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CompletedNCT01623115ODYSSEY FH IUpdated Feb 8, 2016Results posted

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

A Phase 3 interventional study of Alirocumab and Placebo (for alirocumab) in Hypercholesterolemia, sponsored by Sanofi. Completed at 91 sites in 14 countries. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2016-02-08.

Sponsored by Sanofi · Phase 3, Interventional, and Treatment

Phase
Phase 3
Study type
Interventional
Enrollment
486
Allocation
Randomized
Ages
18 Years and older
Sex
All
01

Study 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
Read the detailed description

The maximum study duration was planned to be 89 weeks per participant including participants who successfully completed the 78-week treatment period had the possibility to join an open-label extension study (LTS13463, NCT01954394) at the end of the treatment period.

02

Conditions studied

  • Hypercholesterolemia

Keywords

  • PCSK9
03

In context

Hypercholesterolemia

1,267 studies on the registry are indexed under Hypercholesterolemia; 105 are open to participants now.

This study's enrollment of 486 is above the median of 100 across 1,015 interventional studies indexed under Hypercholesterolemia.

Browse Hypercholesterolemia studies →

Lead sponsor

Sanofi is the lead sponsor of 1,547 studies on the registry; 88 are open to participants now.

Of its 204 completed or terminated interventional studies of FDA-regulated products, 122 (60%) have results posted.

Browse Sanofi studies →

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
18 Years and older
Sexes eligible
All
Accepts healthy volunteers
No

Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.

Inclusion criteria

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

Exclusion criteria

Exclusion criteria:

  • Age \< 18 years or legal age of adulthood, whichever is greater
  • LDL-C \< 70 mg/dL (1.81 mmol/L) and with cardiovascular disease
  • LDL-C \< 100 mg/dL (2.59 mmol/L) and without cardiovascular disease
  • Fasting serum triglycerides > 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.

05

Study design

Phase
Phase 3
Primary purpose
Treatment
Allocation
Randomized
Intervention model
Parallel assignment
Masking
Quadruple (Participant, Care provider, Investigator, Outcomes assessor)
Enrollment
486 participants (actual)

Study arms

  • Placebo comparator
    Placebo

    Placebo for alirocumab every 2 weeks (Q2W) on top of stable lipid-modifying therapy (LMT) for 78 weeks.

    Drug: Placebo (for alirocumab) · Drug: Lipid Modifying Therapy (LMT)

  • Experimental
    Alirocumab 75 mg/Up to 150 mg Q2W

    Alirocumab 75 mg Q2W on top of stable LMT for 78 weeks. Alirocumab dose up-titrated to 150 mg from Week 12 when low-density lipoprotein cholesterol (LDL-C) levels ≥ 70 mg/dL (1.81 mmol/L) at Week 8.

    Drug: Alirocumab · Drug: Lipid Modifying Therapy (LMT)

Interventions

  • DrugAlirocumab

    Solution for subcutaneous injection in the abdomen, thigh, or outer area of the upper arm by self-injection or by another designated person using auto-injector (also known as pre filled pen).

    Also known as: SAR236553, REGN727, Praluent

  • DrugPlacebo (for alirocumab)

    Solution for subcutaneous injection in the abdomen, thigh, or outer area of the upper arm by self-injection or by another designated person using auto-injector (also known as pre filled pen).

  • DrugLipid Modifying Therapy (LMT)

    Statin (rosuvastatin, simvastatin or atorvastatin) at stable dose with or without other LMT as clinically indicated.

06

What researchers measure

Primary outcomes

  1. Percent Change From Baseline in Calculated LDL-C at Week 24 - Intent-to-Treat (ITT) Analysis

    Adjusted Least-squares (LS) means and standard errors at Week 24 were obtained from a mixed-effect model with repeated measures (MMRM) to account for missing data. All available post-baseline data from Week 4 to Week 52 regardless of status on- or off-treatment were used in the model (ITT analysis).

    Time frame: From Baseline to Week 52

Secondary outcomes

  1. Percent Change From Baseline in Calculated LDL-C at Week 24 - On-Treatment Analysis

    Adjusted LS means and standard errors at Week 24 from MMRM model including available post-baseline on-treatment data from Week 4 to Week 52 (i.e. up to 21 days after last injection) (on-treatment analysis).

    Time frame: From Baseline to Week 52

  2. Percent Change From Baseline in Calculated LDL-C at Week 12 - ITT Analysis

    Adjusted LS means and standard errors at Week 12 from MMRM including all available post-baseline data from Week 4 to Week 52 regardless of status on-or off-treatment.

    Time frame: From Baseline to Week 52

  3. Percent Change From Baseline in Calculated LDL-C at Week 12 - On-Treatment Analysis

    Adjusted LS means and standard errors at Week 12 from MMRM model including available post-baseline on-treatment data from Week 4 to Week 52 (i.e. up to 21 days after last injection).

    Time frame: From Baseline to Week 52

  4. Percent Change From Baseline in Apolipoprotein B (Apo B) at Week 24 - ITT Analysis

    Adjusted LS means and standard errors at Week 24 from MMRM model including all available post-baseline data from Week 4 to Week 52 regardless of status on- or off-treatment.

    Time frame: From Baseline to Week 52

  5. Percent Change From Baseline in Apo B at Week 24 - On-Treatment Analysis

    Adjusted LS means and standard errors at Week 24 from MMRM model including available post-baseline on-treatment data from Week 4 to Week 52 (i.e. up to 21 days after last injection).

    Time frame: From Baseline to Week 52

  6. Percent Change From Baseline in Non-High Density Lipoprotein Cholesterol (Non-HDL-C) at Week 24 - ITT Analysis

    Adjusted LS means and standard errors at Week 24 from MMRM model including all available post-baseline data from Week 4 to Week 52 regardless of status on-or off-treatment.

    Time frame: From Baseline to Week 52

  7. Percent Change From Baseline in Non-HDL-C at Week 24 - On-Treatment Analysis

    Adjusted LS means and standard errors at Week 24 from MMRM model including available post-baseline on-treatment data from Week 4 to Week 52 (i.e. up to 21 days after last injection).

    Time frame: From Baseline to Week 52

  8. Percent Change From Baseline in Total Cholesterol (Total-C) at Week 24 - ITT Analysis

    Adjusted LS means and standard errors at Week 24 from MMRM model including all available post-baseline data from Week 4 to Week 52 regardless of status on-or off-treatment.

    Time frame: From Baseline to Week 52

  9. Percent Change From Baseline in Apo B at Week 12 - ITT Analysis

    Adjusted LS means and standard errors at Week 12 from MMRM model including all available post-baseline data from Week 4 to Week 52 regardless of status on-or off-treatment.

