Table 1.
Color | 6MWT (m): Δ = 35 m | 2MWT (m): Δ = 22 m | CPE‐walkinga: Δ = 2 | MPS HAQ‐walkinga: Δ = 2 | 3MSC (steps): Δ = 20 | CPE‐stairsa: Δ = 1 | MPS HAQ‐stairsa: Δ = 3 | BOT2‐running: Δ = 5 | CPE‐runninga: Δ = 1 | BOT2‐balance: Δ = 5 | CPE‐grossa: Δ = 1 | MPS HAQ‐movementa: Δ = 2 | CHAQ‐paina: Δ = 17 | PROMIS‐paina: Δ = 27 |
---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
[−105, −71] | [−66, −45] | [−5, −4] | [−5, −4] | [−60, −41] | −3 | [−8, −6] | [−13, −9] | −3 | [−13, −9] | −3 | [−4, −3] | [−49, −33] | [−80, −54] | |
[−70, −36] | [−44, −23] | [−3, −2] | [−3, −2] | [−40, −21] | −2 | [−5, −3] | [−8, −5] | −2 | [−8, −5] | −2 | −2 | [−32, −17] | [−53, −27] | |
[−35, −1] | [−22, −1] | −1 | −1 | [−20, −1] | −1 | [−2, −1] | [−4, −1] | −1 | [−4, −1] | −1 | −1 | [−16, −1] | [−26, −1] | |
0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | |
[1, 35] | [1, 22] | 1 | 1 | [1, 20] | 1 | [1, 2] | [1, 4] | 1 | [1, 4] | 1 | 1 | [1, 16] | [1, 26] | |
[36, 70] | [23, 44] | [2, 3] | [2, 3] | [21, 40] | 2 | [3, 5] | [5, 8] | 2 | [5, 8] | 2 | 2 | [17, 32] | [27, 53] | |
[71, 105] | [45, 66] | [4, 5] | [4, 5] | [41, 60] | 3 | [6, 8] | [9, 13] | 3 | [9, 13] | 3 | [3, 4] | [33, 49] | [54, 80] |
Note: White cells (not shown in table, but included in Figure 1) indicate that the assessment was not completed.
Abbreviations: 2MWT, 2‐minute walk test; 3MSC (steps), 3‐minute stair climb test; 6MWT, 6‐minute walk test; BOT2, Bruininks‐Oseretsky test of motor proficiency; CHAQ, childhood health assessment questionnaire; CPE, clinical problem evaluation; MPS HAQ, MPS health assessment questionnaire; PROMIS, PROMIS health assessment questionnaire.
The negative changes (from randomization for placebo period and from baseline for UX003 period) are displayed since decreases in these scores indicate improvement.