NCT04652726

Study to Evaluate Efficacy and Safety of Inclisiran in Adolescents With Heterozygous Familial Hypercholesterolemia

Two Part (Double-blind Inclisiran Versus Placebo [Year 1] Followed by Open-label Inclisiran [Year 2]) Randomized Multicenter Study to Evaluate Safety, Tolerability, and Efficacy of Inclisiran in Adolescents (12 to Less Than 18 Years) With Heterozygous Familial Hypercholesterolemia and Elevated LDL-cholesterol (ORION-16)

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 141 (actual)
• Sponsor: Novartis Pharmaceuticals · Industry
• Sex: All
• Age: 12 Years – 17 Years
• Healthy volunteers: Not accepted

Summary

This was a pivotal phase III study designed to evaluate safety, tolerability, and efficacy of inclisiran in adolescents with heterozygous familial hypercholesterolemia (HeFH) and elevated low density lipoprotein cholesterol (LDL-C).

Detailed description

This was a two-part (1 year double-blind inclisiran versus placebo / 1 year open-label inclisiran) multicenter study designed to evaluate safety, tolerability, and efficacy of inclisiran in adolescents with heterozygous familial hypercholesterolemia (HeFH) and elevated low density lipoprotein cholesterol (LDL-C) on stable standard of care background lipid-lowering therapy. The primary objective was to demonstrate superiority of inclisiran compared to placebo in reducing LDL-C (percent change) at Day 330.

Conditions

• Familial Hypercholesterolemia - Heterozygous

Interventions

Type	Name	Description
DRUG	Inclisiran	Inclisiran sodium 300 mg (equivalent to 284 mg inclisiran) in 1.5 mL solution for subcutaneous injection
DRUG	Placebo	Sterile normal saline (0.9% sodium chloride in water for subcutaneous injection)

Timeline

• Start date: 2021-01-27
• Primary completion: 2023-11-09
• Completion: 2024-11-27
• First posted: 2020-12-03
• Last updated: 2026-01-13
• Results posted: 2025-06-10

Locations

59 sites across 26 countries: United States, Argentina, Brazil, Canada, Czechia, France, Germany, Greece, Hungary, Israel, Italy, Jordan, Lebanon, Malaysia, Netherlands, Norway, Poland, Russia, Slovakia, Slovenia, South Africa, Spain, Switzerland, Taiwan, Turkey (Türkiye), United Kingdom

Regulatory

• FDA-regulated drug study