Once-Weekly Administration of Sustained-Release Growth Hormone in Korean Prepubertal Children with Idiopathic Short...

Area of Science:

• Pediatric Endocrinology
• Pharmacology
• Clinical Trial Research

Background:

• Idiopathic short stature (ISS) affects children's growth and requires effective treatment.
• Daily recombinant human growth hormone (rhGH) is a standard therapy for ISS.
• Developing less frequent rhGH formulations can improve patient adherence and quality of life.

Purpose of the Study:

• To determine the optimal dose of LB03002, a novel sustained-release, once-weekly rhGH formulation.
• To compare the efficacy and safety of LB03002 with daily rhGH in children with ISS.
• To evaluate height velocity (HV) and height standard deviation score (SDS) changes.

Main Methods:

• A multicenter, randomized, open-label, phase II study.
• GH-naïve, prepubertal children with ISS were randomized into three groups.
• Groups received daily rhGH 0.37 mg/kg/week (control), LB03002 0.5 mg/kg/week, or LB03002 0.7 mg/kg/week.

Main Results:

• At 26 weeks, the least square means for HV change were 5.08 cm/year (control), 3.65 cm/year (LB03002 0.5 mg/kg/week), and 4.38 cm/year (LB03002 0.7 mg/kg/week).
• The LB03002 0.7 mg/kg/week dose demonstrated noninferiority to the daily rhGH control, with a lower bound of the 90% confidence interval for HV change difference at -1.72, meeting the noninferiority margin of -1.75.
• Changes in height SDS were 0.65 (control), 0.49 (LB03002 0.5 mg/kg/week), and 0.58 (LB03002 0.7 mg/kg/week).

Conclusions:

• Once-weekly LB03002 at 0.7 mg/kg/week is noninferior to daily rhGH (0.37 mg/kg/week) for increasing height velocity in children with ISS.
• LB03002 was well-tolerated, with a safety profile comparable to daily rhGH.
• The 0.7 mg/kg/week dose of LB03002 represents a potentially effective alternative for ISS treatment.