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

NCT03510884 · Status: COMPLETED · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 153

Last updated 2023-05-06

Study results available
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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.

Conditions

  • Hypercholesterolaemia

Interventions

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

Sponsors & Collaborators

Principal Investigators

  • Clinical Sciences & Operations · Sanofi

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
TRIPLE
Model
PARALLEL

Eligibility

Min Age
8 Years
Max Age
17 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2018-05-31
Primary Completion
2021-01-14
Completion
2022-08-05
FDA Drug
Yes

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)

Study Locations

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Entities

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT03510884 on ClinicalTrials.gov