Pausing wilson disease drugs for better monitoring?
NCT ID NCT07301216
First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This study looks at whether briefly stopping Wilson disease medication can give a clearer picture of copper levels in the body. Researchers will monitor 30 stable patients for 4 days off treatment, measuring urine copper and blood markers. The goal is to find a better way to track disease stability without relying on long-term treatment changes.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- What this could lead to
- If successful, this could give doctors a simpler, more reliable way to monitor Wilson disease stability without needing frequent blood tests.
- What could go wrong
- This is a small, early study with only 30 people. Stopping medication temporarily carries risks of copper buildup, and the findings may not apply to all patients.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Participants
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About 30 people
The number the study aims to enrol. It can still change while the study runs.
- Started
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Jan 2026
- Expected to finish
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Sep 2028
An estimate. End dates often move.
- Lead sponsor
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Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
Who is studied
Patients with confirmed Wilson Disease (Leipzig score ≥4), and stable disease will be screened and recruited for this pilot. This will include patients 18 years old and above as the intervention for this study is a temporary discontinuation of therapy, and this group of patients will tolerate this intervention with minimal risk.
- Ages
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18 years and older
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: * 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 Criteria: * 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. Childbearing aged patients recruited from the registry who meet inclusion criteria and may move directly to the study intervention will be required to perform a urine pregnancy test as close as possible to the time prior to the initiation of the study protocol (discontinuation of treatment).
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
How to take part
Only the study team decides who joins. These are the ways to reach them.
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The places running it
1 site. The list below names each one and where it is.
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The official record
ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.
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A doctor treating you
A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.
Contacts and locations
Locations
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Yale School of Medicine
RECRUITINGNew Haven, Connecticut, 06520, United States
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