Ascendis Pharma has reached its target for patient enrolment in a Phase lll trial called heiGHt that seeks to evaluate a treatment for paediatric growth hormone deficiency (GHD).
The randomised, open-label, active-controlled trial expects to assess treatment-naive children with GHD who receive either once-weekly TransCon Growth Hormone or daily Genotropin.
The trial's primary endpoint is annualised height velocity at 52 weeks, while primary statistical analysis will be a non-inferiority analysis comparing the two arms.
Ascendis plans to randomise more than 160 subjects in the trial.
Ascendis Pharma president and CEO Jan Mikkelsen said: “We have exceeded our target enrolment in this pivotal trial and we look forward to announcing top-line results early next year.
“Our Phase ll data suggested that TransCon Growth Hormone was comparable to a daily growth hormone therapy in terms of efficacy, safety, and tolerability.
“As a long-acting growth hormone that delivers unmodified growth hormone, we believe TransCon Growth Hormone can help address the ongoing unmet need for a convenient long-acting growth hormone therapy with efficacy, safety, and tolerability similar to daily alternatives.”
Ascendis added that patients completing the heiGHt trial could be enrolled in the enliGHten trial, which is the company’s open-label long-term extension study.
In addition, Ascendis is carrying out a trial, fliGHt, to examine TransCon Growth Hormone in patients who have previously been treated with daily growth hormone therapy.