IMPE2023 Poster Presentations GH and IGFs (14 abstracts)
Children with Growth Hormone Deficiency Who Completed Treatment with Lonapegsomatropin Approached or Exceeded Average Parental Height at Week 130 in the enliGHten Trial
Paul Thornton 1 , Paul Hofman 2 , Ulhas Nadgir 3 , Paul Saenger 4 , Elena Chertok 5 , Elena Aghajanova 6 , Wenjie Song 7 , Meng Mao 7 , Allison Komirenko 7 , Michael Beckert 7 , Aimee Shu 7 & Aristides Maniatis 8
1Cook Children’s Medical Center, Fort Worth, USA. 2Liggins Institute, University of Auckland, Auckland, New Zealand. 3Center of Excellence in Diabetes and Endocrinology, Sacramento, USA. 4NYU Langone Health, New York, USA. 5Voronezh State Medical University, Voronezh, Russia. 6Yerevan State Medical University, Yerevan, Armenia. 7Ascendis Pharma, Palo Alto, USA. 8Rocky Mountain Pediatric Endocrinology, Centennial, USA
Background: Lonapegsomatropin (TransCon hGH) is a once-weekly prodrug of somatropin, approved for the treatment of pediatric growth hormone deficiency (pGHD) by the US FDA and EMA. In the pivotal phase 3 heiGHt trial, lonapegsomatropin demonstrated noninferior and superior annualized heigh velocity (AHV) and a comparable safety profile to daily somatropin in children with GHD. The ongoing open-label extension trial, enliGHten, enrolled participants from the heiGHt or fliGHt trial (switch study) to assess the long-term safety and efficacy of lonapegsomatropin treatment. At enliGHten Week 130, the height standard deviation score (SDS) mean (standard deviation [SD]) was -0.64 (0.85) compared with baseline value of -1.56 (0.88), with an annualized height velocity mean (SD) of 7.6 cm/year (1.85) (ENDO 2022, Thornton et al, presentation RF26|PMON332).
Methods: Upon entry into enliGHten, all participants received lonapegsomatropin via vial/syringe, with subsequent switch to TransCon hGH Auto-Injector when available. Participants were determined to have completed the trial either by achieving near adult height (bone age >14 years [females] or >16 years [males]) or because treatment for pGHD was no longer necessary in these children based on investigator judgement.
Results: At Week 130 (data snapshot date: September 01, 2021), 36 participants had completed the trial per investigator judgement that treatment for pGHD was no longer necessary. Of these, 14 participants had reached bone age of >14 years (girls) or >16 years (boys). All participants who completed the trial had been previously treated with daily GH prior to beginning lonapegsomatropin and the mean (SD) age at baseline was 14.5 (1.44) years (range 10.8- 17.4 years). The mean (SD) duration of treatment with lonapegsomatropin was 2.2 (0.66) years and total treatment duration with GH was 3.5 (0.88) years. The mean (SD) height SDS at last visit was -0.38 (0.74), close to the average parental height SDS of -0.33 (0.83). The majority of completers (61.1%) met or exceeded average parental height SDS. Participants who completed the trial were Tanner stage 4 or 5. The adverse event profile remained consistent with what was observed in the heiGHt and fliGHt trials, with no new safety signals.
Conclusions: In this analysis, 36 children and adolescents who completed therapy with lonapegsomatropin in the enliGHten trial approached or exceeded average parental height SDS. While this is an interim snapshot of the enliGHten trial, additional data from over 60 participants who have completed lonapegsomatropin treatment are anticipated in a future final analysis.
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