Long-term safety and efficacy of the recombinant human growth hormone Omnitrope® in the treatment of Spanish growth hormone deficient children: results of a phase III study.

This phase III study in growth hormone (GH) deficient (GHD) children with growth retardation was designed to demonstrate the safety and efficacy of longterm treatment with the recombinant human GH Omnitrope® (Sandoz BioPharmaceuticals, Holzkirchen, Germany). Treatment-naïve, prepubertal Spanish children (n=70) with isolated GHD were treated with Omnitrope 0.03 mg/kg/day subcutaneously. Changes in height, height standard deviation score (HSDS), height velocity (HV), HV standard deviation score (HVSDS), serum insulin-like growth factor (IGF)-1, and insulin-like growth factor binding protein (IGFBP)-3 levels were recorded. Omnitrope treatment provided a good growth response after 4 years, shown by a significant increase in mean body height (31.1 cm [95% CI: 29.6-32.6]), HSDS (Tanner) (1.42 [1.13-1.70]), HV (2.4 cm [1.7-3.1]), and HVSDS values (3.5 [2.7-4.3]). Mean IGF-1 and IGFBP-3 serum levels also increased significantly. At a dose of 0.03 mg/kg/day, Omnitrope was safe, effective, and well tolerated during long-term treatment of children with GHD.