Table 3.
Timed function test scores (corrected ITT population).
| Mean time and change from baseline to week 48 (Δ) in seconds | ||||||
|---|---|---|---|---|---|---|
| Placebo | 40 mg/kg/day ataluren | 80 mg/kg/day ataluren | ||||
| Endpoint* | Baseline | Week 48 | Baseline | Week 48 | Baseline | Week 48 |
| Climb 4 stairs, s | 6.0 | 10.8 | 6.9 | 9.3 | 7.7 | 11.2 |
| Δ=4.8 | Δ=2.4 | Δ=3.5 | ||||
| Descend 4 stairs | 5.5 | 9.6 | 6.1 | 8.5 | 6.8 | 9.7 |
| Δ=4.0 | Δ=2.4 | Δ=3.0 | ||||
| Run/walk 10 m | 6.8 | 9.8 | 7.4 | 9.1 | 7.8 | 10.2 |
| Δ=3.0 | Δ=1.7 | Δ=2.4 | ||||
| Supine to stand | 11.4 | 14.6 | 10.8 | 14.0 | 12.4 | 15.4 |
| Δ=3.2 | Δ=3.2 | Δ=3.0 | ||||
| Comparison of change from baseline to week 48 between ataluren and placebo, mean (95% CI) | ||
|---|---|---|
| 40 mg/kg/day ataluren vs Placebo | 80 mg/kg/day ataluren vs Placebo | |
| Climb 4 stairs | −2.4 (−4.8, 0.0) | −1.3 (−4.0, 1.4) |
| Descend 4 stairs | −1.6 (−4.2, 1.0) | −1.1 (−3.9, 1.7) |
| Run/walk 10 m | −1.4 (−3.7, 0.9) | −0.7 (−3.0, 1.7) |
| Supine to stand | −0.0 (−2.5, 2.4) | −0.2 (−2.6, 2.2) |
*
For timed function tests, negative differences between ataluren and placebo represent better outcomes in ataluren-treated patients.
CI, confidence interval; Corrected ITT, corrected intent-to-treat population.