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

• Status: Unknown status
• Trial end date: 2020-01-01
• Target enrollment: 0
• Participant gender: All

Summary

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

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: GeneScience Pharmaceuticals Co., Ltd.
• Collaborators: Beijing Children's Hospital; Children's Hospital of Fudan University; Peking Union Medical College Hospital; Peking University First Hospital; The Children's Hospital of Zhejiang University School of Medicine; Tongji medical college huazhong university of science & technology
• Treatments: Hormones

Criteria

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