Cardiovasculorenal Phenotyping in Fabry Disease Through Noninvasive Testing
试验速览
- 阶段
- 不适用
- 状态
- 终止
- 入组人数
- 4
- 试验地点
- 1
- 主要终点
- Peak systolic blood pressure
研究概览
简要总结
A longitudinal pilot study will be conducted to determine if there are additional testing modalities that are effective in broadly phenotyping subclinical dysfunction in patients with Fabry disease. Individual patients will undergo serial testing over a two-year period to evaluate for changes in their cardiovasculaorenal function during this period. Novel modalities evaluated will include measures of arterial stiffness, ambulatory blood pressure monitoring, cardiopulmonary exercise testing (CPET), and novel serum and urine biomarkers. The benefit of these measures being evaluated is that they are noninvasive, can be performed rapidly, and have reduced costs compared to the current standard screening modalities. Results from these evaluations will be compared to cMRI and standard urine and serum biomarkers performed clinically per local standard of care. The results will also be compared to both published normative data and data from patients with diabetes mellitus, who have a similar microvascular disease process to patients with Fabry disease.
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
- 8 Years 至 —(Child, Adult, Older Adult)
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Fabry patients with classical disease
- •English speaking, which is needed to assist with obtaining a maximal effort CPET
- •No medical contraindications to cardiopulmonary exercise testing or cMRI
- •Either treatment naïve or current taking ERT
排除标准
- •Physical limitation that would preclude exercise
- •Currently prescribed non-ERT treatments for Fabry disease
结局指标
主要结局
Peak systolic blood pressure
时间窗: through study completion, approximately 22 months
The mean peak systolic blood pressure for Fabry disease patients is assumed to be 160 mmHg
次要结局
未报告次要终点
