A Phase 2 Study of PEGylated Recombinant Human Growth Hormone for 52 Weeks in Short Children Born Small for Gestational Age in China
Primary result · Change in HT-SDSResult — go to the resultLeast-squares means difference 0.410 95% CI 0.270–0.551Authors' conclusion
PEG-rhGH at either dose for 52 weeks was effective and well tolerated in children born SGA. Patients in the high-dose group achieved greater improvement in HT-SDS than in the low-dose group.
Study context
- Population
- Short children born small for gestational age (SGA), China
- Participants
- 96 children
- Comparison
- PEGylated-recombinant human growth hormone (PEG-rhGH) 0.2 mg/kg/week vs 0.1 mg/kg/week
- Primary endpoint
- Change in height SDS (height standard deviation score (HT-SDS))
- Follow-up
- Week 52
Methodological detailsmulticentre, open-label, phase 2, nine clinical sites in China
- Multicentre
- Open-label
- Phase 2
- Nine clinical sites in China
Primary result
Change in HT-SDS
Least-squares means difference
0.410
95% CI 0.270–0.551
P < 0.0001
Change by group
High dose
0.923
± 0.352
Low dose
0.511
± 0.336
High dosevs Low dose
least-squares means difference 0.410 (95% CI 0.270–0.551); P < 0.0001
As reported in the abstract
Further findings
Height velocity · Week 52 · significantly higher with high dose
P < 0.0001
| Outcome | High dose | Low dose | P value |
|---|---|---|---|
| Change in IGF-1 SDS · Week 52 | 1.867 ± 1.747 | 1.168 ± 1.193 | P = 0.0189 |
IGF-1/IGFBP-3 and bone maturity · Week 52
- IGF-1/IGF binding protein-3 and bone maturity improved in both groups
Safety
Treatment-emergent adverse events
- Most TEAEs mild to moderate; safety profile similar in both groups
Original publication
A Phase 2 Study of PEGylated Recombinant Human Growth Hormone for 52 Weeks in Short Children Born Small for Gestational Age in China. Clin Endocrinol (Oxf). Published 8 November 2024. doi:10.
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