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Description of Renal Involvement in Wilson's Disease (WILKID)

F

Fondation Ophtalmologique Adolphe de Rothschild

Status

Not yet enrolling

Conditions

Wilson Disease

Treatments

Other: full renal workup

Study type

Observational

Funder types

NETWORK

Identifiers

NCT07075393
APS_2025_1

Details and patient eligibility

About

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.

Enrollment

150 estimated patients

Sex

All

Ages

7+ years old

Volunteers

No Healthy Volunteers

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

Trial design

150 participants in 1 patient group

Wilson disease patients
Description:
Patients followed for wilson disease
Treatment:
Other: full renal workup

Trial contacts and locations

1

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Central trial contact

Amelie Yavchitz, Dr

Data sourced from clinicaltrials.gov

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