NCT04233918

Evaluate the Efficacy and Safety of Evinacumab in Pediatric Patients With Homozygous Familial Hypercholesterolemia

A Three-Part, Single-Arm, Open-Label Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of Evinacumab in Pediatric Patients With Homozygous Familial Hypercholesterolemia

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 20 (actual)
• Sponsor: Regeneron Pharmaceuticals · Industry
• Sex: All
• Age: 5 Years – 11 Years
• Healthy volunteers: Not accepted

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

Detailed description

Part A is Phase 1b Part B is Phase 3

Conditions

• Homozygous Familial Hypercholesterolemia

Interventions

Type	Name	Description
DRUG	Evinacumab	Part A: Single IV dose Part B \& C: IV dose Q4W

Timeline

• Start date: 2020-06-29
• Primary completion: 2022-01-31
• Completion: 2023-05-30
• First posted: 2020-01-18
• Last updated: 2024-07-16
• Results posted: 2023-06-07

Locations

11 sites across 6 countries: United States, Australia, Austria, Netherlands, Taiwan, Ukraine

Regulatory

• FDA-regulated drug study