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news.Ascendis Pharma has started its phase 3 registration trial for TransCon Growth Hormone in children with growth hormone deficiency (GHD).
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The heiGHt trial is similar to the company’s earlier phase 2 study in pediatric GHD that showed comparable safety, efficacy, and tolerability of once-weekly TransCon Growth Hormone to a daily growth hormone therapy.
The Phase 3 study, like the phase 2, is a randomized, open-label, active-controlled 52-week trial that is designed to enroll about 150 children diagnosed with GHD who have not previously been treated.
Participants will receive either a once-weekly injection of TransCon Growth Hormone (0.24/mg/kg/week) or daily injections of Genotropin at 34 µg/kg/day (0.24 mg/kg/week) with a 2:1 randomization in a non-inferiority design.
The primary endpoint is height velocity after 12 months of treatment.
Patients completing therapy could then enroll in a proposed open-label extension study.
Ascendis intends to conduct the trial at sites in
Ascendis Pharma senior vice president and chief medical officer Jonathan Leff said: "The Phase 3 heiGHt Trial initiation marks a major milestone for Ascendis and our TransCon Growth Hormone program as we move into the next stage of development.”
The company is applying its TransCon technology to develop an internal pipeline of therapeutics to address unmet medical needs in rare disease indications that carry substantial commercial potential.