According to a story from healio.com, an oral medication being evaluated in a phase 2 study was able to increase annualized height velocity in children living with growth hormone deficiency, a rare condition. This medication is called ibutamoren, which is being developed by Lumos Pharma. These findings were based on interim analysis of the trial data and was presented at the Pediatric Endocrine Society Annual Meeting.
About Growth Hormone Deficiency
Growth hormone deficiency is a medical condition in which the body’s pituitary gland does not release sufficient levels of growth hormone. It is characterized primarily by abnormally short height. Causes of the condition are varied; it may be the result of a genetic abnormality (the genes GH1, BTK, or GHRHR are typically involved), treatment with radiation therapy, injuries, tumors, or infections. However, the cause is unclear in about a third of cases. Symptoms in children include developmental delays, jaundice, small penis size, low hair growth, and low blood sugar. Symptoms in adults include poor bone density, high cholesterol, reduced muscle mass, baldness (in men), heart problems, increased body fat, and issues with fatigue and concentration. Treatment is typically with supplementation of growth hormone via injection; however, this treatment can cause unpleasant side effects. To learn more about growth hormone deficiency, click here.
Trial Results
In the trial, patients saw an increase in annualized height velocity, which reached an average of 8.6 cm after six months. These patients were being treated with a 1.6 mg/kg dose of ibutamoren each day. This result was comparable to children that were receiving subcutaneous growth hormone supplementation. The trial included 80 pediatric patients with growth hormone deficiency of moderate severity.
The interim data included findings from 41 of the participants. Follow up data revealed a durable response to the therapy at nine months and one year. The full cohort is expected to achieve the primary trial endpoint later in the year, with the full results of the trial available by the conclusion of 2023. If results continue to be promising, then a larger phase 3 trial will then begin development.
