Background/Aims: To determine the optimal dose of LB03002, a sustained-release, once-weekly formulation of recombinant human growth hormone (rhGH), and to compare its efficacy and safety with daily rhGH in children with idiopathic short stature (ISS). Methods: This multicenter, randomized, open-label, phase II study included GH-naïve, prepubertal children with ISS, randomized to receive daily rhGH 0.37 mg/kg/week (control, n = 16), LB03002 0.5 mg/kg/week (n = 14), or LB03002 0.7 mg/kg/week (n = 16). The primary endpoint was height velocity (HV) change at week 26. Results: At week 26, the least square (LS) means for HV change (cm/year) with control, LB03002 0.5 mg/kg/week, and LB03002 0.7 mg/kg/week were 5.08, 3.65, and 4.38, and the LS means for the change in height standard deviation score were 0.65, 0.49, and 0.58, respectively. The lower bound of the 90% confidence interval for the difference between LB03002 0.7 mg/kg/week and the control in the LS mean for HV change (-1.72) satisfied the noninferiority margin (-1.75). Adverse events were generally mild and short-lived. Conclusion: A once-weekly regimen of LB03002 0.7 mg/kg demonstrated noninferiority to the daily regimen of rhGH 0.37 mg/kg/week in terms of HV increments. LB03002 was well tolerated and its safety profile was comparable with that of daily rhGH.
Bibliographical noteFunding Information:
This study was sponsored by LG Life Sciences, Ltd., which is now merged with LG Chem, Ltd., Korea. S.H. is employed by LG Chem, Ltd. J.S.H., K.-H.L., H.-W.Y., B.-K.S., C.H.S., H.-S.H., H.-S.K., and C.H.S. are the Steering Committee members of LGS (LG Growth Study). Otherwise, the authors have no competing interest to declare that could be perceived as prejudicing the impartiality of the research reported.
We thank the patients and their caregivers; Suin Lee of LG Chem, Ltd., for her support in clinical operations; and Hye-Ryon Kim and Szymon Bruzewicz (SciencePro) for editorial assistance in the preparation of the manuscript.
© 2018 The Author(s). Published by S. Karger AG, Basel.
- Height velocity
- Idiopathic short stature
- Sustained-release growth hormone
ASJC Scopus subject areas
- Pediatrics, Perinatology, and Child Health
- Endocrinology, Diabetes and Metabolism