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Completed

NCT Number: NCT02624869

Safety, Tolerability and Efficacy of Evolocumab (AMG 145) in Children With Inherited Elevated Low-density Lipoprotein Cholesterol (Familial Hypercholesterolemia)

The main purpose of this study is to describe the safety and tolerability of 80 weeks of subcutaneous (SC) evolocumab when added to standard of care in children 10 to 17 years of age with familial hypercholesterolemia.

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Key information

Conditions

Familial Hypercholesterolemia Congenital, Hereditary, and Neonatal Diseases and Abnormalities Dyslipidemias Genetic Diseases, Inborn Homozygous Familial Hypercholesterolemia Hypercholesterolemia Hyperlipidemias Hyperlipoproteinemia Type II Hyperlipoproteinemias Lipid Metabolism Disorders Lipid Metabolism, Inborn Errors Metabolic Diseases Metabolism, Inborn Errors 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

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

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

Inclusion criteria

Heterozygous Familial Hypercholesterolemia (HeFH):

-Completed Study 20120123 (NCT02392559) while still on assigned investigational product and did not experience a treatment-related serious adverse event

Homozygous Familial Hypercholesterolemia (HoFH):

  • Male or female, ≥ 10 to ≤ 17 years of age at time of enrollment
  • Diagnosis of HoFH
  • On a low-fat diet and receiving background lipid-lowering therapy
  • Lipid-lowering therapy unchanged for ≥ 4 weeks prior to LDL-C screening; fibrates must be stable for at least 6 weeks prior to screening.
  • Fasting LDL-C at screening ≥ 130 mg/dL (3.4 mmol/L)
  • Fasting triglycerides ≤ 400 mg/dL (4.5 mmol/L)

Exclusion criteria

-Currently receiving treatment in another investigational device or drug study, or less than 30 days since ending treatment on another investigational device or drug study(s); except Study 20120123

HoFH:

  • Moderate to severe renal dysfunction
  • Active liver disease or hepatic dysfunction,
  • Creatine kinase > 3 times the upper limit of normal (ULN) at screening

Treatment and study plan

Evolocumab

Biological

Administered by subcutaneous injection

Other names: Repatha®, AMG 145

Primary outcomes

  1. Number of Participants With Treatment-emergent Adverse Events (TEAEs)

    Time frame: From first dose of evolocumab in this study up to and including 30 days after the last dose or up to the end of study date, whichever was earlier; up to 80 weeks.

    An adverse event is defined as any untoward medical occurrence in a clinical trial participant, not necessarily having a causal relationship with study treatment.

    A serious AE is as an AE that met at least 1 of the following criteria:

    • fatal;
    • life threatening;
    • required in-patient hospitalization or prolongation of existing hospitalization;
    • resulted in persistent or significant disability/incapacity;
    • congenital anomaly/birth defect;
    • other medically important serious event.

    AEs were graded for severity using the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) version 4.0:

    Grade 1: Mild; asymptomatic or mild symptoms; Grade 2: Moderate; minimal, local or noninvasive intervention indicated; Grade 3: Severe or medically significant but not immediately life-threatening; hospitalization or prolongation of hospitalization indicated; Grade 4: Life-threatening consequences; urgent intervention indicated; Grade 5: Death related to AE.

Secondary outcomes

  1. Percent Change From Baseline to Week 80 in Low-density Lipoprotein Cholesterol (LDL-C) in HeFH Participants

    Time frame: Baseline and week 80

    For HeFH participants baseline was defined as the baseline value of the parent study 20120123.

  2. Percent Change From Baseline to Week 80 in Low-density Lipoprotein Cholesterol (LDL-C) in HoFH Participants

    Time frame: Baseline and week 80

    For HoFH participants baseline was defined as the baseline value in this study (20120124).

  3. Percent Change From Baseline to Week 80 in Non-HDL-C in HeFH Participants

    Time frame: Baseline and week 80

    For HeFH participants baseline was defined as the baseline value of the parent study 20120123.

  4. Percent Change From Baseline to Week 80 in Non-HDL-C in HoFH Participants

    Time frame: Baseline and week 80

    For HoFH participants baseline was defined as the baseline value in this study (20120124).

  5. Percent Change From Baseline to Week 80 in Apolipoprotein B in HeFH Participants

    Time frame: Baseline and week 80

    For HeFH participants baseline was defined as the baseline value in the parent study 20120123.

  6. Percent Change From Baseline to Week 80 in Apolipoprotein B in HoFH Participants

    Time frame: Baseline and week 80

    For HoFH participants baseline was defined as the baseline value in this study (20120124).

  7. Percent Change From Baseline to Week 80 in Total Cholesterol/HDL-C Ratio in HeFH Participants

    Time frame: Baseline and week 80

    For HeFH participants baseline was defined as the baseline value in the parent study 20120123.

  8. Percent Change From Baseline to Week 80 in Total Cholesterol/HDL-C Ratio in HoFH Participants

    Time frame: Baseline and week 80

    For HoFH participants baseline was defined as the baseline value in this study (20120124).

