First Year Growth Response Associated Genetic Markers Validation Phase IV Open-label Study in Growth Hormone Deficient and Turner Syndrome Pre-pubertal Children: the PREDICT Pharmacogenetics Validation Study
试验速览
- 阶段
- 4 期
- 状态
- 已完成
- 入组人数
- 458
- 试验地点
- 31
- 主要终点
- Change From Baseline in Height at Year 1
研究概览
简要总结
PREDICT Validation is a validation pharmacogenetic trial. The purpose of this study is to confirm that some genes can be used to predict how well a subject diagnosed with idiopathic growth hormone deficiency (IGHD) or turner syndrome (TS) will respond to a treatment with recombinant human growth hormone (r-hGH).
详细描述
This study is an open-label, interventional, retrospective, multicenter, international study, single-arm, non-randomized, and non-controlled study. The subject's trial participation includes a single visit. During the visit, subjects who give consent to participate in the trial will undergo blood sampling for genetic markers testing and retrospective data will be collected relative to the first year of the subject's r-hGH treatment.
The r-hGH treatment followed by the subject is indicated the pediatric population, therefore most of the subjects included into the trial will be below 18 years old.
This study is a non-investigational medicinal product (IMP) trial therefore no drug product data is provided.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Single Group
- 盲法
- None
入排标准
- 年龄范围
- — 至 18 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Pre-established diagnosis of IGHD or TS based on classical criteria with at least 1 year of r-hGH therapy and with Tanner stage 1 at treatment start
- •Retrospective availability of a complete set of clinical, auxological and biological parameters necessary for building the predictive model
- •Other protocol defined inclusion criteria could apply
排除标准
- •Acquired growth hormone deficiency (GHD)
- •Any drug or disease that could affect growth during the first year of r-hGH treatment
- •Other protocol defined exclusion criteria could apply
结局指标
主要结局
Change From Baseline in Height at Year 1
时间窗: Baseline and Year 1
Change from baseline in height at year 1 was one of the growth parameter to assess the first year growth response to r-hGH treatment.
Height Velocity Standard Deviation Score (SDS) at Year 1
时间窗: Year 1
Height velocity SDS was calculated as height velocity minus reference mean height velocity divided by standard deviation of the reference population. Height velocity SDS reflects the height velocity relative to a reference population of the same age and gender. Height velocity SDS at Year 1 was one of the growth parameter to assess the first year growth response to r-hGH treatment.
Change From Baseline in Height Standard Deviation Score (SDS) at Year 1
时间窗: Baseline and Year 1
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. Change from baseline in height SDS at Year 1 was one of the growth parameter to assess the first year growth response to r-hGH treatment.
次要结局
- Evaluation of the Contribution of Validated Genetic Markers to the Amplitude of First Year Growth Response to r-hGH Therapy in IGHD Children Using Growth Hormone Deficiency Kabi-Pharmacia International Growth Study (GHD KIGS) Predictive Model(Year 1)
- Evaluation of the Contribution of Validated Genetic Markers to the Amplitude of First Year Growth Response to r-hGH Therapy in TS Girls Using Turner Syndrome Kabi-Pharmacia International Growth Study (TS KIGS) Predictive Model(Year 1)