an 8-week dose-finding study to evaluate the efficacy and safety of alirocumab in children and adolescents with heterozygous familial hypercholesterolemia

Conditions: Hypercholesterolaemia

Summary: primary objective: to evaluate the effect of alirocumab on low-density lipoprotein cholesterol (ldl-c) levels after 8 weeks of treatment in heterozygous familial hypercholesterolemia (hefh) participants aged of 8 to 17 years, with ldl-c \>=130 milligrams per deciliter (mg/dl) (3.37 millimoles per litre \[mmol/l\]) 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 for at least 4 weeks prior to the screening period. secondary objective: * to evaluate the safety and tolerability of alirocumab. * to evaluate the pharmacokinetics profile of alirocumab. * to evaluate the effects of alirocumab on other lipid parameters.

for cohorts 1 to 3, a study duration of approximately 16-23 weeks (screening period: up to 6 (+1) weeks, open-label dose finding treatment period: 8 weeks, follow up period: 6-8 weeks). for cohort 4, a study duration of approximately 14-19 weeks (screening period up to 6 \[+1\] weeks, open-label dose finding treatment period: 12 weeks). optional extension period: up to a maximum of 2 years for the first participants enrolled in cohorts 1 to 3, but a maximum of approximately 5 months for the first participants enrolled in cohort 4. for all participants who declined participation in the phase 3 study, their last alirocumab injection was on december 2018.

Eligibility criteria:
inclusion criteria : * children and adolescent male and female participants aged of 8 to 17 years at the time of signed informed consent. for russia only: male and female participants aged \>=12 and \<=17 years at the time of signed informed consent. * participants with diagnosis of heterozygous familial hypercholesterolemia (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. * participants with body weight greater than or equal to 25 kg. * participants aged of 8 to 9 years to be at tanner stage 1 and participants aged of 10 to 17 years to be at least at tanner stage 2 in their development. * a signed informed consent indicating parental permission with or without participant assent. exclusion criteria: * participant 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. * known history of type 1 or type 2 diabetes mellitus. * known history of thyroid disease. * known history of hypertension. * fasting triglycerides \>350 mg/dl (3.95 mmol/l). * severe renal impairment (i.e., estimated glomerular filtration rate \[egfr\] \<30 ml/min/1.73 m\^2). * alanine aminotransferase (alt) or aspartate aminotransferase (ast) \>2 x upper limit of normal (uln). * creatinine phosphokinase (cpk) \>3 x uln. the above information is not intended to contain all considerations relevant to a participant's potential participation in a clinical trial.