NCT07037771

A Phase 3 Study of Zodasiran in Adolescent and Adult Subjects With Homozygous Familial Hypercholesterolemia (YOSEMITE)

Phase 3 Study to Evaluate the Efficacy and Safety of Zodasiran in Adolescent and Adult Subjects With Homozygous Familial Hypercholesterolemia (YOSEMITE)

• Status: Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 60 (estimated)
• Sponsor: Arrowhead Pharmaceuticals · Industry
• Sex: All
• Age: 12 Years
• Healthy volunteers: Not accepted

Summary

This multicenter, randomized, placebo-controlled study will evaluate the efficacy and safety of zodasiran subcutaneous (SC) injection in subjects 12 years of age and older with genetically or clinically diagnosed Homozygous familial hypercholesterolemia (HoFH). After completion of the double blind (DB) treatment period subjects will be eligible to continue in the optional open-label extension (OLE) period of the study. All placebo subjects who opt to continue will transition to active drug during the OLE Period.

Conditions

• Homozygous Familial Hypercholesterolemia

Interventions

Type	Name	Description
DRUG	zodasiran Injection	ARO-ANG3 Injection
DRUG	Placebo	sterile normal saline (0.9% NaCl)

Timeline

• Start date: 2025-06-17
• Primary completion: 2027-08-20
• Completion: 2027-08-20
• First posted: 2025-06-25
• Last updated: 2026-03-27

Locations

22 sites across 8 countries: United States, Australia, Canada, Georgia, Israel, Japan, New Zealand, South Africa

Regulatory

• FDA-regulated drug study