Evaluate the Efficacy and Safety of Evinacumab in Pediatric Patients With Homozygous Familial Hypercholesterolemia
Status:
Recruiting
Trial end date:
2023-04-05
Target enrollment:
Participant gender:
Summary
The primary objective for Part A of the study is to assess the pharmacokinetics (PK) of
evinacumab in pediatric patients with homozygous familial hypercholesterolemia (HoFH).
The primary objective for Part B of the study is to demonstrate a reduction of low-density
lipoprotein cholesterol (LDL-C) by evinacumab in pediatric (5 to 11 years of age) patients
with HoFH.
The secondary objective for Part A of the study is to evaluate the safety and tolerability of
evinacumab administered intravenous (IV) in pediatric patients with HoFH.
The secondary objectives for Part B of the study are:
- To evaluate the effect of evinacumab on other lipid parameters (ie, apolipoprotein B
(Apo B), non-high-density lipoprotein cholesterol (non-HDL-C), total cholesterol (TC),
lipoprotein a [Lp(a)]) in pediatric patients with HoFH
- To evaluate the safety and tolerability of evinacumab administered IV in pediatric
patients with HoFH
- To assess the PK of evinacumab in pediatric patients with HoFH
- To assess the immunogenicity of evinacumab in pediatric patients with HoFH over time
- To evaluate patient efficacy by mutation status