  9. Percent Change From Baseline to Week 80 in Apolipoprotein B / Apolipoprotein A1 Ratio in HeFH Participants

    Time frame: Baseline and week 80

    For HeFH participants baseline was defined as the baseline value in the parent study 20120123.

  10. Percent Change From Baseline to Week 80 in Apolipoprotein B/Apolipoprotein A1 Ratio in HoFH Participants

    Time frame: Baseline and week 80

    For HoFH participants baseline was defined as the baseline value in this study (20120124).

  11. Change From Baseline to Week 80 in LDL-C in HeFH Participants

    Time frame: Baseline and week 80

    For HeFH participants baseline was defined as the baseline value of the parent study 20120123.

  12. Change From Baseline to Week 80 in LDL-C in HoFH Participants

    Time frame: Baseline and week 80

    For HoFH participants baseline was defined as the baseline value in this study (20120124).

  13. Change From Baseline to Week 80 in Estradiol Levels

    Time frame: Baseline and week 80

    For HeFH participants baseline was defined as the baseline value in the parent study 20120123. For HoFH participants baseline was defined as the baseline value in this study (20120124).

  14. Change From Baseline to Week 80 in Testosterone Levels

    Time frame: Baseline and week 80

    For HeFH participants baseline was defined as the baseline value in the parent study 20120123. For HoFH participants baseline was defined as the baseline value in this study (20120124).

  15. Change From Baseline to Week 80 in Follicle Stimulating Hormone (FSH) Levels

    Time frame: Baseline and week 80

    For HeFH participants baseline was defined as the baseline value in the parent study 20120123. For HoFH participants baseline was defined as the baseline value in this study (20120124).

  16. Change From Baseline to Week 80 in Luteinizing Hormone (LH) Levels

    Time frame: Baseline and week 80

    For HeFH participants baseline was defined as the baseline value in the parent study 20120123. For HoFH participants baseline was defined as the baseline value in this study (20120124).

  17. Change From Baseline to Week 80 in Adenocorticotropic Hormone (ACTH) Levels

    Time frame: Baseline and week 80

    For HeFH participants baseline was defined as the baseline value in the parent study 20120123. For HoFH participants baseline was defined as the baseline value in this study (20120124).

  18. Change From Baseline to Week 80 in Dehydroepiandrosterone Sulfate (DHEA-S) Levels

    Time frame: Baseline and week 80

    For HeFH participants baseline was defined as the baseline value in the parent study 20120123. For HoFH participants baseline was defined as the baseline value in this study (20120124).

  19. Change From Baseline to Week 80 in Cortisol Levels

    Time frame: Baseline and week 80

    For HeFH participants baseline was defined as the baseline value in the parent study 20120123. For HoFH participants baseline was defined as the baseline value in this study (20120124).

  20. Number of Participants With Liver Function Test Abnormalities at Week 80

    Time frame: Week 80

    Liver function tests included alanine aminotransferase (ALT) levels, aspartate aminotransferase (AST) levels and total bilirubin levels.

  21. Number of Participants With Abnormalities in Levels of Creatine Kinase (CK) at Week 80

    Time frame: Week 80

    The number of participants with levels of creatine kinase greater than 5 times the upper limit of normal (ULN) and greater than 10 times the ULN, measured by the central laboratory.

  22. Change From Baseline to Week 80 in Carotid Intima-media Thickness (cIMT)

    Time frame: Baseline and week 80

    Carotid intima-media thickness measures the thickness of the intima and media, the inner two layers of the carotid artery, and is used to determine the extent of plaque buildup in the walls of the arteries (atherosclerosis) supplying blood to the head.

    CIMT was measured by ultrasonography and analyzed at a core laboratory. The largest values measured in the left common carotid artery (LCCA) and the right common carotid artery (RCCA) are averaged in this analysis.

  23. Change From Baseline in Height at Weeks 24, 48, and 80

    Time frame: Baseline and weeks 24, 48, and 80

  24. Change From Baseline in Weight at Weeks 24, 48, and 80

    Time frame: Baseline and weeks 24, 48, and 80

  25. Number of Participants With Change in Tanner Staging From Baseline to Week 80

    Time frame: Baseline and week 80

    Pubertal growth and sexual maturity was assessed separately for males and females using the 5 Tanner stages where stage 1 = prepubertal and stage 5 = mature.

    The number of participants with any change in Tanner Stage from baseline is reported.

Sponsors and collaborators

Lead sponsor

Amgen

Industry

Registry information

Official study title

Open-label, Single-Arm, Multicenter Study to Evaluate the Safety, Tolerability and Efficacy of Evolocumab for LDL-C Reduction, as Add-on to Diet and Lipid-lowering Therapy, in Pediatric Subjects From 10 to 17 Years of Age With Heterozygous Familial Hypercholesterolemia (HeFH) or Homozygous Familial Hypercholesterolemia (HoFH)

Acronym: HAUSER-OLE

Important dates

Study start
2016
Primary completion
2021
Study completion
2021
First posted
Dec 9, 2015
Registry last updated
May 29, 2024

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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