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Once-Weekly Lonapegsomatropin Was Efficacious and Well Tolerated in Chinese Children with Growth Hormone Deficiency:
Yanqin Ying1, Haiyan Wei2, Yan Zhong3
1Department of Pediatrics, Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology, Wuhan, China.
Insights
Weekly lonapegsomatropin showed superior efficacy compared to daily somatropin in treating Chinese children with growth hormone deficiency (GHD). Both treatments were found to be safe and well-tolerated, offering a new therapeutic option.
Area of Science:
- Pediatric Endocrinology
- Pharmacology
- Clinical Trials
Background:
- Lonapegsomatropin is an approved once-weekly treatment for pediatric growth hormone deficiency (GHD).
- This study is the first to compare weekly lonapegsomatropin with daily somatropin in treatment-naive Chinese children with GHD.
Purpose of the Study:
- To assess the efficacy and safety of weekly lonapegsomatropin versus daily somatropin in Chinese children with GHD.
- To evaluate lonapegsomatropin's non-inferiority and superiority in improving growth parameters.
Main Methods:
- A 52-week, randomized, open-label, active-controlled Phase 3 trial (briGHt trial) in China.
- 153 treatment-naive, prepubertal children with GHD were randomized to weekly lonapegsomatropin or daily somatropin.
- Primary endpoint: annualized height velocity (AHV) at week 52; Secondary endpoints: change in height SDS, IGF-1, and safety.
Main Results:
- Weekly lonapegsomatropin demonstrated non-inferiority and superiority over daily somatropin, with a significantly higher AHV (10.66 cm/year vs. 9.75 cm/year, p=0.0010).
- Lonapegsomatropin also showed a greater improvement in height SDS from week 13 onwards (p < 0.05).
- Safety profiles were comparable between the two treatment groups.
Conclusions:
- Weekly lonapegsomatropin is non-inferior and superior in efficacy to daily somatropin for treating naive Chinese children with GHD.
- Lonapegsomatropin offers a comparable safety and tolerability profile to daily somatropin.
- Weekly lonapegsomatropin presents a promising alternative treatment for pediatric GHD.
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.
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