    Time frame: From Baseline to Week 52

  10. Percent Change From Baseline in Non-HDL-C at Week 12 - ITT Analysis

    Adjusted LS means and standard errors at Week 12 from MMRM model including all available post-baseline data from Week 4 to Week 52 regardless of status on- or off-treatment.

    Time frame: From Baseline to Week 52

  11. Percent Change From Baseline in Total-C at Week 12 - ITT Analysis

    Adjusted LS means and standard errors at Week 12 from MMRM model including all available post-baseline data from Week 4 to Week 52 regardless of status on- or off-treatment.

    Time frame: From Baseline to Week 52

  12. Percent Change From Baseline in Calculated LDL-C at Week 52 - ITT Analysis

    Adjusted LS means and standard errors at Week 52 from MMRM model including all available post-baseline data from Week 4 to Week 52 regardless of status on- or off-treatment.

    Time frame: From Baseline to Week 52

  13. 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

    Very high CV risk participants: Heterozygous Familial Hypercholesterolemia (heFH) participants with coronary heart disease (CHD) or CHD risk equivalents. High CV risk participants: heFH participants without CHD or CHD risk equivalents. CHD risk equivalent: peripheral arterial disease, ischemic stroke, moderate chronic kidney disease (estimated glomerular filtration rate, 30 to \<60 ml/minute/1.73 m\^2 of body-surface area), or diabetes mellitus plus 2 or more additional risk factors (hypertension; ankle-brachial index of ≤0.90; microalbuminuria, macroalbuminuria, or a urinary dipstick result of \>2+ protein; preproliferative or proliferative retinopathy or laser treatment for retinopathy; or a family history of premature CHD). Adjusted percentages at Week 24 were obtained from multiple imputation approach for handling of missing data. All available post-baseline data from Week 4 to Week 52 regardless of status on- or off-treatment were included in the imputation model.

    Time frame: Up to Week 52

  14. 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

    Adjusted percentages at Week 24 from multiple imputation approach including available post-baseline on-treatment data from Week 4 to Week 52 i.e. up to 21 days after last injection.

    Time frame: Up to Week 52

  15. Percentage of Participants Achieving Calculated LDL-C <70 mg/dL (1.81 mmol/L) at Week 24 - ITT Analysis

    Adjusted percentages at Week 24 from multiple imputation approach model including all available post-baseline data from Week 4 to Week 52 regardless of status on- or off-treatment.

    Time frame: Up to Week 52

  16. Percentage of Participants Achieving Calculated LDL-C <70 mg/dL (1.81 mmol/L) at Week 24 - On-Treatment Analysis

    Adjusted percentages at Week 24 from multiple imputation approach model including available post-baseline on-treatment data from Week 4 to Week 52 i.e. up to 21 days after last injection.

    Time frame: Up to Week 52

  17. Percent Change From Baseline in Lipoprotein (a) at Week 24 - ITT Analysis

    Adjusted means and standard errors at Week 24 from multiple imputation approach followed by robust regression model including all available post-baseline data from Week 4 to Week 52 regardless of status on- or off-treatment.

    Time frame: From Baseline to Week 52

  18. Percent Change From Baseline in HDL-C at Week 24 - ITT Analysis

    Adjusted LS means and standard errors at Week 24 from MMRM model including all available post-baseline data from Week 4 to Week 52 regardless of status on- or off-treatment.

    Time frame: From Baseline to Week 52

  19. Percent Change From Baseline in Fasting Triglycerides at Week 24 - ITT Analysis

    Adjusted means and standard errors at Week 24 from multiple imputation approach followed by robust regression model including all available post-baseline data from Week 4 to Week 52 regardless of status on- or off-treatment.

    Time frame: From Baseline to Week 52

  20. Percent Change From Baseline in Apolipoprotein A-1 (Apo A-1) at Week 24 - ITT Analysis

    Adjusted LS means and standard errors at Week 24 from MMRM model including all available post-baseline data from Week 4 to Week 52 regardless of status on- or off-treatment.

    Time frame: From Baseline to Week 52

  21. Percent Change From Baseline in Lipoprotein (a) at Week 12 - ITT Analysis

    Adjusted means and standard errors at Week 12 from multiple imputation approach followed by robust regression model including all available post-baseline data from Week 4 to Week 52 regardless of status on- or off-treatment.

    Time frame: From Baseline to Week 52

  22. Percent Change From Baseline in HDL-C at Week 12 - ITT Analysis

    Adjusted LS means and standard errors at Week 12 from MMRM model including all available post-baseline data from Week 4 to Week 52 regardless of status on- or off-treatment.

    Time frame: From Baseline to Week 52

  23. Percent Change From Baseline in Fasting Triglycerides at Week 12 - ITT Analysis

    Adjusted means and standard errors at Week 12 from multiple imputation approach followed by robust regression model including all available post-baseline data from Week 4 to Week 52 regardless of status on- or off-treatment.

    Time frame: From Baseline to Week 52

  24. Percent Change From Baseline in Apo A-1 at Week 12 - ITT Analysis

    Adjusted LS means and standard errors at Week 12 from MMRM model including all available post-baseline data from Week 4 to Week 52 regardless of status on- or off-treatment.

    Time frame: From Baseline to Week 52

Other outcomes

  1. Percent Change From Baseline in Calculated LDL-C at Week 52 - On-Treatment Analysis

    Adjusted LS means and standard errors at Week 52 from MMRM model including available post-baseline on-treatment data from Week 4 to Week 52 (i.e. up to 21 days after last injection).

    Time frame: From Baseline to Week 52

  2. Percent Change From Baseline in Calculated LDL-C at Week 78 - ITT Analysis

    Adjusted LS means and standard errors at Week 78 from MMRM including all available post-baseline data from Week 4 to Week 78 regardless of status on-or off-treatment.

    Time frame: From Baseline to Week 78

  3. Percent Change From Baseline in Calculated LDL-C at Week 78 - On-Treatment Analysis

    Adjusted LS means and standard errors at Week 78 from MMRM model including available post-baseline on-treatment data from Week 4 to Week 78 (i.e. up to 21 days after last injection).

    Time frame: From Baseline to Week 78

07

Results

Posted Nov 4, 2015
Limitations and caveats
Manual reclassification was done by the Sponsor for the "other reasons" of non-completion of study as specified in the electronic case report form (eCRF).

Participant flow

The study was conducted at 89 centers in 14 countries. A total of 597 participants were screened between July 2012 and April 2013, 111 of whom were screen failures.

