Long-acting PEGylated recombinant human growth hormone (Jintrolong) for children with growth hormone deficiency: phase II and phase III multicenter, randomized studies

Xiaoping Luo, Ling Hou, Li Liang, Guanping Dong, Shuixian Shen, Zhuhui Zhao, Chun Xiu Gong, Yuchuan Li, Min-Lian Du, Zhe Su, Hongwei Du, Chaoying Yan, Xiaoping Luo, Ling Hou, Li Liang, Guanping Dong, Shuixian Shen, Zhuhui Zhao, Chun Xiu Gong, Yuchuan Li, Min-Lian Du, Zhe Su, Hongwei Du, Chaoying Yan

Abstract

Objective: We assessed the efficacy and safety of a weekly pegylated human growth hormone (PEG-rhGH) (Jintrolong) vs daily rhGH for children with growth hormone deficiency (GHD).

Design: Phase II and III, multicenter, open-label, randomized controlled trials.

Methods: 108 and 343 children with treatment-naive GHD from 6 hospitals in China were enrolled in the phase II and III studies respectively. Patients in the phase II study were randomized 1:1:1 to weekly Jintrolong (0.1 mg/kg/week PEG-rhGH complex), weekly Jintrolong (0.2 mg/kg/week PEG-rhGH complex) or daily rhGH (0.25 mg/kg/week) for 25 weeks. Patients in the phase III study were randomized in a 2:1 ratio to weekly Jintrolong (0.2 mg/kg/week) or daily rhGH (0.25 mg/kg/week) for 25 weeks. The primary endpoint for both studies was height velocity (HV) increase at the end of treatment. Other growth-related parameters, safety and compliance were also monitored.

Results: The phase II study established the preliminary efficacy, safety and recommended dose of Jintrolong PEG-rhGH. In the phase III study, we demonstrated significantly greater HV increases in patients receiving Jintrolong treatment (from 2.26 ± 0.87 cm/year to 13.41 ± 3.72 cm/year) vs daily rhGH (from 2.25 ± 0.82 cm/year to 12.55 ± 2.99 cm/year) at the end of treatment (P < 0.05). Additionally, significantly greater improvement in the height standard deviation scores was associated with Jintrolong throughout the treatment (P < 0.05). Adverse event rates and treatment compliance were comparable between the two groups.

Conclusion: Jintrolong PEG-rhGH at a dose of 0.2 mg/kg/week for 25 weeks is effective and safe for GHD treatment and is non-inferior to daily rhGH.

© 2017 The authors.

Figures

Figure 1
Figure 1
HTSDS for the PEG-rhGH and daily rhGH groups at baseline, weeks 4, 13 and 25 of the phase III study. The dotted line represents the lower reference limit.
Figure 2
Figure 2
Mean IGF1 standard deviation score (SDS) for the PEG-rhGH and daily rhGH groups at baseline, weeks 4, 13 and 25 of the phase III study. The dotted lines represent the lower and upper reference limits.

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Source: PubMed

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