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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT03508947 | Safety and Tolerability of WVE-210201 in Patients With Duchenne Muscular Dystrophy | PHASE1 | COMPLETED | 36 | — | — | Jan 24, 2018 | Mar 6, 2019 | Apr 8, 2019 | 13 | United States, Belgium +5 |
| Arm | Type | Description |
|---|---|---|
| WVE-210201 (Dose A) or placebo | EXPERIMENTAL | - |
| WVE-210201 (Dose B) or placebo | EXPERIMENTAL | - |
| WVE-210201 (Dose C) or placebo | EXPERIMENTAL | - |
| WVE-210201 (Dose D) or placebo | EXPERIMENTAL | - |
| WVE-210201 (Dose E) or placebo | EXPERIMENTAL | - |
| Name | Type | Description |
|---|---|---|
| WVE-210201 | DRUG | WVE-210201 is a stereopure antisense oligonucleotide (ASO) |
| Placebo | DRUG | Sodium Chloride |
Inclusion Criteria: * Diagnosis of Duchenne muscular dystrophy (DMD) based on clinical phenotype with increased serum creatine kinase * Documented mutation in the Dystrophin gene associated with DMD that is amenable to exon 51 skipping * Ambulatory or non-ambulatory male patients aged ≥5 - ≤18 year...