12-months of Treatment With Burosumab in Children and Adolescents With X-linked Hypophosphatemia: a Prospective Longitudinal Cohort Study
试验速览
- 阶段
- 4 期
- 发起方
- 入组人数
- 57
- 试验地点
- 1
- 主要终点
- Radiological changes in rachitic lesions evaluated with knee MRI
研究概览
简要总结
In this prospective longitudinal cohort study we studied the efficacy and safety of burosumab in real-clinical practice for <13- and >13-years old children affected with X-linked hypophosphatemia.
57 children with XLH were switched from conventional treatment to burosumab. After 12 months we assessed the efficacy and safety of treatment with burosumab on the whole cohort and separately on the cohort of >13-years old adolescents.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 1 Year 至 20 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •insufficient response or refractory to conventional therapy;
- •complications of conventional therapy: hypercalciuria and/or nephrocalcinosis, and/or persistent secondary hyperparathyroidism;
- •need for rapid restoration of phosphate metabolism, e.g., late diagnosis (aged >8 years) and/or preparation for planned orthopaedic surgery.
排除标准
- 未提供
研究组 & 干预措施
The whole cohort
Children affected with X-linked hypophosphatemia of average age of 9.8 years were switch from conventional therapy to burosumab
干预措施: Burosumab Injection (Drug)
结局指标
主要结局
Radiological changes in rachitic lesions evaluated with knee MRI
时间窗: 12 months
maximum width of the physis and transverse extent of widening
次要结局
- incidence of dental abcesses(12 months)
- incidence of hyperparathyroidism(12 months)
- height(12 months)
- incidence of hearing problems(12 months)
- incidence of neurological problems (craniosynostosis, Chiari I malformation)(12 months)
- incidence of nephrocalcinosis(12 months)
- 1,25(OH)vitaminD(12 months)
- incidence of any side effects(12 months)
- alkaline phosphatase(12 months)
- functional capacity(12 months)
- serum phosphate(12 months)
- renal phosphate reabsorption(12 months)
- parathyroid hormone(12 months)
研究者
Volha Zhukouskaya
MD, PhD
Bicetre Hospital