Table 1.
Baseline characteristics in tadalafil DMD trial and ACT DMD placebo arms
| Tadalafil DMD trial placebo arm | ACT DMD placebo arm | |||||
|---|---|---|---|---|---|---|
| Total (N = 116) | Deflazacort (N = 58) | Prednisone/prednisolone (N = 58) | Total (N = 115) | Deflazacort (N = 53) | Prednisone/prednisolone (N = 62) | |
| Age (years) | 9.5 (1.8)* | 9.8 (2.0) | 9.1 (1.4) | 9.0 (1.7) | 9.2 (1.7) | 8.8 (1.6) |
| Corticosteroid duration before baseline, months [n (%)] | ||||||
| 6 to <12 months | 14 (12.1) | 6 (10.3) | 8 (13.8) | 19 (16.5) | 7 (13.2) | 12 (19.4) |
| ≥12 months | 102 (87.9) | 52 (89.7) | 50 (86.2) | 96 (83.5) | 46 (86.8) | 50 (80.6) |
| Daily corticosteroid use [n (%)] | 85 (73.3)* | 50 (86.2) | 35 (60.3) | 84 (73.0)* | 45 (84.9) | 39 (62.9) |
| 6MWD (meters) | 335.0 (49.6) | 343.5 (45.1) | 326.5 (52.8) | 362.7 (81.4) | 361.3 (87.7) | 363.9 (76.4) |
| NSAA total score | 20.2 (7.1) | 20.4 (6.7) | 20.0 (7.4) | 21.7 (8.1) | 20.7 (7.1) | 23.0 (9.0) |
| NSAA linearized score | 55.9 (14.8) | 56.0 (13.6) | 55.7 (16.1) | 60.0 (18.4) | 57.2 (15.4) | 63.3 (21.4) |
| Rise from supine (seconds) | 13.3 (10.0) | 13.3 (10.4) | 13.2 (9.7) | 9.8 (8.0) | 8.7 (7.7) | 10.7 (8.2) |
| 10‐meter walk/run (seconds) | 6.6 (1.9) | 6.6 (1.9) | 6.7 (2.0) | 6.8 (2.9) | 6.6 (3.2) | 7.1 (2.6) |
| 4‐stair climb (seconds) | 7.2 (5.8) | 7.1 (5.7) | 7.3 (6.0) | 6.5 (5.7) | 6.4 (6.9) | 6.5 (4.4) |
Note: Characteristics are presented as mean (standard deviation), unless otherwise specified.
Abbreviations: 6MWD, 6‐minute walk distance; ACT DMD, Ataluren Confirmatory Trial in Duchenne Muscular Dystrophy; DMD, Duchenne muscular dystrophy; NSAA, North Star Ambulatory Assessment.
*
P < .05 for difference between deflazacort and prednisone/prednisolone groups.