Pegylated Somatropin (PEG Somatropin) in the Treatment of Children With Idiopathic Short Stature: A Controlled, Prospective, Randomized, Multicenter Phase-II Study With An Untreated Control Group.
试验速览
- 阶段
- 2 期
- 入组人数
- 360
- 试验地点
- 7
- 主要终点
- Change in Height Standard Deviation Score for Chronological Age (ΔHtSDSCA)
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 with idiopathic short stature (ISS), evaluate its safety and efficacy, and provide scientific and reliable evidence for the medication dosage in Phase III clinical study.
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
- 4 Years 至 9 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Boys are between 4 and 9 years of age and girls are between 4 and 8 years of age.
- •Height <-2 SD for chronological age.
- •Growth velocity<5.0 cm/yr.
- •GH peak concentration ≥10.0 ng/mL in two different stimulation tests.
- •The difference of bone age (BA) and chronological age (CA) is within -2 to +
- •IGF-1 concentration is between -2 SDS to +2 SDS.
- •Prepubertal Status(Tanner Stage I).
- •Birth weight within the normal range.
- •Growth hormone treatment-naive.
- •Subjects are willing and able to cooperate to complete scheduled visits, treatment plans and laboratory tests and other procedures, to sign informed consent.
排除标准
- •Subjects with abnormal liver and kidney functions (ALT > upper limit of normal value; Cr > upper limit of normal value).
- •Subjects are positive for anti-HBc, HbsAg or HbeAg in Hepatitis B virus tests.
- •Subjects with known highly allergic constitution or allergy to investigational product or its excipient.
- •Subjects with systemic chronic disease and immune deficiency.
- •Patients diagnosed with tumor.
- •Patients with mental disease.
- •Patients with other types of abnormal growth and development.
- •Growth hormone deficiency (GHD) (confirmed by GH stimulation test);
- •Turner syndrome (confirmed by karyotype test of girls);
- •Noonan syndrome (hypertelorism, pectus carinatum, hypophrenia, frequently with skin disease and congenital heart disease, missense mutation of the protein tyrosine phosphatase, non-receptor type 11 (PTPN11) gene on chromosome 12 for half of the patients, for both male and female patients);
- •Laron sydrome (confirmed by IGF-1 generation test);
- •Small for gestational age ( the birth height or weight is below the tenth percentile or 2 SD, with catch-up growth uncompleted at 2 years old).
- •Growth disorders caused by malnutrition or hypothyroidism (thyroid function test).
- •Congenital skeletal abnormalities or scoliosis, claudication.
- •Subjects with impaired glucose regulation (IGR) (including impaired fasting glucose (IFG) and/or impaired glucose tolerance (IGT) ) or diabetes).
- •Subjects with abnormal electrolyte, blood gas analysis (vein), creatine kinase.
- •Subjects who took part in other clinical trials within 3 months.
- •Subjects who received medications which may interfere GH secretion or GH function, or other hormones within 3 months (such as sex steroids, glucocorticoids, etc.).
- •For patients with potential high tumor risks such as tumor markers exceed normal range and some other relative information, they may be excluded from the treatment.
- •Other conditions which is inappropriate for this study in the opinion of the investigator.
研究组 & 干预措施
Jintrolong® low dose group
PEG-rhGH Injection (27IU/4.5mg/0.5ml/bottle) 0.1 mg/kg/w by subcutaneous injection for 52 weeks.
干预措施: Jintrolong® low dose group (Drug)
Jintrolong® low dose group
PEG-rhGH Injection (27IU/4.5mg/0.5ml/bottle) 0.1 mg/kg/w by subcutaneous injection for 52 weeks.
干预措施: Jintrolong® high dose group (Drug)
Jintrolong® high dose group
PEG-rhGH Injection (27IU/4.5mg/0.5ml/bottle) 0.2 mg/kg/w by subcutaneous injection for 52 weeks.
干预措施: Jintrolong® low dose group (Drug)
Jintrolong® high dose group
PEG-rhGH Injection (27IU/4.5mg/0.5ml/bottle) 0.2 mg/kg/w by subcutaneous injection for 52 weeks.
干预措施: Jintrolong® high dose group (Drug)
结局指标
主要结局
Change in Height Standard Deviation Score for Chronological Age (ΔHtSDSCA)
时间窗: Baseline，52 weeks
Change in Height Standard Deviation Score for Chronological Age (ΔHtSDSCA) from Baseline to 52 weeks；ΔHtSDSCA=(height Yx - reference mean for CA Yx) / reference SD for CA Yx (Yx refers to the height value at particular timepoint x)
次要结局
- Change in Bone Maturation(Baseline，52 weeks)
- Change in Annualized Height Velocity(Baseline，52 weeks)
- Change in IGF-1 Standard Deviation Score (IGF-1 SDS)(Baseline，52 weeks)
- IGF-1/IGFBP-3 molar ratio at 52 weeks(Baseline，52 weeks)