NCT03851705

A Study of Inclisiran in Participants With Homozygous Familial Hypercholesterolemia (HoFH)

A Two-Part (Double-Blind Placebo Controlled/Open-Label) Multicenter Study to Evaluate Safety, Tolerability, and Efficacy of Inclisiran in Subjects With Homozygous Familial Hypercholesterolemia (Hofh) (ORION-5)

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 56 (actual)
• Sponsor: Novartis Pharmaceuticals · Industry
• Sex: All
• Age: 18 Years – 80 Years
• Healthy volunteers: Accepted

Summary

This study was a Phase III,A two-part (double-blind placebo-controlled/open-label) multicenter study to evaluate safety, tolerability, and efficacy of inclisiran in subjects with homozygous familial hypercholesterolemia (HoFH).

Detailed description

This study had two sequential parts: * Part 1: 6-month double-blind period in which subjects were randomized to receive either inclisiran or placebo * Part 2: 18-month open-label follow-up period; placebo-treated subjects from Part 1 were transitioned to inclisiran at Day 180 and all subjects who participated in an open-label follow-up period of inclisiran only

Conditions

• Homozygous Familial Hypercholesterolemia

Interventions

Type	Name	Description
DRUG	Inclisiran Sodium for injection	Inclisiran is a synthetic, chemically modified small interfering ribonucleic acid (siRNA) targeting proprotein convertase subtilisin kexin type 9 (PCSK9) messenger ribonucleic acid (mRNA) with a covalently attached triantennary N-acetylgalactosamine (GalNAc) ligand.
DRUG	Placebo	Sterile normal saline (0.9% sodium chloride in water for injection)
DRUG	Placebos	Sterile normal saline (0.9% sodium chloride in water for injection)

Timeline

• Start date: 2019-02-06
• Primary completion: 2020-03-02
• Completion: 2021-09-09
• First posted: 2019-02-22
• Last updated: 2023-01-30
• Results posted: 2022-11-10

Locations

13 sites across 8 countries: Hong Kong, Israel, Russia, Serbia, South Africa, Taiwan, Turkey (Türkiye), Ukraine

Regulatory

• FDA-regulated drug study