TransCon hGH weekly dosing evaluated against daily hGH in Phase 3 GHD trial design

Children diagnosed with Growth Hormone Deficiency (GHD) require long-term recombinant human growth hormone (hGH) therapy to achieve normal growth and final adult height. The current standard of care involves daily subcutaneous injections, which can be a significant burden for children and their families, often leading to poor adherence and suboptimal treatment outcomes. This adherence challenge underscores the critical need for less frequent dosing regimens that maintain efficacy while improving patient quality of life. Developing a long-acting hGH formulation, such as TransCon hGH, aims to address this gap by reducing injection frequency and potentially enhancing treatment compliance.

This Phase 3, open-label, randomized, parallel-group, multi-center trial was designed to compare the safety, tolerability, and efficacy of once-weekly TransCon hGH against daily hGH (Genotropin) in prepubertal, hGH-treatment naïve children with GHD. Approximately 150 children were randomized in a 2:1 ratio to receive either TransCon hGH (equivalent to 0.02 mg hGH/kg/wk subcutaneous, weekly) or daily hGH. The trial duration was 52 weeks, following a 14-21 day washout period for any prior daily growth hormone therapy. The primary endpoints included safety, tolerability, and efficacy, likely assessed through height velocity and other growth parameters.

This abstract describes the design of a completed Phase 3 trial, but does not present specific efficacy or safety results. The study aimed to compare the once-weekly administration of TransCon hGH against daily human growth hormone (hGH) in children with growth hormone deficiency (GHD). While the trial is complete, detailed findings regarding height velocity, adverse events, or other primary/secondary endpoints are not provided in this record. Therefore, specific numerical outcomes such as p-values, percentages, or fold-changes cannot be reported here. The trial's completion suggests that data has been collected, but the outcomes are not yet publicly available in this abstract.