Last updated on August 2019

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

Brief description of study

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 patients 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 Objective:

  • To evaluate the efficacy of alirocumab versus placebo on low-density lipoprotein cholesterol (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.

Detailed Study Description

The study duration is 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 treatment period: 80 weeks).

Clinical Study Identifier: NCT03510884

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Investigational Site Number 4220003

Room Hospital Street, Achrafie, Lebanon
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