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临床试验/NCT07075393
NCT07075393尚未招募不适用

Description of Renal Involvement in Wilson's Disease

Fondation Ophtalmologique Adolphe de Rothschild1 个研究点 分布在 1 个国家目标入组 150 人开始时间: 2026年2月1日最近更新:

试验速览

阶段
不适用
状态
尚未招募
入组人数
150
试验地点
1
主要终点
Prevalence of renal involvement in Wilson's disease

研究概览

简要总结

Wilson's disease (WD) is a rare genetic disorder that leads to copper accumulation in various tissues, including the liver, nervous system, heart, and kidneys. Renal involvement in WD has been poorly studied, and systematic screening is not currently recommended.

Indirect renal complications are the most common, such as hepatorenal and cardiorenal syndromes, as well as severe complications like hemolysis or rhabdomyolysis. However, literature suggests that copper may exert a direct toxic effect on renal tubular cells, leading to both proximal and distal tubular dysfunction. These may manifest through often subtle signs, such as aminoaciduria, glycosuria, hypouricemia, and low-molecular-weight proteinuria. Electrolyte imbalances of varying severity may also occur, including hypokalemia, which can cause muscle cramps and cardiac arrhythmias, as well as acid-base disorders (proximal or distal renal tubular acidosis), and/or phosphate-calcium metabolism abnormalities (phosphate diabetes and hypercalciuria). These latter issues may lead to complications such as urinary stones, nephrocalcinosis, and even fracture-related osteoporosis.

In addition, long-term treatment with D-penicillamine (DPA), a common therapy for WD, can cause renal damage in 10-20% of cases, mainly affecting the glomeruli. This includes membranous nephropathy, severe proliferative glomerulonephritis, or nephrotic syndrome with minimal change disease.

Without appropriate monitoring and preventive care, both direct and indirect renal complications can lead to acute or chronic kidney failure. It is likely that the prevalence and systemic impact of renal involvement in WD are currently underestimated.

研究设计

研究类型
Observational
观察模型
Case Only
时间视角
Cross Sectional

入排标准

年龄范围
7 Years 至 —(Child, Adult, Older Adult)
性别
All
接受健康志愿者

入选标准

  • Patient aged 7 years and older
  • Diagnosed with Wilson's disease, with a Leipzig score ≥ 4

排除标准

  • Patient who has undergone organ transplantation
  • Known renal comorbidity unrelated to Wilson's disease
  • Pregnant or breastfeeding woman

结局指标

主要结局

Prevalence of renal involvement in Wilson's disease

时间窗: Day 0

Renal involvement is defined by the presence of at least one of the following criteria: * Chronic kidney disease, defined by an estimated glomerular filtration rate (eGFR) \< 60 mL/min/1.73 m² and/or proteinuria \> 0.5 g/g of creatinine. * Markers of proximal tubulopathy, defined by at least one of the following: phosphate diabetes (renal phosphate wasting), hypokalemia, normoglycemic glycosuria, β2-microglobulinuria, hypouricemia, or metabolic acidosis. * Hypercalciuria and/or urolithiasis.

次要结局

未报告次要终点

研究者

申办方类型
Network
责任方
Sponsor

研究点 (1)

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