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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT06547216 | Phase 2 Open-label Extension Study of AOC 1020 in Participants With Facioscapulohumeral Muscular Dystrophy (FSHD) | PHASE2 | ACTIVE NOT_RECRUITING | 84 | — | — | Jul 25, 2024 | Jun 1, 2030 | May 14, 2026 | 17 | United States, Canada +1 |
| NCT05747924 | Phase 1/2 Study of AOC 1020 in Participants With Facioscapulohumeral Muscular Dystrophy (FSHD) | PHASE1 | COMPLETED | 90 | — | — | Apr 4, 2023 | Mar 20, 2026 | Apr 24, 2026 | 17 | United States, Canada +1 |
Ratio to Baseline
| Arm | Type | Description |
|---|---|---|
| AOC 1020 Regimen | EXPERIMENTAL | AOC 1020 Dose Regimen; Thirty-two doses administered intravenously over 46 months. All participants will receive AOC 1020 at a dose level of 2mg/kg. |
| AOC 1020 Regimen 1 | EXPERIMENTAL | Cohort A: AOC 1020 Dose Regimen 1; Five doses administered intravenously over 9 months |
| AOC 1020 Regimen 2 | EXPERIMENTAL | Cohort B1: AOC 1020 Dose Regimen 2; Five doses administered intravenously over 9 months |
| AOC 1020 Regimen 3 | EXPERIMENTAL | Cohort C: AOC 1020 Dose Regimen 3; Eight doses administered intravenously over approximately 10 months |
| Placebo (Saline) Regimen 1 | PLACEBO_COMPARATOR | Cohort A \& B: Placebo; Five doses administered intravenously over 9 months |
| Placebo (Saline) Regimen 2 | PLACEBO_COMPARATOR | Cohort C: Placebo; Eight doses administered intravenously over approximately 10 months |
| Name | Type | Description |
|---|---|---|
| AOC 1020 | DRUG | AOC 1020 will be administered via intravenous (IV) infusion |
| Placebo | DRUG | Placebo will be administered via intravenous (IV) infusion |
Inclusion Criteria: 1. Ability to provide written informed consent (signed and dated) and any authorizations required by local law and be willing and able to comply with all study requirements. When enrolling participants who are minors, it is necessary to also obtain consent from a legally designa...