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

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Brief Title

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

Official Title

Multi-Center Study for the Assessment of Copper Parameters in Wilson Disease Subjects on Standard of Care Treatment

Brief Summary

      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.
    



Study Type

Observational


Primary Outcome

Percentage Of Participants Who Achieved Or Maintained Normalized Concentrations Of Non-ceruloplasmin-bound Copper (NCC) Or Reached A Reduction Of ≥ 25% In NCC During 6 Months Of Treatment

Secondary Outcome

 Percentage Of Participants Who Achieved Or Maintained Normalized Concentrations Of NCC Or Reached A Reduction Of ≥ 25% In NCC Through Last Assessment

Condition

Wilson Disease

Intervention

Standard of Care Medications

Study Arms / Comparison Groups

 Total
Description:  

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Drug

Estimated Enrollment

64

Start Date

May 19, 2016

Completion Date

January 21, 2019

Primary Completion Date

January 21, 2019

Eligibility Criteria

        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.
      

Gender

All

Ages

18 Years - N/A

Accepts Healthy Volunteers

No

Contacts

Alexion Pharmaceuticals, Inc., , 

Location Countries

Austria

Location Countries

Austria

Administrative Informations


NCT ID

NCT02763215

Organization ID

WTX101-203


Responsible Party

Sponsor

Study Sponsor

Alexion Pharmaceuticals


Study Sponsor

Alexion Pharmaceuticals, Inc., Study Director, Alexion Pharmaceuticals


Verification Date

October 2020