NCT03875534已完成不适用
ACHieve: A Multi-center, Longitudinal, Observational Study of Children With Achondroplasia
适应症
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 260
- 试验地点
- 1
- 主要终点
- Annualized height velocity (centimeters/year) in children with achondroplasia
研究概览
简要总结
This is a long-term, multi-center, longitudinal, observational study in children with achondroplasia (ACH). The aim is to study height velocity and comorbidities in children with ACH. This is a natural history study and 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
- 0 Years 至 8 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Legally authorized representative is willing and able to provide written, signed informed consent (with a written assent from the child when appropriate per local requirements)
- •Willing and able to comply with study protocol per investigator judgement
- •Clinical diagnosis of achondroplasia (confirmed by the investigator)
- •Age between 0 to 8 years old at enrollment
- •Able to stand without assistance (if the child is 24 months or older)
排除标准
- •Have received chronic treatment (> 3 months) of human growth hormone (hGH) or other medicinal products intended to affect stature or body proportionality at any time
- •Have received any dose of medicinal products intended to affect stature or body proportionality within the previous 6 months of screening
- •Have received any investigational medicinal product or device intended to affect stature or body proportionality at any time
- •History or presence of injury or disease of the growth plate(s), other than ACH, that affects growth potential of long bones
- •History of any bone-related surgery that affects growth potential of long bones, such as orthopedic reconstructive surgery and osteotomy (foramen magnum decompression, and laminectomy with full recovery are allowed with minimum of 6 months of bone healing. Limb-lengthening with full recovery is allowed with a minimum of 12 months of bone healing.)
- •Have forms of skeletal dysplasias other than achondroplasia or medical conditions that result in short stature or abnormal bone growth [such as severe achondroplasia with developmental delay and acanthosis nigricans (SADDAN), hypochondroplasia, growth hormone deficiency, Turner syndrome, pseudoachondroplasia. uncontrolled hypothyroidism, uncontrolled diabetes mellitus, autoimmune disease requiring corticosteroid therapy, inflammatory bowel disease, and chronic renal insufficiency]
- •History or presence of malignant disease, other than basal cell epithelioma/carcinoma or completely resected squamous skin cancer with no recurrence for 12 months per medical records
结局指标
主要结局
Annualized height velocity (centimeters/year) in children with achondroplasia
时间窗: Up to 5 years
Subjects will undergo a series of height measurements (in centimeters) on Day 1 and then every 6 months over the study period with height velocity reported in cm/year
次要结局
- Collection of natural history of achondroplasia symptoms in children with achondroplasia(Up to 5 years)
研究者
研究点 (1)
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