Clinical Trial

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

Active, Not Recruiting Phase 3
View on ClinicalTrials.gov →
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.
Protocol Amendment History 5 changes
critical Enrollment closed, study ongoing 2026-08-13
notable Enrollment increased: 60 -> 70 participants 2026-08-13
notable Trial sites expanded: 35 -> 42 locations 2026-05-29
notable Trial sites expanded: 32 -> 35 locations 2026-05-07
notable Trial sites expanded: 22 -> 32 locations 2026-05-05
Trial Details
NCT Number NCT07037771
Lead Sponsor Arrowhead Pharmaceuticals
Conditions Homozygous Familial Hypercholesterolemia
Enrollment 70 participants
Start Date 2025-06-17
Primary Completion 2027-08-20 (estimated)
Study Completion 2027-08-20 (estimated)
Updated on ClinicalTrials.gov 2026-08-12