Prader-Willi Syndrome
Conditions
Brief summary
To evaluate the effectiveness of rhGH (Recombinant human growth hormone) injection for improving motor development in patients with PWS.
Interventions
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.
Sponsors
Children's Hospital of Fudan University
Tongji medical college huazhong university of science & technology
Beijing Children's Hospital
Peking University First Hospital
Peking Union Medical College Hospital
The Children's Hospital of Zhejiang University School of Medicine
Changchun GeneScience Pharmaceutical Co., Ltd.
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
1 Months to 5 Years
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | 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
| Measure | Time frame |
|---|---|
| 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 |
| Gross motor quotient and fine motor quotient calculated by Peabody Developmental Motor Scale before and after treatment | 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 |
| 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 |
Countries
China
Contacts
Primary ContactXiaohua Feng
Outcome results
None listed