A Single-Arm, Phase III Study to Assess Efficacy and Safety after 6-Month-Treatment of Eutropin(TM) Inj. (Recombinant Human Growth Hormone) in Prepubertal Children with Short Stature due to Small for Gestational Age / 대한소아내분비학회지

ABSTRACT

PURPOSE: Recombinant human growth hormone is an effective therapy for short-statured children born small for their gestational age (SGA). This single-arm, multicenter, phase III clinical study of such children was designed to assess the efficacy and safety of treating them with recombinant human-growth-hormone (Eutropin(TM) Inj.) for 6 months. METHODS: Between 2005 and 2007, 30 treatment naive, prepubertal, short-statured SGA-born children were recruited as participants. Eutropin(TM) Inj. was administered for 6 months with a subcutaneous dose of 0.48 mg/kg/wk. The primary endpoint was the change in height velocity from the baseline to month 6. Various parameters were checked to obtain secondary outcome measures and to meet safety criteria. RESULTS: Height velocity significantly increased from 5.36 +/- 1.59 cm/yr at baseline to 10.66 +/- 2.03 cm/yr at month 6 (P < 0.0001). Secondary outcome measures (height velocity at month 3, height SDS for chronological age (CA), weight SDS for CA, bone maturation, and IGF-I and IGFBP-3 levels) were also significantly increased. Eutropin(TM) Inj. was well tolerated and safe over 6 months of treatment. CONCLUSION: The clinical efficacy and safety of Eutropin(TM) Inj. was demonstrated for the 6 month treatment of prepubertal children with short stature due to SGA. Further long-term study is needed.