Table 1. Characteristics of the Participants at Baseline (Modified Intention-to-Treat Population).
| Characteristic | Mean (SD) | |||
|---|---|---|---|---|
| Vamorolone | Prednisone 0.75 mg/kg/d (n = 31) | Control (placebo) group (n = 28) | ||
| 6 mg/kg/d (n = 28) | 2 mg/kg/d (n = 30) | |||
| Quality rating scheme = 1 | ||||
| Age, y | 5.4 (0.9) | 5.3 (0.9) | 5.5 (0.9) | 5.4 (0.8) |
| Height, cm | 107 (7) | 108 (9) | 111 (6) | 109 (9) |
| Height percentile | 23 (25) | 30 (29) | 37 (29) | 33 (29) |
| Weight, kg | 19 (3) | 19 (4) | 21 (3) | 20 (3) |
| BMIa | 16.6 (1.4) | 16.2 (1.2) | 16.8 (1.3) | 16.3 (1.1) |
| TTSTAND velocity, event/s | 0.19 (0.06) | 0.18 (0.05) | 0.22 (0.06) | 0.20 (0.06) |
| 6MWT, m | 313 (56) | 316 (58) | 343 (56) | 355 (78) |
| TTRW velocity, m/s | 1.6 (0.4) | 1.6 (0.3) | 1.9 (0.4) | 1.7 (0.3) |
| NSAA score | 18.9 (4.1) | 17.2 (4.7) | 21.2 (5.5) | 18.9 (5.3) |
| TTCLIMB velocity, event/s | 0.21 (0.09) | 0.20 (0.05) | 0.29 (0.11) | 0.25 (0.09) |
| Osteocalcin level, ng/mLb | 59.7 (14.8) | 57.2 (18.3) | 55.9 (12.9) | 55.0 (13.8) |
| P1NP level, μg/L | 490 (145) | 521 (204) | 480 (116) | 483 (161) |
| CTX1 level, pg/mLc | 1074 (206) | 1128 (382) | 1125 (162) | 1079 (258) |
| Morning cortisol level, nmol/Ld | 235 (67) | 238 (83) | 212 (66) | 199 (62) |
| Standard dose ACTH stimulation test | ||||
| Serum cortisol level, nmol/L | ||||
| 30 min (Normal range >500) | 547 (119) | 555 (86) | 532 (101) | 550 (104) |
| 60 min (Normal range >500) | 659 (105) | 648 (94) | 612 (97) | 628 (112) |
| <500 nmol/L at 30 and 60 min, No. (%) | 0 (0) | 2 (7.4) | 5 (17.2) | 3 (10.7) |
Abbreviations: 6MWT, 6-minute walk test; ACTH, corticotropin; BMI, body mass index; CTX1, type 1 collagen cross-linked C-telopeptide; NSAA, NorthStar Ambulatory Assessment; P1NP, procollagen 1 intact N-terminal propeptide; TTCLIMB, time to climb 4 stairs; TTRW, time to run/walk 10 m; TTSTAND, time to stand from supine.
SI conversion factor: To convert osteocalcin to micrograms per liter, multiply by 1; to convert serum cortisol to micrograms per deciliter, divide by 27.588.
a
Calculated as weight in kilograms divided by height in meters squared.
b
Normal range, 39-121 ng/mL.
c
Normal range, 500-1700 pg/mL.
d
Normal range, 138-690 nmol/L.