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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT06100887 | Phase 2 Study of EDG-5506 in Children and Adolescents With Duchenne Muscular Dystrophy Previously Treated With Gene Therapy | PHASE2 | ACTIVE NOT_RECRUITING | 43 | — | — | Mar 22, 2024 | Mar 1, 2027 | Nov 6, 2025 | 7 | United States |
| NCT05540860 | A Study of EDG-5506 in Children With Duchenne Muscular Dystrophy (LYNX) | PHASE2 | ACTIVE NOT_RECRUITING | 76 | — | — | Oct 24, 2022 | Jan 1, 2027 | Nov 12, 2025 | 14 | United States |
All participants
All participants
| Arm | Type | Description |
|---|---|---|
| Cohort 1 | EXPERIMENTAL | Drug: Sevasemten Drug: Placebo |
| Cohort 2 | EXPERIMENTAL | Drug: Sevasemten Drug: Placebo |
| Cohort 3 | EXPERIMENTAL | Drug: Sevasemten Drug: Placebo |
| Cohort 4 | EXPERIMENTAL | Drug: Sevasemten Drug: Placebo |
| Cohort 5 | EXPERIMENTAL | Drug: Sevasemten Drug: Placebo |
| Cohort 2NS | EXPERIMENTAL | Drug: Sevasemten Drug: Placebo |
| Name | Type | Description |
|---|---|---|
| Sevasemten Dose 1 | DRUG | Sevasemten is administered orally once per day |
| Sevasemten Dose 2 | DRUG | Sevasemten is administered orally once per day |
| Sevasemten Dose 3 | DRUG | Sevasemten is administered orally once per day |
| Placebo | DRUG | Placebo is administered orally once per day |
| Sevasemten Dose 4 | DRUG | Sevasemten is administered orally once per day |
| Sevasemten Dose 5 | DRUG | Sevasemten is administered orally once per day |
Key Inclusion Criteria: * Aged 6 to 17 with a documented mutation on the DMD gene and phenotype consistent with DMD. * Prior receipt of an AAV-based gene therapy (≥ 2 years after documented receipt of gene therapy administration or ≥ 3 years after randomization in a randomized study). * Able to com...