Table 2.
Functional Endpoint Assessments for the Full Analysis Population Overall and by Age Group
| Baseline Mean (SD) | Week 52 Change from Baseline Mean (SD) | |||||
| Edasalonexent Overall n = 81 ≤6.0 yr. n = 51 > 6.0 yr. n = 30 | Placebo Overall n = 38 ≤6.0 yr. n = 26 > 6.0 yr. n = 12 | Edasalonexent | Placebo | LS Mean Δa | p-value | |
| NSAA Score | ||||||
| Overall | 21.5 (4.5) | 19.5 (5.0) | –1.5 (4.4) | –1.8 (3.8) | 0.3 | 0.67 |
| ≤6.0 Years | 20.2 (3.6) | 18.9 (4.9) | 0.0 (3.3) | –1.0 (3.6) | 1.4 | 0.08 |
| > 6.0 Years | 23.9 (5.1) | 20.8 (5.4) | –4.2 (4.9) | –3.8 (3.7) | –0.2 | 0.88 |
| Stand from Supine Time (sec) | ||||||
| Overall | 5.27 (1.89) | 5.37 (2.05) | 1.74 (3.35) | 3.09 (4.81) | ||
| ≤6.0 Years | 5.32 (1.90) | 5.11 (1.74) | 0.72 (2.45) | 2.93 (4.42) | ||
| > 6.0 Years | 5.18 (1.91) | 5.93 (2.59) | 3.70 (3.99) | 3.4 6 (5.89) | ||
| Speed (sec–1) | ||||||
| Overall | 0.211 (0.072) | 0.212 (0.073) | –0.039 (0.067) | –0.046 (0.062) | 0.0011 | 0.92 |
| ≤6.0 Years | 0.208 (0.073) | 0.218 (0.071) | –0.015 (0.060) | –0.045 (0.059) | 0.0271 | 0.046 |
| > 6.0 Years | 0.221 (0.072) | 0.199 (0.079) | –0.086 (0.055) | –0.048 (0.071) | –0.0350 | 0.12 |
| 4-Stair Climb Time (sec) | ||||||
| Overall | 4.29 (1.86) | 4.48 (1.68) | 1.56 (3.91) | 2.56 (4.85) | ||
| ≤6.0 Years | 4.75 (1.89) | 4.60 (1.78) | 0.89 (3.87) | 2.68 (5.16) | ||
| > 6.0 Years | 3.51 (1.52) | 4.21 (1.47) | 2.93 (3.72) | 2.30 (4.36) | ||
| Speed (sec-1) | ||||||
| Overall | 0.271 (0.102) | 0.254 (0.091) | –0.022 (0.089) | –0.039 (0.074) | 0.009 | 0.58 |
| ≤6.0 Years | 0.238 (0.080) | 0.249 (0.092) | 0.003 (0.077) | –0.038 (0.080) | 0.044 | 0.021 |
| > 6.0 Years | 0.332 (0.109) | 0.265 (0.090) | –0.073 (0.093) | –0.042 (0.060) | –0.054 | 0.11 |
| 10-meter Run/Walk Time (sec) | ||||||
| Overall | 5.71 (1.14) | 5.84 (1.16) | 0.32 (1.21) | 0.64 (1.37) | ||
| ≤6.0 Years | 5.96 (1.17) | 5.92 (1.12) | 0.00 (0.94) | 0.40 (1.19) | ||
| > 6.0 Years | 5.28 (0.97) | 5.68 (1.27) | 1.05 (1.46) | 1.18 (1.66) | ||
| Speed (sec-1) | ||||||
| Overall | 0.182 (0.035) | 0.178 (0.36) | –0.006 (0.030) | –0.009 (0.025) | –0.0013 | 0.79 |
| ≤6.0 Years | 0.174 (0.033) | 0.175 (0.035) | 0.002 (0.024) | –0.004 (0.024) | 0.0048 | 0.41 |
| > 6.0 Years | 0.198 (0.034) | 0.184 (0.040) | –0.024 (0.035) | –0.022 (0.025) | –0.0051 | 0.67 |
aLeast squares (LS) mean difference between groups (edasalonexent minus placebo) without multiplicity adjustments. For prespecified subgroup analyses by age, nominal p-values are reported without adjustment of multiplicity and statistical significance is defined as nominal P < 0.05.