After careful consideration, the Endocrine Society canceled its annual meeting (ENDO 2020), which was set to take place in San Francisco, California, from March 28 to 31, 2020, because of concerns regarding coronavirus disease 2019 (COVID-19). Research findings that were scheduled to be presented at the meeting have been published in a supplemental issue of the Journal of the Endocrine Society.
Switching from daily human growth hormone (hGH; somatropin) therapy to once-weekly TransCon hGH resulted in a similar safety and efficacy profile in children and adolescents, according to study results intended to be presented at the annual meeting of the Endocrine Society (ENDO 2020).
Once-weekly TransCon hGH, an investigational long-acting prodrug consisting of hGH, an inert protective carrier, and a linker that temporarily binds them, was assessed in the phase 3 Flight trial (ClinicalTrials.gov Identifier: NCT03305016).
The study aimed to assess the safety, tolerability, and efficacy of once-weekly hGH over 26 weeks. The main efficacy outcomes were annualized height velocity, height standard deviation score (SDS), and insulinlike growth factor-1 (IGF-1) SDS.
The study cohort included 146 participants aged 1 to 17 years (mean age, 10.6 years) who were treated with open-label once-weekly TransCon hGH at dose of 0.24 mg/kg. Most patients had previously received treatment with daily hGH and only 3 were treatment naive.
Treatment-emergent adverse events were documented in 56.8% patients, but only 4.1% experienced an event that was considered to be related to the study drug. The type and frequency of the treatment-emergent adverse events were in line with the known safety profile of daily hGH and none led to discontinuation of the study drug. Although low-titer anti-hGH binding antibodies were detected in 2.8% of treated patients, no neutralizing antibodies were detected.
At 26 weeks, the least-squares mean annualized height velocity was 8.7 cm/y (95% CI, 8.2-9.2) and the least-squares mean height SDS change from baseline was +0.25 (95% CI, 0.21-0.29). The mean observed annualized height velocity ranged from 8.2 to 16.2 cm/y and mean observed height SDS change ranged from +0.23 to +0.96.
In accordance with previous trials in treatment-naive patients, the linear association between average IGF-1 SDS and TransCon hGH doses was also evident in this cohort of children with prior daily hGH treatment who had dose titrations.
Overall, comparing the daily vs weekly treatment regimens, the researchers concluded that “TransCon hGH treatment outcomes, including [annualized height velocity] and height SDS, were as expected across a diversity of ages, disease characteristics, and treatment experiences, reflective of a real-world setting. Dose titrations of TransCon hGH demonstrated a predictable IGF-1 response.”
Disclosure: Several study authors declared affiliations with the pharmaceutical industry. Please see the original reference for a full list of authors’ disclosures.
Maniatis AK, Nadgir U, Saenger P, et al. Phase 3 FliGHt trial: experience of switching from daily growth hormone therapy to once-weekly TransCon hGH in children with growth hormone deficiency. J Endocr Soc. 2020;4(suppl 1):OR10-05.
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