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Phase 3 Zodasiran for Homozygous Familial Hypercholesterolemia (YOSEMITE)

This study, called YOSEMITE, is testing a drug called zodasiran (ARO-ANG3 Injection) for people aged 12 and older with Homozygous Familial Hypercholesterolemia (HoFH). HoFH is a genetic condition that causes very high cholesterol. You might qualify if you have a genetic test or a clinical diagnosis of HoFH, with very high cholesterol levels. Participants will receive either zodasiran or a placebo (a substance with no active drug, like sterile saline). The main goal is to see how much zodasiran changes your "bad" cholesterol (LDL-C) levels after 12 months. After this initial period, you may be able to continue receiving zodasiran in an optional extension part of the study. The study aims to enroll 60 participants, but its current recruitment status is unclear.

Study design
This is a randomized, placebo-controlled study, meaning participants are randomly assigned to receive either zodasiran or a placebo. It is a double-blind study, so neither you nor your doctor will know which treatment you are receiving initially. It plans to enroll 60 participants.
What's involved
Not specified in the trial record.
Compensation
Not stated in the trial record.
Follow-up
The primary endpoint measures changes in cholesterol levels from the beginning of the study to Month 12.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

NCT07037771

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

Active, Not Recruiting
PHASE3Ages 12+InterventionalTreatment
Arrowhead Pharmaceuticals
~70 participants
Updated 2026-08-12 on ClinicalTrials.gov
What's tested:zodasiran InjectionPlacebo

At a glance

Recruiting sites
0 of 43 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Percent Change from Baseline to Month 12 in Fasting Low Density Lipoprotein Cholesterol (LDL-C) (Randomized period)
Measured over Baseline, Month 12

Conditions

Homozygous Familial Hypercholesterolemia

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Familial hypercholesterolemia - homozygous

NCT07037771

Where you'd take part

This study runs at 43 sites. They're the same protocol — you choose where, and that choice sets who your contact draft is addressed to.

  • Research Site 1

    Cincinnati, Ohiono site contact published

  • Research Site 10

    Jerusalem, Israelno site contact published

  • Research Site 11

    Parktown, Johannesburg, South Africano site contact published

  • Research Site 12

    Christchurch, New Zealandno site contact published

  • Research Site 13

    Camperdown, New South Wales, Australiano site contact published

  • Research Site 14

    Pittsburgh, Pennsylvaniano site contact published

  • Research Site 15

    Tel Litwinsky, Israelno site contact published

  • Research Site 16

    Okayama, Japanno site contact published

Sites open and close at different times, so the status above is per site — it can differ from the study's overall status.

Who to contact

This trial hasn't published a contact. View it on ClinicalTrials.gov

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Eligibility criteria

Inclusion

Age ≥12 years, non pregnant, non lactating, do not plan to become pregnant during the study
Body weight ≥35 kg at Screening as patients could theoretically be \<35 kg as the study continues.
HoFH based on a supportive genetic test or a clinical diagnosis (total cholesterol \>500 mg/dL\[13 mmol/L\] OR treated LDL-C concentration of ≥300 mg/dL \[≥8 mmol/L\] either accompanied by TGs \<300 mg/dL \[3.4 mmol/L\] AND both parents with documented total cholesterol \>250 mg/dL \[6.5 mmol/L\] OR cutaneous or tendinous xanthoma before 10 years of age)
LDL-C ≥70 mg/dL (1.8 mmol/L). For adolescents 12 to \<18 years of age, screening LDL-C ≥116 mg/dL (3 mmol/L).
Hemoglobin A1c (HbA1c) ≤9.5%
Total bilirubin \<2xULN, unless in previously confirmed cases of Gilbert's syndrome
Alanine aminotransferase or aspartate aminotransferase \<3×ULN
On standard of care, maximally tolerated lipid-lowering therapy to include a maximally tolerated statin, ezetimibe, and a PCSK9 inhibitor

Exclusion

Use of a hepatocyte-targeted siRNA within 365 days before Day 1 (except inclisiran, which is permitted; administration of inclisiran and study drug must be separated by at least 4 weeks)
Use of an antisense oligonucleotide molecule within 3 months before Day 1 (except inclisiran, which is permitted; administration of inclisiran and study drug must be separated by at least 4 weeks)
Use of evinacumab within 3 months before Day 1. Evinacumab use is prohibited during the study.
Non-response to evinacumab, defined as LDL-C reduction \<15% from baseline after 2 doses
Use of any other investigational agent or device within 30 days or 5 half-lives (whichever is longer) before Day 1
Use of systemic corticosteroids (unless used as replacement therapy for pituitary/adrenal disease with a stable regimen)
Estimated glomerular filtration rate \<30 mL/min

What this trial measures

  • Percent Change from Baseline to Month 12 in Fasting Low Density Lipoprotein Cholesterol (LDL-C) (Randomized period)Baseline, Month 12