Phase 3
Completed N=107
Efficacy and Safety of Alirocumab (SAR236553/REGN727) Versus Placebo on Top of Lipid-Modifying Therapy in Patients With Heterozygous Familial Hypercholesterolemia (ODYSSEY HIGH FH)
Hypercholesterolaemia
Source: ClinicalTrials.gov NCT01617655 ↗
Enrolled (actual)
107
Serious AEs
13.1%
Results posted
Nov 2015
Primary outcomePrimary: Percent Change From Baseline in Calculated LDL-C at Week 24 - ITT Analysis — -6.6; -45.7 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 - ITT Analysis |
-6.6; -45.7 | <0.0001 sig |
| SECONDARY Percent Change From Baseline in Calculated LDL-C at Week 24 - On-Treatment Analysis |
-6.6; -45.5 | <0.0001 sig |
| SECONDARY Percent Change From Baseline in Calculated LDL-C at Week 12 - ITT Analysis |
-6.6; -46.9 | <0.0001 sig |
| SECONDARY Percent Change From Baseline in Calculated LDL-C at Week 12 - On-Treatment Analysis |
-6.6; -46.9 | <0.0001 sig |
| SECONDARY Percent Change From Baseline in Apolipoprotein B (Apo B) at Week 24 - ITT Analysis |
-8.7; -39 | <0.0001 sig |
| SECONDARY Percent Change From Baseline in Apo B at Week 24 - On-Treatment Analysis |
-8.7; -38.9 | <0.0001 sig |
| SECONDARY Percent Change From Baseline in Non-High Density Lipoprotein Cholesterol (Non-HDL-C) at Week 24 - ITT Analysis |
-6.2; -41.9 | <0.0001 sig |
| SECONDARY Percent Change From Baseline in Non-HDL-C at Week 24 - On-Treatment Analysis |
-6.1; -41.7 | <0.0001 sig |
| SECONDARY Percent Change From Baseline in Total Cholesterol (Total-C) at Week 24 - ITT Analysis |
-4.8; -33.2 | <0.0001 sig |
| SECONDARY Percent Change From Baseline in Apo B at Week 12 - ITT Analysis |
-9; -39.2 | <0.0001 sig |
| SECONDARY Percent Change From Baseline in Non-HDL-C at Week 12 - ITT Analysis |
-6.9; -41.4 | <0.0001 sig |
| SECONDARY Percent Change From Baseline in Total-C at Week 12 - ITT Analysis |
-5.2; -33 | <0.0001 sig |
| SECONDARY Percent Change From Baseline in Calculated LDL-C at Week 52 - ITT Analysis |
-3; -42.1 | <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 |
5.7; 41.0 | 0.0016 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 |
5.7; 41.4 | 0.0014 sig |
| SECONDARY Percent Change From Baseline in Lipoprotein (a) at Week 24 - ITT Analysis |
-8.7; -23.5 | 0.0164 sig |
| SECONDARY Percent Change From Baseline in HDL-C at Week 24 - ITT Analysis |
3.9; 7.5 | 0.2745 |
| SECONDARY Percent Change From Baseline in Fasting Triglycerides at Week 24 - ITT Analysis |
-1.9; -10.5 | — |
| SECONDARY Percent Change From Baseline in Apolipoprotein A-1 (Apo A-1) at Week 24 - ITT Analysis |
2; 5.6 | — |
| SECONDARY Percent Change From Baseline in Lipoprotein (a) at Week 12 - ITT Analysis |
-1.5; -23.2 | — |
| SECONDARY Percent Change From Baseline in HDL-C at Week 12 - ITT Analysis |
8.0; 7.9 | — |
| SECONDARY Percent Change From Baseline in Fasting Triglycerides at Week 12 - ITT Analysis |
-4.4; -9.4 | — |
| SECONDARY Percent Change From Baseline in Apo A-1 at Week 12 - ITT Analysis |
1.1; 4.6 | — |
| SECONDARY Percentage of Participants Achieving Calculated LDL-C <70 mg/dL (1.81 mmol/L) at Week 24 - ITT Analysis |
2.9; 32.4 | — |
| SECONDARY Percentage of Participants Achieving Calculated LDL-C <70 mg/dL (1.81 mmol/L) at Week 24 - On-Treatment Analysis |
2.9; 32.6 | — |
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) during the screening period.
- 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.
Data sourced from ClinicalTrials.gov (NCT01617655). 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.