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CompletedNo phase

Real World Evidence Study in Subjects With Wilson's Disease

Key facts

Who runs it
Orphalan
Where
Ann Arbor, Baltimore, Chicago, Miami, Nashville, New Haven, New York, Orlando, and Sacramento, United StatesSee the 9 sites
Trial code
Status
Completed
People taking part
50 people enrolled
Started
28 Jun 2023
Ended
12 Sept 2025
Type of study
Observational, 4 groups
Treatments tested
  • NCC-Sp Assay
Record updated
6 April 2026

Who can take part

Age
18 years and older
Sex
Women and men

The registry criteria, word for word

Show all 9 criteriaShow less

Who can take part

  • 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

Who cannot take part

  • 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

Where in United States

  • University of MichiganAnn Arbor, United States
  • John Hopkins HospitalBaltimore, United States
  • Northwestern University Chicago (Hepatologist)Chicago, United States
  • University of MiamiMiami, United States
  • Vanderbilt University Medical CentreNashville, United States
  • Yale University School Of MedicineNew Haven, United States
  • New York Presbyterian Hospital-Columbia University Medical CentreNew York, United States
  • Advent Health OrlandoOrlando, United States
  • UC Davis Midtown Ambulatory Care CentreSacramento, United States

About the trial

What the trial wants to find out

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.

The 4 groups in the trial

  • A - Newly diagnosed

    Newly diagnosed (DPA/Trientine/Zinc for \< 28 days)

    Treatment: NCC-Sp Assay

  • B - D-penicillamine

    D-penicillamine

    Treatment: NCC-Sp Assay

  • C - Zinc

    Zinc

    Treatment: NCC-Sp Assay

  • D - Trientine (2HCl or 4HCl)

    Trientine (2HCl or 4HCl)

    Treatment: NCC-Sp Assay

Data and sources

Registry record
Listed on ClinicalTrials.gov.
Official title
RWE Study of Subjects With Wilson's Disease to Assess Clinical Utility of Measuring Copper Parameters, Including a Novel Non-ceruloplasmin Bound Copper Assay Based on Copper Protein Speciation (NCC-Sp), Standard of Care Clinical and Biochemical Assessments
Record updated
6 April 2026