Table 3.
Effect of burosumab on select laboratory values and Rickets Severity Score in pediatric clinical trials [7, 50, 72]
| Open-label, phase 2 burosumab Q2W (N = 13) | Open-label, dose-finding phase 2 (N = 52) | Randomized, double-blind, placebo-controlled phase 3 | ||
|---|---|---|---|---|
| Conventional therapy (n = 32) | Burosumab Q2W (n = 29) | |||
| Mean (SE) change from baseline in fasting serum phosphorus, mg/dL | 0.89 (0.11) (week 40) | 0.84 (NR) (week 64) | 0.21 (0.06) (week 64) | 0.91 (0.08) (week 64) |
| Mean (SE) change from baseline in 1,25(OH)2D, pg/mL | 12 (3) (week 40) | 18 (NR) (week 64) | 1 (3) (week 64) | 10 (2) (week 64) |
| Mean (SE) change from baseline in ALP | –213 U/L (14) (week 40) | –90 U/L (NR) (week 64) | –33% (13a) (week 64) | –5% (21a) (week 64) |
| Least squares mean (SE) change from baseline in total RSS | –2.0 (0.1a) (week 64) | –0.92 (0.7) (week 64) | –1.0 (0.2) (week 64) | –2.2 (0.1) (week 64) |
| Patients with substantial healing of rickets (RGI-C ≥ 2.0), % | 100 (week 40) | 54 (week 64) | 19 (week 64) | 87 (week 64) |
Abbreviations: 1,25(OH)2D, 1,25-dihydroxyvitamin D; ALP, alkaline phosphatase; NR, not reported; Q2W, every 2 weeks; RGI-C, radiographic global impression of change; RSS, Rickets Severity Score.
aSD reported.