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Observational Study: Off-Treatment Urinary Copper Excretion in Wilson Disease

This observational study aims to find the best time to measure copper in your urine after you temporarily stop your Wilson Disease (WD) medication. Researchers want to see if this "off-treatment urinary copper excretion" (OT-UCE) measurement matches another copper level called non-ceruloplasmin-bound copper (NCC). If it does, OT-UCE could become a simpler way to check how well your WD treatment is working and if your disease is stable. You can join if you are an adult with Wilson Disease (Leipzig score ≥4) and are willing to follow study procedures, including collecting urine samples. The study plans to enroll 30 participants. Success would mean showing that OT-UCE is a reliable way to monitor WD.

Study design
This is an observational study with 30 planned participants. It is designed to determine the optimal timing for measuring urinary copper excretion after temporarily stopping standard Wilson Disease treatments.
What's involved
You will provide multiple 24-hour urine samples, have blood drawn for copper and liver function tests, and attend an in-person end-of-study visit. You will briefly stop your current Wilson Disease treatment and communicate daily with study staff during this time.
Compensation
Not stated in the trial record.
Follow-up
Measurements for the primary endpoints are taken at days 1, 2, 3, and 4 after stopping Wilson Disease medications.

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NCT07301216

Off Treatment Urinary Copper Excretion in Wilson Disease, Pilot Study

Recruiting
Not specifiedAges 18+Observational
Yale University
~30 participants
Updated 2026-03-05 on ClinicalTrials.gov

At a glance

Recruiting sites
1 of 1 listed site is recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Mean concentration of OT-UCE for each standard of care WD treatment
Measured over days 1, 2, 3 and 4 post stopping WD meds
+1 more outcome measured

Conditions

Wilson Disease

Explore related trials

Hepatolenticular Degeneration

Where it's being run

1 sites across 1 states
Connecticut1

Study leadership

  • Michael L Schilsky, MD FAASLD · PRINCIPAL_INVESTIGATOR · Yale University

Who to contact

Sefa Keserci, MD
[email protected]
203 737 2702
Email the study team

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Eligibility criteria

Inclusion

Patients with Wilson Disease as defined by Leipzig score ≥4.
Provision of signed and dated informed consent form.
Stated willingness to comply with all study procedures (serial 24 h urine collections and local collection of samples for NCC, liver function and estimated GFR) and availability for the duration of the study.
Treated WD for at least 12 months prior to study entry.
Aminotransferase values (ASAT and ALAT) \< 2 times the upper limit of normal (ULN).
INR \< 1.5 or stable INR for those with initial elevated INR for at least six months prior to study entry in the absence of anticoagulation therapy.
Renal function defined as eGFR \> 30 cc/min.
No change of WD therapy during the previous 6 months of study enrollment.

Exclusion

Current dual / mixed therapy for WD (i.e. zinc and d-penicillamine or trientine at the same time)
Current Pregnancy or lactation. \*
Recent estrogen-based treatment (in the last month).
Cirrhosis with recent hepatic decompensation (within the last 6 months) - new onset of ascites, spontaneous bacterial peritonitis, esophageal variceal bleeding, or hepatic encephalopathy
Investigator believes the patient will be unable to do the required 24-hour urine studies and participate in the follow up visits as expected.
Previous non-compliance for therapy and/or to low-copper diet that would compromise the evaluation of previous UCE and/ or results from the off-treatment period.
Childbearing aged patients recruited outside of the registry will be reviewed, and the patients will be asked to perform an initial urine pregnancy test prior to the recommended blood testing (approximately 60 to 90 days prior to intervention). They will be permitted to continue with the screening process if the result is negative. They will be asked to perform a second urine pregnancy test as close as possible prior to study intervention (discontinuation of treatment). If the result of the second pregnancy test is negative they will be permitted to continue with the protocol, but if the result is positive they will be excluded from further participation at that time.

What this trial measures

  • Mean concentration of OT-UCE for each standard of care WD treatmentdays 1, 2, 3 and 4 post stopping WD meds

    Urine samples will be collected daily for 4 days after stopping WD medications. The sequential evaluation of OT-UCE over a maximum of 4 days after treatment withdrawal will allow investigators to define the optimal ranges for UCE and select the best time-point for OT-UCE evaluations for WD patients on the 3 different therapies.

  • Mean NCC concentration for each WD treatmentdays 1, 2, 3 and 4 post stopping WD meds

    Measure NCC and assess the correlation between NCC and OT-UCE Urine samples will be collected daily for 4 days after stopping WD medications. The sequential evaluation of NCC over a maximum of 4 days after treatment withdrawal will allow investigators to assess the correlation between NCC and OT-UCE.