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Completed

NCT Number: NCT02392559

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

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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Key information

Conditions

Heterozygous Familial Hypercholesterolemia Dyslipidemias Hypercholesterolemia Hyperlipidemias Lipid Metabolism Disorders Metabolic Diseases Nutritional and Metabolic Diseases

Age range

10 year–17 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

Research Site, Camperdown, New South Wales, Australia

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About this study

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).

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Evolocumab

Drug

Dose of subcutaneous evolocumab every 4 weeks

Other names: AMG 145; EvoMab

Placebo

Drug

Dose of subcutaneous placebo treatment every 4 weeks

Primary outcomes

  1. Percent Change From Baseline to Week 24 in LDL-C

    Time frame: Baseline, Week 24

    Least squares mean is from the repeated measures model which includes treatment group, stratification factors of age and screening LDL-C (from interactive voice response system [IVRS]), scheduled visit and the interaction of treatment with scheduled visit as covariates. The model uses an unstructured covariance.

Secondary outcomes

  1. Mean Percent Change From Baseline to Mean of Weeks 22 and 24 in LDL-C

    Time frame: Baseline, Week 22, Week 24

    Least squares mean is from the repeated measures model which includes treatment group, stratification factors of age and screening LDL-C (from IVRS), scheduled visit and the interaction of treatment with scheduled visit as covariates.

  2. Change From Baseline to Week 24 in LDL-C

    Time frame: Baseline, Week 24

    Least squares mean is from the repeated measures model which includes treatment group, stratification factors of age and screening LDL-C (from IVRS), scheduled visit and the interaction of treatment with scheduled visit as covariates.

  3. Percent Change From Baseline to Week 24 in Non-HDL-C

    Time frame: Baseline, Week 24

    Least squares mean is from the repeated measures model which includes treatment group, stratification factors of age and screening LDL-C (from IVRS), scheduled visit and the interaction of treatment with scheduled visit as covariates

  4. Percent Change From Baseline to Week 24 in Apoliprotein-B (ApoB)

    Time frame: Baseline, Week 24

    Least squares mean is from the repeated measures model which includes treatment group, stratification factors of age and screening LDL-C (from IVRS), scheduled visit and the interaction of treatment with scheduled visit as covariates.

  5. Percent Change From Baseline to Week 24 in Total Cholesterol/HDL-C Ratio

    Time frame: Baseline, Week 24

    Least squares mean is from the repeated measures model which includes treatment group, stratification factors of age and screening LDL-C (from IVRS), scheduled visit and the interaction of treatment with scheduled visit as covariates.

  6. Percent Change From Baseline to Week 24 in ApoB:ApoA1 Ratio

    Time frame: Baseline, Week 24

    Least squares mean is from the repeated measures model which includes treatment group, stratification factors of age and screening LDL-C (from IVRS), scheduled visit and the interaction of treatment with scheduled visit as covariates.

  7. Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Serious TEAEs, TEAEs Leading to Discontinuation (DC), Fatal TEAEs, and Device-Related TEAEs

    Time frame: From first dose of study drug up to and including 30 days after the last dose or end of study date (Week 24), whichever was earlier. Mean (SD) duration on study was 5.664 (0.278) and 5.608 (0.137) months for Placebo and EvoMab arms, respectively.

    An adverse event (AE) is defined as any untoward medical occurrence that does not necessarily have a causal relationship with study treatment. An SAE is defined as an adverse event that: is fatal; is a life threatening; requires in-patient hospitalization or prolongation of existing hospitalization; results in persistent or significant disability/incapacity; is a congenital anomaly/birth defect; or other medically important serious event. Events were graded by the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) grading scale (1=mild; 2=moderate; 3=severe; 4=life-threatening; 5=death). Events were defined as treatment emergent if they occurred after the first dose of study drug and up to and including 30 days after the last dose or the end of study date, whichever is earlier.

  8. Number of Participants With Maximum Post-Baseline Laboratory Toxicities of Grade ≥ 3

    Time frame: Week 24

    Laboratory toxicity grading was based on NCI CTCAE grading. Grade 3 indicates severe toxicity and Grade 4 indicates life-threatening toxicity. Values representing a worsening from baseline are shown.

  9. Change From Baseline Over Time in Systolic Blood Pressure

    Time frame: Baseline, Week 4, Week 12, Week 20, Week 22, Week 24

  10. Change From Baseline Over Time in Diastolic Blood Pressure

    Time frame: Baseline, Week 4, Week 12, Week 20, Week 22, Week 24

  11. Change From Baseline Over Time in Heart Rate

    Time frame: Baseline, Week 4, Week 12, Week 20, Week 22, Week 24

  12. Number of Participants Testing Positive for Anti-Evolocumab Antibodies

    Time frame: up to Week 24

  13. Serum Evolocumab Concentrations Over Time

    Time frame: Week 12, Week 22, Week 24

Sponsors and collaborators

Lead sponsor

Amgen

Industry

Registry information

Official study title

Double-blind, Randomized, Multicenter, Placebo-Controlled Study to Characterize the Efficacy, Safety, and Tolerability of 24 Weeks of Evolocumab for LDL-C Reduction in Pediatric Subjects 10 to 17 Years of Age With HeFH

Acronym: HAUSER-RCT

Important dates

Study start
2016
Primary completion
2019
Study completion
2019
First posted
Mar 19, 2015
Registry last updated
Nov 8, 2022

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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