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Efficacy and Safety of Alirocumab Versus Placebo on Top of Maximally Tolerated Lipid Lowering Therapy in Patients With Hypercholesterolemia Who Have Type 1 or Type 2 Diabetes and Are Treated With Insulin (ODYSSEY DM - Insulin)

Phase 3
Completed
Conditions
Hypercholesterolaemia
Interventions
Drug: Placebo
Drug: Lipid-Modifying Therapy (LMT)
Drug: Antihyperglycemic Drug
Registration Number
NCT02585778
Lead Sponsor
Sanofi
Brief Summary

Primary Objectives:

* To demonstrate the superiority of alirocumab in comparison with placebo in the reduction of calculated low-density lipoprotein cholesterol (LDL-C) in participants with diabetes treated with insulin and with hypercholesterolemia at high cardiovascular risk not adequately controlled on maximally tolerated LDL-C lowering therapy.

* To evaluate the safety and tolerability of alirocumab in participants with diabetes treated with insulin.

Secondary Objective:

To demonstrate that alirocumab was superior in comparison to placebo in its effects on other lipid parameters (i.e., measured LDL-C, non-high-density lipoprotein cholesterol \[non-HDL-C\], apolipoprotein B \[Apo B\], total cholesterol \[TC\], lipoprotein a \[Lp(a)\], high density lipoprotein cholesterol \[HDL-C\], triglyceride \[TG\] levels, triglyceride rich lipoproteins \[TGRL\], apolipoprotein A-1 \[Apo A-1\], apolipoprotein C-III \[Apo C-III\], and LDL particle number and size).

Detailed Description

The maximum study duration was approximately 9 months per participant, including a 6 month treatment period, a screening period of up to 3 weeks, and an 8 week safety observation period.

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
517
Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
Alirocumab 75 mg Q2W/Up to 150 mg Q2WAntihyperglycemic DrugAlirocumab 75 mg subcutaneous (SC) injection every 2 weeks (Q2W) added to stable, maximally tolerated dose of statin therapy with or without other lipid-modifying therapy (LMT), insulin alone or with other antihyperglycemic drugs for 24 weeks. Alirocumab dose up-titrated to 150 mg Q2W from Week 12 when LDL-C levels ≥70 mg/dL (1.81 mmol/L) at Week 8.
Placebo Q2WPlaceboPlacebo (for alirocumab) SC injection Q2W added to stable, maximally tolerated dose of statin therapy with or without other LMT, insulin alone or with other antihyperglycemic drugs for 24 weeks.
Alirocumab 75 mg Q2W/Up to 150 mg Q2WLipid-Modifying Therapy (LMT)Alirocumab 75 mg subcutaneous (SC) injection every 2 weeks (Q2W) added to stable, maximally tolerated dose of statin therapy with or without other lipid-modifying therapy (LMT), insulin alone or with other antihyperglycemic drugs for 24 weeks. Alirocumab dose up-titrated to 150 mg Q2W from Week 12 when LDL-C levels ≥70 mg/dL (1.81 mmol/L) at Week 8.
Placebo Q2WLipid-Modifying Therapy (LMT)Placebo (for alirocumab) SC injection Q2W added to stable, maximally tolerated dose of statin therapy with or without other LMT, insulin alone or with other antihyperglycemic drugs for 24 weeks.
Placebo Q2WAntihyperglycemic DrugPlacebo (for alirocumab) SC injection Q2W added to stable, maximally tolerated dose of statin therapy with or without other LMT, insulin alone or with other antihyperglycemic drugs for 24 weeks.
Alirocumab 75 mg Q2W/Up to 150 mg Q2WAlirocumabAlirocumab 75 mg subcutaneous (SC) injection every 2 weeks (Q2W) added to stable, maximally tolerated dose of statin therapy with or without other lipid-modifying therapy (LMT), insulin alone or with other antihyperglycemic drugs for 24 weeks. Alirocumab dose up-titrated to 150 mg Q2W from Week 12 when LDL-C levels ≥70 mg/dL (1.81 mmol/L) at Week 8.
Primary Outcome Measures
NameTimeMethod
Percent Change From Baseline in Calculated LDL-C at Week 24 - Intent-to-treat (ITT) AnalysisFrom Baseline to Week 24

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 24 regardless of status on- or off-treatment were used in the model (ITT analysis).

