NCT05353192已完成4 期
A Multicenter, Open-label, Single Arm Phase IV Clinical Study to Evaluate the Efficacy and Safety of Recombinant Human Growth Hormone in Children With Achondroplasia
适应症
干预措施
相关药物
试验速览
- 阶段
- 4 期
- 状态
- 已完成
- 入组人数
- 39
- 试验地点
- 1
- 主要终点
- Change From Baseline in Annualized Height Velocity (AHV) at Week 52
研究概览
简要总结
To Evaluate the Efficacy and Safety of Recombinant Human Growth Hormone in Children with Achondroplasia
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
- 2 Years 至 10 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •2-10 years old;
- •In Tanner I stage;
- •ACH, documented and confirmed by genetic testing;
- •Short stature;
- •Ambulatory and able to stand or walk without assistance;
- •Parent(s) or guardian(s) consent；
- •Had never been treated with growth hormone
排除标准
- •Short stature condition other than ACH;
- •Evidence of growth plate closure (proximal tibia, distal femur);
- •Had a fracture of the long bones within 6 months prior to screening;
- •Planned or expected bone-related surgery;
- •Chronic diseases condition that affect bone metabolism and weight;
- •Severe intracranial hypertension
研究组 & 干预措施
Recombinant human growth hormone
Experimental
Recombinant human growth hormone Injection (15IU/5mg/3ml/bottle)；0.05 mg/kg/d by subcutaneous injection for 52 weeks
干预措施: Recombinant human growth hormone (Drug)
结局指标
主要结局
Change From Baseline in Annualized Height Velocity (AHV) at Week 52
时间窗: week 52
The change in AHV after 52 weeks treatment
次要结局
- Change From Baseline in BMI Standard Deviation Score (BMI SDS)(week 4, week 13, week 26, week 39, week 52)
- Change From Baseline in Sitting Height/Leg Length ratio Standard Deviation Score (SH/LL SDS)(week 4, week 13, week 26, week 39, week 52)
- Change From Baseline in IGF-1 Standard Deviation Score (IGF -1 SDS)(week 4, week 13, week 26, week 39, week 52)
- Change From Baseline in IGF-1/IGFBP-3 ratio(week 4, week 13, week 26, week 39, week 52)
- Change From Baseline in Bone Age/Chronological Age ratio (BA/CA)(week 52)
- Change From Baseline in Height Standard Deviation Score (Ht SDS)(week 4, week 13, week 26, week 39, week 52)
- Change From Baseline in AHV(week 4, week 13, week 26, week 39)
研究者
研究点 (1)
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