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

November 10, 2022 updated by: Amgen

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

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).

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

168

Phase

  • Phase 2

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years to 75 years (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / 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:
  • Repatha
  • AMG 145
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:
  • Repatha
  • AMG 145

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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.
Baseline and Week 12

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Percent Change From Baseline in Apolipoprotein B at Week 12
Time Frame: Baseline and Week 12
Baseline and Week 12
Percent Change From Baseline in the Total Cholesterol/HDL-C Ratio at Week 12
Time Frame: Baseline and Week 12
Baseline and Week 12
Absolute Change From Baseline in LDL-C at Week 12
Time Frame: Baseline and Week 12
LDL-C was measured using ultracentrifugation.
Baseline and Week 12
Percent Change From Baseline in Non-High Density Lipoprotein Cholesterol (HDL-C) at Week 12
Time Frame: Baseline and Week 12
Baseline and Week 12
Percent Change From Baseline in Apolipoprotein B /Apolipoprotein A-1 Ratio at Week 12
Time Frame: Baseline and Week 12
Baseline and Week 12

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Amgen

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

August 2, 2011

Primary Completion (Actual)

May 16, 2012

Study Completion (Actual)

May 16, 2012

Study Registration Dates

First Submitted

June 16, 2011

First Submitted That Met QC Criteria

June 16, 2011

First Posted (Estimate)

June 17, 2011

Study Record Updates

Last Update Posted (Actual)

November 15, 2022

Last Update Submitted That Met QC Criteria

November 10, 2022

Last Verified

November 1, 2022

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 20090158

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

Clinical Trials on Hypercholesterolemia, Familial

Clinical Trials on Evolocumab

Search Similar Trials

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Israel

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations

3
Subscribe