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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT07573631 | An Open-Label Extension Study to Evaluate the Long-term Safety and Efficacy of BMN 351 in Participants With Duchenne Muscular Dystrophy | PHASE2 | ENROLLING BY_INVITATION | 18 | — | — | Apr 1, 2026 | Dec 1, 2031 | May 14, 2026 | 7 | Italy, Netherlands +3 |
The safety and tolerability of BMN 351 will be assessed based on the incidence of adverse and serious adverse events.
| Arm | Type | Description |
|---|---|---|
| BMN 351 | EXPERIMENTAL | Participants from 351-201 enrolling in 351-202 will initially receive BMN 351 at the dose level at which they completed 351-201. Once all 351-201 participants have completed their Week 25 visit in 351-202, available safety and PD data will be analyzed and a single optimal dose level will be selected for 351-202. All participants from 351-201 will transition to that dose level at their next applicable visit. |
| Name | Type | Description |
|---|---|---|
| BMN 351 | DRUG | Anti-sense Oligonucleotide BMN 351 will be administered intravenously |
Inclusion Criteria: * Participants must have completed 351-201 without permanent discontinuation of the investigational medicinal product (IMP) or withdrawal from the study * Currently receiving treatment with oral corticosteroids, on a stable dose regimen during 351-201, and must remain on a consi...