NCT03399786

Efficacy and Safety of Evinacumab in Patients With Homozygous Familial Hypercholesterolemia

A Randomized, Double-blind, Placebo-controlled, Parallel-group Study to Evaluate the Efficacy and Safety of Evinacumab in Patients With Homozygous Familial Hypercholesterolemia

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 65 (actual)
• Sponsor: Regeneron Pharmaceuticals · Industry
• Sex: All
• Age: 12 Years
• Healthy volunteers: Not accepted

Summary

The primary objective of the study is to demonstrate the reduction of low-density lipoprotein cholesterol (LDL-C) by evinacumab intravenously (IV) in comparison to placebo after 24 weeks in patients with homozygous familial hypercholesterolemia (HoFH). The secondary objectives of the study are to evaluate the effect of evinacumab IV on other lipid parameters, evaluate the effect of evinacumab on LDL-C goal attainment, assess the effect of evinacumab on eligibility for apheresis (using German and US apheresis criteria), evaluate the safety and tolerability of evinacumab in patients with HoFH, assess the pharmacokinetics (PK) of evinacumab in patients with HoFH and evaluate the potential development of anti-evinacumab antibodies.

Conditions

• Homozygous Familial Hypercholesterolemia

Interventions

Type	Name	Description
DRUG	evinacumab	IV administration of evinacumab
DRUG	Placebo	IV administration of placebo

Timeline

• Start date: 2018-01-18
• Primary completion: 2019-06-10
• Completion: 2020-03-17
• First posted: 2018-01-16
• Last updated: 2021-05-18
• Results posted: 2021-05-18

Locations

30 sites across 11 countries: United States, Australia, Austria, Canada, France, Greece, Italy, Japan, Netherlands, South Africa, Ukraine

Regulatory

• FDA-regulated drug study