Effect of Cinacalcet on the Long-term Treatment of Familial Hypophosphatemic Rickets
试验速览
- 阶段
- 不适用
- 状态
- 撤回
- 发起方
- 试验地点
- 1
- 主要终点
- doses of phosphate and calcitriol required to sustain goals of blood and urine chemistries
研究概览
简要总结
Currently, large oral doses of phosphate and 1,25(OH)2D (calcitriol) are the standard treatment of patients with familial hypophosphatemic rickets (XLH). While this therapy is effective in healing the rickets, it is often limited by development of complications due to the high dose of medications required to achieve cure. Among them are the development of calcifications in the kidneys and secondary hyperparathyroidism (HPT) which in some patients may cause complications like high blood calcium level, high blood pressure and damage to the kidney. A drug to treat secondary hyperparathyroidism was just developed. In a short term study we found that it might help the treatment of XLH, by allowing the use of lower doses of the both phosphate and calcitriol. In the present study we will learn if indeed the addition of this new medicine (Cinacalcet) to the long-term treatment will allow the use of lower doses of both phosphate and calcitriol and consequently lower the risk of complications.
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
- 5 Years 至 21 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 是
入选标准
- •Patients with XLH
- •Age 5-21 years old
- •From the Renal and Endocrinology Clinics who have been in a stable clinical, biochemical and radiological condition for the preceding 9 months
- •Who have not received Cinacalcet before
排除标准
- •Treatment with growth hormone
- •Inability to swallow pills.
- •An orthopedic surgery or braces in the past 9 months, or scheduled within 9 months of start.
- •History of seizure disorder
- •Abnormal liver functions (which may change the drug's AUC)
研究组 & 干预措施
Cinacalcet
Stable patients with XLH already treated with Phosphate and calcitriol will add Cinacalcet to their treatment regimen. Sequential monitoring of blood and urine biochemical variables will follow, based on which adjustments to the doses of the 3 medications will be done.
干预措施: Cinacalcet (Drug)
结局指标
主要结局
doses of phosphate and calcitriol required to sustain goals of blood and urine chemistries
时间窗: 9 months
次要结局
未报告次要终点