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Weekly Lonapegsomatropin in Treatment-Naïve Children With Growth Hormone Deficiency: The Phase 3 heiGHt Trial

Paul S Thornton1, Aristides K Maniatis2, Elena Aghajanova3

  • 1Cook Children's Medical Center, Fort Worth, TX, USA.

The Journal of Clinical Endocrinology and Metabolism
|July 17, 2021
PubMed

Insights

Once-weekly lonapegsomatropin demonstrated noninferiority and superiority in annualized height velocity compared to daily somatropin in children with growth hormone deficiency (GHD). Safety profiles were similar between the two treatments.

Area of Science:

  • Pediatric endocrinology
  • Pharmacology

Background:

  • Daily somatropin injections for growth hormone deficiency (GHD) present a high treatment burden for children, potentially impacting adherence and outcomes.
  • Lonapegsomatropin (TransCon hGH) is an investigational long-acting, once-weekly human growth hormone (hGH) prodrug designed to reduce treatment burden.

Purpose of the Study:

  • To evaluate the efficacy and safety of once-weekly lonapegsomatropin compared to daily somatropin in treatment-naïve, prepubertal children with GHD.

Main Methods:

  • A 52-week, randomized, open-label, active-controlled Phase 3 trial (heiGHt trial) involving 161 patients.
  • Patients received either lonapegsomatropin (0.24 mg hGH/kg/week) or daily somatropin at an equivalent weekly dose.
  • Primary endpoint was annualized height velocity (AHV); secondary endpoints included changes in height standard deviation scores (SDS).

Main Results:

  • Lonapegsomatropin achieved a significantly higher mean AHV (11.2 cm/year) compared to daily somatropin (10.3 cm/year), demonstrating both noninferiority and superiority (P=0.009).
  • Height SDS increased significantly more from baseline with lonapegsomatropin (1.10) versus somatropin (0.96) (P=0.01).
  • Bone age progression, adverse events, tolerability, and immunogenicity were comparable between treatment groups.

Conclusions:

  • Once-weekly lonapegsomatropin met its primary objective, showing noninferiority and superiority in AHV compared to daily somatropin.
  • Lonapegsomatropin offers a potentially improved treatment option for children with GHD, with a similar safety profile to daily somatropin.
Abstract

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