NCT02434497

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

An Open-Label Long-Term Extension to the 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: 9 (actual)
• Sponsor: AstraZeneca · Industry
• Sex: All
• Age: 6 Years – 18 Years
• Healthy volunteers: Not accepted

Summary

The purpose of the study is to evaluate the safety of Rosuvastatin in Children and Adolescents with Homozygous Familial Hypercholesterolemia.

Detailed description

This is a long-term extension (LTE) to the randomized, double-blind, cross-over study of rosuvastatin 20 mg once daily (QD) versus placebo QD in children and adolescents (aged from 6 to \<18 years) with homozygous familial hypercholesterolemia (HoFH) (Study D3561C00004). The study is designed to assess the long-term safety and tolerability of rosuvastatin 20 mg in pediatric patients with HoFH. In this study all patients will receive rosuvastatin 20 mg QD. Investigators will also be permitted to titrate the dose of rosuvastatin from 20 to 40 mg per day if they feel it is warranted to more aggressively treat patients' elevated LDL-C levels. This up-titration will not be permitted in Asian patients. Pharmacokinetic data of the trough plasma exposure of rosuvastatin will also be assessed for the pediatric patients with HoFH taking a daily dose of rosuvastatin 40 mg. The primary outcome measures to be assessed include 1) Adverse events, including: * The frequency and severity of adverse events, * Rate of discontinuations due to adverse events, * Abnormal serum and urine laboratory values, electrocardiograms (ECGs), physical examinations, and vital signs; and 2) Assessments of growth.

Conditions

• Homozygous Familial Hypercholesterolemia (HoFH)

Interventions

Type	Name	Description
DRUG	Rosuvastatin 20mg	Active drug 1 or 2 tablets will be taken taken orally, QD, either in the morning or in the evening

Timeline

• Start date: 2015-06-06
• Primary completion: 2016-11-17
• Completion: 2016-11-17
• First posted: 2015-05-05
• Last updated: 2018-02-27
• Results posted: 2018-02-27

Locations

6 sites across 6 countries: Belgium, Canada, Denmark, Israel, Malaysia, Taiwan