Participant flow — Overall Study
MilestonePlaceboAlirocumab 75 mg/Up to 150 mg Q2W
Started163323
Treated163322
Completed130246
Not completed3377
Withdrew: Randomized but not treated01
Withdrew: Physician decision10
Withdrew: Participants moved15
Withdrew: Adverse event1013
Withdrew: Poor compliance to protocol410
Withdrew: Consent withdrawn by participant26
Withdrew: Related to study drug administration02
Withdrew: Last visit outside protocol visit window1426
Withdrew: Site closure17
Withdrew: Death04
Withdrew: Other than specified above03

Outcome measures

PrimaryPercent Change From Baseline in Calculated LDL-C at Week 24 - Intent-to-Treat (ITT) Analysis

Adjusted Least-squares (LS) means and standard errors at Week 24 were obtained from a mixed-effect model with repeated measures (MMRM) to account for missing data. All available post-baseline data from Week 4 to Week 52 regardless of status on- or off-treatment were used in the model (ITT analysis).

Time frame:
From Baseline to Week 52
Reported as:
Least squares mean · percent change
Percent Change From Baseline in Calculated LDL-C at Week 24 - Intent-to-Treat (ITT) Analysis
percent changePlaceboAlirocumab 75 mg/Up to 150 mg Q2W
Percent Change From Baseline in Calculated LDL-C at Week 24 - Intent-to-Treat (ITT) Analysis9.1 ± 2.2-48.8 ± 1.6
Statistical analysis
  • Placebo vs Alirocumab 75 mg/Up to 150 mg Q2W · Mixed Models Analysis · p = <0.0001 (Threshold for significance ≤ 0.05.) · Ls mean difference: -57.9 · 95% CI -63.3 to -52.6Alirocumab vs Placebo
SecondaryPercent Change From Baseline in Calculated LDL-C at Week 24 - On-Treatment Analysis

Adjusted LS means and standard errors at Week 24 from MMRM model including available post-baseline on-treatment data from Week 4 to Week 52 (i.e. up to 21 days after last injection) (on-treatment analysis).

Time frame:
From Baseline to Week 52
Reported as:
Least squares mean · percent change
Percent Change From Baseline in Calculated LDL-C at Week 24 - On-Treatment Analysis
percent changePlaceboAlirocumab 75 mg/Up to 150 mg Q2W
Percent Change From Baseline in Calculated LDL-C at Week 24 - On-Treatment Analysis8.8 ± 2.2-49.3 ± 1.6
Statistical analysis
  • Placebo vs Alirocumab 75 mg/Up to 150 mg Q2W · Mixed Models Analysis · p = <0.0001 (Threshold for significance ≤ 0.05.) · Ls mean difference: -58.1 · 95% CI -63.5 to -52.7Alirocumab vs Placebo
SecondaryPercent Change From Baseline in Calculated LDL-C at Week 12 - ITT Analysis

Adjusted LS means and standard errors at Week 12 from MMRM including all available post-baseline data from Week 4 to Week 52 regardless of status on-or off-treatment.

Time frame:
From Baseline to Week 52
Reported as:
Least squares mean · percent change
Percent Change From Baseline in Calculated LDL-C at Week 12 - ITT Analysis
percent changePlaceboAlirocumab 75 mg/Up to 150 mg Q2W
Percent Change From Baseline in Calculated LDL-C at Week 12 - ITT Analysis5.7 ± 2-43.5 ± 1.4
Statistical analysis
  • Placebo vs Alirocumab 75 mg/Up to 150 mg Q2W · Mixed Models Analysis · p = <0.0001 (Threshold for significance ≤ 0.05.) · Ls mean difference: -49.2 · 95% CI -53.9 to -44.5Alirocumab vs Placebo
SecondaryPercent Change From Baseline in Calculated LDL-C at Week 12 - On-Treatment Analysis

Adjusted LS means and standard errors at Week 12 from MMRM model including available post-baseline on-treatment data from Week 4 to Week 52 (i.e. up to 21 days after last injection).

Time frame:
From Baseline to Week 52
Reported as:
Least squares mean · percent change
Percent Change From Baseline in Calculated LDL-C at Week 12 - On-Treatment Analysis
percent changePlaceboAlirocumab 75 mg/Up to 150 mg Q2W
Percent Change From Baseline in Calculated LDL-C at Week 12 - On-Treatment Analysis5.7 ± 2-43.9 ± 1.4
Statistical analysis
  • Placebo vs Alirocumab 75 mg/Up to 150 mg Q2W · Mixed Models Analysis · p = <0.0001 (Threshold for significance ≤ 0.05.) · Ls mean difference: -49.5 · 95% CI -54.2 to -44.8Alirocumab vs Placebo
SecondaryPercent Change From Baseline in Apolipoprotein B (Apo B) at Week 24 - ITT Analysis

Adjusted LS means and standard errors at Week 24 from MMRM model including all available post-baseline data from Week 4 to Week 52 regardless of status on- or off-treatment.

Time frame:
From Baseline to Week 52
Reported as:
Least squares mean · percent change
Percent Change From Baseline in Apolipoprotein B (Apo B) at Week 24 - ITT Analysis
percent changePlaceboAlirocumab 75 mg/Up to 150 mg Q2W
Percent Change From Baseline in Apolipoprotein B (Apo B) at Week 24 - ITT Analysis4.7 ± 1.6-41.1 ± 1.2
Statistical analysis
  • Placebo vs Alirocumab 75 mg/Up to 150 mg Q2W · Mixed Models Analysis · p = <0.0001 (Threshold for significance ≤ 0.05.) · Ls mean difference: -45.8 · 95% CI -49.8 to -41.8Alirocumab vs Placebo
SecondaryPercent Change From Baseline in Apo B at Week 24 - On-Treatment Analysis

Adjusted LS means and standard errors at Week 24 from MMRM model including available post-baseline on-treatment data from Week 4 to Week 52 (i.e. up to 21 days after last injection).

Time frame:
From Baseline to Week 52
Reported as:
Least squares mean · percent change
Percent Change From Baseline in Apo B at Week 24 - On-Treatment Analysis
percent changePlaceboAlirocumab 75 mg/Up to 150 mg Q2W
Percent Change From Baseline in Apo B at Week 24 - On-Treatment Analysis4.5 ± 1.7-41.4 ± 1.2
Statistical analysis
  • Placebo vs Alirocumab 75 mg/Up to 150 mg Q2W · Mixed Models Analysis · p = <0.0001 (Threshold for significance ≤ 0.05.) · Ls mean difference: -45.9 · 95% CI -49.9 to -41.8Alirocumab vs Placebo
SecondaryPercent Change From Baseline in Non-High Density Lipoprotein Cholesterol (Non-HDL-C) at Week 24 - ITT Analysis

Adjusted LS means and standard errors at Week 24 from MMRM model including all available post-baseline data from Week 4 to Week 52 regardless of status on-or off-treatment.

