Collaborative Study to Assess the Effects of Treatment With Recombinant Growth Hormone Saizen® in the Prevention of Short Stature in Young Girls Suffering From Turner Syndrome Before the Age of 4 Years. Original French Title: Etude Collaborative Pour apprécier Les Effets du Traitement Par l'Hormone de Croissance Recombinante SAIZEN® Dans le Retard de Croissance de la Fillette Atteinte de Syndrome de Turner Avant l'âge de 4 Ans
试验速览
- 阶段
- 4 期
- 状态
- 已完成
- 入组人数
- 115
- 试验地点
- 1
- 主要终点
- Height SDS at Year 4
研究概览
简要总结
The objective of this study is to evaluate the efficacy and safety of recombinant human growth hormone (r-hGH) treatment in girls with Turner Syndrome under the age of 4 years. After 4 years of treatment, height in these girls will be compared with an historical control group of untreated girls with Turner Syndrome, matched for age and height at baseline.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- — 至 4 Years（Child）
- 性别
- Female
- 接受健康志愿者
- 否
入选标准
- •Young girls with turner syndrome proved by karyotype
- •Growth hormone secretion confirmed with ornithin stimulation test
- •Normal glucidic metabolism confirmed by assessment of HbA1c
- •None associated severe pathology which could have impact on growth (i.e. renal insufficiency, decompensated heart failure)
- •No previous or associated treatment with anabolic or sexual steroids
- •Known parental height
排除标准
- •Severe associated pathology with impact on growth
- •Concomitant treatment with impact on growth
- •Previous or associated treatment with anabolic steroids
- •Associated growth hormone deficiency
研究组 & 干预措施
r-hGH
Participants (girls) will receive r-hGH as a subcutaneous injection administered by a parent in the evening. During Years 1-2, the dose of r-hGH received will depend on participants' baseline height standard deviation score (SDS) relative to the general population standard: participants with a height SDS of -2 standard deviation (SD) or lower will receive 0.05 milligrams per kilogram (mg/kg) per day r-hGH and those with a height SDS between -1 and -2 SD will receive 0.035 mg/kg per day r-hGH. After 2 years of treatment, all participants will receive a fixed dose of 0.05 mg/kg per day for a further 2 years.
干预措施: r-hGH (Drug)
结局指标
主要结局
Height SDS at Year 4
时间窗: Year 4
Height SDS was calculated as height minus reference mean height divided by standard deviation of the reference population. Height SDS reflects the height relative to a reference population of the same age and gender.
次要结局
- Difference Between Bone Age (BA) and Chronological Age (CA) (BA-CA)(Baseline, Year 1, Year 2)
- Number of Participants With Anti r-hGH Antibodies(Baseline up to Year 2)
- Number of Participants With Abnormal Insulin-Like Growth Factor 1 (IGF1) Levels(Baseline up to Year 2)
- Number of Participants Who Reached Normal Height at Year 4(Year 4)
- Number of Participants With Abnormal Glycated Hemoglobin (HbA1c) Levels(Baseline up to Year 2)