NCT03156621

Study in Participants With Homozygous Familial Hypercholesterolemia (HoFH)

A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate the Efficacy and Safety of Alirocumab in Patients With Homozygous Familial Hypercholesterolemia

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 69 (actual)
• Sponsor: Regeneron Pharmaceuticals · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

The primary objective of the study is to demonstrate the reduction of low-density lipoprotein cholesterol (LDL-C) with alirocumab subcutaneous (SC) every 2 weeks (Q2W) in comparison to placebo after 12 weeks of treatment. The secondary objectives of the study are: * To evaluate the effect of alirocumab Q2W on other lipid parameters (ie, apolipoprotein \[Apo\] A-1 and B, non-high-density lipoprotein cholesterol \[non-HDL-C\], total-cholesterol \[TC\], proportion of participants with 15%, 30%, and 50% LDL-C reductions, Lp(a), HDL-C, triglycerides \[TG\]) in participants with HoFH * To evaluate the safety and tolerability of alirocumab SC Q2W in participants with HoFH * To assess the pharmacokinetics of alirocumab SC Q2W in participants with HoFH * To assess the potential development of anti-drug (alirocumab) antibodies

Conditions

• Homozygous Familial Hypercholesterolemia

Interventions

Type	Name	Description
DRUG	Alirocumab	Alirocumab SC Q2W
DRUG	Placebo	Matching placebo SC Q2W

Timeline

• Start date: 2017-10-03
• Primary completion: 2019-09-27
• Completion: 2020-02-13
• First posted: 2017-05-17
• Last updated: 2021-06-29
• Results posted: 2021-06-29

Locations

28 sites across 13 countries: United States, Austria, Canada, Czechia, France, Germany, Greece, Italy, Japan, South Africa, Taiwan, Turkey (Türkiye), Ukraine

Regulatory

• FDA-regulated drug study