Time frame:
From Baseline to Week 52
Reported as:
Least squares mean · percent change
Percent Change From Baseline in Non-High Density Lipoprotein Cholesterol (Non-HDL-C) at Week 24 - ITT Analysis
percent changePlaceboAlirocumab 75 mg/Up to 150 mg Q2W
Percent Change From Baseline in Non-High Density Lipoprotein Cholesterol (Non-HDL-C) at Week 24 - ITT Analysis9.6 ± 2-42.8 ± 1.4
Statistical analysis
  • Placebo vs Alirocumab 75 mg/Up to 150 mg Q2W · Mixed Models Analysis · p = <0.0001 (Threshold for significance ≤ 0.05.) · Ls mean difference: -52.4 · 95% CI -57.2 to -47.6Alirocumab vs Placebo
SecondaryPercent Change From Baseline in Non-HDL-C at Week 24 - On-Treatment Analysis

Adjusted LS means and standard errors at Week 24 from MMRM model including available post-baseline on-treatment data from Week 4 to Week 52 (i.e. up to 21 days after last injection).

Time frame:
From Baseline to Week 52
Reported as:
Least squares mean · percent change
Percent Change From Baseline in Non-HDL-C at Week 24 - On-Treatment Analysis
percent changePlaceboAlirocumab 75 mg/Up to 150 mg Q2W
Percent Change From Baseline in Non-HDL-C at Week 24 - On-Treatment Analysis9.4 ± 2-43.3 ± 1.4
Statistical analysis
  • Placebo vs Alirocumab 75 mg/Up to 150 mg Q2W · Mixed Models Analysis · p = <0.0001 (Threshold for significance ≤ 0.05.) · Ls mean difference: -52.6 · 95% CI -57.5 to -47.8Alirocumab vs Placebo
SecondaryPercent Change From Baseline in Total Cholesterol (Total-C) at Week 24 - ITT Analysis

Adjusted LS means and standard errors at Week 24 from MMRM model including all available post-baseline data from Week 4 to Week 52 regardless of status on-or off-treatment.

Time frame:
From Baseline to Week 52
Reported as:
Least squares mean · percent change
Percent Change From Baseline in Total Cholesterol (Total-C) at Week 24 - ITT Analysis
percent changePlaceboAlirocumab 75 mg/Up to 150 mg Q2W
Percent Change From Baseline in Total Cholesterol (Total-C) at Week 24 - ITT Analysis7.3 ± 1.5-31.4 ± 1.1
Statistical analysis
  • Placebo vs Alirocumab 75 mg/Up to 150 mg Q2W · Mixed Models Analysis · p = <0.0001 (Threshold for significance ≤ 0.05.) · Ls mean difference: -38.7 · 95% CI -42.4 to -35Alirocumab vs Placebo
SecondaryPercent Change From Baseline in Apo B at Week 12 - ITT Analysis

Adjusted LS means and standard errors at Week 12 from MMRM model including all available post-baseline data from Week 4 to Week 52 regardless of status on-or off-treatment.

Time frame:
From Baseline to Week 52
Reported as:
Least squares mean · percent change
Percent Change From Baseline in Apo B at Week 12 - ITT Analysis
percent changePlaceboAlirocumab 75 mg/Up to 150 mg Q2W
Percent Change From Baseline in Apo B at Week 12 - ITT Analysis3.1 ± 1.5-34.5 ± 1.1
Statistical analysis
  • Placebo vs Alirocumab 75 mg/Up to 150 mg Q2W · Mixed Models Analysis · p = <0.0001 (Threshold for significance ≤ 0.05.) · Ls mean difference: -37.5 · 95% CI -41.2 to -33.9Alirocumab vs Placebo
SecondaryPercent Change From Baseline in Non-HDL-C at Week 12 - ITT Analysis

Adjusted LS means and standard errors at Week 12 from MMRM model including all available post-baseline data from Week 4 to Week 52 regardless of status on- or off-treatment.

Time frame:
From Baseline to Week 52
Reported as:
Least squares mean · percent change
Percent Change From Baseline in Non-HDL-C at Week 12 - ITT Analysis
percent changePlaceboAlirocumab 75 mg/Up to 150 mg Q2W
Percent Change From Baseline in Non-HDL-C at Week 12 - ITT Analysis5.3 ± 1.8-38.4 ± 1.3
Statistical analysis
  • Placebo vs Alirocumab 75 mg/Up to 150 mg Q2W · Mixed Models Analysis · p = <0.0001 (Threshold for significance ≤ 0.05.) · Ls mean difference: -43.7 · 95% CI -48 to -39.4Alirocumab vs Placebo
SecondaryPercent Change From Baseline in Total-C at Week 12 - ITT Analysis

Adjusted LS means and standard errors at Week 12 from MMRM model including all available post-baseline data from Week 4 to Week 52 regardless of status on- or off-treatment.

Time frame:
From Baseline to Week 52
Reported as:
Least squares mean · percent change
Percent Change From Baseline in Total-C at Week 12 - ITT Analysis
percent changePlaceboAlirocumab 75 mg/Up to 150 mg Q2W
Percent Change From Baseline in Total-C at Week 12 - ITT Analysis4.1 ± 1.4-28.3 ± 1
Statistical analysis
  • Placebo vs Alirocumab 75 mg/Up to 150 mg Q2W · Mixed Models Analysis · p = <0.0001 (Threshold for significance ≤ 0.05.) · Ls mean difference: -32.5 · 95% CI -35.7 to -29.2Alirocumab vs Placebo
SecondaryPercent Change From Baseline in Calculated LDL-C at Week 52 - ITT Analysis

Adjusted LS means and standard errors at Week 52 from MMRM model including all available post-baseline data from Week 4 to Week 52 regardless of status on- or off-treatment.

Time frame:
From Baseline to Week 52
Reported as:
Least squares mean · percent change
Percent Change From Baseline in Calculated LDL-C at Week 52 - ITT Analysis
percent changePlaceboAlirocumab 75 mg/Up to 150 mg Q2W
Percent Change From Baseline in Calculated LDL-C at Week 52 - ITT Analysis9 ± 2.6-47.1 ± 1.9
Statistical analysis
  • Placebo vs Alirocumab 75 mg/Up to 150 mg Q2W · Mixed Models Analysis · p = <0.0001 (Threshold for significance ≤ 0.05.) · Ls mean difference: -56.2 · 95% CI -62.4 to -50Alirocumab vs Placebo
SecondaryPercentage 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

Very high CV risk participants: Heterozygous Familial Hypercholesterolemia (heFH) participants with coronary heart disease (CHD) or CHD risk equivalents. High CV risk participants: heFH participants without CHD or CHD risk equivalents. CHD risk equivalent: peripheral arterial disease, ischemic stroke, moderate chronic kidney disease (estimated glomerular filtration rate, 30 to \<60 ml/minute/1.73 m\^2 of body-surface area), or diabetes mellitus plus 2 or more additional risk factors (hypertension; ankle-brachial index of ≤0.90; microalbuminuria, macroalbuminuria, or a urinary dipstick result of \>2+ protein; preproliferative or proliferative retinopathy or laser treatment for retinopathy; or a family history of premature CHD). Adjusted percentages at Week 24 were obtained from multiple imputation approach for handling of missing data. All available post-baseline data from Week 4 to Week 52 regardless of status on- or off-treatment were included in the imputation model.

