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

Phase 3

Completed

• Conditions: Hyperlipidemia
• Interventions: Drug: Placebo; Biological: Evolocumab

• Registration Number: NCT01763918
• Lead Sponsor: Amgen

Brief Summary

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).

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2013-01-07
• Study First Submitted QC: 2013-01-08
• Study First Posted: 2013-01-09
• Study Start: 2013-02-07
• Primary Completion: 2013-11-27
• Study Completion: 2013-12-19
• Disposition First Submitted: 2014-05-02
• Disposition First Submitted QC: 2014-05-02
• Disposition First Posted: 2014-05-20
• Results First Submitted: 2015-09-02
• Results First Submitted QC: 2015-11-18
• Results First Posted: 2015-12-22
• Status Verified: 2019-06
• Last Update Submitted: 2019-06-03
• Last Update Posted: 2019-06-17

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 331

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Placebo Q2W	Placebo	Participants received placebo subcutaneous injection once every 2 weeks (Q2W) for up to 12 weeks.
Placebo QM	Placebo	Participants received placebo subcutaneous injection once every month (QM) for up to 12 weeks.
Evolocumab Q2W	Evolocumab	Participants received 140 mg evolocumab by subcutaneous injection once every 2 weeks for up to 12 weeks.
Evolocumab QM	Evolocumab	Participants received 420 mg evolocumab by subcutaneous injection once a month for up to 12 weeks.

Primary Outcome Measures

Name	Time	Method
Percent Change From Baseline in LDL-C at Week 12	Baseline and Week 12
Percent Change From Baseline in LDL-C at the Mean of Weeks 10 and 12	Baseline and Weeks 10 and 12

Secondary Outcome Measures

Name	Time	Method
Change From Baseline in LDL-C at Week 12	Baseline and Week 12
Percent Change From Baseline in Non-High-Density Lipoprotein Cholesterol (Non-HDL-C) at the Mean of Weeks 10 and 12	Baseline and Weeks 10 and 12
Percent Change From Baseline in Lipoprotein (a) at the Mean of Weeks 10 and 12	Baseline and Weeks 10 and 12
Percent Change From Baseline in Lipoprotein (a) at Week 12	Baseline and Week 12
Percent Change From Baseline in Triglycerides at the Mean of Weeks 10 and 12	Baseline and Weeks 10 and 12
Percent Change From Baseline in Triglycerides at Week 12	Baseline and Week 12
Percent Change From Baseline in HDL-C at the Mean of Weeks 10 and 12	Baseline and Weeks 10 and 12
Percent Change From Baseline in HDL-C at Week 12	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)	Weeks 10 and 12
Percentage of Participants With LDL-C < 70 mg/dL (1.8 mmol/L) at Week 12	Week 12
Change From Baseline in LDL-C at the Mean of Weeks 10 and 12	Baseline and Weeks 10 and 12
Percent Change From Baseline in Non-HDL-C at Week 12	Baseline and Week 12
Percent Change From Baseline in Apolipoprotein B at the Mean of Weeks 10 and 12	Baseline and Weeks 10 and 12
Percent Change From Baseline in Apolipoprotein B at Week 12	Baseline and Week 12
Percent Change From Baseline in the Total Cholesterol/HDL-C Ratio at the Mean of Weeks 10 and 12	Baseline and Weeks 10 and 12
Percent Change From Baseline in the Total Cholesterol/HDL-C Ratio at Week 12	Baseline and Week 12
Percent Change From Baseline in Apolipoprotein B/Apolipoprotein A1 Ratio at the Mean of Weeks 10 and 12	Baseline and Weeks 10 and 12
Percent Change From Baseline in Apolipoprotein B/Apolipoprotein A1 Ratio at Week 12	Baseline and Week 12
Percent Change From Baseline in Very Low-Density Lipoprotein Cholesterol (VLDL-C) at the Mean of Weeks 10 and 12	Baseline and Weeks 10 and 12
Percent Change From Baseline in VLDL-C at Week 12	Baseline and Week 12

Trial Locations

Locations (1)

Research Site; 🇬🇧 Manchester, United Kingdom