ESPEYB19 4. Growth and Growth Factors Long-acting growth hormone (LAGH) (2 abstracts)
4.8. Weekly lonapegsomatropin in treatment-naive children with growth hormone deficiency: the phase 3 height trial
Thornton PS , Maniatis AK , Aghajanova E , Chertok E , Vlachopapadopoulou E , Lin Z , Song W , Dam Christoffersen E , Breinholt VM , Kovalenko T , Giorgadze E , Korpal-Szczyrska M , Hofman PL , Karpf DB , Shu AD & Michael Beckert M.
J Clin Endocrinol Metab, 2021. 106(11): p. 3184-3195. PMID: 34272849
Brief Summary: This randomized, open-label, controlled, phase 3 trial compared the effects of once-weekly long-acting GH lonapegsomatropin versus daily GH in GHD children over a period of 52 weeks. Efficacy was evaluated by height velocity (HV) and height gain from baseline to end. The long-acting formulation showed not only non-inferiority but also superior efficacy compared to daily GH. Adverse events (AEs), bone age advancement and immunogenicity were not different between the 2 groups of treatment. Lonapegsomatropin may represent a promising alternative to daily GH treatment in children with GHD.
Lonapegsomatropin is a once-weekly long-acting GH formulation for children and adults with GHD consisting of the parent drug, somatropin, an inert methyoxy polyethelene glycol carrier, and a TransCon® linker (1). This study reports the results of the Phase 3 randomized, open-label, controlled trial comparing weekly lonapegsomatropin to daily somatropin (heiGHt; NCT02781727). The enrolled children with GHD were randomized to receive either once-weekly lonapegsomatropin (n=105; 0.24 mg/kg/wk) or daily rhGH (n= 56; 0.034 mg/kg/d) for 52 weeks. Treatment was completed by 104 patients in the lonapegsomatropin arm and by 55 patients in the daily rhGH arm.
At week 52, mean height velocity was higher in children treated with lonapegsomatropin compared with those treated with daily GH (11.2 vs 10.3 cm/year, p<0.001). Mean height gain was 1.10 SDS on lonapegsomatropin vs 0.96 SDS on daily GH (p=0.01). On lonapegsomatropin, IGF-1 levels increased between 0 to +2 SDS earlier than on daily GH, and showed a higher average IGF-1 SDS throughout the study. 7.6% of lonapegsomatropin treated patients vs 3.6% of daily GH treated children showed IGF-1 levels > +2 SDS at least once during the trial. No notable difference in bone age maturation was observed between the two treatment groups. No increase in adverse events, immunogenicity and local reactions was observed in patients treated with lonapegsomatropin compared to daily somatropin. In the rapidly evolving landscape of long-acting GH formulations, lonapegsomatropin appears as a valid alternative to daily GH treatment in children with GHD.
Reference: 1. Chatelain P, Malievskiy O, Radziuk K, et al; TransCon GH Working Group. A randomized Phase 2 study of long-acting TransCon GH vs daily GH in childhood GH deficiency. J Clin Endocrinol Metab.2017;102(5):1673–1682.
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ESPE Yearbook of Paediatric Endocrinology
ISSN 1662-4009 (Online)
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