Background: TransCon hGH is a long-acting prodrug of recombinant human GH (hGH) that releases into the blood compartment fully active unmodified hGH. TransCon hGH was shown in two Phase 1 studies and a Phase 2 study in AGHD to be safe and well tolerated and inducing an IGF-I pharmacodynamic response within the normal range throughout the dosing period. This interim analysis consists of 25 patients completing all 6 months of treatment.
Objectives: The objective of this study is to investigate i) safety and tolerability, ii) pharmacokinetics and pharmacodynamics, and iii) efficacy of TransCon hGH in children with GHD.
Design and methods: Pre-pubertal, treatment naïve GHD children received s.c. injections of one of three once-weekly TransCon hGH doses (0.14, 0.21 or 0.30 mg hGH/kg per week) or daily hGH (Genotropin; 0.03 mg hGH/kg per day=0.21 mg/kg per week) over a 6-month treatment period.
Results: Mean annualised height velocities ranged from 11.9 cm for the 0.14 mg/kg per week dose to 14.5 cm for the 0.30 mg/kg per week dose, which were comparable to 11.5 cm for the active comparator, Genotropin at a 0.21 mg/kg per week dose. No SAEs occurred and injection site reactions were generally mild and transient and occurred in only a few patients. Maximum hGH blood concentrations are comparable between equivalent weekly doses of TransCon hGH and daily hGH; and a doseproportional increase in IGF-I levels into the normal range was observed for the three TransCon hGH dose levels.
Conclusions: To date, TransCon hGH has demonstrated efficacy and safety comparable to that observed with daily hGH. Injection site reactions have been mild and similar to what is expected with daily hGH injections, with no nodule formation or lipoatrophy noted. This TransCon hGH Phase 2 study conducted in a paediatric population confirms the excellent safety and efficacy profile observed in previous clinical trials.
Disclosure: This study was sponsored by Ascendis Pharma A/S.