Peer-reviewed article
Once-Weekly Lonapegsomatropin Was Efficacious and Well Tolerated in Chinese Children with Growth Hormone Deficiency: Results from a Phase 3 Randomized Trial.
Ying Y, Wei H, Zhong Y, Li Y, Yang Y et al. ·
Summary
Written by AI, not yet AICS's view · claude-haiku-4-5-20251001This study tested a new once-weekly growth hormone treatment (lonapegsomatropin) against the standard daily treatment (somatropin) in Chinese children with growth hormone deficiency. Children who received the weekly injection grew taller and faster than those on daily injections over the 52-week study. Both treatments were safe and well tolerated, with no serious safety concerns reported.
Key points
- Weekly lonapegsomatropin produced greater height gain (10.66 cm/year) compared to daily somatropin (9.75 cm/year)
- The difference of 0.91 cm/year represented both non-inferiority and statistical superiority
- Height standard deviation score improvement favored lonapegsomatropin from week 13 onwards
- Safety profiles were similar between treatment groups
- Study included 153 treatment-naive prepubertal children across 17 sites in China
Limitations
The study was open-label rather than blinded, which could introduce bias in assessment. The 52-week timeframe provides only short-term efficacy and safety data; longer-term outcomes and effects into adulthood are unknown.
Abstract
Introduction 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. Methods 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. Results 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. Conclusions 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.