Neuromuscular Changes In Small For Gestational Age (SGA) Children During Somatropin Therapy - A Prospective Randomized, Controlled, Open-Label Multicenter Trial
试验速览
- 阶段
- 3 期
- 状态
- 终止
- 发起方
- Pfizer
- 入组人数
- 23
- 试验地点
- 1
- 主要终点
- Change From Baseline in Maximum Jump Velocity (Vmax; Two-leg-jump) in Per Protocol (PP) Population at Month 6
研究概览
简要总结
The planned study focuses on the effect of a one year Somatropin treatment (0.035 mg/kg/d or 0.067 mg/kg/d) in short children born SGA on neuromuscular function and cognitive performance.
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
- 6 Years 至 10 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Pre-pubertal boys between 6 and 10 years of age or girls between 6 and 9 years of age.
- •Birth length- and/or birth weight-SDS adjusted to gestational age < -2.0 (Voigt et al. 2002, Voigt et al. 2006 or Lawrence et al. 1989).
- •Current height-SDS < -2.5 (Brandt/Reinken 1992) and parental adjusted height-SDS below -1 (Hermanussen and Cole 2003).
- •Growth velocity SDS < 0 during the last year before inclusion (Brandt/Reinken 1988).
排除标准
- •Severe SGA (birth weight or length < -4 SD) and clinically relevant dysmorphic features.
- •Severe pre-maturity (GA < 32 weeks of gestation).
- •Severe perinatal complications like asphyxia, sepsis, necrotizing enterocolitis (NEC), respiratory distress syndrome, if associated with long-term sequelae (like short bowel syndrome, bronchopulmonary dysplasia (BPD), cerebral palsy etc).
- •Inability to perform one- or two leg jumps from a standing position.
- •Prior GH treatment.
研究组 & 干预措施
Treatment Group
Somatropin for 12 months
干预措施: Somatropin (Drug)
Control Group
In the first 6 months no intervention, afterwards Somatropin for 12 months
干预措施: Somatropin (Drug)
结局指标
主要结局
Change From Baseline in Maximum Jump Velocity (Vmax; Two-leg-jump) in Per Protocol (PP) Population at Month 6
时间窗: Baseline and Month 6
Vmax was measured by Leonardo Jumping Platform during two-leg jump.
Change From Baseline in Peak Jump Power Standard Deviation Score (PJP-SDS; Two-leg-jump) in Full Analysis Set (FAS) Population at Month 6
时间窗: Baseline and Month 6
Peak jump power (PJP) was defined as the peak of the calculated power (force multiplied by velocity). It was measured by Leonardo Jumping Platform during two-leg jump. The participant performs 3 jumps and the highest peak (PJP) of the 3 recordings was selected for further calculations. The SDS indicates how similar the participant was to the reference population.
Change From Baseline in Peak Jump Force Standard Deviation Score (PJF-SDS; Two-leg-jump) in Per Protocol (PP) Population at Month 6
时间窗: Baseline and Month 6
PJF was defined as the maximum of force of the ascending part of the jump which the participant performed as a counter-movement jump with freely moving arms and as high as possible with the head and chest. It was measured by Leonardo Jumping Platform during two-leg jump. The SDS indicates how similar the participant was to the reference population.
Change From Baseline in Peak Jump Power Standard Deviation Score (PJP-SDS; Two-leg-jump) in Per Protocol (PP) Population at Month 6
时间窗: Baseline and Month 6
PJP was defined as the peak of the calculated power (force multiplied by velocity). It was measured by Leonardo Jumping Platform during two-leg jump. The participant performs 3 jumps and the highest peak (PJP) of the 3 recordings was selected for further calculations. The SDS indicates how similar the participant was to the reference population.
Change From Baseline in Maximum Jump Velocity (Vmax; Two-leg-jump) in Full Analysis Set (FAS) Population at Month 6
时间窗: Baseline and Month 6
Vmax was measured by Leonardo Jumping Platform during two-leg jump.
Change From Baseline in Peak Jump Force Standard Deviation Score (PJF-SDS; Two-leg-jump) in Full Analysis Set (FAS) Population at Month 6
时间窗: Baseline and Month 6
PJF was defined as the maximum of force of the ascending part of the jump which the participant performed as a counter-movement jump with freely moving arms and as high as possible with the head and chest. It was measured by Leonardo Jumping Platform during two-leg jump. The SDS indicates how similar the participant was to the reference population.
