Reduction of LDL-C With PCSK9 Inhibition in Heterozygous Familial Hypercholesterolemia Disorder Study-2 (RUTHERFORD-2)

Amgen

Status and phase: Completed; Phase 3

Conditions: Hyperlipidemia

Treatments: Drug: Placebo; Biological: Evolocumab

Study type: Interventional

Funder types: Industry

Identifiers

NCT01763918

20110117

2012-001365-32 (EudraCT Number)

Details and patient eligibility

About

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).

Enrollment: 331 patients

Sex: All

Ages: 18 to 80 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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331 participants in 4 patient groups, including a placebo group

Placebo Q2W

Placebo Comparator group

Description:

Participants received placebo subcutaneous injection once every 2 weeks (Q2W) for up to 12 weeks.

Treatment:

Drug: Placebo

Placebo QM

Placebo Comparator group

Description:

Participants received placebo subcutaneous injection once every month (QM) for up to 12 weeks.

Treatment:

Drug: Placebo

Evolocumab Q2W

Experimental group

Description:

Participants received 140 mg evolocumab by subcutaneous injection once every 2 weeks for up to 12 weeks.

Treatment:

Biological: Evolocumab

Evolocumab QM

Experimental group

Description:

Participants received 420 mg evolocumab by subcutaneous injection once a month for up to 12 weeks.

Treatment:

Biological: Evolocumab