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

Part of paid clinical trials in Park Ridge, Illinois.

Sponsor
Arrowhead Pharmaceuticals
Study ID
NCT07037771
Phase
PHASE3
Status
Recruiting
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Conditions

  • Homozygous Familial Hypercholesterolemia

Eligibility Criteria

Sex
ALL
Age
12 Years - N/A
Healthy Volunteers
Not accepted

Interventions

  • zodasiran Injection — DRUG
    ARO-ANG3 Injection
  • Placebo — DRUG
    sterile normal saline (0.9% NaCl)

Study Details

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.

Key Dates

Start date
Jun 17, 2025
Status verified
Jun 2026
Primary completion
Aug 20, 2027
Completion
Aug 20, 2027

Study Design

Enrollment
60 participants (estimated)
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT

Arms

  • Experimental: zodasiran
    5 doses of zodasiran by subcutaneous (sc) injection (randomized period). 4 or 5 doses of zodasiran by sc injection (optional open-label period).
  • Placebo Comparator: Placebo
    Calculated volume to match active treatment by sc injection (randomized period). 4 or 5 doses of zodasiran by sc injection (optional open-label period)

Primary Outcome Measure

Percent Change from Baseline to Month 12 in Fasting Low Density Lipoprotein Cholesterol (LDL-C) (Randomized period) [ Time Frame: Baseline, Month 12 ]

Central Contacts

Locations (4)

FacilityCityStateZIPSite coordinators
Research Site 7Park RidgeIllinois60068-
Research Site 2New YorkNew York10029-
Research Site 1CincinnatiOhio45227-
Research Site 14PittsburghPennsylvania15213-

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