NCT02265952

Study of REGN1500 in Participants With Homozygous Familial Hypercholesterolemia (HoFH)

An Open-Label, Single-Arm, Proof-of-Concept Study to Evaluate the Safety and Efficacy of Single and Multiple Doses of REGN1500 in Patients With Homozygous Familial Hypercholesterolemia

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 9 (actual)
• Sponsor: Regeneron Pharmaceuticals · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

This is an open-label, single-arm study to assess the reduction of low-density lipoprotein cholesterol (LDL-C) by REGN1500 in patients with homozygous familial hypercholesterolemia (HoFH).

Conditions

• Homozygous Familial Hypercholesterolemia

Interventions

Type	Name	Description
DRUG	REGN1500 250 mg SC/15 mg/kg IV/450 mg SC	Participants received single dose of REGN1500 subcutaneous (SC) injection of 250 milligrams (mg) at Week 0 (Day 1), followed by single dose of REGN1500 intravenous (IV) injection of 15 milligrams per kilogram (mg/kg) at Week 2 (Day 15) and then followed by 4 doses of REGN1500 SC injection of 450 mg once weekly starting from Week 12 (Day 85). Only the first 2 enrolled participants received 4 weekly REGN1500 450 mg SC doses at weeks 12, 13, 14, and 15 per the protocol. This dose regimen was removed under protocol amendment 4. Participants were followed for a period of 24 weeks (through Week 26 \[Day 183\]) after the last dose of study drug in the main study period.

Timeline

• Start date: 2015-02-04
• Primary completion: 2016-11-21
• Completion: 2018-07-23
• First posted: 2014-10-16
• Last updated: 2019-12-09
• Results posted: 2019-12-09

Locations

5 sites across 3 countries: United States, Canada, Netherlands