Percentage of Participants Who Experienced Treatment-Emergent Adverse Events (AEs)From Baseline up to 10 weeks after last study drug administration (maximum of 32 weeks)

Reported adverse events are treatment-emergent adverse events that is AEs that developed/worsened during the 'treatment-emergent period' (the time from the first dose of study drug up to the last dose of study drug +70 days).

Secondary Outcome Measures
NameTimeMethod
Percentage of Participants Reaching Calculated LDL-C <70 mg/dL (1.81 mmol/L) at Week 24 - On-Treatment AnalysisUp to Week 24

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

Percentage of Participants Reaching Calculated Non-HDL-C <100 mg/dL at Week 24 - On-Treatment AnalysisUp to Week 24

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

Absolute Change From Baseline in Insulin Daily Dose/Kg at Weeks 12 and 24 - On-Treatment AnalysisBaseline, Weeks 12 and 24

Absolute change = daily insulin dose/kg at specified weeks minus baseline value.

Percent Change From Baseline in Calculated LDL-C at Week 12 - ITT AnalysisFrom Baseline to Week 24

Adjusted LS means and standard errors at Week 12 from MMRM model including available post-baseline data from Week 4 to Week 24 regardless of status on- or off-treatment.

Percent Change From Baseline in Non-High Density Lipoprotein Cholesterol (Non-HDL-C) at Week 24 - ITT AnalysisFrom Baseline to Week 24

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

Percent Change From Baseline in HDL-C at Week 24 - ITT AnalysisFrom Baseline to Week 24

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

Percent Change From Baseline in Measured LDL-C at Week 12 - ITT AnalysisFrom Baseline to Week 24

Measured LDL-C values via beta quantification method. Adjusted LS means and standard errors at Week 12 from MMRM model including available post-baseline data from Week 4 to Week 24 regardless of status on- or off-treatment.

Percent Change From Baseline in Lipoprotein(a) at Week 24 - ITT AnalysisFrom Baseline to Week 24

Adjusted means and standard errors at Week 24 were obtained from multiple imputation approach for handling of missing data followed by robust regression model. All available post-baseline data from Week 4 to Week 24 regardless of status on- or off-treatment were included in the imputation model.

Percent Change From Baseline in Fasting Triglycerides at Week 24 - ITT AnalysisFrom Baseline to Week 24

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

Percent Change From Baseline in Calculated LDL-C at Week 24 - On-Treatment AnalysisFrom Baseline to Week 24

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

Percentage of Participants Reaching Calculated Non-HDL-C <80 mg/dL at Week 24 - On-Treatment AnalysisUp to Week 24

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

Percent Change From Baseline in Measured LDL-C at Week 24 - ITT AnalysisFrom Baseline to Week 24

Measured LDL-C values via beta quantification method. Adjusted LS means and standard errors at Week 24 from MMRM model including available post-baseline data from Week 4 to Week 24 regardless of status on- or off-treatment.

Percent Change From Baseline in Apolipoprotein B (Apo-B) at Week 24 - ITT AnalysisFrom Baseline to Week 24

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

Percent Change From Baseline in Total Cholesterol (Total-C) at Week 24 - ITT AnalysisFrom Baseline to Week 24

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

Percentage of Participants Reaching Calculated LDL-C <50 mg/dL (1.3 mmol/L) at Week 24 - On-Treatment AnalysisUp to Week 24

Adjusted percentages at Week 24 from last observation carried forward (LOCF) approach (for T1DM participants) and multiple imputation approach model (for T2DM participants) including available post-baseline data from Week 4 to Week 24 (i.e. up to 21 days after last injection). The maximum likelihood estimate did not exist as response rate was zero in a treatment group of T1DM participants.

