NCT04035811进行中（未招募）不适用
Prospective Clinical Assessment Study in Children With Achondroplasia: The PROPEL Trial
适应症
试验速览
- 阶段
- 不适用
- 状态
- 进行中（未招募）
- 入组人数
- 271
- 试验地点
- 32
- 主要终点
- Annualized height velocity (cm/year)
研究概览
简要总结
This is a long-term, multi-center, observational study in children 2.5 to <17 years with achondroplasia (ACH). The objective is to evaluate growth, ACH-related medical complications, assessments of health-related quality of life, body pain, functional abilities, cognitive functions, and treatments of study participants. No study medication will be administered.
研究设计
- 研究类型
- Observational
- 观察模型
- Case Only
- 时间视角
- Prospective
入排标准
- 年龄范围
- 30 Months 至 17 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Signed informed consent by study participant or parent(s) or legally authorized representative (LAR) and signed informed assent by the study participant (when applicable)
- •Aged 2.5 to <17 years at study entry
- •Diagnosis of ACH
- •Study participants and parent(s) or LAR(s) are willing and able to comply with study visits and study procedures
排除标准
- •Have hypochondroplasia or short stature condition other than ACH (e.g. trisomy 21, pseudoachondroplasia, psychosocial short stature)
- •In females, having had their menarche
- •Height < -2 or > +2 standard deviations for age and sex based on reference tables on growth in children with ACH
- •Annualized height growth velocity ≤1.5 cm/year over a period ≥6 months prior to screening
- •Current evidence of corneal or retinal disorder/keratopathy
- •Current evidence of endocrine alterations of calcium/phosphorus homeostasis
- •Have a concurrent disease or condition that in the view of the Investigator and/or Sponsor, may impact growth or where the treatment is known to impact growth.
- •Significant abnormality in screening laboratory results.
- •Have been treated with growth hormone, insulin-like growth factor 1 (IGF 1), or anabolic steroids in the previous 6 months or long-term treatment (>3 months) at any time
- •Have had regular long-term treatment (>1 month) with oral corticosteroids (low-dose ongoing inhaled steroid for asthma is acceptable)
- •Have had previous guided growth surgery or limb-lengthening surgery within 12 months prior to screening.
结局指标
主要结局
Annualized height velocity (cm/year)
时间窗: Up to 2 years
次要结局
未报告次要终点
研究者
研究点 (32)
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