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

• Status: Completed
• Trial end date: 2019-11-25
• Target enrollment: 0
• Participant gender: All

Summary

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

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Amgen
• Treatments: Evolocumab

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