A Study to Evaluate the Efficacy and Safety of Recombinant Human Growth Hormone Injection in Patients With Prader-Willi Syndrome
Phase 3
- Conditions
- Prader-Willi Syndrome
- Interventions
- Drug: Recombinant Human Growth Hormone (rhGH) Injection
- Registration Number
- NCT03554031
- Lead Sponsor
- Changchun GeneScience Pharmaceutical Co., Ltd.
- Brief Summary
To evaluate the effectiveness of rhGH (Recombinant human growth hormone) injection for improving motor development in patients with PWS.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- UNKNOWN
- Sex
- All
- Target Recruitment
- 30
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
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description rhGH injection/Jintropin AQ Recombinant Human Growth Hormone (rhGH) Injection 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.
- Primary Outcome Measures
Name Time Method 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
Name Time Method 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 Bone maturation ( bone age/ chronological age: BA/CA) Baseline, 52 weeks Gross motor quotient and fine motor quotient calculated by Peabody Developmental Motor Scale before and after treatment Baseline, 26 weeks, 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 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
Trial Locations
- Locations (6)
Peking union medical college hospital
🇨🇳Beijing, China
Beijing Children's Hospital, Capital Medical University
🇨🇳Beijing, China
Peking university first hospital
🇨🇳Beijing, China
Children's Hospital of Fudan University
🇨🇳Shanghai, China
Tongji medical college huazhong university of science & technology
🇨🇳Wuhan, Hubei, China
The Children's Hospital,Zhejiang University School of Medicine
🇨🇳Hangzhou, Zhejiang, China