Clinical Study of Pegylated Somatropin (PEG Somatropin) to Treat SGA Children With Short Stature

Study Overview
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Brief Summary
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The study was evaluated as the optimal dose for the treatment of SGA short stature children by Pegylated Somatropin, initially evaluated its efficiency and safety for the treatment of SGA short stature children and provided scientific, reliable basis for phase III clinical trials for dose selection.

Detailed Description
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This was a multicenter, randomized phase II study, half of participants will receive the high dose, while the other half will receive the low dose.

Conditions
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Small for Gestational Age Infant

Intervention / Treatment
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* Biological: PEG-somatropin


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: The patients are diagnosed as being clinically full term small for gestational age infant. Girl are 3-6 years old, boys are 3-7 years old. Be in preadolescence (Tanner stage 1). The child did not achieve catch-up growth when he/she entered the group (the definition of catch-up growth is that the height is higher the third percentile with the same age and gender) (Appendix 2 and 3). The height of child is shorter than -2SDS of the median of normal children with the same age and gender when he/she entered the group (the mean height and height standard deviation of normal children with the same age and gender of normal children regard the height data in the physical development investigation data of children aged 0-18 in 9 cities of China (2005) as standard [13], Appendix 4 and 5). Within a year before entering the group, after any growth hormone stimulation test, the peak concentration of growth hormone in serum>10 µg/L. Bone age≤ the actual age+1. The function of glucose regulation is normal: fasting blood glucose < 5.6mmol/L. Birth gestational age ≥ 37 weeks. The subjects and their guardians sign the informed consent (if the subjects is lack of ability for signing the informed consent, his legal guardian can write the subjects name instead). Exclusion Criteria: People with abnormal liver or kidney function (ALT> 2 times the upper limit of normal value, Cr> the upper limit of normal value). Patients are positive for hepatitis B core antigen (HBc), hepatitis B surface antigen (HBsAg) or hepatitis B e antigen (HBeAg). People with known highly allergic constitution or allergy to the drug or the excipient of the study. People with diabetes, severe cardiopulmonary, hematological system and malignant tumors diseases or general infection, immune deficiency and patients with mental disease. Other abnormal growth and development, such as Turner syndrome, Laron syndrome, growth hormone receptor deficiency. Potential tumor patients (family history). Patients who used growth hormone for treatment. Subjects took part in other clinical trial study within 3 months. Patients used other hormonal for treatment within 3 months (such as sex hormone, glucocorticoid and etc., treat for more than a month) and received the drug treatment which may interfere with the secretion of GH or GH function (oxandrolone, growth hormone releasing hormone and etc.); Other conditions which in the opinion of the investigator preclude enrollment into the study.

Ages Eligible for Study
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Minimum Age: 3 Years
Maximum Age: 7 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: Double


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: PEG-somatropin: Low dose<br>0.1 mg/(kg.w), once per week for 52 weeks. | Biological: PEG-somatropin<br> <br> |
| Experimental: PEG-somatropin: High dose<br>0.2 mg/(kg.w), once per week for 52 weeks. | Biological: PEG-somatropin<br> <br> |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Actual height of the patient after treatment compared with the mean height of the population and the standard deviation (SD) of the height of the population for that chronological age. |  | 52 weeks | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Height |  | 52 weeks | 
| Bone maturity (bone age change / actual age change: △BA/△CA) |  | 52 weeks | 
| The mole ratio of IGF-1/IGFBP-3: （[IGF-1(ng/ml)/7.6]/[IGFBP-3 (ng/ml)/25.75]） |  | 52 weeks |