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

Phase 3

• Conditions: Pituitary Diseases; Growth Hormone Deficiency; Endocrine System Diseases; Hormones; Pituitary Disease, Anterior
• Interventions: Drug: TransCon hGH; Drug: daily hGH

• Registration Number: NCT04326374
• Lead Sponsor: Visen Pharmaceuticals (Shanghai) Co., Ltd.

Brief 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).

Detailed Description

Not Provided

Study Timeline

• Study Start: 2019-12-30
• Status Verified: 2020-03
• Study First Submitted: 2020-03-26
• Study First Submitted QC: 2020-03-27
• Last Update Submitted: 2020-03-27
• Study First Posted: 2020-03-30
• Last Update Posted: 2020-03-30
• Primary Completion: 2022-04
• Study Completion: 2022-04

Recruitment & Eligibility

• Status: UNKNOWN
• Sex: All
• Target Recruitment: 150

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
TransCon hGH	TransCon hGH	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.
Daily hGH	daily hGH	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.

Primary Outcome Measures

Name	Time	Method
Annualized height velocity(AHV) at Week 52	52 Weeks	Annualized height velocity at Week 52 for weekly TransCon hGH treatment and the daily hGH treatment groups

Secondary Outcome Measures

Name	Time	Method
AHV and the change from baseline over 52 weeks at each visit	52 Weeks	AHV and the change from baseline for the TransCon hGH and the daily hGH treatment group over 52 weeks at each visit
Height SDS and the change from baseline over 52 weeks	52 weeks	Height SDS and the change from baseline for the TransCon hGH and the daily hGH treatment group over 52 weeks
The plasma concentration	52 weeks	pharmacokinetics measure of hGH over 52 weeks in TransCon hGH group
Serum IGF-1 and IGFBP-3 levels, and IGF-1 SDS, IGFBP-3 SDS, and the change from baseline over 52 weeks at each visit	52 weeks	Serum IGF-1 and IGFBP-3 levels, and IGF-1 SDS, IGFBP-3 SDS, and the change from baseline for the TransCon hGH and the daily hGH treatment group over 52 weeks at each visit
The percentage of participants achieving IGF-1 SDS 0 to +2.0	52 weeks	The normalization of IGF-1 SDS (percentage of participants achieving IGF-1 SDS 0 to +2.0 over 52 weeks for the TransCon hGH and the daily hGH treatment group
Incidence of Treatment-Emergent Adverse Events	52 weeks

Trial Locations

Locations (1)

Department of Pediatrics, Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology,; 🇨🇳 Wuhan, Hubei, China