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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT04281485 | Study to Evaluate the Safety and Efficacy of PF-06939926 for the Treatment of Duchenne Muscular Dystrophy | PHASE3 | ACTIVE NOT_RECRUITING | 114 | — | — | Nov 5, 2020 | Apr 15, 2039 | Mar 2, 2026 | 53 | United States, Australia +13 |
The NSAA was a 17-item test that graded performance of various functional skills using the following scale: 0 (unable to achieve independently), 1 (modified method but achieves goal independent of physical assistance from another), and 2 ("normal"- no obvious modification of activity). Total score was calculated as the sum of all 17 individual item responses and ranged from 0 (worst) to 34 (fully independent function) with higher scores indicating better function. Baseline NSAA total score is defined as the last non-missing NSAA total score collected prior to Year 1 drug administration.
| Arm | Type | Description |
|---|---|---|
| Cohort 1 | OTHER | Approximately two thirds of participants will be randomized to Cohort 1. |
| Cohort 2 | OTHER | Approximately one third of participants will be randomized to Cohort 2. |
| Name | Type | Description |
|---|---|---|
| PF-06939926 | GENETIC | PF-06939926 will be administered as a single IV infusion at Year 1 for Cohort 1. |
| Placebo | OTHER | Placebo will be administered as a single IV infusion at Year 1 for Cohort 2. |
Key inclusion criteria: 1. Confirmed diagnosis of Duchenne muscular dystrophy by prior genetic testing 2. Receiving a stable daily dose (at least 0.5 mg/kg/day prednisone or prednisolone, or at least 0.75 mg/kg/day deflazacort) for at least 3 months prior to Screening 3. Ambulatory, as assessed by ...