline, Week 4, Week 12, Week 20, Week 22, Week 24 |
1 to 10 of 14Source ClinicalTrials.gov
See all 14 registered outcomesOutcomes14
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 to Week 24 in LDL-C | Baseline, Week 24 | Full Analysis Set: all randomized participants who received at least 1 dose of study drug. | percent change | LEAST_SQUARES_MEAN |
| SECONDARY | Mean Percent Change From Baseline to Mean of Weeks 22 and 24 in LDL-C | Baseline, Week 22, Week 24 | Full Analysis Set: all randomized participants who received at least 1 dose of study drug. | percent change | LEAST_SQUARES_MEAN |
| SECONDARY | Change From Baseline to Week 24 in LDL-C | Baseline, Week 24 | Full Analysis Set: all randomized participants who received at least 1 dose of study drug. | mg/dL | LEAST_SQUARES_MEAN |
| SECONDARY | Percent Change From Baseline to Week 24 in Non-HDL-C | Baseline, Week 24 | Full Analysis Set: all randomized participants who received at least 1 dose of study drug. | percent change | LEAST_SQUARES_MEAN |
| SECONDARY | Percent Change From Baseline to Week 24 in Apoliprotein-B (ApoB) | Baseline, Week 24 | Full Analysis Set: all randomized participants who received at least 1 dose of study drug. | percent change | LEAST_SQUARES_MEAN |
| SECONDARY | Percent Change From Baseline to Week 24 in Total Cholesterol/HDL-C Ratio | Baseline, Week 24 | Full Analysis Set: all randomized participants who received at least 1 dose of study drug. | percent change | LEAST_SQUARES_MEAN |
| SECONDARY | Percent Change From Baseline to Week 24 in ApoB:ApoA1 Ratio | Baseline, Week 24 | Full Analysis Set: all randomized participants who received at least 1 dose of study drug. | percent change | LEAST_SQUARES_MEAN |
| SECONDARY | Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Serious TEAEs, TEAEs Leading to Discontinuation (DC), Fatal TEAEs, and Device-Related TEAEs | From first dose of study drug up to and including 30 days after the last dose or end of study date (Week 24), whichever was earlier. Mean (SD) duration on study was 5.664 (0.278) and 5.608 (0.137) months for Placebo and EvoMab arms, respectively. | Full Analysis Set: all randomized participants who received at least 1 dose of study drug. | participants | NUMBER |
| SECONDARY | Number of Participants With Maximum Post-Baseline Laboratory Toxicities of Grade ≥ 3 | Week 24 | Full Analysis Set: all randomized participants who received at least 1 dose of study drug. | participants | NUMBER |
| SECONDARY | Change From Baseline Over Time in Systolic Blood Pressure | Baseline, Week 4, Week 12, Week 20, Week 22, Week 24 | Full Analysis Set: all randomized participants who received at least 1 dose of study drug. Participants with an assessment at given time point. | mmHg | MEAN |
1 to 10 of 14Source ClinicalTrials.gov
See all 14 outcomesResults by group83
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 to Week 24 in LDL-C | Placebo | -6.23 | Standard Error 3.08 | percent change | |
| Percent Change From Baseline to Week 24 in LDL-C | EvoMab 420 mg QM | -44.53 | Standard Error 2.17 | percent change | |
| Mean Percent Change From Baseline to Mean of Weeks 22 and 24 in LDL-C | Placebo | -5.87 | Standard Error 2.66 | percent change | |
| Mean Percent Change From Baseline to Mean of Weeks 22 and 24 in LDL-C | EvoMab 420 mg QM | -47.95 | Standard Error 1.92 | percent change | |
| Change From Baseline to Week 24 in LDL-C | Placebo | -9.0 | Standard Error 6.2 | mg/dL | |
| Change From Baseline to Week 24 in LDL-C | EvoMab 420 mg QM | -77.5 | Standard Error 4.4 | mg/dL | |
| Percent Change From Baseline to Week 24 in Non-HDL-C | Placebo | -6.14 | Standard Error 2.87 | percent change | |
| Percent Change From Baseline to Week 24 in Non-HDL-C | EvoMab 420 mg QM | -41.19 | Standard Error 2.01 | percent change | |
| Percent Change From Baseline to Week 24 in Apoliprotein-B (ApoB) | Placebo | -2.37 | Standard Error 2.70 | percent change | |
| Percent Change From Baseline to Week 24 in Apoliprotein-B (ApoB) | EvoMab 420 mg QM | -34.85 | Standard Error 1.88 | percent change |
1 to 10 of 83Source ClinicalTrials.gov
See all 83 results by groupParticipant flow8
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Placebo | 53 | |
| Overall Study | STARTED | EvoMab 420 mg QM | 105 | |
| Overall Study | Randomized and Dosed | Placebo | 53 | |
| Overall Study | Randomized and Dosed | EvoMab 420 mg QM | 104 | |
| Overall Study | COMPLETED | Placebo | 53 | |
| Overall Study | COMPLETED | EvoMab 420 mg QM | 104 | |
| Overall Study | NOT COMPLETED | Placebo | 0 | |
| Overall Study | NOT COMPLETED | EvoMab 420 mg QM | 1 |
Source ClinicalTrials.gov
Adverse events28
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 | Constipation | Gastrointestinal disorders | Placebo SC QM | 0 | 53 | |
| other | Constipation | Gastrointestinal disorders | EvoMab 420 mg SC QM | 3 | 104 | |
| other | Influenza like illness | General disorders | Placebo SC QM | 0 | 53 | |
| other | Influenza like illness | General disorders | EvoMab 420 mg SC QM | 3 | 104 | |
| other | Pyrexia | General disorders | Placebo SC QM | 3 | 53 | |
| other | Pyrexia | General disorders | EvoMab 420 mg SC QM | 3 | 104 | |
| other | Gastroenteritis | Infections and infestations | Placebo SC QM | 4 | 53 | |
| other | Gastroenteritis | Infections and infestations | EvoMab 420 mg SC QM | 5 | 104 | |
| other | Influenza | Infections and infestations | Placebo SC QM | 2 | 53 | |
| other | Influenza | Infections and infestations | EvoMab 420 mg SC QM | 6 | 104 |
1 to 10 of 28Source ClinicalTrials.gov
See all 28 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Placebo SC QM | 0 | 53 |
| Total, all-cause mortality | EvoMab 420 mg SC QM | 0 | 105 |
| Total, other adverse events | Placebo SC QM | 19 | 53 |
| Total, other adverse events | EvoMab 420 mg SC QM | 41 | 104 |
| Total, serious adverse events | Placebo SC QM | 0 | 53 |
| Total, serious adverse events | EvoMab 420 mg SC QM | 1 | 104 |
Source ClinicalTrials.gov
Papers4
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 comparisons2
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| EvoMab 420 mg SC QM vs Placebo SC QM: deaths from any cause | Trial comparisons |
| EvoMab 420 mg SC QM vs Placebo SC QM: 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/NCT02392559
GET https://conductscience.org/api/trials/v1/NCT02392559/results?page=2