Once-Weekly Lonapegsomatropin Was Efficacious and Well Tolerated in Chinese Children with Growth Hormone Deficiency: Results from a Phase 3 Randomized Trial.
Lonapegsomatropin showed a statistically significant improvement in growth velocity compared to daily somatropin in children with GHD, but the clinical relevance of the difference should be considered carefully.
Where it sits
this study against the rest of the hgh (somatropin) corpusSummary and findings
This study measured the efficacy and safety of weekly lonapegsomatropin compared to daily somatropin in treatment-naive Chinese children with growth hormone deficiency (GHD). A total of 153 participants were randomized to receive either 0.24 mg hGH/kg/week of lonapegsomatropin or an equivalent weekly dose of daily somatropin. The results indicated that lonapegsomatropin was both non-inferior and superior in terms of annualized height velocity (AHV).
Abstract
<h4>Introduction</h4>Lonapegsomatropin, a prodrug of somatropin, is approved for once-weekly treatment of paediatric growth hormone deficiency (GHD) in the USA and Europe. Here, we report the first trial to assess the efficacy and safety of weekly lonapegsomatropin compared to daily somatropin in treatment-naive Chinese children with GHD.<h4>Methods</h4>The briGHt trial was a randomized, open-label, active-controlled, 52-week, phase 3 trial (NCT04326374; CTR20200399) conducted at 17 sites across China. Treatment-naive, prepubertal children with GHD were enrolled and randomized 2:1 to either lonapegsomatropin 0.24 mg hGH/kg/week or equivalent weekly dose of daily somatropin 0.034 mg/kg/day. The primary endpoint was annualized height velocity (AHV) at week 52. Secondary endpoints included change in height standard deviation score (ΔHT SDS) from baseline, insulin-like growth factor 1, and safety.<h4>Results</h4>A total of 153 participants received treatment. Least squares (LS) mean ± standard error (±SE) of AHV at week 52 was 10.66 ± 0.22 cm/year for weekly lonapegsomatropin and 9.75 ± 0.26 cm/year for daily somatropin; weekly lonapegsomatropin demonstrated non-inferiority and superiority over daily somatropin, with a difference of 0.91 ± 0.28 cm/year (95% confidence interval: 0.37-1.45; p = 0.0010). LS mean (±SE) of ΔHT SDS was 1.01 ± 0.04 for weekly lonapegsomatropin and 0.83 ± 0.05 for daily somatropin at week 52, favouring lonapegsomatropin from week 13 (p < 0.05) onward. The safety profile was similar between treatment groups.<h4>Conclusions</h4>Weekly lonapegsomatropin demonstrated non-inferiority and superiority in efficacy compared to daily somatropin among treatment-naive Chinese children with GHD. The treatment groups showed comparable safety and tolerability profiles.
Background
The paper addresses the treatment of growth hormone deficiency in children, a condition that can lead to significant growth and developmental issues. Prior studies have established the role of growth hormone therapy in promoting growth, but the specific efficacy and safety of lonapegsomatropin in a Chinese pediatric population had not been fully evaluated. This study aims to fill that gap by providing data from a phase 3 randomized trial.
Methods
Not reported in abstract.
Results
Not reported in abstract.
Interpretation
Not reported in abstract.
Key findings
- Not reported in abstract.
Limitations
- Not reported in abstract.