Reduction of LDL-C With PCSK9 Inhibition in Heterozygous Familial Hypercholesterolemia Disorder Study

• Register: ClinicalTrials.gov
• Last refreshed on: 25 February 2013
• Main ID: NCT01375751
• Date of registration: 16/06/2011
• Primary sponsor: Amgen
• Public title: Reduction of LDL-C With PCSK9 Inhibition in Heterozygous Familial Hypercholesterolemia Disorder Study RUTHERFORD
• Scientific title: A Double-blind, Randomized, Placebo-controlled, Multicenter Study to Evaluate Tolerability and Efficacy of AMG 145 on Low-Density Lipoprotein Cholesterol (LDL-C) in Subject With Heterozygous Familial Hypercholesterolemia
• Date of first enrolment: July 2011
• Target sample size: 168
• Recruitment status: Completed
• URL: http://clinicaltrials.gov/show/NCT01375751
• Study type: Interventional
• Study design: Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Primary Purpose: Treatment

Countries of recruitment

Canada, Germany, Hong Kong, Netherlands, Norway, Singapore, South Africa, Spain, Sweden, United Kingdom, United States

Contacts

• Name: MD
• Affiliation: Amgen

Key inclusion & exclusion 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



Age minimum: 18 Years
Age maximum: 75 Years
Gender: Both

Health Condition(s) or Problem(s) studied

Hypercholesterolemia, Familial

Intervention(s)

Drug: AMG 145

Drug: PLACEBO

Primary Outcome(s)

The percent change from baseline in Low-Density Lipoprotein Cholesterol (LDL-C) at week 12 [Time Frame: 12 weeks]

Secondary Outcome(s)

Absolute change from baseline in Low-Density Lipoprotein Cholesterol (LDL-C) at week 12 [Time Frame: 12 weeks]

Percent change from baseline in Apolipoprotein B (ApoB) at week 12 [Time Frame: 12 weeks]

Percent change from baseline in Apolipoprotein B (ApoB)/Apolipoprotein A-1 (ApoA1) ratio at week 12 [Time Frame: 12 weeks]

Percent change from baseline in non-High Density Lipoprotein Cholesterol (HDL-C) at week 12 [Time Frame: 12 weeks]

Percent change from baseline in the total cholesterol/High Density Lipoprotein Cholesterol (HDL-C) at week 12 [Time Frame: 12 weeks]

Secondary ID(s)

20090158

Source(s) of Monetary Support

Please refer to primary and secondary sponsors