Evinacumab in Pediatric Patients With Homozygous FH

Clinical Trial

Offered by: Nemours Children's
Location: Delaware Valley

Trial Name

Evinacumab in Pediatric Patients With Homozygous FH

What is the trial about?

Evinacumab lowers LDL-c (the “bad” cholesterol) in adults. This study evaluates the safety and efficacy of evinacumab in children 5 to 11 years of age with homozygous Familial Hypercholesterolemia (HoFH) who may be taking other medications to lower cholesterol.

Who can participate?

This registry will include children who are 5-11 years of age and who have homozygous familial hypercholesterolemia.

What is involved?

The study consists of 3 parts (A, B, C). If your child completes Part A or Part B, then your child may continue to Part C.

This is an open-label study (for Parts A, B, C). This means that you, your child, and the study doctor will know what treatment your child will receive. All participants will get evinacumab. If your child is eligible, they will receive the study drug (evinacumab). The study drug is given by intravenous (IV) infusion (injection into a vein in the arm). In addition to receiving the study treatment, your child will continue with his/her current cholesterol-lowering therapy during the study.