A Phase 2 Study of Pegylated Recombinant Human Growth Hormone Injection to Treat Children of Turner Syndrome
试验速览
- 阶段
- 2 期
- 入组人数
- 180
- 试验地点
- 9
- 主要终点
- Change of height standard deviation score before and after treatment (ΔHT SDS)
研究概览
简要总结
This study aims to explore the optimal dose of pegylated recombinant human growth hormone (PEG-rhGH) injection to treat children of Turner syndrome (TS), preliminarily evaluate its safety and efficacy and provide scientific and reliable evidence for the medication dosage in Phase 3 clinical trial.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 2 Years 至 18 Years（Child, Adult）
- 性别
- Female
- 接受健康志愿者
- 否
入选标准
- •Bone age <12 years
- •Karyotype: 45, X; 45, X / 46, XXqi; 45, X / 46, XXr; 45, X / 46, XX; 46, XXqi; 46, XXpi; 45, X / 47, XXX; 46, XXp-; 45, X / 46, XXp-; 46, XXq-; 45X / 46, XXq-; 45, X / 46, XX / 47, XXX, etc. (count 50 or more cells);
- •Facial appearance and abnormalities: Patients with at least one of the following signs, which include but are not limited to facial pigmented nevus, short neck, webbed neck, low posterior hairline, low-set ears, micrognathia, high-voulted arch, shield-like chest, cubitus valgus, genu valgum, short 4th and 5th metacarpals, nail dysplasia, scoliosis, ptosis and strabismus, cardiovascular abnormalities (such as aortic stenosis, bicuspid aortic valve and hypertension), reproductive abnormalities (such as primary gonadal dysfunction), renal abnormalities, thyroid hypofunction, middle ear lesion, etc.
- •Short stature: height below -2.5SD of the mean height of the same age and gender.
- •Pre-pubertal (Tanner Stage I ) patients
- •No history of growth hormone treatment
- •The subject and his/her guardian sign the informed consent (if the subject is incapable to sign the informed consent, his/her legal guardian shall sign the name of the subject instead)
排除标准
- •Subjects with abnormal liver and kidney functions (ALT > upper limit of normal value; Cr > upper limit of normal value)
- •Subjects positive for anti-HBc, HbsAg or HbeAg in Hepatitis B virus tests;
- •Subjects with highly allergic constitution or allergy to proteins or investigational product or its excipient
- •Subjects with systemic chronic disease and immune deficiency
- •Patients diagnosed with tumor
- •For patients whose tumor markers exceeding normal range in combination with other information, considering as potential high risks of tumor, they may be excluded from the treatment.
- •Patients with mental disease
- •Subjects with impaired glucose regulation (IGR) (including impaired fasting glucose (IFG) and/or impaired glucose tolerance (IGT) ) or diabetes
- •Subjects who took part in other clinical trials within 3 months
- •Subjects who received medicines which may interfere GH secretion or GH function, or other hormones within 3 months (such as sex steroids, glucocorticoids, etc.)
- •Other conditions which are unsuitable for this study in the opinion of the investigator.
研究组 & 干预措施
PEG-rhGH low dose
PEG-rhGH Injection (27IU/4.5mg/0.5ml/bottle) 0.1 mg/kg/w by subcutaneous injection for 52 weeks.
干预措施: PEG-rhGH low dose (Biological)
PEG-rhGH low dose
PEG-rhGH Injection (27IU/4.5mg/0.5ml/bottle) 0.1 mg/kg/w by subcutaneous injection for 52 weeks.
干预措施: PEG-rhGH high dose (Biological)
PEG-rhGH low dose
PEG-rhGH Injection (27IU/4.5mg/0.5ml/bottle) 0.1 mg/kg/w by subcutaneous injection for 52 weeks.
干预措施: Non-treatment control group (Other)
PEG-rhGH high dose
PEG-rhGH Injection (27IU/4.5mg/0.5ml/bottle) 0.2 mg/kg/w by subcutaneous injection for 52 weeks.
干预措施: Non-treatment control group (Other)
PEG-rhGH high dose
PEG-rhGH Injection (27IU/4.5mg/0.5ml/bottle) 0.2 mg/kg/w by subcutaneous injection for 52 weeks.
干预措施: PEG-rhGH low dose (Biological)
PEG-rhGH high dose
PEG-rhGH Injection (27IU/4.5mg/0.5ml/bottle) 0.2 mg/kg/w by subcutaneous injection for 52 weeks.
干预措施: PEG-rhGH high dose (Biological)
Non-treatment control group
结局指标
主要结局
Change of height standard deviation score before and after treatment (ΔHT SDS)
时间窗: 52 weeks
The change of height standard deviation score of chronological age before and after treatment
次要结局
- ΔBA/ΔCA(52 weeks)
- Height Velocity(52 weeks)
- IGF-1(Insulin-like growth factor 1) SDS(52 weeks)