NCT06410976招募中不适用
Prospective Clinical Assessment Study in Children With Hypochondroplasia: ACCEL
适应症
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 入组人数
- 150
- 试验地点
- 47
- 主要终点
- Annualized height velocity
研究概览
简要总结
This is a long-term, multicenter, non-interventional study of children ages 2.5 to <17 years with hypochondroplasia (HCH).
详细描述
The objective is to evaluate growth, HCH-related medical complications, health-related quality of life, functional abilities and cognitive functions of study participants. Data collected will contribute to the characterization of the natural history of children with HCH. 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 16 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Signed informed consent.
- •Aged 2.5 to <17 years at study entry.
- •Diagnosis of HCH documented clinically by the presence of disproportionate short stature and confirmed with a molecular test.
- •Participants are ambulatory and able to stand without assistance.
- •Study participants and parent(s), guardian(s), or caregiver(s) are willing and able to comply with study visits and study procedures.
排除标准
- •Have ACH or short stature condition other than HCH.
- •In females, having had their menarche. Annualized height growth velocity ≤1.5 cm/year over a period ≥6 months prior to screening.
- •Having a clinically significant disease or condition that in view of the investigator or Sponsor will interfere with the evaluation of growth, with study participation or not be in the best interest of the participant.
- •Clinically significant abnormality in any laboratory test result at screening
- •Current evidence of corneal or retinal disorders.
- •Have used any other investigational or approved product or medical device for the treatment of HCH or short stature for ≥ 30 days or with the last dose <6 months before screening.
- •Have had regular long-term treatment (>1 month) with oral corticosteroids (low-dose ongoing inhaled steroid for asthma is acceptable).
- •Previous limb-lengthening surgery or guided growth surgery with plates still in place or removed within the 6 months prior to screening.
- •Having had a fracture of the long bones or spine within 12 months of screening.
- •History and/or current evidence of extensive ectopic tissue calcification.
- •History of malignancy.
- •Concurrent circumstance, disease, or condition that, in the view of the investigator and/or sponsor, would interfere with study participation, and/or would place the participant at high risk for poor compliance with study activities or for not completing the study.
- •Current participation in any other ongoing clinical study with another sponsor.
结局指标
主要结局
Annualized height velocity
时间窗: up to 3 years
次要结局
- HCH-related surgical procedures(up to 3 years)
- Cognitive functions(up to 3 years)
- Change over time in height Z-score, upper arm to forearm ratio, and upper leg to lower leg ratio(up to 3 years)
- HCH-related medical events reported as medical history or NT-AEs(up to 3 years)
- Change from BL in the Physical Functioning dimension of the Pediatric Quality of Life Generic Core Scale Short Form.(up to 3 years)
研究者
研究点 (47)
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