NCT03510884

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

A Randomized, Double-Blind, Placebo-Controlled Study Followed by an Open Label Treatment Period to Evaluate the Efficacy and Safety of Alirocumab in Children and Adolescents With Heterozygous Familial Hypercholesterolemia

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 153 (actual)
• Sponsor: Sanofi · Industry
• Sex: All
• Age: 8 Years – 17 Years
• Healthy volunteers: Not accepted

Summary

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.

Detailed description

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).

Conditions

• Hypercholesterolaemia

Interventions

Type	Name	Description
DRUG	Alirocumab SAR236553 (REGN727)	Pharmaceutical form:solution Route of administration: subcutaneous injection
DRUG	Rosuvastatin	Pharmaceutical form:tablet Route of administration: oral
DRUG	Atorvastatin	Pharmaceutical form:Tablet Route of administration: Oral
DRUG	Simvastatin	Pharmaceutical form:Tablet Route of administration: Oral
DRUG	Pravastatin	Pharmaceutical form:Tablet Route of administration: Oral
DRUG	Lovastatin	Pharmaceutical form:Tablet Route of administration: Oral
DRUG	Fluvastatin	Pharmaceutical form:Capsule Route of administration: Oral
DRUG	Ezetimibe	Pharmaceutical form:Tablet Route of administration: Oral
DRUG	Cholestyramine	Pharmaceutical form:oral suspension Route of administration: oral
DRUG	Nicotinic acid	Pharmaceutical form:Tablet Route of administration: Oral
DRUG	Fenofibrate	Pharmaceutical form:Tablet Route of administration: Oral
DRUG	Omega-3 fatty acids	Pharmaceutical form:capsule Route of administration: oral
DRUG	Placebo	Pharmaceutical form:solution Route of administration: subcutaneous injection

Timeline

• Start date: 2018-05-31
• Primary completion: 2021-01-14
• Completion: 2022-08-05
• First posted: 2018-04-27
• Last updated: 2023-05-06
• Results posted: 2023-05-06

Locations

43 sites across 24 countries: United States, Argentina, Austria, Brazil, Bulgaria, Canada, Czechia, Denmark, Finland, France, Hungary, Italy, Lebanon, Mexico, Netherlands, Norway, Poland, Russia, Slovenia, South Africa, Spain, Sweden, Taiwan, Turkey (Türkiye)

Regulatory

• FDA-regulated drug study