Table 1. Participant Demographic and Baseline Characteristics for External Comparisonsa.
| Characteristic | Participants | ||
|---|---|---|---|
| Higher-dose VBP15-LTE (N = 23)b | GC in CINRG DNHS (N = 75) | GC in NorthStar UK Network (N = 110)c | |
| Age at baseline comparison visit, y | |||
| Mean (SD) | 5.83 (0.88) | 6.08 (0.81) | 6.00 (0.77) |
| Median (range) | 5.65 (4.60-7.29) | 5.97 (4.50-7.48) | 5.94 (NA) |
| Steroid exposure at baseline comparison visit, mean (SD), d | 200.57 (7.54) | 227.73 (61.91) | 264.84 (57.39) |
| Duration of follow up from baseline visit, mean (SD), yd | 1.85 (0.46) | 1.36 (0.49) | NA |
| Participants with >18 mo follow-up after initial 6 mo of steroid exposure, No. (%) | 21 (91.3) | 30 (40.0) | NA |
| Weight (kg), No. | 23 | 73 | NA |
| Mean (SD) | 21.98 (3.78) | 20.35 (3.55) | NA |
| Median (range) | 22.50 (16.30-32.60) | 20.10 (14.80-32.60) | NA |
| Height (cm), No. | 22 | 73 | NA |
| Mean (SD) | 111.80 (6.94) | 109.86 (6.86) | NA |
| Median (range) | 113.05 (100.60-123.00) | 110.00 (96.10-126.30) | NA |
| Body Mass Index, No. | 22 | 72 | NA |
| Mean (SD) | 17.68 (1.23) | 16.68 (1.55) | NA |
| Median (range) | 17.65 (15.90-21.50) | 16.56 (12.19-20.44) | NA |
Abbreviations: CINRG, Cooperative International Neuromuscular Research Group; DNHS, Duchenne Natural History Study; GC, glucocorticoid; LTE, long-term extension; NA, not applicable; VBP, vamorolone.
a
Based on baseline comparator visit, which corresponds to approximately 6 months of steroid exposure for participants in the NorthStar UK Network and CINRG DNHS.
b
The higher-dose cohort refers to participants assigned to vamorolone 2.0 and 6.0 mg/kg/d from initial dose-finding (VBP15-002/-003) studies and maintained at 2.0 mg/kg/d or more from long-term extension (VBP15-LTE) baseline.
c
Owing to NorthStar UK Network participant-level data-sharing restrictions, limited summary information was available.
d
Reported for time to stand velocity.