Table 2.

Height, BMI, and Bone Biomarker Comparisons (Safety-2 [Height and BMI] and mITT-2 [Biomarker] Populations)

End-point	LSM difference (SE)	95% CI for LSM difference	p Value
Dose dependency at week 48
Height z score VAM6 vs VAM2	0.123 (0.0805)	−0.0382 to 0.285	0.1312
BMI z score VAM6 vs VAM2	0.0463 (0.183)	−0.320 to 0.413	0.8010
Intragroup change from week 24 (prednisone) to week 48 (vamorolone)
Height z score PRED-VAM6	0.228 (0.104)	0.0157 to 0.44	0.0362
Height z score PRED-VAM2	0.0959 (0.0993)	−0.108 to 0.299	0.3430
BMI z score PRED-VAM6	−0.0064 (0.104)	−0.22 to 0.207	0.952
BMI z score PRED-VAM2	−0.187 (0.097)	−0.387 to 0.0129	0.0655
Osteocalcin ng/mL PRED-VAM6	26.8 (5.14)	16.1 to 37.5	<0.0001
Osteocalcin ng/mL PRED-VAM2	38.5 (5.24)	27.6 to 49.5	<0.0001
CTX1 pg/mL PRED-VAM6	570 (80.3)	404 to 737	<0.0001
CTX1 pg/mL PRED-VAM2	667 (78.1)	505 to 829	<0.0001
P1NP μg/L PRED-VAM6	184 (36.2)	109 to 259	<0.0001
P1NP μg/L PRED-VAM2	317 (36.5)	242 to 393	<0.0001

Abbreviations: 6MWD = 6-minute walk distance; BMI = body mass index; CTX1 = type 1 collagen cross-linked C-telopeptide; LSM = least squares mean; NSAA = North Star Ambulatory Assessment; P1NP = procollagen 1 intact N-terminal propeptide; PRED-VAM2 = prednisone → vamorolone 2 mg/kg/d; PRED-VAM6 = prednisone → vamorolone 6 mg/kg/d; SE = standard error; VAM2 = vamorolone 2 mg/kg/d; VAM6 = vamorolone 6 mg/kg/d.