Biotechnology firm Ascendis Pharma has revealed the positive top-line results from its six-month Phase II paediatric trial of once-weekly TransCon Growth Hormone.
The study has been designed to assess the safety and efficacy of the TransCon Growth Hormone in 53 treatment-naïve and pre-pubertal children to treat growth hormone deficiency (GHD).
The Phase II paediatric study coordinating investigator Dr Pierre Chatelain said: “I am highly encouraged by the mean annualized height velocity achieved by once-weekly TransCon Growth Hormone in the Phase II paediatric study.
“Based on these top-line data, once-weekly TransCon Growth Hormone appears comparable to gold-standard daily growth hormone therapies in terms of efficacy, safety and tolerability, with reduced injection frequency that may allow for improved patient compliance and treatment outcomes.”
The trial evaluated the safety, tolerability, pharmacokinetics, pharmacodynamics, and efficacy of the TransCon Growth Hormone in treatment-naïve pre-pubertal children with GHD.
The firm enrolled 53 patients in the treatment phase of the trial, which was a six-month multi-center, randomised and open-label study that compared three dose levels of TransCon Growth Hormone against the active control Genotropin.
Three dose levels of TransCon Growth Hormone, including 0.14, 0.21, and 0.30mg hGH / kg / week, were administered once per week, while the active control Genotropin with 0.21 mg hGH / kg / week was administered as a daily injection.
In the trial, mean annualised height velocities among the three dosing levels administered weekly ranged from 11.9cm for the 0.14 mg/kg/week dose to 13.9cm for the 0.30mg / kg / week dose, which were comparable to 11.6cm for the active comparator, daily injections of Genotropin at a 0.21mg / kg / week dose, said the company.