NCT02226198

A Study to Evaluate the Efficacy and Safety of Rosuvastatin in Children and Adolescents With Homozygous Familial Hypercholesterolemia

A Randomized, Double-blind, Placebo-controlled, Multi-center, Cross-over Study of Rosuvastatin in Children and Adolescents (Aged 6 to <18 Years) With Homozygous Familial Hypercholesterolemia (HoFH)

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 20 (actual)
• Sponsor: AstraZeneca · Industry
• Sex: All
• Age: 6 Years – 17 Years
• Healthy volunteers: Not accepted

Summary

The purpose of the study is to establish the efficacy, safety and tolerability of rosuvastatin in children and adolescents with homozygous familial hypercholesterolemia.

Detailed description

This is a randomized, double-blind, placebo-controlled, multi-center, cross-over study of the efficacy, safety and tolerability rosuvastatin in children and adolescents (aged 6 to \<18 years) with homozygous familial hypercholesterolemia (HoFH). The study is designed to assess the efficacy of rosuvastatin 20 mg compared to placebo on lipids, lipoproteins and apolipoproteins in pediatric patients with HoFH. The outcome measures to be assessed include low density lipoprotein cholesterol (LDL-C), high density lipoprotein cholesterol (HDL-C), total cholesterol (TC), triglycerides, non-HDL-C, LDL-C/HDL-C, TC/HDL-C, non-HDL-C/HDL-C, apolipoprotein B (ApoB), apolipoprotein A 1 (ApoA-1) and ApoB/ApoA-1 following 6 weeks of treatment with rosuvastatin 20 mg or placebo. Pharmacokinetic data of the trough plasma exposure of rosuvastatin will also be assessed in these pediatric patients with HoFH.

Conditions

• Homozygous Familial Hypercholesterolemia (HoFH)

Interventions

Type	Name	Description
DRUG	Rosuvastatin 20mg	Active drug will be taken taken orally, QD, either in the morning or in the evening
DRUG	Placebo	Will be taken taken orally, QD, either in the morning or in the evening

Timeline

• Start date: 2014-11-01
• Primary completion: 2015-07-01
• Completion: 2015-07-01
• First posted: 2014-08-27
• Last updated: 2016-07-04
• Results posted: 2016-07-04

Locations

9 sites across 7 countries: Belgium, Canada, Denmark, Israel, Malaysia, Netherlands, Taiwan