The Impact of Phosphate Metabolism on Healthy Aging
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 入组人数
- 30
- 试验地点
- 2
- 主要终点
- Parathyroid Hormone (PTH) levels
研究概览
简要总结
Determine the association between duration and dose of chronic conventional therapy with Pi and renal (nephrocalcinosis/nephrolithiasis), vascular (endothelial function), and cardiovascular function (echo- cardiography) in patients with hereditary hypophosphatemic rickets with hypercalciuria (HHRH) and patients with X-linked hypophosphatemia (XLH).
详细描述
The central hypothesis of this proposal is that patients with X-linked hypophosphatemia (XLH), when matched for duration and dose of phosphate (Pi) therapy to patients with hereditary hypophosphatemic rickets with hypercalciuria (HHRH), will evidence greater cardiovascular and vascular debility than patients with HHRH. The overall objectives of this project are to utilize our existing longitudinal databases for individuals with XLH and HHRH through an interdisciplinary collaboration between pediatric and adult endocrinology to: i) quantify the impact of exposure to Pi therapy across the lifespan on cardiovascular and renal complications, which are key aging endpoints, ii) determine the acute response to Pi loading in XLH and HHRH by studying the changes in surrogate markers of cardiovascular and renal function.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
盲法说明
crossover parallel
入排标准
- 年龄范围
- 13 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Children above the age of 13 years
- •Younger and older adults with XLH and HHRH with confirmed NPT2c mutations affecting both copies of the NPT2c gene (HHRH) or one copy of the PHEX gene (XLH)
- •Be willing to provide access to prior medical records to determine eligibility including imaging, biochemical, medical, and surgical history data
- •Be willing and able to complete all aspects of the study
- •Be willing to adhere to the study visit schedule and comply with the assessments (in the opinion of the investigator).
排除标准
- •Subjects will be excluded, if they are children younger than age 13 years
- •Subjects that have other diseases likely to impact bone and mineral metabolism (e.g. renal, hepatic, gastrointestinal disorders, and malignancy),
- •Subjects that are currently pregnant,
- •Subjects that received medical therapy or developed any condition, which in the opinion of the investigator, could present a concern for either subject safety or difficulty with data interpretation.
- •Subjects will be excluded from Aim 2, if they are unable to tolerate supplemental phosphate.
研究组 & 干预措施
Patients with hereditary hypophosphatemic rickets with HHRH
Hereditary hypophosphatemic rickets with hypercalciuria (HHRH)
干预措施: phosphate (Drug)
15 Patients Hereditary hypophosphatemic rickets with HHRH
15 patients withHereditary hypophosphatemic rickets with hypercalciuria (HHRH) that will receive phosphate treatment for 30 days.
干预措施: phosphate (Drug)
Patients with X-linked Hypophosphatemia
Patients with X-linked hypophosphatemia
干预措施: phosphate (Drug)
15 Patients with X-linked Hypophosphatemia
15 Patients with X-linked Hypophosphatemia that will receive phosphate treatment for 30 days.
干预措施: phosphate (Drug)
结局指标
主要结局
Parathyroid Hormone (PTH) levels
时间窗: 30 days
PTH levels are expected to increase over baseline after phosphate supplement (Pi).
Fibroblast Growth Factor 23 (FGF23) levels
时间窗: 30 days
FGF23 levels are expected to increase over baseline after phosphate supplement (Pi).
次要结局
未报告次要终点