次要结局
- Change From Baseline in Intellectual Performance of Children Using Kinderversion Der Testbatterie Zur Aufmerksamkeitsprüfung für Kinder (KITAP) Test at Month 6(Baseline and Month 6)
- Change From Baseline in Peak Jump Power Standard Deviation Score (PJP-SDS; One-leg-jump) at Months 6, 12 and 18(Baseline, Month 6 , Month 12 and Month 18)
- Change From Baseline in Peak Jump Force Standard Deviation Score (PJF-SDS; One-leg-jump) at Months 6, 12 and 18(Baseline, Month 6, Month 12 and Month 18)
- Change From Baseline in Five-chair Rising Test- Peak Jump Power (PJP) at Month 6(Baseline and Month 6)
- Change From Baseline in Five-chair Rising Test-Peak Jump Force (PJF) at Months 12 and 18(Baseline, Month 12 and Month 18)
- Change From Baseline in Intellectual Performance of Children Using Kaufmann-Assessment Battery for Children (K-ABC) Test Global Scales at Months 12 and 18(Baseline, Month 12 and Month 18)
- Change From Baseline in Intellectual Performance of Children Using Child Behavior Checklist 4-18 Years (CBCL 4-18) at Months 6, 12 and 18(Baseline, Month 6, Month 12 and Month 18)
- Change From Baseline in Five-chair Rising Test-Peak Jump Power (PJP) at Months 12 and 18(Baseline, Month 12 and Month 18)
- Change From Baseline in Five-chair Rising Test-Peak Jump Force (PJF) at Month 6(Baseline and Month 6)
- Change From Baseline in One-chair Rising Test-Peak Jump Power (PJP) at Months 6, 12 and 18(Baseline, Month 6 , Month 12 and Month 18)
- Mean Thigh Circumference(Baseline, Month 6, Month 12 and Month 18)
- Mean Height at Month 6(Month 6)
- Change From Baseline in Intellectual Performance of Children Using Non-verbal Learning Test (NVLT) at Months 12 and 18(Baseline, Month 12 and Month 18)
- Change From Baseline in Maximum Jump Velocity (Vmax; One-leg-jump) at Months 6, 12 and 18(Baseline, Month 6, Month 12 and Month 18)
- Mean Growth Velocity at Months 12 and 18(Month 12 and Month 18)
- Mean Height-Standard Deviation Score (SDS) at Month 6(Month 6)
- Change From Baseline in Intellectual Performance of Children Using Kaufmann-Assessment Battery for Children (K-ABC) Test Global Scales at Month 6(Baseline and Month 6)
- Change From Baseline in Intellectual Performance of Children Using Non-verbal Learning Test (NVLT) at Month 6(Baseline and Month 6)
- Change From Baseline in Five-chair Rising Test-Maximum Jump Velocity (Vmax) at Month 6(Baseline and Month 6)
- Change From Baseline in Five-chair Rising Test (Time to Perform the Tasks) at Months 12 and 18(Baseline, Month 12 and Month 18)
- Change From Baseline in One-chair Rising Test-Peak Jump Force (PJF) at Months 6, 12 and 18(Baseline, Month 6 , Month 12 and Month 18)
- Mean Growth Velocity at Month 6(Month 6)
- Mean Growth Velocity-Standard Deviation Score (SDS) at Month 6(Month 6)
- Mean Growth Velocity-Standard Deviation Score (SDS) at Months 12 and 18(Month 12 and Month 18)
- Change From Baseline in Intellectual Performance of Children Using Kinderversion Der Testbatterie Zur Aufmerksamkeitsprüfung für Kinder (KITAP) Test at Months 12 and 18(Baseline, Month 12 and Month 18)
- Change From Baseline in Five-chair Rising Test-Maximum Jump Velocity (Vmax) at Months 12 and 18(Baseline, Month 12 and Month 18)
- Change From Baseline in Five-chair Rising Test (Time to Perform the Tasks) at Month 6(Baseline and Month 6)
- Change From Baseline in Maximal Isometric Grip Force-Standard Deviation Score (MIGF-SDS) at Month 6(Baseline and Month 6)
- Mean Calf Circumference(Baseline, Month 6, Month 12 and Month 18)
- Mean Height at Months 12 and 18(Month 12 and Month 18)
- Change From Baseline in Skinfold Thickness-Standard Deviation Score (SDS) at Month 6(Baseline and Month 6)
- Mean Height-Standard Deviation Score (SDS) at Months 12 and 18(Month 12 and Month 18)
- Change From Baseline in One-chair Rising Test (Time to Perform the Tasks) at Months 6, 12 and 18(Baseline, Month 6 , Month 12 and Month 18)
- Change From Baseline in Maximal Isometric Grip Force-Standard Deviation Score (MIGF-SDS) at Months 12 and 18(Baseline, Month 12 and Month 18)
- Mean Upper Arm Circumference(Baseline, Month 6, Month 12 and Month 18)
- Change From Baseline in Growth Velocity-Standard Deviation Score (SDS) at Month 6(Baseline and Month 6)
- Change From Baseline in Head Circumference-Standard Deviation Score (SDS) at Months 6, 12 and 18(Baseline, Month 6, Month 12 and Month 18)
- Change From Baseline in Height-Standard Deviation Score (SDS) at Months 12 and 18(Baseline, Month 12 and Month 18)
- Change From Baseline in Height-Standard Deviation Score (SDS) at Month 6(Baseline and Month 6)
- Change From Baseline in Growth Velocity-Standard Deviation Score (SDS) at Months 12 and 18(Baseline, Month 12 and Month 18)
- Sitting Height-Standard Deviation Score (SDS)(Baseline, Month 6, Month 12 and Month 18)
- Change From Baseline in Head Circumference at Months 6, 12 and 18(Baseline, Month 6, Month 12 and Month 18)
- Change From Baseline in Skinfold Thickness-Standard Deviation Score (SDS) at Months 12 and 18(Baseline, Month 12 and Month 18)
- Body Mass Index-Standard Deviation Score (BMI-SDS)(Baseline, Month 6, Month 12 and Month 18)