NCT06597006

Study to Evaluate Safety, Tolerability and Efficacy of Inclisiran in Children 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 Children (2 to Less Than 12 Years) With Homozygous Familial Hypercholesterolemia and Elevated LDL-cholesterol

• Status: Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 9 (estimated)
• Sponsor: Novartis Pharmaceuticals · Industry
• Sex: All
• Age: 2 Years – 11 Years
• Healthy volunteers: Not accepted

Summary

This is a pivotal phase III study designed to evaluate safety, tolerability, and efficacy of inclisiran in children (aged 2 to \<12 years) with homozygous familial hypercholesterolemia (HoFH) and elevated low density lipoprotein cholesterol (LDLC).

Detailed description

This is 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 children (aged 2 to \<12 years) with homozygous familial hypercholesterolemia (HoFH) and elevated low density lipoprotein cholesterol (LDL-C) on stable standard of care background lipid-lowering therapy.

Conditions

• Familial Hypercholesterolemia - Homozygous

Interventions

Type	Name	Description
DRUG	Inclisiran	Inclisiran (inclisiran sodium 300 mg subcutaneous (s.c.) for participants with body weight ≥23 kg, inclisiran sodium 180 mg s.c. for participants with body weight \<23 kg to ≥16 kg, or inclisiran sodium 100 mg s.c. for participants with body weight \<16 kg. The dose level is based on the participant's body weight on Day 1 (for Part 1) and Day 360 (for Part 2), respectively.
DRUG	Placebo	Sterile normal saline (0.9% sodium chloride in water for subcutaneous injection)

Timeline

• Start date: 2025-02-28
• Primary completion: 2028-03-23
• Completion: 2029-04-15
• First posted: 2024-09-19
• Last updated: 2026-02-20

Locations

18 sites across 11 countries: United States, Austria, China, Germany, Greece, Malaysia, Netherlands, South Africa, Taiwan, Turkey (Türkiye), United Kingdom

Regulatory

• FDA-regulated drug study