NCT03554031Unknown3 期
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
适应症
干预措施
相关药物
试验速览
- 阶段
- 3 期
- 入组人数
- 30
- 试验地点
- 6
- 主要终点
- The change of the total motor quotient calculated by Peabody Developmental Motor Scale before and after treatment
研究概览
简要总结
To evaluate the effectiveness of rhGH (Recombinant human growth hormone) injection for improving motor development in patients with PWS.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 1 Month 至 5 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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.
排除标准
- •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
研究组 & 干预措施
rhGH injection/Jintropin AQ
Experimental
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.
干预措施: Recombinant Human Growth Hormone (rhGH) Injection (Drug)
结局指标
主要结局
The change of the total motor quotient calculated by Peabody Developmental Motor Scale before and after treatment
时间窗: Baseline, 26 weeks, 52 weeks
次要结局
- 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)
- 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)
研究者
研究点 (6)
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