Optimization of the Dosage Regimen With Growth Hormone Therapy in Children Born Small for Gestational Age. An Open Label, Randomized, Pilot Study, Comparing in Children Treated for 3 Years, the Efficacy of a Saizen® Treatment at the Same Dose Versus a Lower Maintenance Dose Prolonged During 1 Additional Year.
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 22
- 主要终点
- Height Velocity
研究概览
简要总结
Multicentric, open-label, randomized, pilot comparative study in parallel groups comparing 1 group of subjects receiving 0.057 milligram/kilogram/day (mg/kg/day) or 0.40 mg/kg/week of Saizen® during 1 year to 1 group receiving 0.035 mg/kg/day (0.24 mg/kg/week) of Saizen® during 1 year after an initial 3-year treatment of recombinant human growth hormone (r-hGH) therapy with 0.057 mg/kg/day in both groups.
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 11 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •To be eligible for inclusion / randomization into this study, the subjects must fulfill all of the following criteria (if there is no inclusion phase, the inclusion criteria will be considered as inclusion criteria for randomization):
- •Written consent form signed by the parents / legal guardian, and child if possible
- •Subject born SGA and receiving a r-hGH therapy for this pathology
- •Recombinant human growth hormone (r-hGH) started at the maximal chronological age of 7 years for girls and 8 years for boys
- •Treatment with r-hGH started for at least 30 months and less than 36 months at 0.057 mg/kg/day
- •Height gain during the first 2 years of GH treatment > 1 SD compared with the initial value
排除标准
- •To be eligible for inclusion in this study the subjects must not meet any of the following criteria:
- •Known hypersensitivity to Somatropin or any of the excipients
- •Active neoplasia (either newly diagnosed or recurrent)
- •Intracranial hypertension
- •Known diabetes mellitus
- •Proliferative or preproliferative diabetic retinopathy
- •Evidence of any progression or recurrence of an underlying intra-cranial space occupying lesion
- •Obesity defined as degree 1 on the corpulence curves
- •Precocious puberty
- •Pubertal status: Tanner breast development stage > 2 for girls, and testicular volume > 4 milliliter (mL) or testicular length > 3 centimeter (cm) and/or testosterone value >1 nanomole/liter [nmol/L] (0.29 gram/mL [g/mL]) for boys For girls > 9 years and Tanner breast development stage 1: uterine size > 35 millimeter (mm)
- •Severe chronic concomitant illness such as chronic renal failure, cystic fibrosis
- •Concomitant corticoid treatment or levothyroxine treatment other than substitutive treatment, topical or inhaled treatment
- •Participation to any clinical study within the 30 days preceding study entry
研究组 & 干预措施
Saizen® 0.057 mg/kg/day
Subjects who met all inclusion/exclusion criteria were randomly assigned to 0.057 mg/kg/day in this multi-center study. Subjects were stratified at randomization according to the Height-Standard Deviation Score (H-SDS) at this time (less than [<] -2 SDS or greater than [>] -2 SDS)
干预措施: Saizen® (Drug)
Saizen® 0.035 mg/kg/day
Subjects who met all inclusion/exclusion criteria were randomly assigned to 0.035 mg/kg/day in this multi-center study. Subjects were stratified at randomization according to the H-SDS at this time (< -2 SDS or > -2 SDS)
干预措施: Saizen® (Drug)
结局指标
主要结局
Height Velocity
时间窗: Month 12
Height Velocity (HV) is the change in height since the previous year´s measurement and more precisely: HV = {(h-hp)/(d-dp)} \* 365.25 \[centimeter (cm)/year\] where h is current height in cm, hp is previous height in cm, closest to 1 year previous, d is the current date and dp is the date of measurement of previous height, closest to 1 year previous. Additionally, d and dp have to be within 0.6 years and 1.5 years. HV is the mean height velocity over the interval between d and dp but is displayed as HV at d.
次要结局
- Change From Baseline in Height-Standard Deviation Score (H-SDS) at Month 6 and Month 12(Baseline (randomization), Month 6 and Month 12)
- Height Velocity-Standard Deviation Score (HV-SDS)(Month 6 and Month 12)
- Insulin Like Growth Factor-1 (IGF-1) Levels(Baseline (randomization), Month 6 and Month 12)
- Number of Participants With Treatment Emergent Adverse Events (TEAEs)(Baseline (randomization) until Month 12)
- Change From Baseline in Bone Age at Month 12(Baseline (randomization) and Month 12)
- Change From Baseline in Height at Month 6(Baseline (randomization) and Month 6)
- Insulin Like Growth Factor Binding Protein-3 (IGFBP-3) Levels(Baseline (randomization), Month 6 and Month 12)