Phase 3 Completed N=486 Randomized Quadruple-blind Treatment

Efficacy and Safety of Alirocumab (SAR236553/REGN727) Versus Placebo on Top of Lipid-Modifying Therapy in Patients With Heterozygous Familial Hypercholesterolemia Not Adequately Controlled With Their Lipid-Modifying Therapy

Hypercholesterolemia

Source: ClinicalTrials.gov NCT01623115 ↗

Enrolled (actual)

486

Serious AEs

13.6%

Results posted

Nov 2015

Primary outcomePrimary: Percent Change From Baseline in Calculated LDL-C at Week 24 - Intent-to-Treat (ITT) Analysis — 9.1; -48.8 percent change — p=<0.0001

Summary

Alirocumab (SAR236553/REGN727) is a fully human monoclonal antibody that binds PCSK9 (proprotein convertase subtilisin/kexin type 9). Primary Objective of the study: To evaluate the effect of alirocumab on low-density lipoprotein cholesterol (LDL-C) levels after 24 weeks of treatment in comparison with placebo. Secondary Objectives: * To evaluate the effect of alirocumab in comparison with placebo on LDL-C at other time points * To evaluate the effects of alirocumab on other lipid parameters * To evaluate the safety and tolerability of alirocumab

Outcome Measures

Outcome	Result	p-value
PRIMARY Percent Change From Baseline in Calculated LDL-C at Week 24 - Intent-to-Treat (ITT) Analysis	9.1; -48.8	<0.0001 sig
SECONDARY Percent Change From Baseline in Calculated LDL-C at Week 24 - On-Treatment Analysis	8.8; -49.3	<0.0001 sig
SECONDARY Percent Change From Baseline in Calculated LDL-C at Week 12 - ITT Analysis	5.7; -43.5	<0.0001 sig
SECONDARY Percent Change From Baseline in Calculated LDL-C at Week 12 - On-Treatment Analysis	5.7; -43.9	<0.0001 sig
SECONDARY Percent Change From Baseline in Apolipoprotein B (Apo B) at Week 24 - ITT Analysis	4.7; -41.1	<0.0001 sig
SECONDARY Percent Change From Baseline in Apo B at Week 24 - On-Treatment Analysis	4.5; -41.4	<0.0001 sig
SECONDARY Percent Change From Baseline in Non-High Density Lipoprotein Cholesterol (Non-HDL-C) at Week 24 - ITT Analysis	9.6; -42.8	<0.0001 sig
SECONDARY Percent Change From Baseline in Non-HDL-C at Week 24 - On-Treatment Analysis	9.4; -43.3	<0.0001 sig
SECONDARY Percent Change From Baseline in Total Cholesterol (Total-C) at Week 24 - ITT Analysis	7.3; -31.4	<0.0001 sig
SECONDARY Percent Change From Baseline in Apo B at Week 12 - ITT Analysis	3.1; -34.5	<0.0001 sig
SECONDARY Percent Change From Baseline in Non-HDL-C at Week 12 - ITT Analysis	5.3; -38.4	<0.0001 sig
SECONDARY Percent Change From Baseline in Total-C at Week 12 - ITT Analysis	4.1; -28.3	<0.0001 sig
SECONDARY Percent Change From Baseline in Calculated LDL-C at Week 52 - ITT Analysis	9; -47.1	<0.0001 sig
SECONDARY Percentage of Very High Cardiovascular (CV) Risk Participants Achieving Calculated LDL-C < 70 mg/dL (<1.81 mmol/L) or High CV Risk Participants Achieving Calculated LDL-C < 100 mg/dL (<2.59 mmol/L) at Week 24 - ITT Analysis	2.4; 72.2	<0.0001 sig
SECONDARY Percentage of Very High CV Risk Participants Achieving Calculated LDL-C < 70 mg/dL (<1.81 mmol/L) or High CV Risk Participants Achieving Calculated LDL-C < 100 mg/dL (<2.59 mmol/L) at Week 24 - On- Treatment Analysis	2.4; 73	<0.0001 sig
SECONDARY Percentage of Participants Achieving Calculated LDL-C <70 mg/dL (1.81 mmol/L) at Week 24 - ITT Analysis	0.8; 59.8	<0.0001 sig
SECONDARY Percentage of Participants Achieving Calculated LDL-C <70 mg/dL (1.81 mmol/L) at Week 24 - On-Treatment Analysis	0.8; 60.1	<0.0001 sig
SECONDARY Percent Change From Baseline in Lipoprotein (a) at Week 24 - ITT Analysis	-7.5; -25.2	<0.0001 sig
SECONDARY Percent Change From Baseline in HDL-C at Week 24 - ITT Analysis	0.8; 8.8	<0.0001 sig
SECONDARY Percent Change From Baseline in Fasting Triglycerides at Week 24 - ITT Analysis	6.3; -9.6	<0.0001 sig
SECONDARY Percent Change From Baseline in Apolipoprotein A-1 (Apo A-1) at Week 24 - ITT Analysis	0.3; 5	= 0.0002 sig
SECONDARY Percent Change From Baseline in Lipoprotein (a) at Week 12 - ITT Analysis	-3.9; -21.2	<0.0001 sig
SECONDARY Percent Change From Baseline in HDL-C at Week 12 - ITT Analysis	2.1; 6.4	0.0031 sig
SECONDARY Percent Change From Baseline in Fasting Triglycerides at Week 12 - ITT Analysis	1.7; -8	0.0003 sig
SECONDARY Percent Change From Baseline in Apo A-1 at Week 12 - ITT Analysis	0.1; 2.9	0.0187 sig

Eligibility Criteria

Inclusion criteria

Participants with heterozygous familial hypercholesterolemia who were not adequately controlled with their lipid-modifying therapy

Exclusion criteria

Age 400 mg/dL (4.52 mmol/L)
Known history of homozygous familial hypercholesterolemia

The above information is not intended to contain all considerations relevant to a participant's potential participation in a clinical trial.

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT01623115). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.