A Multicenter Multinational Observational Study of Children With Hypochondroplasia
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 入组人数
- 400
- 试验地点
- 43
- 主要终点
- Change in BMI
研究概览
简要总结
This study will assess growth over time and the clinical course of HCH in children by collecting growth measurements and other variables of interest.
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
- — 至 15 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Participants must be ≤ 15 years old at the time of signing the informed consent
- •Participants must have genetic confirmation of Hypochondroplasia diagnosis
排除标准
- •Have a diagnosis of another genetic short stature condition other than Hypochondroplasia or a genetic variant known to cause another genetic syndrome associated with short stature
- •Received an investigational product or medical device within 6 months before the Screening visit
结局指标
主要结局
Change in BMI
时间窗: Every 6 months through end of study, up to 15 years
Change in ratios of upper to lower body segments
时间窗: Every 6 months through end of study, up to 15 years
Change in Height
时间窗: Every 6 months through end of study, up to 15 years
Change in Height Z-score
时间窗: Every 6 months through end of study, up to 15 years
Change in BMI Z-score
时间窗: Every 6 months through end of study, up to 15 years
Change in ratios of upper and lower leg length ratio
时间窗: Every 6 months through end of study, up to 15 years
Change in annualized growth velocity (AGV)
时间窗: Every 6 months through end of study, up to 15 years
Change in ratio of arms span to standing height ratio
时间窗: Every 6 months through end of study, up to 15 years
次要结局
- Change in patient global impression of severity (PGI-S)(Every 52 weeks through end of study, up to 15 years)
- Change in caregiver global impression of severity (CaGI-S)(Every 52 weeks through end of study, up to 15 years)
- Change in Quality of Life in Short Statured Youth (QoLISSY) physical domain(Every 52 weeks through end of study, up to 15 years)
- Proportion of children who report use of growth hormone, treatment patterns and impact on growth(Every 6 months through end of study, up to 15 years)
- Frequency of event rates of medical events of interest(Every 6 months through end of study, up to 15 years)
- Proportion of children who report limb lengthening surgery(Every 6 months through end of study, up to 15 years)
- Change in Quality of Life in Short Statured Youth (QoLISSY) total score(Every 52 weeks through end of study, up to 15 years)