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 (HAUSER-RCT)

Amgen

Status and phase: Completed; Phase 3

Conditions: Heterozygous Familial Hypercholesterolemia

Treatments: Drug: Placebo; Drug: Evolocumab

Study type: Interventional

Funder types: Industry

Identifiers

NCT02392559

20120123

2014-002277-11 (EudraCT Number)

Details and patient eligibility

About

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

Full description

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 heterozygous familial hypercholesterolemia (HeFH).

Enrollment: 158 patients

Sex: All

Ages: 10 to 17 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Triple Blind

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158 participants in 2 patient groups, including a placebo group

Placebo

Placebo Comparator group

Description:

Matching subcutaneous injection every 4 weeks (QM)

Treatment:

Drug: Placebo

EvoMab 420 mg QM

Experimental group

Description:

Evolocumab subcutaneous injection QM

Treatment:

Drug: Evolocumab