NCT03510715

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

An Open-Label Study to Evaluate the Efficacy and Safety of Alirocumab in Children and Adolescents With Homozygous Familial Hypercholesterolemia

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 18 (actual)
• Sponsor: Sanofi · Industry
• Sex: All
• Age: 8 Years – 17 Years
• Healthy volunteers: Not accepted

Summary

Primary Objective: To evaluate the efficacy of alirocumab (75 or 150 milligrams \[mg\] depending on body weight \[BW\]), administered every 2 weeks (Q2W), on low-density lipoprotein cholesterol (LDL-C) levels at Week 12 of treatment in children and adolescents with homozygous familial hypercholesterolemia (hoFH) of 8 to 17 years of age on top of background treatments. Secondary Objectives: * To evaluate the efficacy of alirocumab after 24 and 48 weeks of treatment on LDL-C levels. * To evaluate the effects of alirocumab on other lipid parameters (eg, apolipoprotein B \[Apo B\], non-high density lipoprotein cholesterol \[non-HDL-C\], total cholesterol \[Total-C\], high density lipoprotein cholesterol \[HDL-C\], lipoprotein a \[Lp (a)\], triglycerides \[TG\], apolipoprotein A-1 \[Apo A-1\] levels) after 12, 24, and 48 weeks of treatment. * To evaluate the safety and tolerability of alirocumab up to 48 weeks of treatment.

Detailed description

The study duration was up to 62 weeks, which included (if needed) a run-in period of up to 4 weeks, a screening period of up to 2 weeks, a treatment period of up to 48 weeks, and a follow-up of 8 weeks.

Conditions

• Hypercholesterolemia

Interventions

Type	Name	Description
DRUG	Alirocumab SAR236553 (REGN727)	Pharmaceutical form: solution for injection in pre-filled syringe, Route of administration: subcutaneous (SC)
DRUG	Atorvastatin	Pharmaceutical form: tablet, Route of administration: oral
DRUG	Simvastatin	Pharmaceutical form: tablet, Route of administration: oral
DRUG	Fluvastatin	Pharmaceutical form: capsule, Route of administration: oral
DRUG	Pravastatin	Pharmaceutical form: tablet, Route of administration: oral
DRUG	Lovastatin	Pharmaceutical form: tablet, Route of administration: oral
DRUG	Rosuvastatin	Pharmaceutical form: tablet, 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

Timeline

• Start date: 2018-08-31
• Primary completion: 2020-02-17
• Completion: 2020-02-17
• First posted: 2018-04-27
• Last updated: 2020-12-29
• Results posted: 2020-12-29

Locations

10 sites across 10 countries: Brazil, Canada, Denmark, Mexico, Netherlands, Russia, Slovenia, Spain, Taiwan, Turkey (Türkiye)

Regulatory

• FDA-regulated drug study