Time frame:
Up to Week 52
Reported as:
Number · percentage of participants
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
percentage of participantsPlaceboAlirocumab 75 mg/Up to 150 mg Q2W
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 Analysis2.472.2
Statistical analysis
  • Placebo vs Alirocumab 75 mg/Up to 150 mg Q2W · Regression, Logistic · p = <0.0001 (Threshold for significance ≤ 0.05.) · Odds ratio (or): 156 · 95% CI 48.9 to 498.1Alirocumab vs Placebo
SecondaryPercentage 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

Adjusted percentages at Week 24 from multiple imputation approach including available post-baseline on-treatment data from Week 4 to Week 52 i.e. up to 21 days after last injection.

Time frame:
Up to Week 52
Reported as:
Number · percentage of participants
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
percentage of participantsPlaceboAlirocumab 75 mg/Up to 150 mg Q2W
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 Analysis2.473
Statistical analysis
  • Placebo vs Alirocumab 75 mg/Up to 150 mg Q2W · Regression, Logistic · p = <0.0001 (Threshold for significance ≤ 0.05.) · Odds ratio (or): 156.6 · 95% CI 49.7 to 493.7Alirocumab vs Placebo
SecondaryPercentage of Participants Achieving Calculated LDL-C <70 mg/dL (1.81 mmol/L) at Week 24 - ITT Analysis

Adjusted percentages at Week 24 from multiple imputation approach model including all available post-baseline data from Week 4 to Week 52 regardless of status on- or off-treatment.

Time frame:
Up to Week 52
Reported as:
Number · percentage of participants
Percentage of Participants Achieving Calculated LDL-C <70 mg/dL (1.81 mmol/L) at Week 24 - ITT Analysis
percentage of participantsPlaceboAlirocumab 75 mg/Up to 150 mg Q2W
Percentage of Participants Achieving Calculated LDL-C <70 mg/dL (1.81 mmol/L) at Week 24 - ITT Analysis0.859.8
Statistical analysis
  • Placebo vs Alirocumab 75 mg/Up to 150 mg Q2W · Regression, Logistic · p = <0.0001 (Threshold for significance ≤ 0.05.) · Odds ratio (or): 244.9 · 95% CI 34.4 to 1744.4Alirocumab vs Placebo
SecondaryPercentage of Participants Achieving Calculated LDL-C <70 mg/dL (1.81 mmol/L) at Week 24 - On-Treatment Analysis

Adjusted percentages at Week 24 from multiple imputation approach model including available post-baseline on-treatment data from Week 4 to Week 52 i.e. up to 21 days after last injection.

Time frame:
Up to Week 52
Reported as:
Number · percentage of participants
Percentage of Participants Achieving Calculated LDL-C <70 mg/dL (1.81 mmol/L) at Week 24 - On-Treatment Analysis
percentage of participantsPlaceboAlirocumab 75 mg/Up to 150 mg Q2W
Percentage of Participants Achieving Calculated LDL-C <70 mg/dL (1.81 mmol/L) at Week 24 - On-Treatment Analysis0.860.1
Statistical analysis
  • Placebo vs Alirocumab 75 mg/Up to 150 mg Q2W · Regression, Logistic · p = <0.0001 (Threshold for significance ≤ 0.05.) · Odds ratio (or): 240 · 95% CI 33.9 to 1700.7Alirocumab vs Placebo
SecondaryPercent Change From Baseline in Lipoprotein (a) at Week 24 - ITT Analysis

Adjusted means and standard errors at Week 24 from multiple imputation approach followed by robust regression model including all available post-baseline data from Week 4 to Week 52 regardless of status on- or off-treatment.

Time frame:
From Baseline to Week 52
Reported as:
Mean · percent change
Percent Change From Baseline in Lipoprotein (a) at Week 24 - ITT Analysis
percent changePlaceboAlirocumab 75 mg/Up to 150 mg Q2W
Percent Change From Baseline in Lipoprotein (a) at Week 24 - ITT Analysis-7.5 ± 2-25.2 ± 1.4
Statistical analysis
  • Placebo vs Alirocumab 75 mg/Up to 150 mg Q2W · Regression, Robust · p = <0.0001 (Threshold for significance ≤ 0.05.) · Adjusted mean difference: -17.7 · 95% CI -22.6 to -12.9Alirocumab vs Placebo
SecondaryPercent Change From Baseline in HDL-C at Week 24 - ITT Analysis

Adjusted LS means and standard errors at Week 24 from MMRM model including all available post-baseline data from Week 4 to Week 52 regardless of status on- or off-treatment.

Time frame:
From Baseline to Week 52
Reported as:
Least squares mean · percent change
Percent Change From Baseline in HDL-C at Week 24 - ITT Analysis
percent changePlaceboAlirocumab 75 mg/Up to 150 mg Q2W
Percent Change From Baseline in HDL-C at Week 24 - ITT Analysis0.8 ± 1.28.8 ± 0.9
Statistical analysis
  • Placebo vs Alirocumab 75 mg/Up to 150 mg Q2W · Mixed Models Analysis · p = <0.0001 (Threshold for significance ≤ 0.05.) · Ls mean difference: 8 · 95% CI 5 to 11Alirocumab vs Placebo
SecondaryPercent Change From Baseline in Fasting Triglycerides at Week 24 - ITT Analysis

Adjusted means and standard errors at Week 24 from multiple imputation approach followed by robust regression model including all available post-baseline data from Week 4 to Week 52 regardless of status on- or off-treatment.

Time frame:
From Baseline to Week 52
Reported as:
Mean · percent change
Percent Change From Baseline in Fasting Triglycerides at Week 24 - ITT Analysis
percent changePlaceboAlirocumab 75 mg/Up to 150 mg Q2W
Percent Change From Baseline in Fasting Triglycerides at Week 24 - ITT Analysis6.3 ± 2.2-9.6 ± 1.6
Statistical analysis
  • Placebo vs Alirocumab 75 mg/Up to 150 mg Q2W · Regression, Robust · p = <0.0001 (Threshold for significance was ≤ 0.05.) · Adjusted mean difference: -16 · 95% CI -21.3 to -10.6Alirocumab vs Placebo
SecondaryPercent Change From Baseline in Apolipoprotein A-1 (Apo A-1) at Week 24 - ITT Analysis

Adjusted LS means and standard errors at Week 24 from MMRM model including all available post-baseline data from Week 4 to Week 52 regardless of status on- or off-treatment.

