Phase 3 N=158 Randomized Triple-blind Treatment

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

Bottom Line

View on ClinicalTrials.gov: NCT02392559 ↗

Enrolled (actual)

158

Serious AEs

0.6%

Results posted

Jul 2020

Primary outcome: Primary: Percent Change From Baseline to Week 24 in LDL-C — -6.23; -44.53 percent change — p=< 0.0001

Study Design & Population

Study type: Interventional
Phase: Phase 3
Interventions: Evolocumab (Drug); Placebo (Drug)
Age: Pediatric · 10+ yrs
Sex: All
Sponsor: Amgen
Primary completion: Nov 2019

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	—

Summary

A study to assess safety and efficacy of evolocumab (AMG-145) in paediatric subjects aged 10-17 years diagnosed with heterozygous familial hypercholesterolemia.

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

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT02392559). 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.