CompletedNot Applicable

Real World Evidence Study in Subjects With Wilson's Disease

United States50 participantsStarted 2023-06-28

Plain-language summary

This non-interventional Real-World Evidence (RWE) study aims to describe non-ceruloplasmin copper values obtained using a new NCC Speciation assay by taking a small (up to 10mLs) volume of additional blood from patients with Wilson's Disease, around the time when routine blood sampling is expected to be scheduled by the treating physician. Data will be collected over an approximate 12-month period.

Who can participate

Age range18 Years

SexALL

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Inclusion criteria

✓. Willing and able to give informed consent for participation in the study
✓. Male or female patients, aged 18 years or older as of signing the Informed Consent Form (ICF)
✓. Able and willing to comply with study procedures and requirements, as judged by the treating physician
✓. Established diagnosis of Wilson's Disease (Leipzig score \>4)
✓. Either newly diagnosed or with elevated liver enzymes (defined as ALT, AST ≥1.5xULN) or 24-hour urinary copper excretion outside of recommended ranges \[chelation range 200-500mcg/24 hr; zinc \< 100mcg/24 hr\]
✓. Adequate venous access to allow collection of blood samples

Exclusion criteria

✕. Major systemic disease or other illness that would, in the opinion of the investigator, compromise patient safety or interfere with the collection or interpretation of the study results.
✕. Patients with a New Wilson's Disease Index Score of \>7
✕. In the opinion of the investigator, the patient is likely to be non-compliant or uncooperative for routine clinical visits during the study.

What they're measuring

The distribution of Non-Ceruloplasmin bound Copper (NCC) in serum as assessed by a NCC-speciation LC-ICP-MS assay (NCC-Sp), at study entry and over an approximate 12-month period from a real-world population of WD patients.

Timeframe: Approx 12 Months

Trial details

NCT IDNCT05783687

SponsorOrphalan

Sponsor typeINDUSTRY

Study typeOBSERVATIONAL

Primary completion2025-09-12

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