A Study to Evaluate the Efficacy and Safety of Recombinant Human Growth Hormone in Short Stature Children Due to Chronic Kidney Disease Before Transplantation

Study Overview
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Brief Summary
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This study aims to evaluate the efficacy and safety of recombinant human growth hormone injection in short stature children due to chronic kidney disease before transplantation.

Official Title
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A Phase 3 Study to Evaluate the Efficacy and Safety of Recombinant Human Growth Hormone in Short Stature Children Due to Chronic Kidney Disease Before Transplantation

Conditions
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Dwarfism

Intervention / Treatment
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* Drug: Recombinant Human Growth Hormone Injection (rhGH)


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Signed informed consent of the subjects or the legal guardian. Subjects and guardian are willing and able to cooperate to complete scheduled visits,treatment plans and laboratory tests and other procedures. Diagnosed as chronic kidney disease. Glomerular filtration rate (GFR) <75ml/per min/1.73m2. After the treatment and adjustment of hypertension, anemia, metabolic acidosis, malnutrition, renal osteopathy, hypothyroidism, etc.. Chronological age: ≥2years and ≤14years. Height Standard Deviation Score(HtSDS) ≤-2SD for chronological age. Bone age: girls≤10 years; boys≤11years. Pre-pubertal (Tanner Stage I ) patients. No history of growth hormone treatment. Exclusion Criteria: Subjects with abnormal liver functions. Subjects positive for anti-HBc, HbsAg or HbeAg in Hepatitis B virus tests. After adjustment heart function,Cardiac ejection fraction(EF) <50%. Subjects with highly allergic constitution or allergy to proteins or investigational product or its excipient. Subjects with systemic chronic disease or general infection or mental disease. Subjects with diabetes or impaired fasting glucose. Subjects with tumor or potential tumor. Subjects who are using glucocorticoid or immunosuppressant. Other causes for growth retardation. Inability to obtain accurate height measurements. Subjects who took part in other clinical trials within 3 months. Concomitant administration of other treatment that may have an effect on growth within 3 months. Other conditions which are unsuitable for this study in the opinion of the investigator.

Ages Eligible for Study
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Minimum Age: 2 Years
Maximum Age: 14 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: rhGH Injection<br>rhGH 0.05mg/kg/d by subcutaneous injection | Drug: Recombinant Human Growth Hormone Injection (rhGH)<br>* rhGH 0.05mg/kg/d by subcutaneous injection<br>|
| No Intervention: Non-treatment control group<br>Only follow-up without treatment | |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| The change of height standard deviation score of chronological age before and after treatment (ΔHT SDS) |  | 52 weeks，104 weeks | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Growth velocity | 365.25*（height at the time of assessment -height at baseline）/duration of treatment(days) | 12 months, 24 months | 
| Bone maturation ( bone age/ chronological age: BA/CA) |  | 12 months, 24 months | 
| IGF-1 (Insulin-like growth factor 1) SDS |  | 12 months, 24 months | 
| IGF-1/IGFBP-3 molar ratio |  | 12 months, 24 months |