TABLE 2.
Baseline NSAA and timed function test scores by pre-specified age subgroup A) Analysis of the 4- to 5-year-old subgroup.
| Motor function assessment | Age 4–5 years | ||
|---|---|---|---|
| Delandistrogene moxeparvovec (n = 8) | Placebo (n = 8) | p-value (vs placebo) | |
| NSAA total score | |||
| Mean (SD) | 20.10 (1.9) | 20.40 (2.7) | 0.8318 |
| Median | 20.50 | 20.50 | |
| 100MWR (seconds) | |||
| Mean (SD) | 58.76 (7.1) | 59.79 (8.2) | 0.7925 |
| Median | 57.90 | 59.70 | |
| 4-stair Climb (seconds) | |||
| Mean (SD) | 3.46 (0.9) | 3.48 (1.3) | 0.9822 |
| Median | 3.50 | 3.00 | |
| Time to Rise (seconds) | |||
| Mean (SD) | 3.89 (0.7) | 3.76 (0.8) | 0.7421 |
| Median | 3.70 | 3.75 | |
| 10MWR (seconds) | |||
| Mean (SD) | 5.01 (0.6) | 5.24 (1.0) | 0.5832 |
| Median | 5.05 | 5.10 | |
| B) Analysis of the 6- to 7-year-old subgroup | |||
| Motor function assessment | Age 6–7 years | ||
| Delandistrogene moxeparvovec (n = 12) | Placebo (n = 13) | p-value (vs placebo) | |
| NSAA total score | |||
| Mean (SD) | 19.60 (4.1) | 24.00 (2.9) | 0.0046 |
| Median | 20.00 | 24.00 | |
| 100MWR (seconds) | |||
| Mean (SD) | 62.56 (15.5) | 50.21 (6.2) | 0.0219 |
| Median | 57.10 | 50.40 | |
| 4-stair Climb (seconds) | |||
| Mean (SD) | 3.83 (1.8) | 2.86 (0.71) | 0.0958 |
| Median | 3.20 | 3.00 | |
| Time to Rise (seconds) | |||
| Mean (SD) | 5.91 (2.5) | 3.44 (0.6) | 0.0053 |
| Median | 5.05 | 3.40 | |
| 10MWR (seconds) | |||
| Mean (SD) | 5.58 (1.4) | 4.58 (0.4) | 0.0313 |
| Median | 5.00 | 4.60 | |
10MWR, 10-m Walk/Run; 100MWR, 100-m Walk/Run; NSAA, North Star Ambulatory Assessment; SD, standard deviation.