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 (ODYSSEY FH I)

Sanofi

Status and phase: Completed; Phase 3

Conditions: Hypercholesterolemia

Treatments: Drug: Alirocumab; Drug: Placebo (for alirocumab); Drug: Lipid Modifying Therapy (LMT)

Study type: Interventional

Funder types: Industry

Identifiers

NCT01623115

2011-005109-56 (EudraCT Number)

U1111-1121-4275 (Other Identifier)

EFC12492

Details and patient eligibility

About

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

Full 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.

Enrollment: 486 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

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

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.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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486 participants in 2 patient groups, including a placebo group

Placebo

Placebo Comparator group

Description:

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

Treatment:

Drug: Placebo (for alirocumab)

Drug: Lipid Modifying Therapy (LMT)

Alirocumab 75 mg/Up to 150 mg Q2W

Experimental group

Description:

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.

Treatment:

Drug: Lipid Modifying Therapy (LMT)

Drug: Alirocumab