Safety, Tolerability and Efficacy of TransCon hGH Weekly Versus Daily hGH in Chinese Pediatric Growth Hormone Deficiency

• Status: Recruiting
• Trial end date: 2022-04-01
• Target enrollment: 0
• Participant gender: All

Summary

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).

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Visen Pharmaceuticals (Shanghai) Co., Ltd.
• Collaborator: Ascendis Pharma A/S
• Treatments: Hormones

Criteria

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.