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Résztvevőt keres itt: Egyesült ÁllamokFázis nélkül

Off Treatment Urinary Copper Excretion in Wilson Disease, Pilot Study

A legfontosabb adatok

Ki végzi
Yale University
Hol
New Haven (Egyesült Államok)A helyszín megnézése
Azonosító
Állapot
Toboroz
Résztvevők
30 fő, tervezett
Kezdés
2026. jan. 8.
Várható befejezés
2028. szept. 30.
A vizsgálat típusa
Megfigyeléses, 3 csoport
A regiszteradat frissítve
2026. március 5.

Ki vehet részt

MI-összefoglaló

A vizsgálat saját részvételi feltételeiből. Gép olvasta ki az oldalon álló regiszterszövegből, ezért tévedhet. Hogy ki vehet részt, azt a lenti regiszterszöveg és a vizsgálat csapata dönti el.

The trial is looking for adults with stable Wilson disease who have been treated for at least 12 months. Their treatment will be paused for a while under close monitoring to measure copper in urine and blood.

Életkor
18 Years and older
Nem
All sexes
A betegség stádiuma
Wilson disease with a Leipzig score of 4 or more. The disease must be stable: liver enzymes (ASAT and ALAT) below 2 times the upper limit of normal, INR below 1.5 or stable for at least six months without blood thinners, and kidney function (eGFR) above 30 cc/min.
Korábbi kezelés
Treatment for Wilson disease for at least 12 months before joining. No change in this treatment in the last 6 months. Only one Wilson disease medicine at a time (zinc, trientine or penicillamine).
Biomarker vagy leletérték
Leipzig score of 4 or more.

A részvétel fő kizáró okai

  • Taking two Wilson disease medicines at the same time, such as zinc together with d-penicillamine or trientine
  • Being pregnant or breastfeeding; people who could become pregnant need negative urine pregnancy tests
  • Estrogen-based treatment in the last month
  • Cirrhosis with a recent worsening of the liver in the last 6 months, such as new fluid in the belly, belly infection, bleeding from veins in the food pipe, or confusion caused by the liver
  • Not taking treatment or not following the low-copper diet in the past, or being unable to do the 24-hour urine collections and follow-up visits

A regiszter feltételei, szó szerint

Mind a 14 feltétel megnézéseKevesebb

Ki vehet részt

  • 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

Ki nem vehet részt

  • Current dual / mixed therapy for WD (i.e. zinc and d-penicillamine or trientine at the same time)
  • 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)
  • Cirrhosis with recent hepatic decompensation (within the last 6 months) - new onset of ascites, spontaneous bacterial peritonitis, esophageal variceal bleeding, or hepatic encephalopathy
  • Current Pregnancy or lactation. *
  • Recent estrogen-based treatment (in the last month)
  • Investigator believes the patient will be unable to do the required 24-hour urine studies and participate in the follow up visits as expected

Helyszínek itt: Egyesült Államok

  • Yale School of MedicineNew Haven, Egyesült ÁllamokContact: Sefa Keserci, MDRésztvevőt keres

A vizsgálatról

Mit akar kideríteni a vizsgálat

This is a prospective study that will determine the optimal timing for 24-hour urinary copper excretion (UCE) measurement after temporary discontinuation of standard therapies in Wilson Disease (WD) patients. The primary objective is to assess whether off-treatment UCE (OT-UCE) correlates with non-ceruloplasmin-bound copper (NCC) levels, aiming to validate OT-UCE as a surrogate marker for systemic copper bioavailability and disease stability. Stable WD patients will be enrolled, temporarily taken off treatment under close monitoring, and undergo UCE and NCC testing. If OT-UCE is validated, it could serve as a practical biomarker for monitoring WD treatment and stability in clinical practice and future trials.

A vizsgálat 3 csoportja

  • OT-UCE and NCC for Zinc treated WD patients

    OT-UCE and NCC will be measured in WD patients on Zinc therapy

  • OT-UCE and NCC for Trientine treated WD patients

    OT-UCE and NCC will be measured in WD patients on trientine therapy

  • OT-UCE and NCC for Penicillamine treated WD patients

    OT-UCE and NCC will be measured in WD patients on Penicillamine therapy

Kérdések a vizsgálat csapatának

  1. Milyen gyakran kell bejárnom, és egy-egy alkalom mennyi ideig tart?
  2. Megtérítik az utazási költséget?
  3. Megtarthatom a saját orvosomat?
  4. Mi történik, ha ki akarok lépni?

Contact the study team: see the registry record → Contacts and Locations

Oldal nyomtatása az orvosomnak

Adatok és források

Regiszteradat
Szerepel a ClinicalTrials.gov-on.
Hivatalos cím
Monitoring of Therapy in Wilson Disease With Off-Treatment Urinary Copper Excretion (OT-UCE): Comparison With Serum Non-Ceruloplasmin Copper (NCC) Assays
MI-összefoglaló
A vizsgálat saját részvételi feltételeiből. Gépi kiolvasás: 2026. november 15.
A regiszteradat frissítve
2026. március 5.