Frontlight ResearchEarly access
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