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

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ClinicalTrials.gov Identifier: NCT01375751
Recruitment Status : Completed
First Posted : June 17, 2011
Results First Posted : September 29, 2015
Last Update Posted : November 2, 2018
Sponsor:
Information provided by (Responsible Party):
Amgen

Brief Summary:
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 or disease Intervention/treatment Phase
Hypercholesterolemia, Familial Biological: Evolocumab Biological: Placebo Phase 2

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 168 participants
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
Actual Study Start Date : August 2, 2011
Actual Primary Completion Date : May 16, 2012
Actual Study Completion Date : May 16, 2012

Resource links provided by the National Library of Medicine

Drug Information available for: Evolocumab

Arm Intervention/treatment
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




Primary Outcome Measures :
  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 Outcome Measures :
  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 ]


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Ages Eligible for Study:   18 Years to 75 Years   (Adult, Older Adult)
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01375751


Sponsors and Collaborators
Amgen
Investigators
Study Director: MD Amgen

Additional Information:
Publications:
Responsible Party: Amgen
ClinicalTrials.gov Identifier: NCT01375751     History of Changes
Other Study ID Numbers: 20090158
First Posted: June 17, 2011    Key Record Dates
Results First Posted: September 29, 2015
Last Update Posted: November 2, 2018
Last Verified: November 2018

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
Antibodies, Monoclonal
Immunologic Factors
Physiological Effects of Drugs