NCT04659863

Study to Evaluate Efficacy and Safety of Inclisiran in Adolescents With Homozygous 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 Homozygous Familial Hypercholesterolemia and Elevated LDL-cholesterol (ORION-13)

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 13 (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 homozygous familial hypercholesterolemia (HoFH) and elevated low density lipoprotein cholesterol (LDL-C).

Detailed description

This was a two-part (double-blind, inclisiran versus placebo \[Year 1\] followed by open-label inclisiran \[Year 2\]) multicenter study in adolescents (aged 12 to \< 18 years) with HoFH and elevated LDL-C (\> 130 mg/dL; 3.4 mmol/L) on stable, individualized, optimal standard of care (SoC) background lipid-lowering therapy (including maximally tolerated statin treatment, at the Investigator's discretion) to evaluate the safety, tolerability, and efficacy of inclisiran in this pediatric patient population. Following an approximately 4-week screening/run-in period, the study had 2 sequential parts as follows: Part 1/Year 1: 12 months double-blind, parallel group period, in which participants were randomized in a 2:1 ratio to receive either inclisiran sodium 300 mg subcutaneous (s.c.) or placebo. The primary endpoint was assessed at Day 330. Part 2/Year 2: 12 months single arm, open-label follow-up period, with all participants receiving inclisiran sodium 300 mg s.c.

Conditions

• Familial Hypercholesterolemia - Homozygous

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 injection) for subcutaneous injection

Timeline

• Start date: 2021-02-16
• Primary completion: 2023-10-30
• Completion: 2024-11-18
• First posted: 2020-12-09
• Last updated: 2026-01-13
• Results posted: 2025-05-31

Locations

11 sites across 8 countries: United States, Canada, France, Greece, Lebanon, Malaysia, Netherlands, Turkey (Türkiye)

Regulatory

• FDA-regulated drug study