Percent Change From Baseline in LDL-C Particle Number at Week 24 - ITT AnalysisFrom Baseline to Week 24

LDL-C particle number was calculated from lipid subfractions by nuclear magnetic resonance (NMR) spectroscopy. Adjusted LS means and standard errors at Week 24 from MMRM model including all available post-baseline data from Week 4 to Week 24 regardless of status on- or off-treatment.

Absolute Change From Baseline in HbA1c at Weeks 12 and 24 - On-Treatment AnalysisBaseline, Weeks 12 and 24

Absolute change = HbA1c value at specified weeks minus HbA1c value at baseline.

Percent Change From Baseline in LDL-C Particle Size at Week 24 - ITT AnalysisFrom Baseline to Week 24

LDL-C particle size was calculated from lipid subfractions by NMR spectroscopy. Adjusted LS means and standard errors at Week 24 from MMRM model including available post-baseline data from Week 4 to Week 24 regardless of status on- or off-treatment.

Absolute Change From Baseline in Glycosylated Hemoglobin (HbA1c) at Weeks 12 and 24 - ITT AnalysisBaseline, Weeks 12 and 24

Absolute change = HbA1c value at specified weeks minus HbA1c value at baseline.

Absolute Change From Baseline in FPG at Weeks 12 and 24 - On-Treatment AnalysisBaseline, Weeks 12 and 24

Absolute change = FPG value at specified weeks minus FPG value at baseline.

Absolute Change From Baseline in Insulin Daily Dose/Kg at Weeks 12 and 24 - ITT AnalysisBaseline, Weeks 12 and 24

Absolute change = daily insulin dose/kg at specified weeks minus baseline value.

Absolute Change From Baseline in Fasting Plasma Glucose (FPG) at Weeks 12 and 24 - ITT AnalysisBaseline, Weeks 12 and 24

Absolute change = FPG value at specified weeks minus FPG value at baseline.

Absolute Change From Baseline in Number of Glucose-Lowering Treatments at Weeks 12 and 24 - ITT AnalysisBaseline, Weeks 12 and 24

Glucose lowering treatment was calculated for non-insulin treatments as one for each unique treatment received and for insulin treatment as one in total for all participants who have taken one or more treatments. Absolute change = number of glucose-lowering treatments at specified weeks minus baseline value.

Absolute Change From Baseline in Number of Glucose-Lowering Treatments at Weeks 12 and 24 - On-Treatment AnalysisBaseline, Weeks 12 and 24

Glucose lowering treatment was calculated for non-insulin treatments as one for each unique treatment received and for insulin treatment as one in total for all participants who have taken one or more treatments. Absolute change = number of glucose-lowering treatments at specified weeks minus baseline value.

Absolute Change From Baseline in Total Daily Insulin Dose at Weeks 12 and 24 - ITT AnalysisBaseline, Weeks 12 and 24

Absolute change = total daily insulin dose at specified weeks minus baseline value.

Absolute Change From Baseline in Total Daily Insulin Dose at Weeks 12 and 24 - On-Treatment AnalysisBaseline, Weeks 12 and 24

Absolute change = total daily insulin dose at specified weeks minus baseline value.

Trial Locations

Locations (110)

Investigational Site Number 840020

🇺🇸

Encino, California, United States

Investigational Site Number 840002

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Fresno, California, United States

Investigational Site Number 840029

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Oakland, California, United States

Investigational Site Number 840027

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Loveland, Colorado, United States

Investigational Site Number 840026

🇺🇸

Atlantis, Florida, United States

Investigational Site Number 840006

🇺🇸

Bradenton, Florida, United States

Investigational Site Number 840023

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Jacksonville, Florida, United States

Investigational Site Number 840028

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Palm Harbor, Florida, United States

Investigational Site Number 840022

🇺🇸

Ponte Vedra Beach, Florida, United States

Investigational Site Number 840021

🇺🇸

Roswell, Georgia, United States

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Investigational Site Number 840020
🇺🇸Encino, California, United States

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