Time frame:
From Baseline to Week 52
Reported as:
Least squares mean · percent change
Percent Change From Baseline in Apolipoprotein A-1 (Apo A-1) at Week 24 - ITT Analysis
percent changePlaceboAlirocumab 75 mg/Up to 150 mg Q2W
Percent Change From Baseline in Apolipoprotein A-1 (Apo A-1) at Week 24 - ITT Analysis0.3 ± 15 ± 0.7
Statistical analysis
  • Placebo vs Alirocumab 75 mg/Up to 150 mg Q2W · Mixed Models Analysis · p = = 0.0002 (Threshold for significance ≤ 0.05.) · Ls mean difference: 4.7 · 95% CI 2.3 to 7.2Alirocumab vs Placebo
SecondaryPercent Change From Baseline in Lipoprotein (a) at Week 12 - ITT Analysis

Adjusted means and standard errors at Week 12 from multiple imputation approach followed by robust regression model including all available post-baseline data from Week 4 to Week 52 regardless of status on- or off-treatment.

Time frame:
From Baseline to Week 52
Reported as:
Mean · percent change
Percent Change From Baseline in Lipoprotein (a) at Week 12 - ITT Analysis
percent changePlaceboAlirocumab 75 mg/Up to 150 mg Q2W
Percent Change From Baseline in Lipoprotein (a) at Week 12 - ITT Analysis-3.9 ± 1.8-21.2 ± 1.3
Statistical analysis
  • Placebo vs Alirocumab 75 mg/Up to 150 mg Q2W · Regression, Robust · p = <0.0001 (Threshold for significance ≤ 0.05.) · Adjusted mean difference: -17.3 · 95% CI -21.5 to -13Alirocumab vs Placebo
SecondaryPercent Change From Baseline in HDL-C at Week 12 - ITT Analysis

Adjusted LS means and standard errors at Week 12 from MMRM model including all available post-baseline data from Week 4 to Week 52 regardless of status on- or off-treatment.

Time frame:
From Baseline to Week 52
Reported as:
Least squares mean · percent change
Percent Change From Baseline in HDL-C at Week 12 - ITT Analysis
percent changePlaceboAlirocumab 75 mg/Up to 150 mg Q2W
Percent Change From Baseline in HDL-C at Week 12 - ITT Analysis2.1 ± 1.26.4 ± 0.8
Statistical analysis
  • Placebo vs Alirocumab 75 mg/Up to 150 mg Q2W · Mixed Models Analysis · p = 0.0031 (Threshold for significance ≤ 0.05.) · Ls mean difference: 4.3 · 95% CI 1.5 to 7.2Alirocumab vs Placebo
SecondaryPercent Change From Baseline in Fasting Triglycerides at Week 12 - ITT Analysis

Adjusted means and standard errors at Week 12 from multiple imputation approach followed by robust regression model including all available post-baseline data from Week 4 to Week 52 regardless of status on- or off-treatment.

Time frame:
From Baseline to Week 52
Reported as:
Mean · percent change
Percent Change From Baseline in Fasting Triglycerides at Week 12 - ITT Analysis
percent changePlaceboAlirocumab 75 mg/Up to 150 mg Q2W
Percent Change From Baseline in Fasting Triglycerides at Week 12 - ITT Analysis1.7 ± 2.2-8 ± 1.6
Statistical analysis
  • Placebo vs Alirocumab 75 mg/Up to 150 mg Q2W · Regression, Robust · p = 0.0003 (Threshold for significance ≤ 0.05.) · Adjusted mean difference: -9.7 · 95% CI -15 to -4.4Alirocumab vs Placebo
SecondaryPercent Change From Baseline in Apo A-1 at Week 12 - ITT Analysis

Adjusted LS means and standard errors at Week 12 from MMRM model including all available post-baseline data from Week 4 to Week 52 regardless of status on- or off-treatment.

Time frame:
From Baseline to Week 52
Reported as:
Least squares mean · percent change
Percent Change From Baseline in Apo A-1 at Week 12 - ITT Analysis
percent changePlaceboAlirocumab 75 mg/Up to 150 mg Q2W
Percent Change From Baseline in Apo A-1 at Week 12 - ITT Analysis0.1 ± 12.9 ± 0.7
Statistical analysis
  • Placebo vs Alirocumab 75 mg/Up to 150 mg Q2W · Mixed Models Analysis · p = 0.0187 (Threshold for significance ≤ 0.05.) · Ls mean difference: 2.8 · 95% CI 0.5 to 5.2Alirocumab vs Placebo
Other pre-specifiedPercent Change From Baseline in Calculated LDL-C at Week 52 - On-Treatment Analysis

Adjusted LS means and standard errors at Week 52 from MMRM model including available post-baseline on-treatment data from Week 4 to Week 52 (i.e. up to 21 days after last injection).

Time frame:
From Baseline to Week 52
Reported as:
Least squares mean · percent change
Percent Change From Baseline in Calculated LDL-C at Week 52 - On-Treatment Analysis
percent changePlaceboAlirocumab 75 mg/Up to 150 mg Q2W
Percent Change From Baseline in Calculated LDL-C at Week 52 - On-Treatment Analysis9.1 ± 2.6-48.1 ± 1.9
Other pre-specifiedPercent Change From Baseline in Calculated LDL-C at Week 78 - ITT Analysis

Adjusted LS means and standard errors at Week 78 from MMRM including all available post-baseline data from Week 4 to Week 78 regardless of status on-or off-treatment.

Time frame:
From Baseline to Week 78
Reported as:
Least squares mean · percent change
Percent Change From Baseline in Calculated LDL-C at Week 78 - ITT Analysis
percent changePlaceboAlirocumab 75 mg/Up to 150 mg Q2W
Percent Change From Baseline in Calculated LDL-C at Week 78 - ITT Analysis11.5 ± 2.8-40.3 ± 2.0
Other pre-specifiedPercent Change From Baseline in Calculated LDL-C at Week 78 - On-Treatment Analysis

Adjusted LS means and standard errors at Week 78 from MMRM model including available post-baseline on-treatment data from Week 4 to Week 78 (i.e. up to 21 days after last injection).

Time frame:
From Baseline to Week 78
Reported as:
Least squares mean · percent change
Percent Change From Baseline in Calculated LDL-C at Week 78 - On-Treatment Analysis
percent changePlaceboAlirocumab 75 mg/Up to 150 mg Q2W
Percent Change From Baseline in Calculated LDL-C at Week 78 - On-Treatment Analysis14.0 ± 2.8-43.7 ± 2.0

Adverse events

Collected over All Adverse Events (AEs) were collected from signature of the informed consent form up to the final visit in the study regardless of seriousness or relationship to study drug.. Non-serious events are listed at a 5% frequency threshold.

