Safety, Tolerability and Efficacy of TransCon hGH Weekly Versus Daily hGH in Chinese Pediatric Growth Hormone Deficiency
The Efficacy, Safety and Tolerability of TransCon hGH Administered Weekly Versus Daily hGH in Prepubertal Children With Growth Hormone Deficiency: a Randomized, Open-lable, Active-controlled, Parallel-group Study in China
Lead SponsorVisen Pharmaceuticals
Intervention/TreatmentTransCon hGH daily hGH
This study is conducted in China only. The purpose is to demonstrate the efficacy and safety of once weekly dosing of TransCon hGH, a long-acting growth hormone product, compare to once-daily dosing of human growth hormone (hGH) after 52 weeks of treatment in prepubertal children with growth hormone deficiency (GHD).
Once weekly subcutaneous injection
Once daily subcutaneous injection
Daily hGH will be self-administered or injected by parents once daily. The dose will be adjusted based on subject's weight. The treatment will continue 52 weeks.
TransCon hGH will be self-administered or injected by parents once weekly. The dose will be adjusted based on subject's weight. The treatment will continue 52 weeks.
Inclusion Criteria: Prepubertal children with GHD in Tanner stage 1, aged of 3 years and below 17 years; Impaired HT defined as at least 2.0 standard deviations (SD) below the mean height for chronological age and sex (HT SDS≤-2.0) according to the Chinese 2005 standard； Diagnosis of GHD confirmed by 2 different GH stimulation tests, defined as a peak GH level of ≤10 ng/mL, determined with a validated assay. Bone age (BA) at least 6 months less than the chronological age； Baseline IGF-1 level of at least 1.0 SD below the mean IGF-1 level standardized for age and sex (IGF-1 SDS ≤-1.0)； Written, signed informed consent of the parent(s) or legal guardian(s) of the subject and written assent of the subject (if the subject is 8 years old or above). Exclusion Criteria: Children with a body weight below 12 kg； Prior exposure to recombinant hGH or IGF-1 therapy； Children with past or present intracranial tumor growth as confirmed by a sellar MRI scan (with contrast dye recommended) at Screening Children born SGA (birth weight ≤10th percentile for gestational age according to the Chinese reference)； Children with psychosocial dwarfism； Children with idiopathic short stature； Other causes of short stature such as coeliac disease, hypothyroidism, or rickets； History or presence of malignant disease; any evidence of present tumor growth; Subjects with diabetes mellitus； Closed epiphyses； Major medical conditions and/or presence of contraindication to hGH treatment； Participation in any other trial of an investigational agent within 3 months prior to Screening.