Oral medication boosts annualized height velocity among children with GH deficiency
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Key takeaways:
- Children with pediatric GH deficiency receiving 1.6 mg/kg per day ibutamoren orally had an annualized height velocity of 8.6 cm per year at 6 months.
- Few treatment-related adverse events were reported.
Children with idiopathic pediatric growth hormone deficiency had an increase in annualized height velocity after 6 months of receiving a daily oral GH therapy, according to a speaker.
During a presentation at the Pediatric Endocrine Society Annual Meeting, Andrew Dauber, MD, MMSc, chief of endocrinology at Children’s National Hospital in Washington, D.C., presented interim data from the phase 2 OraGrowtH210 trial, which analyzed the effects of ibutamoren (LUM-201, Lumos Pharma) among a small group of children with pediatric GH deficiency.
“The interim analysis of the data shows that children with isolated GH deficiency treated with the oral medication LUM-201 at the dose of 1.6 mg/kg were able to increase their growth rates similar to what is expected for similar children treated with subcutaneous GH,” Dauber told Healio. “This data is very exciting because it shows promise that there could be an oral treatment for children with mild, isolated GH deficiency.”
In the trial, 80 children with moderate idiopathic pediatric GH deficiency and a positive predictive enrichment marker for response to ibutamoren were randomly assigned, 1:1:1:1, to 0.8 mg/kg, 1.6 mg/kg or 3.2 mg/kg ibutamoren daily or a daily recombinant human GH injection. Participants were enrolled at 45 international sites. The trial began in the fourth quarter of 2020. The primary endpoint was annualized height velocity. The interim analysis examined data collected at 6 months.
Interim data were presented for 41 participants, including 11 children who received 0.8 mg/kg ibutamoren per day and 10 children from the other three groups. Children receiving 1.6 mg/kg ibutamoren per day had a mean annualized height velocity of 8.57 cm per year, which was on pace with the predicted annualized height velocity of 8.3 cm per year based on historical data from prior human GH studies. Children receiving recombinant human GH had a mean annualized height velocity of 11.05 cm per year. However, Dauber noted, this growth was higher than previously reported in historical studies due to differences in baseline characteristics. The human GH group was younger, had a higher midparental height and a had a higher BMI standard deviation score than the three groups receiving ibutamoren.
Response to ibutamoren was deemed durable in follow-up data at 9 and 12 months. The group receiving 1.6 mg/kg per day ibutamoren had an annualized height velocity of 8.48 cm per year at 9 months and 8.14 cm per year at 12 months.
There were no treatment-related serious adverse events in the trial. Of all participants receiving ibutamoren, 9.3% reported any treatment-related adverse event compared with 13.3% in the human GH group.
Dauber said the full OraGrowtH210 cohort will reach the primary endpoint later in 2023 and researchers anticipate to have the full results of the trial by the end of the year.
“If those are positive, there will need to be a larger phase 3 study [that] is adequately powered to compare LUM-201 to subcutaneous GH therapy,” Dauber said.
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Dauber A, et al. Abstract 6178. Presented at: Pediatric Endocrine Society Annual Meeting; May 5-8, 2023; San Diego.
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