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

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Amgen
ClinicalTrials.gov Identifier:
NCT01375751
First received: June 16, 2011
Last updated: August 28, 2015
Last verified: August 2015
  Purpose
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).

Condition Intervention Phase
Hypercholesterolemia, Familial Biological: Evolocumab Biological: Placebo Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official 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

Resource links provided by NLM:


Further study details as provided by Amgen:

Primary Outcome Measures:
  • 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 Outcome Measures:
  • Absolute Change From Baseline in LDL-C at Week 12 [ Time Frame: Baseline and Week 12 ]
    LDL-C was measured using ultracentrifugation.

  • Percent Change From Baseline in Non-High Density Lipoprotein Cholesterol (HDL-C) at Week 12 [ Time Frame: Baseline and Week 12 ]
  • Percent Change From Baseline in Apolipoprotein B at Week 12 [ Time Frame: Baseline and Week 12 ]
  • Percent Change From Baseline in the Total Cholesterol/HDL-C Ratio at Week 12 [ Time Frame: Baseline and Week 12 ]
  • Percent Change From Baseline in Apolipoprotein B /Apolipoprotein A-1 Ratio at Week 12 [ Time Frame: Baseline and Week 12 ]

Enrollment: 168
Study Start Date: August 2011
Study Completion Date: May 2012
Primary Completion Date: May 2012 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Placebo Comparator: Placebo
Participants received placebo subcutaneous injection once every 4 weeks for 12 weeks.
Biological: Placebo
d by subcutaneous injection
Experimental: Evolocumab 350 mg
Participants received 350 mg evolocumab by subcutaneous injection once every 4 weeks for 12 weeks.
Biological: Evolocumab
Administered by subcutaneous injection
Other Names:
  • AMG 145
  • Repatha
Experimental: Evolocumab 420 mg
Participants received 420 mg evolocumab by subcutaneous injection once every 4 weeks for 12 weeks.
Biological: Evolocumab
Administered by subcutaneous injection
Other Names:
  • AMG 145
  • Repatha

  Eligibility

Ages Eligible for Study:   18 Years to 75 Years   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01375751

Sponsors and Collaborators
Amgen
Investigators
Study Director: MD Amgen
  More Information

Additional Information:
Publications:
Responsible Party: Amgen
ClinicalTrials.gov Identifier: NCT01375751     History of Changes
Other Study ID Numbers: 20090158
Study First Received: June 16, 2011
Results First Received: August 28, 2015
Last Updated: August 28, 2015

Keywords provided by Amgen:
Heterozygous Familial Hypercholesterolemia
Proprotein convertase subtilisin/kexin type 9 (PCSK9)

Additional relevant MeSH terms:
Hypercholesterolemia
Hyperlipoproteinemia Type II
Hyperlipidemias
Dyslipidemias
Lipid Metabolism Disorders
Metabolic Diseases
Lipid Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Hyperlipoproteinemias

ClinicalTrials.gov processed this record on September 19, 2017