Reduction of LDL-C With PCSK9 Inhibition in Heterozygous Familial Hypercholesterolemia Disorder Study-2 (RUTHERFORD-2)
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ClinicalTrials.gov Identifier: NCT01763918 |
Recruitment Status
:
Completed
First Posted
: January 9, 2013
Results First Posted
: December 22, 2015
Last Update Posted
: March 15, 2017
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Condition or disease | Intervention/treatment | Phase |
---|---|---|
Hyperlipidemia | Biological: Evolocumab Drug: Placebo | Phase 3 |
Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 331 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 Safety, Tolerability and Efficacy of AMG 145 on LDL-C in Subjects With Heterozygous Familial Hypercholesterolemia |
Study Start Date : | February 2013 |
Actual Primary Completion Date : | November 2013 |

Arm | Intervention/treatment |
---|---|
Placebo Comparator: Placebo Q2W
Participants received placebo subcutaneous injection once every 2 weeks (Q2W) for up to 12 weeks.
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Drug: Placebo
Administered by subcutaneous injection
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Placebo Comparator: Placebo QM
Participants received placebo subcutaneous injection once every month (QM) for up to 12 weeks.
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Drug: Placebo
Administered by subcutaneous injection
|
Experimental: Evolocumab Q2W
Participants received 140 mg evolocumab by subcutaneous injection once every 2 weeks for up to 12 weeks.
|
Biological: Evolocumab
Administered by subcutaneous injection
Other Names:
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Experimental: Evolocumab QM
Participants received 420 mg evolocumab by subcutaneous injection once a month for up to 12 weeks.
|
Biological: Evolocumab
Administered by subcutaneous injection
Other Names:
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- Percent Change From Baseline in LDL-C at Week 12 [ Time Frame: Baseline and Week 12 ]
- Percent Change From Baseline in LDL-C at the Mean of Weeks 10 and 12 [ Time Frame: Baseline and Weeks 10 and 12 ]
- Change From Baseline in LDL-C at the Mean of Weeks 10 and 12 [ Time Frame: Baseline and Weeks 10 and 12 ]
- Change From Baseline in LDL-C at Week 12 [ Time Frame: Baseline and Week 12 ]
- Percentage of Participants With Mean LDL-C at Weeks 10 and 12 of Less Than 70 mg/dL (1.8 mmol/L) [ Time Frame: Weeks 10 and 12 ]
- Percentage of Participants With LDL-C < 70 mg/dL (1.8 mmol/L) at Week 12 [ Time Frame: Week 12 ]
- Percent Change From Baseline in Non-High-Density Lipoprotein Cholesterol (Non-HDL-C) at the Mean of Weeks 10 and 12 [ Time Frame: Baseline and Weeks 10 and 12 ]
- Percent Change From Baseline in Non-HDL-C at Week 12 [ Time Frame: Baseline and Week 12 ]
- Percent Change From Baseline in Apolipoprotein B at the Mean of Weeks 10 and 12 [ Time Frame: Baseline and Weeks 10 and 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 the Mean of Weeks 10 and 12 [ Time Frame: Baseline and Weeks 10 and 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 A1 Ratio at the Mean of Weeks 10 and 12 [ Time Frame: Baseline and Weeks 10 and 12 ]
- Percent Change From Baseline in Apolipoprotein B/Apolipoprotein A1 Ratio at Week 12 [ Time Frame: Baseline and Week 12 ]
- Percent Change From Baseline in Lipoprotein (a) at the Mean of Weeks 10 and 12 [ Time Frame: Baseline and Weeks 10 and 12 ]
- Percent Change From Baseline in Lipoprotein (a) at Week 12 [ Time Frame: Baseline and Week 12 ]
- Percent Change From Baseline in Triglycerides at the Mean of Weeks 10 and 12 [ Time Frame: Baseline and Weeks 10 and 12 ]
- Percent Change From Baseline in Triglycerides at Week 12 [ Time Frame: Baseline and Week 12 ]
- Percent Change From Baseline in HDL-C at the Mean of Weeks 10 and 12 [ Time Frame: Baseline and Weeks 10 and 12 ]
- Percent Change From Baseline in HDL-C at Week 12 [ Time Frame: Baseline and Week 12 ]
- Percent Change From Baseline in Very Low-Density Lipoprotein Cholesterol (VLDL-C) at the Mean of Weeks 10 and 12 [ Time Frame: Baseline and Weeks 10 and 12 ]
- Percent Change From Baseline in VLDL-C at Week 12 [ Time Frame: Baseline and Week 12 ]

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Ages Eligible for Study: | 18 Years to 80 Years (Adult, Senior) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- Male or female ≥ 18 to ≤ 80 years of age
- Diagnosis of heterozygous familial hypercholesterolemia
- On a stable dose of an approved statin and lipid regulating medication
- Fasting LDL-C ≥ 100 mg/dL (2.6 mmol/L)
- Fasting triglycerides ≤ 400 mg/dL (4.5 mmol/L)
Exclusion Criteria:
- Homozygous familial hypercholesterolemia
- LDL or plasma apheresis
- New York Heart Association (NYHA) III or IV heart failure
- Uncontrolled cardiac arrhythmia
- Uncontrolled hypertension
- Type 1 diabetes, poorly controlled type 2 diabetes
- Uncontrolled hypothyroidism or hyperthyroidism

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): NCT01763918

Study Director: | MD | Amgen |
Additional Information:
Publications:
Responsible Party: | Amgen |
ClinicalTrials.gov Identifier: | NCT01763918 History of Changes |
Other Study ID Numbers: |
20110117 2012-001365-32 ( EudraCT Number ) |
First Posted: | January 9, 2013 Key Record Dates |
Results First Posted: | December 22, 2015 |
Last Update Posted: | March 15, 2017 |
Last Verified: | February 2017 |
Keywords provided by Amgen:
High cholesterol, Treatment for high cholesterol, Lowering cholesterol, Lowering high cholesterol, Hypercholesterolemia |
Additional relevant MeSH terms:
Hypercholesterolemia Hyperlipidemias Hyperlipoproteinemias Hyperlipoproteinemia Type II Dyslipidemias Lipid Metabolism Disorders Metabolic Diseases |
Lipid Metabolism, Inborn Errors Metabolism, Inborn Errors Genetic Diseases, Inborn Antibodies, Monoclonal Immunologic Factors Physiological Effects of Drugs |