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

Study Overview
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Brief Summary
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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
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Not Provided

Official Title
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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

Conditions
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Growth Hormone Deficiency, Endocrine System Diseases, Hormones, Pituitary Diseases, Pituitary Disease, Anterior

Intervention / Treatment
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* Drug: TransCon hGH
* Drug: daily hGH


Participation Criteria
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Eligibility Criteria
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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.

Ages Eligible for Study
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Minimum Age: 3 Years
Maximum Age: 17 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Interventional Model Description: Eligible subjects will be randomized to 1 of the 2 treatment groups: either weekly TransCon hGH or daily hGH, in a 2:1 ratio
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: TransCon hGH<br>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. | Drug: TransCon hGH<br>* Once weekly subcutaneous injection<br>* Other names: ACP-011;|
| Active Comparator: Daily hGH<br>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. | Drug: daily hGH<br>* Once daily subcutaneous injection<br>* Other names: somatropin (rDNA orgin) for injection;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Annualized height velocity(AHV) at Week 52 | Annualized height velocity at Week 52 for weekly TransCon hGH treatment and the daily hGH treatment groups | 52 Weeks | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| AHV and the change from baseline over 52 weeks at each visit | AHV and the change from baseline for the TransCon hGH and the daily hGH treatment group over 52 weeks at each visit | 52 Weeks | 
| Height SDS and the change from baseline over 52 weeks | Height SDS and the change from baseline for the TransCon hGH and the daily hGH treatment group over 52 weeks | 52 weeks | 
| 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 | 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 | 52 weeks | 
| The percentage of participants achieving IGF-1 SDS 0 to +2.0 | 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 | 52 weeks | 
| Incidence of Treatment-Emergent Adverse Events |  | 52 weeks | 
| The plasma concentration | pharmacokinetics measure of hGH over 52 weeks in TransCon hGH group | 52 weeks | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Growth Hormone Deficiency, TransCon hGH, human growth hormone, hGH