Table 1.
Patient characteristics.
| Characteristic | Treatment arm | ||
|---|---|---|---|
| Placebo N=57 | 40 mg/kg/day N=57 | 80 mg/kg/day N=60 | |
| Demographics | |||
| Age, years | |||
| Mean (SD) | 8.3 (2.33) | 8.8 (2.91) | 8.4 (2.53) |
| Median | 8.0 | 8.0 | 8.0 |
| Range | 5–15 | 5–20 | 5–16 |
| Race, n (%) | |||
| Caucasian | 54 (94.7) | 53 (93.0) | 50 (83.3) |
| Black | 0 (0.0) | 1 (1.8) | 1 (1.7) |
| Asian | 1 (1.8) | 1 (1.8) | 4 (6.7) |
| Hispanic | 1 (1.8) | 1 (1.8) | 2 (3.3) |
| Other | 1 (1.8) | 1 (1.8) | 3 (5.0) |
| Body height, cm | |||
| Mean (SD) | 123 (11.8) | 125 (15.3) | 126 (13.8) |
| Median | 122 | 121 | 126 |
| Range | 104–163 | 99–173 | 99–173 |
| Body weight, kg | |||
| Mean (SD) | 29 (9.1) | 31 (12.1) | 32 (12.8) |
| Median | 26 | 27 | 28 |
| Range | 16–55 | 16–76 | 17–84 |
| Stop codon type, n (%) | |||
| UGA | 31 (54.4) | 29 (50.9) | 23 (38.3) |
| UAG | 12 (21.1) | 17 (29.8) | 19 (31.7) |
| UAA | 14 (24.6) | 11 (19.3) | 18 (30.0) |
| Stratification factors | |||
| Age group, n (%) | |||
| <9 years | 32 (56) | 32 (56) | 34 (57) |
| ≥9 years | 25 (44) | 25 (44) | 26 (43) |
| Glucocorticoid use, n (%) | |||
| Yes | 40 (70) | 41 (72) | 43 (72) |
| No | 17 (30) | 16 (28) | 17 (28) |
| Baseline 6MWD, n (%) | |||
| ≥350 m | 35 (61) | 32 (56) | 33 (55) |
| <350 m | 22 (39) | 25 (44) | 27 (45) |
| Functional characteristics | |||
| 6MWD, m, mean (SD) | 361 (87.5) | 350 (97.6) | 361 (99.7) |
| %-predicted 6MWD, mean (SD) | 61.9 (16.26) | 59.6 (18.06) | 61.6 (17.78) |
| Climb 4 stairs, s, mean (SD) | 6.0 (5.67) | 6.9 (6.47) | 7.5 (7.46) |
| Descend 4 stairs, s, mean (SD) | 5.5 (5.75) | 6.1 (5.98) | 6.7 (7.21) |
| 10-m run/walk, s, mean (SD) | 6.7 (2.67) | 7.4 (4.37) | 7.4 (4.36) |
| Supine to stand, s, mean (SD) | 11.5 (11.44) | 10.8 (9.92) | 12.3 (11.19) |
| Falls/day*, mean (SD) | 0.5 (0.94) | 0.3 (0.48) | 0.4 (0.60) |
*
Baseline falls/day data were available for 48, 48, and 54 patients in the placebo, ataluren 40 mg/kg/day, and ataluren 80 mg/kg/day treatment arms, respectively.
SD, standard deviation; UAA, uridine-adenosine-adenosine; UAG, uridine-adenosine-guanosine; UGA, uridine-guanosine-adenosine