NCT06597019

Study to Evaluate Efficacy and Safety of Inclisiran in Children 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 Children (6 to Less Than 12 Years) With Heterozygous Familial Hypercholesterolemia and Elevated LDL- Cholesterol

• Status: Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 51 (estimated)
• Sponsor: Novartis Pharmaceuticals · Industry
• Sex: All
• Age: 6 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 6 to \<12 years) with heterozygous familial hypercholesterolemia (HeFH) 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 6 to \<12 years) with heterozygous familial hypercholesterolemia (HeFH) and elevated low density lipoprotein cholesterol (LDL-C) on stable standard of care background lipid-lowering therapy.

Conditions

• Familial Hypercholesterolemia - Heterozygous

Interventions

Type	Name	Description
DRUG	Inclisiran	Inclisiran (inclisiran sodium 300 mg subcutaneous (s.c.) for participants with body weight ≥23 kg and inclisiran sodium 180 mg s.c. for participants with body weight \<23 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: 2024-12-09
• Primary completion: 2028-03-21
• Completion: 2029-04-15
• First posted: 2024-09-19
• Last updated: 2025-11-21

Locations

66 sites across 23 countries: United States, Argentina, Austria, Belgium, Brazil, China, Czechia, France, Germany, Greece, Hong Kong, Hungary, Israel, Italy, Malaysia, Netherlands, Poland, Portugal, South Africa, Spain, Taiwan, Turkey (Türkiye), United Kingdom

Regulatory

• FDA-regulated drug study