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An 8-Week Dose-Finding Study to Evaluate the Efficacy and Safety of Alirocumab in Children and Adolescents With Heterozygous Familial Hypercholesterolemia

NCT02890992

CompletedPhase 2

Sponsor: Sanofi

Conditions: Hypercholesterolaemia

Interventions: alirocumab SAR236553 (REGN727), statins, ezetimibe, cholestyramine, fenofibrate

Countries: United States, Canada, Czechia, France, Netherlands, Norway, Russia, South Africa

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.

Eligibility overview

Sex: ALL

Age: 8 Years to 17 Years

Healthy volunteers: No

Study type: INTERVENTIONAL

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.

Locations (16)

St Louis, Missouri, United States
Charlotte, North Carolina, United States
Cincinnati, Ohio, United States
Québec, Canada
Brno, Czechia
Praha 5 - Motol, Czechia
Zlín, Czechia
Bron, France
Amsterdam, Netherlands
Oslo, Norway
Kemerovo, Russia
Saint Petersburg, Russia
Parow, South Africa
A Coruña, Spain
Madrid, Spain
Stockholm, Sweden