X-linked Hypophosphatemia Disease Monitoring Program (XLH-DMP)
试验速览
- 阶段
- 不适用
- 状态
- 进行中（未招募）
- 入组人数
- 782
- 试验地点
- 73
- 主要终点
- Long-Term Safety of Burosumab
研究概览
简要总结
The objectives of this observational study are to characterize XLH disease presentation and progression and to assess long-term effectiveness and safety of burosumab.
详细描述
The XLH-DMP is a global, prospective, multicenter, longitudinal, long-term outcomes program for subjects on or off any treatment designed to characterize XLH disease presentation and progression, assess long-term safety and effectiveness of burosumab, as well as prospectively investigate longitudinal change over time across biomarker(s), clinical assessments, and patient/caregiver-reported outcome measures in a representative population. The XLH-DMP will collect demographic, biochemical, physiologic, disease severity, and progression data in patients taking burosumab and those not taking burosumab. In this DMP, patients will only have access to burosumab through authorized prescribed use. The Sponsor will not provide any treatments as part of the DMP.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Willing and able to provide informed consent or, in the case of patients under the age of 18 years (or 16 years, depending on the region), provide assent (if required) and informed consent by a legally authorized representative after the nature of the study has been explained, and prior to any research-related procedures.
- •Clinical diagnosis of XLH based on family history, OR confirmed PHEX mutation, OR biochemical profile consistent with XLH.
- •Willing and able to comply with the study visit schedule and study procedures.
排除标准
- •Concurrent enrollment in an Ultragenyx-sponsored clinical trial is NOT permitted.
- •Serious medical or psychiatric comorbidity.
- •Less than one year of life expectancy.
研究组 & 干预措施
Prior Burosumab Clinical Trial Participants
Patients who participated in burosumab clinical trials and continue to receive burosumab via prescription from their physician.
干预措施: No intervention (Other)
Not from Prior Burosumab Clinical Trial
Patients may take other treatments for XLH and may start burosumab treatment at any time as prescribed by a physician.
干预措施: No intervention (Other)
结局指标
主要结局
Long-Term Safety of Burosumab
时间窗: 10 years
To assess the long-term safety of burosumab treatment in adult and pediatric patients with XLH, including overall renal health, the presence and/or progression of nephrocalcinosis and spinal stenosis, and pregnancy outcomes.
Long-Term Effectiveness of Burosumab
时间窗: 10 years
To evaluate the long-term effectiveness of burosumab treatment on key manifestations of XLH, including skeletal health, stiffness, mobility and physical functioning.
Clinical Course of XLH Disease
时间窗: 10 years
To illustrate the clinical, radiological, biochemical manifestations and progression of XLH over time in both untreated and treated patients with XLH.
次要结局
未报告次要终点