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Clinical Trial
. 2022;95(3):233-243.
doi: 10.1159/000524003. Epub 2022 Mar 9.

Switching to Weekly Lonapegsomatropin from Daily Somatropin in Children with Growth Hormone Deficiency: The fliGHt Trial

Aristides K Maniatis  1 , Ulhas Nadgir  2 , Paul Saenger  3 , Kent L Reifschneider  4 , Jennifer Abuzzahab  5 , Larry Deeb  6 , Larry A Fox  7 , Katie A Woods  8 , Wenjie Song  9 , Meng Mao  9 , Steven D Chessler  9 , Allison S Komirenko  9 , Aimee D Shu  9 , Samuel J Casella  10 , Paul S Thornton  11
Affiliations
Clinical Trial

Switching to Weekly Lonapegsomatropin from Daily Somatropin in Children with Growth Hormone Deficiency: The fliGHt Trial

Aristides K Maniatis et al. Horm Res Paediatr. 2022.

Abstract

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.

Keywords: Growth hormone; Growth hormone deficiency; Growth hormone replacement therapy; Lonapegsomatropin; Long-acting growth hormone; TransCon human growth hormone.

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Conflict of interest statement

A.K.M. has received research funding and is an Advisory Board Consultant for Ascendis Pharma, Novo Nordisk, OPKO, and Pfizer and is a speaker for Ascendis Pharma and Novo Nordisk. P.S. has been a speaker for Ascendis Pharma and Novo Nordisk and has received research funding from Ascendis Pharma, OPKO, Novo Nordisk, and Aeterna Zentaris, and is a member of the editorial board of Hormone Research in Paediatrics. K.L.R. has received research funding from Ascendis Pharma, is a speaker for Ascendis, and serves as a board member of The Human Growth Foundation. J.A. has received research funding from Ascendis Pharma, Novo Nordisk, Lumos, Soleno, Rhythm, and Saniona, and has served as a consultant for Consynance Therapeutics and Rhythm. K.A.W. has served as a consultant for Ascendis Pharma. S.J.C. has received research funding from Ascendis Pharma. W.S., M.M., S.D.C., A.S.K., and A.D.S. are employees of Ascendis Pharma, Inc. P.S.T. has received research funding from Ascendis Pharma, Novo Nordisk, Pfizer, and OPKO. U.N., L.D., and L.A.F. have nothing to declare.

Figures

Fig. 1
Fig. 1
HSDS from GHD diagnosis to Week 26 of the fliGHt Trial. *Based on N = 143 at fliGHt baseline. Values in graph are observed means.
Fig. 2
Fig. 2
a–d Subgroup analyses of AHV and ∆HSDS by baseline characteristics following 26 weeks of lonapegsomatropin treatment.
See this image and copyright information in PMC

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