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临床试验/NCT05699265
NCT05699265终止不适用

Cardiovasculorenal Phenotyping in Fabry Disease Through Noninvasive Testing

Children's Hospital Medical Center, Cincinnati1 个研究点 分布在 1 个国家目标入组 4 人开始时间: 2023年2月1日最近更新:
适应症

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

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

研究者

申办方类型
Other
责任方
Sponsor

研究点 (1)

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