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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT07587242 | A Phase 3 Study to Evaluate the Safety and Efficacy of AOC 1044 (Also Referred to as Delpacibart Zotadirsen) in Participants With DMD With Gene Mutations Amenable to Exon 44 Skipping | PHASE3 | NOT YET_RECRUITING | 70 | — | — | Jun 1, 2026 | Jul 1, 2030 | May 14, 2026 | - | — |
| Arm | Type | Description |
|---|---|---|
| AOC 1044 | EXPERIMENTAL | Participants will receive AOC 1044 intravenously (IV) every 6 weeks (Q6W) during the 54-week double-blind treatment period, followed by a 54-week open-label treatment period in which the same participants will continue to receive AOC 1044 IV Q6W |
| Placebo | PLACEBO_COMPARATOR | Participants will receive placebo (saline) IV Q6W during the 54-week double-blind treatment period, followed by a 54-week open-label treatment period in which all participants will then receive AOC 1044 IV Q6W |
| Name | Type | Description |
|---|---|---|
| AOC 1044 | DRUG | AOC 1044 will be administered by intravenous (IV) infusion |
| Placebo | DRUG | Placebo will be administered by intravenous (IV) infusion |
Key Inclusion Criteria: * Ambulatory males with clinical and genetic diagnosis of DMD * Acceptable genetic test confirming dystrophin gene mutation amenable to exon 44 skipping * 7 to 16 years of age at time of consent * TTR and NSAA assessment completed within the protocol specified parameters at ...