Adverse event summary by group
GroupDeathsSeriousOther
Placebo—22/163 (13.5%)70/163 (42.9%)
Alirocumab 75/Up to 150 mg Q2W—44/322 (13.7%)142/322 (44.1%)
Most frequent serious events
Showing 10 of 85
Most frequent serious events
EventPlaceboAlirocumab 75/Up to 150 mg Q2W
Coronary artery diseaseCardiac disorders0/1634/322
Angina unstableCardiac disorders2/1632/322
Atrial fibrillationCardiac disorders2/1631/322
Acute myocardial infarctionCardiac disorders1/1633/322
OsteoarthritisMusculoskeletal and connective tissue disorders0/1633/322
SyncopeNervous system disorders1/1632/322
Angina pectorisCardiac disorders1/1632/322
Cardiac failure congestiveCardiac disorders1/1632/322
BradycardiaCardiac disorders0/1632/322
Miscarriage of partnerSocial circumstances0/1632/322
Most frequent other events
Most frequent other events
EventPlaceboAlirocumab 75/Up to 150 mg Q2W
Injection site reactionGeneral disorders18/16340/322
NasopharyngitisInfections and infestations12/16336/322
Upper respiratory tract infectionInfections and infestations14/16322/322
MyalgiaMusculoskeletal and connective tissue disorders11/1636/322
InfluenzaInfections and infestations9/16320/322
ArthralgiaMusculoskeletal and connective tissue disorders9/16320/322
Back painMusculoskeletal and connective tissue disorders7/16318/322
BronchitisInfections and infestations9/16310/322
HeadacheNervous system disorders9/16315/322
SinusitisInfections and infestations7/16317/322

Baseline characteristics

Age, Continuous
Age, Continuous(years)PlaceboAlirocumab 75 mg/Up to 150 mg Q2WTotal
Mean51.7 ± 12.352.1 ± 12.951.9 ± 12.7
Sex: Female, Male
Sex: Female, Male(Participants)PlaceboAlirocumab 75 mg/Up to 150 mg Q2WTotal
Female69143212
Male94180274
Calculated LDL-C in mg/dL
Calculated LDL-C in mg/dL(mg/dL)PlaceboAlirocumab 75 mg/Up to 150 mg Q2WTotal
Mean144.4 ± 46.8144.8 ± 51.1144.6 ± 49.7
Calculated LDL-C in mmol/L
Calculated LDL-C in mmol/L(mmol/L)PlaceboAlirocumab 75 mg/Up to 150 mg Q2WTotal
Mean3.739 ± 1.2133.749 ± 1.3253.746 ± 1.287
08

Study locations

91 sites
  • Investigational Site Number 840417
    Bell Gardens, California 90201, United States
  • Investigational Site Number 840429
    Long Beach, California 90801, United States
  • Investigational Site Number 840419
    Los Angeles, California 90048, United States
  • Investigational Site Number 840421
    Mission Viejo, California 92691, United States
  • Investigational Site Number 840412
    Newport Beach, California 92660, United States
  • Investigational Site Number 840428
    Newport Beach, California, United States
  • Investigational Site Number 840461
    Northridge, California 91324, United States
  • Investigational Site Number 840452
    Washington D.C., District of Columbia 20037, United States
  • Investigational Site Number 840456
    Miami, Florida 33165, United States
  • Investigational Site Number 840418
    Ponte Vedra, Florida 32081, United States
  • Investigational Site Number 840455
    Evanston, Illinois 60201, United States
  • Investigational Site Number 840415
    Kansas City, Kansas 66160-7321, United States
  • Investigational Site Number 840425
    Auburn, Maine 04210, United States
  • Investigational Site Number 840411
    Boston, Massachusetts 02114, United States
  • Investigational Site Number 840409
    St Louis, Missouri 63110, United States
  • Investigational Site Number 840407
    Morristown, New Jersey 07962, United States
  • Investigational Site Number 840408
    New York, New York 10032, United States
  • Investigational Site Number 840401
    Charlotte, North Carolina 28207, United States
  • Investigational Site Number 840410
    Durham, North Carolina 27710, United States
  • Investigational Site Number 840430
    Cincinnati, Ohio 45219, United States
  • Investigational Site Number 840424
    Portland, Oregon 97201-3098, United States
  • Investigational Site Number 840404
    Philadelphia, Pennsylvania 19104, United States
  • Investigational Site Number 840426
    Philadelphia, Pennsylvania 19104, United States
  • Investigational Site Number 840406
    Nashville, Tennessee 37232, United States
  • Investigational Site Number 840460
    Dallas, Texas 75216, United States
  • Investigational Site Number 840422
    Bountiful, Utah 84010, United States
  • Investigational Site Number 040403
    Graz, 8036, Austria
  • Investigational Site Number 040402
    Vienna, 1130, Austria
  • Investigational Site Number 040405
    Vienna, Austria
  • Investigational Site Number 124404
    Chicoutimi, G7H 5H6, Canada
  • Investigational Site Number 124401
    Montreal, H2W 1R7, Canada
  • Investigational Site Number 124403
    Québec, G1V 4M6, Canada
  • Investigational Site Number 124406
    Sherbrooke, J1H 5N4, Canada
  • Investigational Site Number 124407
    Toronto, M5C 2T2, Canada
  • Investigational Site Number 203401
    Prague, 140 00, Czechia
  • Investigational Site Number 203403
    Prague, 180 00, Czechia
  • Investigational Site Number 203405
    Uherské Hradiště, Czechia
  • Investigational Site Number 203402
    Zlín, 760 00, Czechia
  • Investigational Site Number 208401
    Copenhagen, Denmark
  • Investigational Site Number 208403
    Esbjerg, 6700, Denmark
  • Investigational Site Number 250403
    Dijon, 21000, France
  • Investigational Site Number 250401
    Paris, 75651, France
  • Investigational Site Number 250402
    Saint-Herblain, 44093, France
  • Investigational Site Number 376402
    Holon, 76100, Israel
  • Investigational Site Number 376405
    Jerusalem, Israel
  • Investigational Site Number 376404
    Safed, 13100, Israel
  • Investigational Site Number 376401
    Tel Litwinsky, 52621, Israel
  • Investigational Site Number 528406
    Amsterdam, 1105 AZ, Netherlands
  • Investigational Site Number 528410
    Amsterdam, Netherlands
  • Investigational Site Number 528408
    Den Helder, 1782 GZ, Netherlands
  • Investigational Site Number 528402
    Groningen, 9728 NT, Netherlands
  • Investigational Site Number 528411
    Leiden, 2333 ZA, Netherlands
  • Investigational Site Number 528416
    Maastricht, 6229 HX, Netherlands
  • Investigational Site Number 528409
    Nieuwegein, 3435 CM, Netherlands
  • Investigational Site Number 528412
    Sliedrecht, Netherlands
  • Investigational Site Number 578401
    Bodø, 8092, Norway
  • Investigational Site Number 643402
    Arkhangelsk, 163000, Russia
  • Investigational Site Number 643407
    Kazan', 420012, Russia
  • Investigational Site Number 643409
    Moscow, 111539, Russia
  • Investigational Site Number 643413
    Moscow, 121552, Russia
  • Investigational Site Number 643401
    Moscow, 129301, Russia
  • Investigational Site Number 643412
    Novisibirsk, Russia
  • Investigational Site Number 643408
    Saint Petersburg, 193079, Russia
  • Investigational Site Number 643406
    Saint Petersburg, 194291, Russia
  • Investigational Site Number 643404
    Saint Petersburg, 197341, Russia
  • Investigational Site Number 643410
    Yaroslavl, 150062, Russia
  • Investigational Site Number 710401
    Bloemfontein, South Africa
  • Investigational Site Number 710405
    Bloemfontein, South Africa
  • Investigational Site Number 710406
    Cap Town, 7530, South Africa
  • Investigational Site Number 710402
    Cape Town, 7708, South Africa
  • Investigational Site Number 710407
    Parktown, 2193, South Africa
  • Investigational Site Number 710403
    Parow, 7500, South Africa
  • Investigational Site Number 710408
    Pretoria, South Africa
  • Investigational Site Number 710404
    Rondebosch, South Africa
  • Investigational Site Number 710409
    Somerset West, 7130, South Africa
  • Investigational Site Number 724403
    A Coruña, 15006, Spain
  • Investigational Site Number 724408
    Barcelona, 08036, Spain
  • Investigational Site Number 724406
    Córdoba, 14004, Spain
  • Investigational Site Number 724407
    L'Hospitalet de Llobregat, 08907, Spain
  • Investigational Site Number 724409
    Madrid, 28029, Spain
  • Investigational Site Number 724401
    Madrid, 28040, Spain
  • Investigational Site Number 724405
    Madrid, 28040, Spain
  • Investigational Site Number 724404
    Reus, 43201, Spain
  • Investigational Site Number 724402
    Zaragoza, 50009, Spain
  • Investigational Site Number 752404
    Gothenburg, 41345, Sweden
  • Investigational Site Number 752401
    Stockholm, 111 35, Sweden
  • Investigational Site Number 826402
    London, United Kingdom
  • Investigational Site Number 826403
    London, United Kingdom
  • Investigational Site Number 826408
    London, United Kingdom
  • Investigational Site Number 826409
    London, United Kingdom
  • Investigational Site Number 826405
    Manchester, M23 9LT, United Kingdom
09

