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

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.

Conditions

• Hypercholesterolemia

Interventions

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

Sponsors & Collaborators

• Regeneron Pharmaceuticals

  collaborator INDUSTRY

• Sanofi

  lead INDUSTRY

Principal Investigators

• Clinical Sciences & Operations · Sanofi

Study Design

Allocation

NA

Purpose

TREATMENT

Masking

NONE

Model

SINGLE_GROUP

Eligibility

Min Age

8 Years

Max Age

17 Years

Sex

ALL

Healthy Volunteers

No

Timeline & Regulatory

Start

2018-08-31

Primary Completion

2020-02-17

Completion

2020-02-17

FDA Drug

Yes

Countries

• Brazil
• Canada
• Denmark
• Mexico
• Netherlands
• Russia
• Slovenia
• Spain
• Taiwan
• Turkey (Türkiye)

Study Locations