A Phase 2 Study Extension Period of Pegylated Somatropin (PEG-somatropin) in the Treatment of Children With Idiopathic Short Stature: An Open, Non-controlled Observational Study.
试验速览
- 阶段
- 2 期
- 状态
- 进行中（未招募）
- 入组人数
- 360
- 试验地点
- 12
- 主要终点
- Change of yearly height velocity (ΔHV)
研究概览
简要总结
After the first stage (52 weeks) of Phase II clinical trial, Pegylated recombinant human growth hormone (PEG-rhGH) injection of appropriate dose in compliance with ISS clinical treatment strategy is used to treat children with ISS (Idiopathic Short Stature). The long-term efficacy and safety of the investigational product are evaluated, which can provide more scientific and reliable medication guidance information for clinical diagnosis and treatment.
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
- 4 Years 至 9 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •All subjects who have completed the first stage (52 weeks) of Phase II clinical trial (including negative controls) with completed follow-up records may be enrolled in the extension period study.
- •Before the extension period study, the investigator shall fully inform the subjects and their guardians of all the information about the extension period study, including detailed follow-up procedure, treatment plan, laboratory examination items during follow-ups and possible benefits and risks. The extension period study shall only be initiated after the subjects and their guardians are well informed, and agree to cooperate and complete the treatment, follow-ups and examinations during the study, and sign the written informed consent.
排除标准
- •Subjects who have taken the following medications within 2 months before entering the extension period study:
- •Aromatase inhibitors (which include but are not limited to Lelrozol and Anastrozole), with continuous medication ≥1 month;
- •Gonadotropin releasing hormone analogues (which include but are not limited to Triptorelin, Leuprorelin and Goserelin),, with continuous medication ≥1 month;
- •Sex steroids (which include but are not limited to any type of estrogen, progestin and androgen) , with continuous medication ≥1 month;
- •Protein anabolic drugs (which include but are not limited to Oxandrolone, Danazol and Strombafort), with continuous medication ≥1 month;
- •Glucocorticoids via oral/intravenous administration for more than 1 month..
研究组 & 干预措施
PEG-somatropin
After the first stage (52 weeks) of Phase II clinical trial, the initial medication dose of this extension period is 0.2 mg/kg weight/week of PEG-rhGH for the high dose group, low dose group and negative control group, and it is adjusted in accordance with yearly height velocity (HV) and IGF-1 SDS of each visit. The maximum dose shall not exceed 0.4 mg/kg weight/week.
干预措施: PEG-somatropin (Drug)
结局指标
主要结局
Change of yearly height velocity (ΔHV)
时间窗: Baseline，the end of 3-year addendum
Change of yearly height velocity before and after treatment. Yearly Height Velocity=12×(Height Yx - Height at Baseline)/(Date of Yx - Date of Baseline)(Yx refers to the height value at particular timepoint x)
次要结局
- Standard deviation score of height at the actual age (ΔHT SDS)(Baseline，every 3 months，the end of 3-year addendum)
- Change of IGF-1 SDS (ΔIGF-1 SDS)(Baseline，every 3 months，the end of 3-year addendum)
- Final height (FH)(Baseline，every 3 months，the end of 3-year addendum)
- The improvement of FH compared with the baseline predicted adult height (PAH)(Baseline，every 3 months，the end of 3-year addendum)
- Change fo Bone maturation(Baseline，every 3 months，the end of 3-year addendum)
- Changes of standard deviation scores of body mass index (ΔBMI SDS)(Baseline，every 3 months，the end of 3-year addendum)
- The yearly average dose of PEG-rhGH injection(Baseline，every 3 months，the end of 3-year addendum)
- Improvement of NAH (near adult height)(Baseline，every 3 months，the end of 3-year addendum)
- The changes of the percentage of body fat (optional)(Baseline，every 3 months，the end of 3-year addendum)
- The changes of bone mineral density (BMD) (optional)(Baseline，every 3 months，the end of 3-year addendum)
- the improvement of PAH(Baseline，every 3 months，the end of 3-year addendum)
- The changes of the scores evaluated by the Quality of Life Scale(Baseline，every 3 months，the end of 3-year addendum)
- The changes of lean body mass (LBM) (optional)(Baseline，every 3 months，the end of 3-year addendum)
- The changes of fat mass (torso) (FM) (optional)(Baseline，every 3 months，the end of 3-year addendum)