Conditions: Homozygous Familial Hypercholesterolemia (HoFH)
Keywords: LDL-C HoFH Hyperlipidemia
Allocation: RANDOMIZED | Intervention model: CROSSOVER
Enrollment: 20
Healthy volunteers: no

Brief 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.

Arms:
arm 1: 6-week treatment period, and after crossover finished a 12-week efficacy maintenance phase for all patients arm 2: 6 weeks treatment during crossover

Interventions: intervention 1: Rosuvastatin 20mg intervention 2: Placebo
Intervention descriptions: intervention 1: Active drug will be taken taken orally, QD, either in the morning or in the evening intervention 2: Will be taken taken orally, QD, either in the morning or in the evening

Protocol document: