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

Completed · Phase 2

Conditions studied: Hypercholesterolaemia

In brief

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.

Key facts

Study ID

NCT02890992

Run by

Sanofi

People needed

42

Starts

2016-09-15

Expected to finish

2019-02-22

Last updated by the study team

2019-09-06

Who can join

Age: 8 and older, up to 17. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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.

You may not qualify if…

• 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.

Where it is running

• Investigational Site Number 8400002 — St Louis, Missouri, United States
• Investigational Site Number 8400005 — Charlotte, North Carolina, United States
• Investigational Site Number 8400001 — Cincinnati, Ohio, United States
• Investigational Site Number 1240001 — Québec, Canada
• Investigational Site Number 2030001 — Brno, Czechia
• Investigational Site Number 2030003 — Praha 5 - Motol, Czechia
• Investigational Site Number 2030002 — Zlín, Czechia
• Investigational Site Number 2500001 — Bron, France
• Investigational Site Number 5280001 — Amsterdam, Netherlands
• Investigational Site Number 5780001 — Oslo, Norway
• Investigational Site Number 6430001 — Kemerovo, Russia
• Investigational Site Number 6430004 — Saint Petersburg, Russia
• Investigational Site Number 7100001 — Parow, South Africa
• Investigational Site Number 7240004 — A Coruña, Spain
• Investigational Site Number 7240001 — Madrid, Spain
• Investigational Site Number 7520001 — Stockholm, Sweden

Full record on ClinicalTrials.gov

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