Phase 3
Completed N=158
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
Heterozygous Familial Hypercholesterolemia
Source: ClinicalTrials.gov NCT02392559 ↗
Enrolled (actual)
158
Serious AEs
0.6%
Results posted
Jul 2020
Primary outcomePrimary: Percent Change From Baseline to Week 24 in LDL-C — -6.23; -44.53 percent change — p=< 0.0001
◆ Published Evidence
Highly cited
194citations · ~32 / year
Evolocumab in Pediatric Heterozygous Familial Hypercholesterolemia.
Summary
A study to assess safety and efficacy of evolocumab (AMG-145) in paediatric subjects aged 10-17 years diagnosed with heterozygous familial hypercholesterolemia.
Linked Publications (3)
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Evolocumab in Pediatric Heterozygous Familial Hypercholesterolemia.
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PCSK9 monoclonal antibodies for the primary and secondary prevention of cardiovascular disease.
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Efficacy, safety, and tolerability of evolocumab in pediatric patients with heterozygous familial hypercholesterolemia: Rationale and design of the HAUSER-RCT study.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Percent Change From Baseline to Week 24 in LDL-C |
-6.23; -44.53 | < 0.0001 sig |
| SECONDARY Mean Percent Change From Baseline to Mean of Weeks 22 and 24 in LDL-C |
-5.87; -47.95 | < 0.0001 sig |
| SECONDARY Change From Baseline to Week 24 in LDL-C |
-9.0; -77.5 | < 0.0001 sig |
| SECONDARY Percent Change From Baseline to Week 24 in Non-HDL-C |
-6.14; -41.19 | < 0.0001 sig |
| SECONDARY Percent Change From Baseline to Week 24 in Apoliprotein-B (ApoB) |
-2.37; -34.85 | < 0.0001 sig |
| SECONDARY Percent Change From Baseline to Week 24 in Total Cholesterol/HDL-C Ratio |
-4.66; -34.96 | < 0.0001 sig |
| SECONDARY Percent Change From Baseline to Week 24 in ApoB:ApoA1 Ratio |
-0.63; -37.02 | < 0.0001 sig |
| SECONDARY Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Serious TEAEs, TEAEs Leading to Discontinuation (DC), Fatal TEAEs, and Device-Related TEAEs |
34; 64; 22; 46; 0; 4 | — |
| SECONDARY Number of Participants With Maximum Post-Baseline Laboratory Toxicities of Grade ≥ 3 |
1; 0; 1; 1; 2; 8 | — |
| SECONDARY Change From Baseline Over Time in Systolic Blood Pressure |
-0.1; -0.7; -0.6; 0.3; -2.1; 0.1 | — |
| SECONDARY Change From Baseline Over Time in Diastolic Blood Pressure |
-2.5; -1.5; -2.2; 0.5; -3.3; -0.6 | — |
| SECONDARY Change From Baseline Over Time in Heart Rate |
2.1; 0.1; -0.5; -1.3; -1.1; 1.1 | — |
| SECONDARY Number of Participants Testing Positive for Anti-Evolocumab Antibodies |
0; 0 | — |
| SECONDARY Serum Evolocumab Concentrations Over Time |
22400; 64900; 25800 | — |
Eligibility Criteria
Inclusion Criteria
- Male or female ≥ 10 to ≤ 17 years of age (before 18th birthday)
- Diagnosis of heterozygous familial hypercholesterolemia
- On an approved statin with stable optimized dose for ≥ 4 weeks
- Other lipid-lowering therapy stable for ≥ 4 weeks (fibrates must be stable for ≥ 6 weeks)
- Fasting LDL-C ≥ 130 mg/dL (3.4 mmol/L)
- Fasting triglycerides ≤ 400 mg/dL (4.5 mmol/L)
Exclusion Criteria
- Type 1 diabetes, or type 2 diabetes that is or poorly controlled
- Uncontrolled hyperthyroidism or hypothyroidism
- Cholesterylester transfer protein (CETP) inhibitor in the last 12 months, or mipomersen or lomitapide in the last 5 months
- Previously received evolocumab or any other investigational therapy to inhibit proprotein convertase subtilisin/kexin type 9 (PCSK9).
- Lipid apheresis within the last 12 weeks prior to screening.
- Homozygous familial hypercholesterolemia
Data sourced from ClinicalTrials.gov (NCT02392559) and the linked publication. Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.