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Ascendis Pharma A/S, a clinical stage biopharmaceutical company that applies its innovative TransCon technology to address significant unmet medical needs, announced the initiation of the global phase 3 TransCon Growth Hormone heiGHt Trial in children with growth hormone deficiency (GHD).
The heiGHt Trial initiation follows end-of-phase 2 discussions with the US Food and Drug Administration (FDA), as well as various discussions with regulatory agencies worldwide.
"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," said Jonathan A. Leff, M.D., senior vice president and chief medical officer at Ascendis. "This pivotal trial mirrors the design of our phase 2 study in pediatric GHD that demonstrated comparable safety, efficacy, and tolerability of once-weekly TransCon Growth Hormone to a daily growth hormone therapy. This global trial is designed to support planned regulatory applications worldwide, including in the United States and Europe."
Dr. Leff continued, "The phase 2 data for our once-weekly sustained-release TransCon Growth Hormone demonstrated the strong efficacy and favorable safety, tolerability, and convenience profile of our product candidate. As the only long-acting growth hormone that provides the same mode of action as daily growth hormone, we believe TransCon Growth Hormone can become a best-in-class option for physicians and patients to address the ongoing unmet need for an effective and convenient long-acting growth hormone therapy."
The heiGHt trial is a randomized, open-label, active-controlled phase 3 registration study that is designed to enroll approximately 150 children with GHD who have not previously been treated. Patients will receive either once-weekly 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 of the trial is height velocity after twelve months of treatment. Patients completing therapy may then enroll in a planned open-label extension study. Ascendis plans to conduct the trial at sites in North and South America, Europe, the Middle East, North Africa, and Oceania (Australia/New Zealand).
