A Phase 3 Study of Zodasiran in Adolescent and Adult Subjects With Homozygous Familial Hypercholesterolemia (YOSEMITE)
NCT ID: NCT07037771
Last Updated: 2025-12-22
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.
RECRUITING
PHASE3
60 participants
INTERVENTIONAL
2025-06-17
2027-08-20
Brief Summary
Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.
Detailed Description
Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.
Conditions
See the medical conditions and disease areas that this research is targeting or investigating.
Study Design
Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.
RANDOMIZED
PARALLEL
TREATMENT
QUADRUPLE
Study Groups
Review each arm or cohort in the study, along with the interventions and objectives associated with them.
zodasiran
5 doses of zodasiran by subcutaneous (sc) injection (randomized period). 4 or 5 doses of zodasiran by sc injection (optional open-label period).
zodasiran Injection
ARO-ANG3 Injection
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)
zodasiran Injection
ARO-ANG3 Injection
Placebo
sterile normal saline (0.9% NaCl)
Interventions
Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.
zodasiran Injection
ARO-ANG3 Injection
Placebo
sterile normal saline (0.9% NaCl)
Other Intervention Names
Discover alternative or legacy names that may be used to describe the listed interventions across different sources.
Eligibility Criteria
Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.
Inclusion Criteria
* 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)
* 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
Exclusion Criteria
* Use of an antisense oligonucleotide molecule within 3 months before Day 1
* Use of evinacumab within 3 months before Day 1
* 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
12 Years
ALL
No
Sponsors
Meet the organizations funding or collaborating on the study and learn about their roles.
Arrowhead Pharmaceuticals
INDUSTRY
Responsible Party
Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.
Locations
Explore where the study is taking place and check the recruitment status at each participating site.
Research Site 7
Park Ridge, Illinois, United States
Research Site 2
New York, New York, United States
Research Site 1
Cincinnati, Ohio, United States
Research Site 3
Nedlands, Western Australia, Australia
Research Site 5
Vancouver, British Columbia, Canada
Research Site 6
Chicoutimi, Quebec, Canada
Research Site 4
Québec, Quebec, Canada
Research Site 8
Cape Town, , South Africa
Countries
Review the countries where the study has at least one active or historical site.
Central Contacts
Reach out to these primary contacts for questions about participation or study logistics.
Other Identifiers
Review additional registry numbers or institutional identifiers associated with this trial.
AROANG3-3001
Identifier Type: -
Identifier Source: org_study_id