The Genetics and Neuroendocrinology of Short Stature International Study (GeNeSIS)
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 22,845
- 主要终点
- Type 2 Diabetes Mellitus in GH-treated Participants
研究概览
简要总结
GeNeSIS is an open-label, multinational, multicenter, observational study to evaluate the safety and effectiveness of Humatrope treatment.
GeNeSIS is a modular program that includes:
- Core study: Evaluating the safety and effectiveness of Humatrope in the observational setting
- Genetic Analysis Sub-study: Investigating the genetic defects underlying growth hormone (GH) deficiency and non-GH-deficient growth disorders
- Growth Prediction Sub-study: Working to validate and refine specific models to accurately predict growth response to GH
- Short Stature Homeobox containing gene (SHOX) Deficiency Sub-study: Elucidating the clinical, endocrine and radiological features of participants with SHOX deficiency due to loss of, or mutation in the SHOX gene (including participants with Turner syndrome)
- Neoplasia Sub-study: To characterize the natural history of neoplastic disease, especially in relation to recurrence/progression of primary neoplasia or development of secondary neoplasia in children with a history of neoplasia
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 年龄范围
- 1 Day 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •All participants participating in GeNeSIS must be enrolled in the core study. Participants for whom written consent to release information is provided may enter the core study if they meet any of the following inclusion guidelines:
- •Treatment with Humatrope for improvement of growth.
- •No treatment with somatropin in participants with a history of neoplasia or in those with any SHOX deficiency-related disorder.
排除标准
- •Participants with closed epiphyses are not eligible for GeNeSIS entry. However, participants may remain in the study if epiphyseal closure occurs during study participation.
研究组 & 干预措施
Treated
Participants treated with somatropin for improvement of growth
干预措施: Somatropin (recombinant deoxyribonucleic acid [rDNA] origin) (Drug)
结局指标
主要结局
Type 2 Diabetes Mellitus in GH-treated Participants
时间窗: Year 15
Final Height (FH) Gain by Diagnostic Group
时间窗: Baseline through Year 15
The standard deviation score (SDS) reports the number of standard deviations from the mean for age and sex for an individual measurement (normal range is -2 to +2 SDS). Height SDS is derived by subtracting the population mean from individual's height value and then dividing that difference by the population standard deviation. Greater height SDS values indicate greater height. Due to the small number of participants involved, untreated and unknown treatment groups, data was not provided and could not be calculated.
Primary Malignancies in Participant Without Previous Cancer History
时间窗: Year 15
Due to the small number of participants involved, untreated and unknown treatment groups, data was not provided and could not be calculated.
次要结局
- Percentage of Participants With Recurrent Neoplasms and Second Neoplasms in Childhood Cancer Survivors(Baseline through Year 15)
- Change From Baseline to Final Height in Anthropometric Measures for Participants With SHOX Deficiency(Baseline, Year 15)
- Percentage of Participants With Defects in Genes Associated With Pituitary Development(Baseline through Year 15)
- Predicted First Year Height Gain Versus Actual First Year Height Gain(Baseline through Year 15)
- Percentage of Participants With De Novo Neoplasms(Baseline through Year 15)
- Diabetes Mellitus (DM) in Somatropin-Treated Children With Different Short Stature Diagnoses(Baseline through Year 15)