TY - JOUR
T1 - Switching to Weekly Lonapegsomatropin from Daily Somatropin in Children with Growth Hormone Deficiency
T2 - The fliGHt Trial
AU - Maniatis, Aristides K.
AU - Nadgir, Ulhas
AU - Saenger, Paul
AU - Reifschneider, Kent L.
AU - Abuzzahab, Jennifer
AU - Deeb, Larry
AU - Fox, Larry A.
AU - Woods, Katie A.
AU - Song, Wenjie
AU - Mao, Meng
AU - Chessler, Steven D.
AU - Komirenko, Allison S.
AU - Shu, Aimee D.
AU - Casella, Samuel J.
AU - Thornton, Paul S.
N1 - Publisher Copyright:
© 2022 The Author(s). Published by S. Karger AG, Basel.
PY - 2022/8/1
Y1 - 2022/8/1
N2 - Introduction: The phase 3 fliGHt Trial evaluated the safety and tolerability of once-weekly lonapegsomatropin, a long-acting prodrug, in children with growth hormone deficiency (GHD) who switched from daily somatropin therapy to lonapegsomatropin. Methods: This multicenter, open-label, 26-week phase 3 trial took place at 28 sites across 4 countries (Australia, Canada, New Zealand, and the USA). The trial enrolled 146 children with GHD, 143 of which were previously treated with daily somatropin. All subjects received once-weekly lonapegsomatropin 0.24 mg human growth hormone/kg/week. The primary outcome measure was safety and tolerability of lonapegsomatropin over 26 weeks. Secondary outcome measures assessed annualized height velocity (AHV), height standard deviation score (SDS), and IGF-1 SDS at 26 weeks. Results: Subjects had a mean prior daily somatropin dose of 0.29 mg/kg/week. Treatment-emergent adverse events (AEs) reported were similar to the published AE profile of daily somatropin therapies. After switching to lonapegsomatropin, the least-squares mean (LSM) AHV was 8.7 cm/year (95% CI: 8.2, 9.2) at Week 26 and LSM height SDS changed from baseline to Week 26 of +0.25 (95% CI: 0.21, 0.29). Among switch subjects, the LSM for average IGF-1 SDS was sustained at Weeks 13 and 26, representing an approximate 0.7 increase from baseline (prior to switching from daily somatropin therapy). Patient-reported outcomes indicated a preference for weekly lonapegsomatropin among both children and their parents. Conclusions: Lonapegsomatropin treatment outcomes were as expected across a range of ages and treatment experiences. Switching to lonapegsomatropin resulted in a similar AE profile to daily somatropin therapy.
AB - Introduction: The phase 3 fliGHt Trial evaluated the safety and tolerability of once-weekly lonapegsomatropin, a long-acting prodrug, in children with growth hormone deficiency (GHD) who switched from daily somatropin therapy to lonapegsomatropin. Methods: This multicenter, open-label, 26-week phase 3 trial took place at 28 sites across 4 countries (Australia, Canada, New Zealand, and the USA). The trial enrolled 146 children with GHD, 143 of which were previously treated with daily somatropin. All subjects received once-weekly lonapegsomatropin 0.24 mg human growth hormone/kg/week. The primary outcome measure was safety and tolerability of lonapegsomatropin over 26 weeks. Secondary outcome measures assessed annualized height velocity (AHV), height standard deviation score (SDS), and IGF-1 SDS at 26 weeks. Results: Subjects had a mean prior daily somatropin dose of 0.29 mg/kg/week. Treatment-emergent adverse events (AEs) reported were similar to the published AE profile of daily somatropin therapies. After switching to lonapegsomatropin, the least-squares mean (LSM) AHV was 8.7 cm/year (95% CI: 8.2, 9.2) at Week 26 and LSM height SDS changed from baseline to Week 26 of +0.25 (95% CI: 0.21, 0.29). Among switch subjects, the LSM for average IGF-1 SDS was sustained at Weeks 13 and 26, representing an approximate 0.7 increase from baseline (prior to switching from daily somatropin therapy). Patient-reported outcomes indicated a preference for weekly lonapegsomatropin among both children and their parents. Conclusions: Lonapegsomatropin treatment outcomes were as expected across a range of ages and treatment experiences. Switching to lonapegsomatropin resulted in a similar AE profile to daily somatropin therapy.
KW - Growth hormone
KW - Growth hormone deficiency
KW - Growth hormone replacement therapy
KW - Lonapegsomatropin
KW - Long-acting growth hormone
KW - TransCon human growth hormone
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U2 - 10.1159/000524003
DO - 10.1159/000524003
M3 - Article
C2 - 35263755
AN - SCOPUS:85135596478
SN - 1663-2818
VL - 95
SP - 233
EP - 243
JO - Hormone Research in Paediatrics
JF - Hormone Research in Paediatrics
IS - 3
ER -