Prader-Willi Syndrome Clinical Trial
Official title:
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
To evaluate the effectiveness of rhGH (Recombinant human growth hormone) injection for improving motor development in patients with PWS.
Status | Recruiting |
Enrollment | 30 |
Est. completion date | January 1, 2020 |
Est. primary completion date | January 2020 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 1 Month to 5 Years |
Eligibility |
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 |
Country | Name | City | State |
---|---|---|---|
China | Beijing Children's Hospital, Capital Medical University | Beijing | |
China | Peking union medical college hospital | Beijing | |
China | Peking university first hospital | Beijing | |
China | The Children's Hospital,Zhejiang University School of Medicine | Hangzhou | Zhejiang |
China | Children's Hospital of Fudan University | Shanghai | |
China | Tongji medical college huazhong university of science & technology | Wuhan | Hubei |
Lead Sponsor | Collaborator |
---|---|
GeneScience Pharmaceuticals Co., Ltd. | Beijing Children's Hospital, Children's Hospital of Fudan University, gwcmc, Peking University First Hospital, The Children's Hospital, Zhejiang University School Of Medicine, Tongji medical college huazhong university of science & technology |
China,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | The change of the total motor quotient calculated by Peabody Developmental Motor Scale before and after treatment | Baseline, 26 weeks, 52 weeks | ||
Secondary | Gross motor quotient and fine motor quotient calculated by Peabody Developmental Motor Scale before and after treatment | Baseline, 26 weeks, 52 weeks | ||
Secondary | 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 | ||
Secondary | Body weight change before and after treatment | Baseline, 4 weeks, 13 weeks, 26 weeks, 39 weeks, 52 weeks | ||
Secondary | Change of BMI (Body mass index) standard deviation score before and after treatment | Baseline, 4 weeks, 13 weeks, 26 weeks, 39 weeks, 52 weeks | ||
Secondary | 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 | ||
Secondary | Bone maturation ( bone age/ chronological age: BA/CA) | Baseline, 52 weeks | ||
Secondary | Change of IGF-1(Insulin-like growth factor 1) SDS | Baseline, 4 weeks, 13 weeks, 26 weeks, 39 weeks, 52 weeks | ||
Secondary | IGF-1/IGFBP-3 molar ratio | Baseline, 4 weeks, 13 weeks, 26 weeks, 39 weeks, 52 weeks |
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