The Assessment of Copper Parameters in Wilson Disease Participants on Standard of Care Treatment

Alexion Pharmaceuticals

Status: Completed

Conditions: Wilson Disease

Treatments: Drug: Standard of Care Medications

Study type: Observational

Funder types: Industry

Identifiers

NCT02763215

WTX101-203

Details and patient eligibility

About

This was a 24-month study to assess copper parameters in participants with Wilson disease (WD) treated with standard of care (SoC) medications.

After providing informed consent, participants meeting all inclusion and no exclusion criteria were enrolled into the study as outpatients. The participants' routine clinic visits were scheduled according to the standard clinical practice at the study center and at the discretion of the treating physician at approximate 6-month intervals.

At the time of enrollment, participants were receiving SoC medications for the treatment of WD, which could include penicillamine, trientine, zinc, or a combination of a copper chelator and zinc. If treatment was interrupted or stopped during the course of the study, participants continued in the study and biological samples and clinical data were continued to be collected for the full 24-month study period. Dosing with SoC agents was individualized and managed by the treating physician at the study center according to standard clinical practice at the site.

Enrollment: 64 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Willing and able to give informed consent for participation in the study.
• Male or female participants, aged 18 years or older as of signing the informed consent form.
• Receiving SoC medications (penicillamine, trientine, zinc, or copper chelators with zinc) for the treatment of WD at the time of enrollment and for no more than 60 months prior to enrollment.
• Able to understand and willing to comply with study procedures and requirements, as judged by the Investigator.
• Established diagnosis of WD.
• Adequate venous access to allow for collection of blood samples.

Exclusion criteria

• Major systemic disease or other illness that would, in the opinion of the Investigator, compromise participant safety or interfere with the collection or interpretation of study results.
• In the opinion of the Investigator, the participant was likely to be non-compliant or uncooperative during the study.

Trial design

64 participants in 1 patient group

Total

Treatment:

Drug: Standard of Care Medications