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NCT Number: NCT07075393

Description of Renal Involvement in Wilson's Disease

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.

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Key information

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

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

Exclusion criteria

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

Treatment and study plan

full renal workup

Other

full renal workup (biology and echography)

Primary outcomes

  1. Prevalence of renal involvement in Wilson's disease

    Time frame: 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.

Study contacts

Contact information is provided by the study sponsor or research team.

Amelie Yavchitz, Dr

CONTACT

[email protected]

+33148036454

Sponsors and collaborators

Lead sponsor

Fondation Ophtalmologique Adolphe de Rothschild

Network

Registry information

Acronym: WILKID

Important dates

Study start
2026
Primary completion
2027
Study completion
2027
First posted
Jul 20, 2025
Registry last updated
Dec 10, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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