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

NCT ID: NCT02763215

Last Updated: 2020-12-01

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Total Enrollment

64 participants

Study Classification

OBSERVATIONAL

Study Start Date

2016-05-19

Study Completion Date

2019-01-21

Brief Summary

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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.

Detailed Description

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Conditions

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Wilson Disease

Study Design

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Observational Model Type

COHORT

Study Time Perspective

PROSPECTIVE

Study Groups

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Total

Standard of Care Medications

Intervention Type DRUG

Standard of care medications could include penicillamine, trientine, zinc, or a combination of a copper chelator and zinc.

Interventions

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Standard of Care Medications

Standard of care medications could include penicillamine, trientine, zinc, or a combination of a copper chelator and zinc.

Intervention Type DRUG

Eligibility Criteria

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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.
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Alexion Pharmaceuticals, Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Alexion Pharmaceuticals, Inc.

Role: STUDY_DIRECTOR

Alexion Pharmaceuticals, Inc.

Locations

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Clinical Trial Site

New Haven, Connecticut, United States

Site Status

Clinical Trial Site

Chicago, Illinois, United States

Site Status

Clinical Trial Site

Ann Arbor, Michigan, United States

Site Status

Clinical Trial Site

Nashville, Tennessee, United States

Site Status

Clinical Trial Site

Seattle, Washington, United States

Site Status

Clinical Trial Site

Vienna, , Austria

Site Status

Clinical Trial Site

Heidelberg, , Germany

Site Status

Clinical Trial Site

Warsaw, , Poland

Site Status

Clinical Trial Site

Birmingham, , United Kingdom

Site Status

Clinical Trial Site

Guildford, , United Kingdom

Site Status

Countries

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United States Austria Germany Poland United Kingdom

Provided Documents

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Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

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WTX101-203

Identifier Type: -

Identifier Source: org_study_id