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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT00264888 | Safety and Efficacy Study of PTC124 in Duchenne Muscular Dystrophy | PHASE2 | COMPLETED | 38 | — | — | Dec 1, 2005 | May 1, 2007 | Jan 14, 2009 | 3 | United States |
| Name | Type | Description |
|---|---|---|
| PTC124 | DRUG | - |
Inclusion Criteria: * Diagnosis of DMD based on a clinical phenotype presenting by age 5, with increased serum CK and decrease of dystrophin on a muscle biopsy * Presence of a nonsense mutation in the dystrophin gene * Physical examination or radiographic imaging documenting the presence of EDB or ...