Resumen

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.

Descripción detallada

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).

Elegibilidad

Edad mínima: 8 YearsEdad máxima: 17 YearsSexo: ALL

Criterios de inclusión

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.

Criterios de exclusión

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.

Intervenciones

drug

Alirocumab SAR236553 (REGN727)

Pharmaceutical form:solution Route of administration: subcutaneous injection

drug