GHLIQUID-1516: A 104-week, Multi-centre, Randomised, Double-blind, Parallel-group, no Treatment Controlled (Open-label) Trial Investigating the Efficacy and Safety of Two Doses of NN-220 in Subjects With Short Stature Born Small for Gestational Age / GHLIQUID-1517: A Long-term, Multi-centre, Randomised, Controlled, Double-blind, Parallel-group Trial, Investigating the Efficacy and Safety of Two Doses of NN-220 in Subjects With Short Stature Born Small for Gestational Age
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 98
- 试验地点
- 1
- 主要终点
- Change in Height Standard Deviation Score (SDS) for Chronological Age (CA) at Week 208 - Subjects Received NN220 Treatment for 4 Years
研究概览
简要总结
This study is conducted in Japan. The aim of this trial is to assess the efficacy and safety of somatropin in children born small for gestational age (SGA) in Japan.
In the main period, subjects will receive either active treatment for 104 weeks (two dosing regimens) or no treatment for 52 weeks followed by an extension period where subjects who received active treatment for 104 weeks (two years) will continue with the same treatment for further 156 weeks (three years) while those subjects who received no treatment for 52 weeks (one year) will be randomised to receive two dosing regimens for 208 weeks (four years). In total, subjects participate in trial for 260 weeks (five years).
Main period is registered internally at Novo Nordisk as GHLIQUID-1516 while the extension period is registered as GHLIQUID-1517.
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
- Double (Participant, Investigator)
入排标准
- 年龄范围
- 3 Years 至 8 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •For MAIN period (GHLIQUID-1516):
- •Born small for gestational age (SGA) with birth weight and birth length below the 10th percentile for gestational age, and additional either birth length below or equal to -2.0 standard deviation score (SDS) or birth weight below or equal to -2.0 SDS for gestational age
- •Growth failure with height at -2.0 SDS or below for chronological age (CA)
- •Normal growth hormone (GH) production, defined as peak GH level > 10 ng/mL in one GH provocation test
- •For EXTENSION period (GHLIQUID-1517):
- •Subjects who completed the main period
- •Chronological age (CA) for boys at least 4 years, but maximum 11 years
- •Chronological age (CA) for girls at least 4 years, but maximum 10 years
排除标准
- •Subjects with diabetes mellitus
- •Subjects suffering from malignancy
- •Several medical conditions
研究组 & 干预措施
0.033 mg / NN-220
In the 156-week main period, subjects received 0.033 mg/kg/day somatropin (NN-220) s.c. (under the skin) injected at bedtime followed by a 104-week extension period where subjects received 0.033 mg/kg/day somatropin (NN-220) s.c. (under the skin) injected at bedtime
干预措施: somatropin (Drug)
0.067 mg / NN-220
In the 156-week main period, subjects received 0.067 mg/kg/day somatropin (NN-220) s.c. (under the skin) injected at bedtime followed by a 104-week extension period where subjects received 0.067 mg/kg/day somatropin (NN-220) s.c. (under the skin) injected at bedtime
干预措施: somatropin (Drug)
No treatment --> 0.033 mg
In the 208-week extension period, subjects received 0.033 mg/kg/day somatropin (NN-220) s.c. (under the skin) injected at bedtime after having received no somatromin (NN-220) treatment in the 52-week main period
干预措施: somatropin (Drug)
No treatment --> 0.067 mg
In the 208-week extension period, subjects received 0.067 mg/kg/day somatropin (NN-220) s.c. (under the skin) injected at bedtime after having received no somatromin (NN-220) treatment in the 52-week main period
干预措施: somatropin (Drug)
结局指标
主要结局
Change in Height Standard Deviation Score (SDS) for Chronological Age (CA) at Week 208 - Subjects Received NN220 Treatment for 4 Years
时间窗: Week 0, week 208
Height SDS for chronological age were derived as follow; {Height - mean (age, sex)}/ SD (age, sex), where mean (age, sex) and SD (age, sex) were mean and SD of height for corresponding chronological age and sex (data of those in 2000). Height SDS was calculated using mean of three height observations at corresponding visit
Change in Height Standard Deviation Score (SDS) for Chronological Age (CA) at Week 260 - Subjects Received NN220 Treatment for 5 Years
时间窗: Week 0, week 260
Height SDS for chronological age were derived as follow; {Height - mean (age, sex)}/ SD (age, sex), where mean (age, sex) and SD (age, sex) were mean and SD of height for corresponding chronological age and sex (data of those in 2000). Height SDS was calculated using mean of three height observations at corresponding visit
次要结局
- Change in Bone Age (Left Hand X-Ray) at Week 208 - Subjects Received NN220 Treatment for 4 Years(Week 0, week 208)
- Adverse Events - Subjects Received NN220 Treatment for 4 Years(Weeks 0-208)
- Yearly Height Velocity SDS for Chronological Age - Subjects Received NN220 Treatment for 5 Years(Weeks 0-260)
- Yearly Height Velocity SDS for Chronological Age - Subjects Received NN220 Treatment for 4 Years(Weeks 0-208)
- Change in Bone Age (Left Hand X-Ray) at Week 260 - Subjects Received NN220 Treatment for 5 Years(Week 0, week 260)
- Adverse Events - Subjects Received NN220 Treatment for 5 Years(Weeks 0-260)