A Study to Investigate Efficacy and Safety of Weekly PEG-somatropin (GenSci004) in Treatment Naive Children With Growth Hormone Deficiency

Study Overview
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Brief Summary
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The purpose of this study is to evaluate the efficacy and safety of weekly GenSci004 compared with daily Genotropin in treatment-naive children with growth failure due to GHD.

Detailed Description
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The purpose of this Phase 3 study is to evaluate the efficacy, safety, and tolerability of weekly GenSci004 compared to daily Genotropin over 52 weeks in prepubertal treatment-naïve children with growth failure due to GHD.

Official Title
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The Efficacy and Safety of Once-weekly PEG-somatropin (GenSci004) in Treatment-naive Children With Growth Hormone Deficiency: A Randomized, Open-label, Parallel-group, Active-Controlled, Non-inferiority Phase 3 Study (ELEVATE)

Conditions
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GHD

Intervention / Treatment
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* Drug: GenSci004
* Drug: Genotropin


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Prepubertal children with GHD in Tanner Stage 1 Baseline HT at least -2.0 SD below the mean HT for CA and sex (HT SDS ≤ 2.0). Body mass index (BMI) within ±2.0 SD of the mean BMI for BA and sex. Growth hormone stimulation tests: ≤10 ng/mL 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) Normal 46 XX karyotype for girls. Children with multiple hormonal deficiencies must be on stable replacement therapy for other hypothalamo-pituitary axes for at least 3 months Written, signed informed consent of the parent(s) or legal guardian(s) of the participant and written assent of the participant Exclusion Criteria: BA≥CA Prior exposure to rhGH, long-acting growth hormones, or IGF 1 therapy. Major medical conditions or presence of contraindication to human growth hormone (hGH) treatment Participation in any other trial of an investigational agent within 3 months prior to Screening. Any reason per investigator's discretion

Ages Eligible for Study
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Minimum Age: 3 Years
Maximum Age: 12 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
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: GenSci004<br> | Drug: GenSci004<br>* GenSci004 is a pegylated rhGH (PEG rhGH) (i.e., PEG-somatropin)<br>|
| Active Comparator: Genotropin<br> | Drug: Genotropin<br>* Genotropin<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Annualized Height Velocity (AHV) for GenSci004 and Genotropin groups | Measured in centimeter per year (cm/year) | 52 weeks | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Annualized Height Velocity (AHV) for GenSci004 and Genotropin groups | Measured in centimeter per year (cm/year) | 104 weeks | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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GHD, growth hormone deficiency