Somapacitan Is Effective and Well Tolerated in Chinese Children with Growth Hormone Deficiency: A Randomized...

Area of Science:

• Pediatric Endocrinology
• Pharmacology
• Clinical Trials

Background:

• REAL6 trial investigated somapacitan, a novel GH derivative, in Chinese children with GHD.
• Somapacitan offers once-weekly administration, potentially improving treatment adherence.
• Growth Hormone Deficiency (GHD) impacts linear growth and metabolic function in children.

Purpose of the Study:

• To evaluate the efficacy and safety of once-weekly somapacitan versus daily GH in Chinese children with GHD.
• To compare treatment burden between somapacitan and daily GH.
• To assess growth parameters and biochemical markers in GHD patients.

Main Methods:

• Phase 3, randomized, open-label, active-controlled trial (NCT04970654) involving 110 treatment-naïve Chinese children.
• Patients were randomized 2:1 to receive somapacitan (0.16 mg/kg/week) or daily GH (0.034 mg/kg/day) for 52 weeks.
• Primary endpoint: annualized height velocity (HV) at week 52. Secondary endpoints included HV standard deviation score (SDS), height SDS, bone age, and IGF-I SDS.

Main Results:

• Somapacitan achieved non-inferiority to daily GH, with mean HV of 11.0 cm/year vs. 10.4 cm/year, respectively.
• Similar improvements in HVSDS, height SDS, bone age, and IGF-I SDS were observed in both groups.
• Somapacitan exhibited a favorable safety profile, comparable to daily GH, with minimal injection site reactions and no reported pain.

Conclusions:

• Once-weekly somapacitan is a safe and effective treatment for Chinese children with GHD, demonstrating comparable efficacy to daily GH.
• Somapacitan significantly reduced treatment burden compared to daily GH, potentially improving patient and caregiver quality of life.
• Both treatments effectively normalized IGF-I levels and reduced disease burden.