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Trial Assessing Efficacy, Saf...

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

Last Updated: 2022-11-08

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

158 participants

Study Classification

INTERVENTIONAL

Study Start Date

2016-03-24

Study Completion Date

2019-11-25

Brief Summary

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

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Detailed Description

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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).

Conditions

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Heterozygous Familial Hypercholesterolemia

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

TRIPLE

Participants Caregivers Investigators

Study Groups

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Placebo

Matching subcutaneous injection every 4 weeks (QM)

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

Dose of subcutaneous placebo treatment every 4 weeks

EvoMab 420 mg QM

Evolocumab subcutaneous injection QM

Group Type EXPERIMENTAL

Evolocumab

Intervention Type DRUG

Dose of subcutaneous evolocumab every 4 weeks

Interventions

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Evolocumab

Dose of subcutaneous evolocumab every 4 weeks

Intervention Type DRUG

Placebo

Dose of subcutaneous placebo treatment every 4 weeks

Intervention Type DRUG

Other Intervention Names

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AMG 145; EvoMab

Eligibility Criteria

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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

Minimum Eligible Age

10 Years

Maximum Eligible Age

17 Years

Eligible Sex

ALL

Accepts Healthy Volunteers