An Efficacy and Safety Study of Alirocumab in Children and Adolescents With Heterozygous Familial Hypercholesterolemia

A Randomized, Double-Blind, Placebo-Controlled Study Followed by an Open Label Treatment Period to Evaluate the Efficacy and Safety of Alirocumab in Children and Adolescents With Heterozygous Familial Hypercholesterolemia

Primary Objective:

To evaluate the efficacy of alirocumab administered every 2 weeks (Q2W) and every 4 weeks (Q4W) versus placebo after 24 weeks of double-blind (DB) treatment on low-density lipoprotein cholesterol (LDL-C) levels in participants with heterozygous familial hypercholesterolemia (heFH) 8 to 17 years of age on optimal stable daily dose of statin therapy ± other lipid modifying therapies (LMTs) or a stable dose of non-statin LMTs in case of intolerance to statins.

Secondary Objectives:

• To evaluate the efficacy of alirocumab versus placebo on LDL-C levels.
• To evaluate the effects of alirocumab versus placebo on other lipid parameters.
• To evaluate the safety and tolerability of alirocumab in comparison with placebo.
• To evaluate the efficacy, safety, and tolerability of alirocumab after open label treatment.
• To evaluate the development of anti-alirocumab antibodies.

The study duration was approximately up to 110 weeks (run-in period [if needed]: up to 4 weeks [+2 days], screening period: up to 2 weeks [+5 days], double-blind treatment period: 24 weeks, open label (OL) treatment period: 80 weeks).

Inclusion criteria:

• Children and adolescent male and female participants 8 to 17 years of age at the time of signed informed consent.
• Participants with diagnosis of heFH through genotyping or clinical criteria.
• Participants treated with optimal dose of statin +/- other LMT(s) or non-statin LMT(s) if statin intolerant at stable dose for at least 4 weeks prior to screening lipid sampling.
• Participants with calculated LDL-C greater than or equal to 130 mg/dL (>=3.37 mmol/L) at the screening visit except for participants who have previously participated in the DFI14223 (NCT02890992) study.
• A signed informed consent indicating parental permission with or without participant assent.

Exclusion criteria:

• Participant with body weight < 25 kg.
• Participants aged of 8 to 9 years not at Tanner stage 1 and participants aged of 10 to 17 years not at least at Tanner stage 2 in their development.
• Participants with secondary hyperlipidemia.
• Diagnosis of homozygous familial hypercholesterolemia.
• Participant who had received lipid apheresis treatment within 2 months prior to the screening period, or has plans to receive it during the study.
• Participants with uncontrolled type 1 or type 2 diabetes mellitus.
• Participants with known uncontrolled thyroid disease.
• Participants with uncontrolled hypertension.
• Fasting triglycerides greater than (>) 350 mg/dL (3.95 mmol/L).
• Severe renal impairment (ie, estimated glomerular filtration rate <30 mL/min/1.73 m^2).
• Alanine aminotransferase or aspartate aminotransferase >2*upper limit of normal (ULN).
• Creatinine phosphokinase (CPK) >3*ULN.

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