Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT07473843 | Study of Zodasiran in Adolescent Participants With Homozygous Familial Hypercholesterolemia | PHASE3 | NOT YET_RECRUITING | 12 | — | — | Aug 1, 2026 | Aug 1, 2028 | May 29, 2026 | - | — |
| NCT07037771 | A Phase 3 Study of Zodasiran in Adolescent and Adult Subjects With Homozygous Familial Hypercholesterolemia (YOSEMITE) | PHASE3 | RECRUITING | 60 | — | — | Jun 17, 2025 | Aug 20, 2027 | May 28, 2026 | 42 | United States, Australia +16 |
| Arm | Type | Description |
|---|---|---|
| Zodasiran | EXPERIMENTAL | - |
| Placebo | PLACEBO_COMPARATOR | Calculated volume to match active treatment by sc injection (randomized period). 4 or 5 doses of zodasiran by sc injection (optional open-label period) |
| Name | Type | Description |
|---|---|---|
| Zodasiran | DRUG | By sc injection |
| zodasiran Injection | DRUG | ARO-ANG3 Injection |
| Placebo | DRUG | sterile normal saline (0.9% NaCl) |
Inclusion Criteria: * Adolescents 12 to \<18 years of age who are nonpregnant, nonlactating, and do not plan to become pregnant during the study * Body weight ≥35 kilograms (kg) at screening * HoFH based on a supportive genetic test (from a source-verifiable medical record or based on screening gen...