References and documents

Publications

  • Kastelein JJ, Robinson JG, Farnier M, Krempf M, Langslet G, Lorenzato C, Gipe DA, Baccara-Dinet MT. Efficacy and safety of alirocumab in patients with heterozygous familial hypercholesterolemia not adequately controlled with current lipid-lowering therapy: design and rationale of the ODYSSEY FH studies. Cardiovasc Drugs Ther. 2014 Jun;28(3):281-9. doi: 10.1007/s10557-014-6523-z. PubMed 24842558 ↗
  • Kastelein JJ, Ginsberg HN, Langslet G, Hovingh GK, Ceska R, Dufour R, Blom D, Civeira F, Krempf M, Lorenzato C, Zhao J, Pordy R, Baccara-Dinet MT, Gipe DA, Geiger MJ, Farnier M. ODYSSEY FH I and FH II: 78 week results with alirocumab treatment in 735 patients with heterozygous familial hypercholesterolaemia. Eur Heart J. 2015 Nov 14;36(43):2996-3003. doi: 10.1093/eurheartj/ehv370. Epub 2015 Sep 1. PubMed 26330422 ↗
  • Mahmood T, Minnier J, Ito MK, Li QH, Koren A, Kam IW, Fazio S, Shapiro MD. Discordant responses of plasma low-density lipoprotein cholesterol and lipoprotein(a) to alirocumab: A pooled analysis from 10 ODYSSEY Phase 3 studies. Eur J Prev Cardiol. 2021 Jul 23;28(8):816-822. doi: 10.1177/2047487320915803. Epub 2020 Apr 10. PubMed 34298554 ↗
  • Leiter LA, Tinahones FJ, Karalis DG, Bujas-Bobanovic M, Letierce A, Mandel J, Samuel R, Jones PH. Alirocumab safety in people with and without diabetes mellitus: pooled data from 14 ODYSSEY trials. Diabet Med. 2018 Dec;35(12):1742-1751. doi: 10.1111/dme.13817. Epub 2018 Oct 9. PubMed 30183102 ↗
  • Defesche JC, Stefanutti C, Langslet G, Hopkins PN, Seiz W, Baccara-Dinet MT, Hamon SC, Banerjee P, Kastelein JJP. Efficacy of alirocumab in 1191 patients with a wide spectrum of mutations in genes causative for familial hypercholesterolemia. J Clin Lipidol. 2017 Nov-Dec;11(6):1338-1346.e7. doi: 10.1016/j.jacl.2017.08.016. Epub 2017 Sep 4. PubMed 28964736 ↗
  • Kastelein JJ, Hovingh GK, Langslet G, Baccara-Dinet MT, Gipe DA, Chaudhari U, Zhao J, Minini P, Farnier M. Efficacy and safety of the proprotein convertase subtilisin/kexin type 9 monoclonal antibody alirocumab vs placebo in patients with heterozygous familial hypercholesterolemia. J Clin Lipidol. 2017 Jan-Feb;11(1):195-203.e4. doi: 10.1016/j.jacl.2016.12.004. Epub 2016 Dec 28. PubMed 28391886 ↗
  • Ray KK, Ginsberg HN, Davidson MH, Pordy R, Bessac L, Minini P, Eckel RH, Cannon CP. Reductions in Atherogenic Lipids and Major Cardiovascular Events: A Pooled Analysis of 10 ODYSSEY Trials Comparing Alirocumab With Control. Circulation. 2016 Dec 13;134(24):1931-1943. doi: 10.1161/CIRCULATIONAHA.116.024604. Epub 2016 Oct 24. PubMed 27777279 ↗
10

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Feb 8, 2016, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
11

Registry details

Key details

Study ID
NCT01623115
Lead sponsor
Sanofi
Collaborators
Regeneron Pharmaceuticals
Responsible party
Sponsor
First posted
Jun 19, 2012
Start date
Jul 2012
Primary completion
Apr 2014
Completion
Dec 2014
Results posted
Nov 4, 2015
Last update
Feb 8, 2016

Study contacts

Clinical Sciences & Operations
study director · Sanofi

Oversight

Data monitoring committee
Yes
View the source record on ClinicalTrials.gov ↗

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