Table 4.
Evaluation of other clinical endpoints at week 24 and week 48
| Lower/Upper Extremity | Week 24 Treatment Comparisons | Week 48 Treatment Comparisons | ||||||
| Functional Endpoints | ||||||||
| 6 g/day Ace-ER (N = 15) | 3 g/day Ace-ER (N = 18) | 6/6 + 0/6 g/day Ace-ER (N = 24) | 6/6 g/day Ace-ER (N = 15) | |||||
| vs Placebo (N = 14) | vs Placebo (N = 14) | vs 3/3 + 0/3 g/day Ace-ER (N = 23) | vs 3/3 g/day Ace-ER (N = 18) | |||||
| LS means | p-value | LS means | p-value | LS means | p-value | LS means | p-value | |
| difference | difference | difference | Difference | |||||
| (95% CI) | (95% CI) | (95% CI) | (95% CI) | |||||
| 6MWT (m) | +0.53 | 0.95 | –6.65 | 0.44 | +2.41 | 0.78 | –2.80 | 0.78 |
| (–16.45, 17.52) | (–23.70, 10.39) | (–14.30, 19.12) | (–22.88, 17.28) | |||||
| Gait Speed: Comfortable Gait (cm/sec) | +0.64 | 0.90 | –5.19 | 0.33 | +1.51 | 0.65 | +3.03 | 0.29 |
| (–9.48, –10.76) | (–15.70, 5.32) | (–5.03, 8.06) | (–2.56, 8.62) | |||||
| Gait Speed Maximum Gait (cm/sec) | +1.65 | 0.76 | –6.16 | 0.23 | +2.01 | 0.59 | +4.37 | 0.22 |
| (–8.86, 12.16) | (–16.21, 3.89) | (–5.24, 9.26) | (–2.65, 11.38) | |||||
| Stair Climb Test | –2.14 | 0.25 | –2.03 | 0.30 | –2.42 | 0.41 | –5.02 | 0.38 |
| (–5.79, 1.51) | (–5.83, 1.78) | (–8.15, 3.31) | (–16.14, 6.10) | |||||
| Sit-to-stand Test | +0.92 | 0.28 | +0.25 | 0.80 | +1.84 | 0.086 | +1.32 | 0.22 |
| (–0.76, 2.61) | (–1.67, 2.17) | (–0.26, 3.93) | (–0.80, 3.44) | |||||
| Weighted Arm-lift Test | –0.59 | 0.80 | +0.35 | 0.89 | +2.37 | 0.41 | +2.90 | 0.076 |
| (–5.19, 4.01) | (–4.45, 5.14) | (–3.23, 7.98) | (–0.31, 6.10) | |||||
Abbreviations: 6MWT = six minute walk test; CI = confidence interval; cm = centimeters; LS = least squares; m = meters; Ace-ER = sialic acid extended release; sec = seconds. Treatment comparisons are based on the LS means and CI derived from the GEE model.