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Completed

NCT Number: NCT01375751

Reduction of Low-Density Lipoprotein Cholesterol (LDL-C) With PCSK9 Inhibition in Heterozygous Familial Hypercholesterolemia Disorder Study

The primary objective of this study was to evaluate the effect of 12 weeks of subcutaneous evolocumab (AMG 145), compared with placebo, on percent change from baseline in LDL-C in adults with heterozygous familial hypercholesterolemia (HeFH).

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Key information

Conditions

Hypercholesterolemia, Familial Congenital, Hereditary, and Neonatal Diseases and Abnormalities Dyslipidemias Genetic Diseases, Inborn Hypercholesterolemia, Autosomal Dominant, 3 Hyperlipidemias Hyperlipoproteinemia Type II Hyperlipoproteinemias Lipid Metabolism Disorders Lipid Metabolism, Inborn Errors Metabolic Diseases Metabolism, Inborn Errors Nutritional and Metabolic Diseases

Age range

18 year–75 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Who can participate

Healthy volunteers accepted: No

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

Inclusion criteria

  • Male or female ≥ 18 to ≤ 75 years of age
  • Diagnosis of heterozygous familial hypercholesterolemia by having met the diagnostic criteria outlined by the Simon Broome Register Group (Scientific Steering Committee 1991)
  • On an approved statin, with or without ezetimibe, with stable dose(s) for at least 4 weeks
  • Fasting Low-Density Lipoprotein Cholesterol (LDL-C) ≥ 100 mg/dL
  • Fasting triglycerides ≤ 400 mg/dL

Exclusion criteria

  • Homozygous familial hypercholesterolemia
  • Low-Density Lipoprotein (LDL) or plasma apheresis within 12 months prior to randomization
  • New York Heart Association (NYHA) III or IV heart failure, or known left ventricular ejection fraction < 30%
  • Uncontrolled cardiac arrhythmia
  • Myocardial infarction, unstable angina, percutaneous coronary intervention (PCI), coronary artery bypass graft (CABG) or stroke within 3 months prior to randomization
  • Type 1 diabetes; newly diagnosed or poorly controlled type 2 diabetes (HbA1c > 8.5%)
  • Uncontrolled hypertension

Treatment and study plan

Evolocumab

Biological

Administered by subcutaneous injection

Other names: AMG 145, Repatha

Placebo

Biological

d by subcutaneous injection

Primary outcomes

  1. Percent Change From Baseline in Low-Density Lipoprotein Cholesterol (LDL-C) at Week 12

    Time frame: Baseline and Week 12

    LDL-C was measured using ultracentrifugation.

Secondary outcomes

  1. Absolute Change From Baseline in LDL-C at Week 12

    Time frame: Baseline and Week 12

    LDL-C was measured using ultracentrifugation.

  2. Percent Change From Baseline in Non-High Density Lipoprotein Cholesterol (HDL-C) at Week 12

    Time frame: Baseline and Week 12

  3. Percent Change From Baseline in Apolipoprotein B at Week 12

    Time frame: Baseline and Week 12

  4. Percent Change From Baseline in the Total Cholesterol/HDL-C Ratio at Week 12

    Time frame: Baseline and Week 12

  5. Percent Change From Baseline in Apolipoprotein B /Apolipoprotein A-1 Ratio at Week 12

    Time frame: Baseline and Week 12

Sponsors and collaborators

Lead sponsor

Amgen

Industry

Registry information

Official study title

A Double-blind, Randomized, Placebo-controlled, Multicenter Study to Evaluate Tolerability and Efficacy of AMG 145 on LDL-C in Subject With Heterozygous Familial Hypercholesterolemia

Acronym: RUTHERFORD

Important dates

Study start
2011
Primary completion
2012
Study completion
2012
First posted
Jun 17, 2011
Registry last updated
Nov 15, 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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