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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT07608432 | Efficacy, Safety, and Tolerability of Zeleciment Rostudirsen (DYNE-251) Administered Intravenously Every 4 Weeks in Ambulatory Participants With Duchenne Muscular Dystrophy (FORZETTO) | PHASE3 | RECRUITING | 90 | — | — | Jun 1, 2026 | Oct 1, 2032 | May 27, 2026 | 1 | United States |
| Arm | Type | Description |
|---|---|---|
| Placebo-Controlled Period: Zeleciment Rostudirsen (DYNE-251) | EXPERIMENTAL | Participants will be randomized to receive zeleciment rostudirsen, once every 4 weeks (Q4W) for up to 72 weeks. |
| Placebo-Controlled Period: Placebo | PLACEBO_COMPARATOR | Participants will be randomized to receive placebo, Q4W for up to 72 weeks. |
| Open-Label Long-Term Extension Period: Zeleciment Rostudirsen (DYNE-251) | EXPERIMENTAL | All participants who complete the Placebo-Controlled Period of the study will receive zeleciment rostudirsen administered Q4W for up to 96 weeks. |
| Name | Type | Description |
|---|---|---|
| Zeleciment Rostudirsen (DYNE-251) | DRUG | Administered by IV infusion |
| Placebo | DRUG | Administered by IV infusion |
Inclusion Criteria: * Ambulatory male with confirmed diagnosis of DMD and with a mutation in the dystrophin gene characterized by exon deletion amenable to exon 51 skipping . * Rise From Floor (RFF) time must be \< 10 seconds for both screening assessments . * Receiving a stable daily or weekend do...