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Reduction of LDL-C With PCSK9 Inhibition in Heterozygous Familial Hypercholesterolemia Disorder Study-2 (RUTHERFORD-2)
This study has been completed.
Sponsor:
Amgen
Information provided by (Responsible Party):
Amgen
ClinicalTrials.gov Identifier:
NCT01763918
First received: January 7, 2013
Last updated: February 7, 2017
Last verified: February 2017
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Purpose
The primary objective was to evaluate the effect of 12 weeks of evolocumab subcutaneously once every 2 weeks (Q2W) and once monthly (QM), compared with placebo, on percent change from baseline in low-density lipoprotein cholesterol (LDL-C) in adults with heterozygous familial hypercholesterolemia (HeFH).
| Condition | Intervention | Phase |
|---|---|---|
| Hyperlipidemia | Biological: Evolocumab Drug: Placebo | Phase 3 |
| 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 Safety, Tolerability and Efficacy of AMG 145 on LDL-C in Subjects With Heterozygous Familial Hypercholesterolemia |
Resource links provided by NLM:
Genetics Home Reference related topics:
hypercholesterolemia
MedlinePlus related topics:
Cholesterol
Drug Information available for:
Evolocumab
U.S. FDA Resources
Further study details as provided by Amgen:
Primary Outcome Measures:
- 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 ]
Secondary Outcome Measures:
- 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 ]
| Enrollment: | 331 |
| Study Start Date: | February 2013 |
| Primary Completion Date: | November 2013 (Final data collection date for primary outcome measure) |
| Arms | Assigned Interventions |
|---|---|
|
Placebo Comparator: Placebo Q2W
Participants received placebo subcutaneous injection once every 2 weeks (Q2W) for up to 12 weeks.
|
Drug: Placebo
Administered by subcutaneous injection
|
|
Placebo Comparator: Placebo QM
Participants received placebo subcutaneous injection once every month (QM) for up to 12 weeks.
|
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:
|
|
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:
|
Eligibility| Ages Eligible for Study: | 18 Years to 80 Years (Adult, Senior) |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | No |
Criteria
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
Contacts and Locations
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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: NCT01763918
Show 39 Study Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01763918
Show 39 Study Locations
Sponsors and Collaborators
Amgen
Investigators
| Study Director: | MD | Amgen |
More Information
Additional Information:
Publications:
| Responsible Party: | Amgen |
| ClinicalTrials.gov Identifier: | NCT01763918 History of Changes |
| Other Study ID Numbers: |
20110117 2012-001365-32 ( EudraCT Number ) |
| Study First Received: | January 7, 2013 |
| Results First Received: | September 2, 2015 |
| Last Updated: | February 7, 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 |
ClinicalTrials.gov processed this record on August 18, 2017