An Efficacy and Safety Study of Alirocumab in Children and Adolescents With Homozygous Familial Hypercholesterolemia
Status:
Completed
Trial end date:
2020-02-17
Target enrollment:
Participant gender:
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.