Trial Assessing Efficacy, Safety and Tolerability of PCSK9 Inhibition in Paediatric Subjects With Genetic LDL Disorders

A study to evaluate the effect of 24 weeks of subcutaneous (SC) evolocumab compared with
placebo,when added to standard of care, on percent change from baseline in low‑density
lipoprotein cholesterol (LDL-C) in pediatric subjects 10 to 17 years of age with HeFH

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 PCSK9.

- Lipid apheresis within the last 12 weeks prior to screening.

- Homozygous Familial Hypercholesterolemia