A Study to Evaluate the Efficacy and Safety of Recombinant Human Growth Hormone Injection in Patients With Prader-Willi Syndrome

Study Overview
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Brief Summary
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To evaluate the effectiveness of rhGH (Recombinant human growth hormone) injection for improving motor development in patients with PWS.

Official Title
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A Single Arm, Multicenter Phase III Clinical Trial to Evaluate the Efficacy and Safety of Recombinant Human Growth Hormone Injection in Patients With Prader-Willi Syndrome

Conditions
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Prader-Willi Syndrome

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


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: 1.Signed informed consent from legal guardian of the subjects; Subjects are willing and able to cooperate to complete scheduled visits, treatment plans and laboratory tests and other procedures; Diagnosed as PWS by gene test; Age: 1 month (30 days after birth) - 5 years of age; Male or female; Calculated by Peabody Developmental Motor Scale, total motor quotient, gross motor quotient or fine motor quotient is less than 90 points; Thyroid function is within the normal reference range or remained within the normal reference range by substitution therapy; No history of rhGH therapy before. Exclusion Criteria: Subjects with abnormal liver or kidney function; Subjects with obvious central sleep apnea and/or moderate or severe obstructive sleep apnea, acute lung infection; Subjects with chronic diseases that have long-term effects on bone metabolism and body composition; Subjects with congenital skeletal dysplasia, or spine scoliosis with moderate and above degree requiring treatment or lameness; Subjects with history of congenital heart disease, or an echocardiogram showing that the structural abnormalities require surgery or interventional therapy or that the left ventricular ejection fraction is <40%, or the abnormal electrocardiogram requiring intervention; Subjects with history of convulsions or epilepsy; Subjects with other systemic chronic diseases; Subjects with diagnosed tumors; Subjects with family history of cancers, a previous history of cancer, or considered to be a high risk of cancer combinating other information; Subjects with mental disease; Subjects with diabetes, or abnormal fasting glucose and researchers believe that may affect the safety of the subject; Subjects with severe obesity; Subjects with highly allergic constitution or allergy to proteins or investigational product or its excipient; Subjects who took part in other clinical trials within 3 months ; Subjects who received drug treatment that may interfere with GH secretion or GH action within 3 months; Other conditions in which the investigator preclude enrollment into the study

Ages Eligible for Study
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Minimum Age: 1 Month
Maximum Age: 5 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
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: rhGH injection/Jintropin AQ<br>Drug: Recombinant Human Growth Hormone Injection /Jintropin AQ, 30IU/10 mg/3ml/kit, 0.5 mg/m2/d for the first 4 weeks, then 1.0 mg/m2/d for subsequent 48 weeks; by subcutaneous injection, once per day for total 52 weeks.No control. | Drug: Recombinant Human Growth Hormone (rhGH) Injection<br>* Drug: Recombinant Human Growth Hormone Injection /Jintropin AQ, 30IU/10 mg/3ml/kit, 0.5 mg/m2/d for the first 4 weeks, then 1.0 mg/m2/d for subsequent 48 weeks; by subcutaneous injection, once per day for total 52 weeks.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| The change of the total motor quotient calculated by Peabody Developmental Motor Scale before and after treatment |  | Baseline, 26 weeks, 52 weeks | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Gross motor quotient and fine motor quotient calculated by Peabody Developmental Motor Scale before and after treatment |  | Baseline, 26 weeks, 52 weeks | 
| Change of height standard deviation score (SDS) by chronological age before and after treatment |  | Baseline, 4 weeks, 13 weeks, 26 weeks, 39 weeks, 52 weeks | 
| Body weight change before and after treatment |  | Baseline, 4 weeks, 13 weeks, 26 weeks, 39 weeks, 52 weeks | 
| Change of BMI (Body mass index) standard deviation score before and after treatment |  | Baseline, 4 weeks, 13 weeks, 26 weeks, 39 weeks, 52 weeks | 
| Global quotient, locomotor quotient, personal-social development quotient, language quotient, eye and hand coordination quotient, performance quotient and practical reasoning change calculated by Griffiths Mental Development Scale |  | Baseline, 26 weeks, 52 weeks | 
| Bone maturation ( bone age/ chronological age: BA/CA) |  | Baseline, 52 weeks | 
| Change of IGF-1(Insulin-like growth factor 1) SDS |  | Baseline, 4 weeks, 13 weeks, 26 weeks, 39 weeks, 52 weeks | 
| IGF-1/IGFBP-3 molar ratio |  | Baseline, 4 weeks, 13 weeks, 26 weeks, 39 weeks, 52 weeks |