NCT07473843

Study of Zodasiran in Adolescent Participants With Homozygous Familial Hypercholesterolemia

Phase 3 Single-Arm Open-Label Study to Evaluate the Efficacy and Safety of Zodasiran in Adolescent Participants (Age 12 to <18 Years) With Homozygous Familial Hypercholesterolemia (SPRUCE)

• Status: Not Yet Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 12 (estimated)
• Sponsor: Arrowhead Pharmaceuticals · Industry
• Sex: All
• Age: 12 Years – 17 Years
• Healthy volunteers: Not accepted

Summary

This study will evaluate the efficacy and safety of zodasiran subcutaneous (sc) injection in participants 12 to \<18 years of age with genetically or clinically diagnosed homozygous familial hypercholesterolemia (HoFH) and low-density lipoprotein cholesterol (LDL-C) ≥116 milligrams per deciliter (mg/dL) on maximally tolerated lipid-lowering therapy.

Conditions

• Homozygous Familial Hypercholesterolemia

Interventions

Type	Name	Description
DRUG	Zodasiran	By sc injection

Timeline

• Start date: 2026-03-01
• Primary completion: 2028-07-01
• Completion: 2029-05-01
• First posted: 2026-03-16
• Last updated: 2026-03-20

Regulatory

• FDA-regulated drug study