Actively Recruiting
A Phase 3 Study of Zodasiran in Adolescent and Adult Subjects With Homozygous Familial Hypercholesterolemia (YOSEMITE)
Led by Arrowhead Pharmaceuticals · Updated on 2026-05-06
60
Participants Needed
35
Research Sites
113 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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
Official Title
A Phase 3 Study of Zodasiran in Adolescent and Adult Subjects With Homozygous Familial Hypercholesterolemia (YOSEMITE)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Age 12 years or older, not pregnant or breastfeeding, and not planning pregnancy during the study
- Body weight of at least 35 kg at screening
- Diagnosis of Homozygous Familial Hypercholesterolemia (HoFH) confirmed by genetic test or clinical diagnosis with specific cholesterol criteria
- LDL cholesterol level of at least 70 mg/dL for adults, or at least 116 mg/dL for adolescents aged 12 to under 18 years
- Hemoglobin A1c level of 9.5% or less
- Total bilirubin less than twice the upper limit of normal unless Gilbert's syndrome is confirmed
- Alanine aminotransferase and aspartate aminotransferase levels less than three times the upper limit of normal
- Currently receiving maximally tolerated standard lipid-lowering therapy including statins, ezetimibe, and a PCSK9 inhibitor
You will not qualify if you...
- Use of hepatocyte-targeted siRNA treatments within 365 days before Day 1, except for inclisiran with at least 4 weeks between treatments
- Use of antisense oligonucleotide molecules within 3 months before Day 1, except inclisiran with at least 4 weeks separation
- Use of evinacumab within 3 months before Day 1; evinacumab is not allowed during the study
- Failure to respond to evinacumab treatment, defined as less than 15% reduction in LDL cholesterol after 2 doses
- Use of any other investigational drugs or devices within 30 days or 5 half-lives before Day 1
- Use of systemic corticosteroids unless as stable replacement therapy for pituitary or adrenal disease
- Estimated glomerular filtration rate below 30 mL/min
- Additional criteria may apply according to protocol
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 35 locations
1
Research Site 7
Park Ridge, Illinois, United States, 60068
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2
Research Site 2
New York, New York, United States, 10029
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3
Research Site 1
Cincinnati, Ohio, United States, 45227
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4
Research Site 14
Pittsburgh, Pennsylvania, United States, 15213
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5
Research Site 13
Camperdown, New South Wales, Australia, 2050
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6
Research Site 9
Saint Leonards, New South Wales, Australia, 2065
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7
Research Site 21
Heidelberg, Victoria, Australia, 3084
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8
Research Site 3
Nedlands, Western Australia, Australia, 6009
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9
Research Site 33
La Louvière, Belgium, 7100
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10
Research Site 34
Leuven, Belgium, 3000
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11
Research Site 24
Cerqueira César, São Paulo, Brazil, CEP-05403-000
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12
Research Site 5
Vancouver, British Columbia, Canada, V6Z 2H2
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13
Research Site 6
Chicoutimi, Quebec, Canada, G7H 7K9
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14
Research Site 4
Québec, Canada, G1V 4W2
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15
Research Site 30
Hradec Králové, Czechia, General E3 500 0
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16
Research Site 23
Paris, France, 75013
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17
Research Site 17
Tbilisi, Georgia, 0159
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18
Research Site 18
Tbilisi, Georgia, 160
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19
Research Site 19
Tbilisi, Georgia, 186
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20
Research Site 35
Berlin, Germany, 13353
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21
Research Site 10
Jerusalem, Israel, 9112001
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22
Research Site 15
Tel Litwinsky, Israel, 5265601
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23
Research Site 26
Kanazawa, Ishikawa-ken, Japan, 920-8641
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24
Research Site 27
Suita, Osaka, Japan, 564-8565
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25
Research Site 20
Fukushima, Japan, 960-1295
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26
Research Site 16
Okayama, Japan, 700-8558
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27
Research Site 22
Saitama, Japan, 350-0495
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28
Research Site 25
Tokyo, Japan, 113-8510
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29
Research Site 12
Christchurch, New Zealand, 8011
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30
Research Site 31
Riyadh, Saudi Arabia, 12713
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31
Research Site 11
Parktown, Johannesburg, South Africa, 2193
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32
Research Site 8
Cape Town, South Africa, 7925
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33
Research Site 28
Córdoba, Spain, 14004
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34
Research Site 29
Madrid, Spain, 28041
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35
Research Site 32
Gothenburg, Sweden, 413 46
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Research Team
M
Medical Monitor
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
QUADRUPLE
Allocation
RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
2
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