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Phase 3 Study to Evaluate the Efficacy and Safety of Zodasiran in Adolescent and Adult Subjects with Homozygous Familial Hypercholesterolemia (YOSEMITE)

Status
Not yet recruiting
Phases
Phase 3
Study type
Interventional
Source
EU CTIS
Registry ID
CTIS2025-521792-31-01
Acronym
AROANG3-3001
Enrollment
13
Registered
2026-01-23
Start date
Unknown
Completion date
Unknown
Last updated
2026-01-26

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Homozygous Familial Hypercholesterolemia (HoFH)

Brief summary

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

Detailed description

1. Percent Change from Baseline to Month 12 in Fasting Apolipoprotein B (ApoB) (Randomized Period), 2. Percent Change from Baseline to Month 12 in Fasting Non-High Density Lipoprotein Cholesterol (non-HDL-C) (Randomized Period), 3. Change from Baseline to Month 12 in Fasting LDL-C (Randomized Period), 4. Area Under the Plasma Concentration Versus the Time Curve (AUC) from Baseline to Month 12 for Fasting LDL-C (Randomized Period), 5. Percent Change from Baseline to Month 12 in Fasting Triglycerides (TGs) (Randomized Period), 6. Percent Change from Baseline to Month 12 in Fasting Angiopoietin-like Protein 3 (ANGPTL3) (Randomized Period), 7. Percent Change from Baseline to Month 12 in Fasting Total Cholesterol (Randomized Period), 8. Percent Change from Baseline to Month 12 in Fasting High-Density Lipoprotein Cholesterol (HDL-C) (Randomized Period), 9. Proportion of Participants who Meet European Union (EU) LDL-C Apheresis Eligibility Criteria (per German Apheresis Working Group) at Month 12 (Randomized Period), 10. Proportion of Participants who Meet United States (US) Apheresis Eligibility Criteria (per National Lipid Association) at Month 12 (Randomized Period), 11. Proportion of Participants with Fasting LDL-C <100 mg/dL (2.6 mmol/L) at Month 12 (Randomized Period), 12. Change from Baseline in Fasting LDL-C Over Time (Randomized Period), 13. Percent Change from Baseline in fasting LDL-C Over Time (Randomized Period), 14. Percent Change from Baseline to Month 12 in Fasting Lipoprotein(a) [Lp(a)] (Randomized Period)

Interventions

DRUGPlacebo of Zodasiran

Sponsors

Arrowhead Pharmaceuticals Inc.
Lead SponsorINDUSTRY

Eligibility

Sex/Gender
All
Age
0 Years to No maximum

Design outcomes

Primary

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

Secondary

MeasureTime frame
1. Percent Change from Baseline to Month 12 in Fasting Apolipoprotein B (ApoB) (Randomized Period), 2. Percent Change from Baseline to Month 12 in Fasting Non-High Density Lipoprotein Cholesterol (non-HDL-C) (Randomized Period), 3. Change from Baseline to Month 12 in Fasting LDL-C (Randomized Period), 4. Area Under the Plasma Concentration Versus the Time Curve (AUC) from Baseline to Month 12 for Fasting LDL-C (Randomized Period), 5. Percent Change from Baseline to Month 12 in Fasting Triglycerides (TGs) (Randomized Period), 6. Percent Change from Baseline to Month 12 in Fasting Angiopoietin-like Protein 3 (ANGPTL3) (Randomized Period), 7. Percent Change from Baseline to Month 12 in Fasting Total Cholesterol (Randomized Period), 8. Percent Change from Baseline to Month 12 in Fasting High-Density Lipoprotein Cholesterol (HDL-C) (Randomized Period), 9. Proportion of Participants who Meet European Union (EU) LDL-C Apheresis Eligibility Criteria (per German Apheresis Working Group) at Mont

Countries

Austria, Belgium, Czechia, Germany, Greece, Italy, Sweden

Outcome results

None listed

Source: EU CTIS · Data processed: Feb 4, 2026