Clinical trial NCT02763215: 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
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Wilson Disease
Lead sponsor: Alexion Pharmaceuticals, Inc.
Sponsor class: INDUSTRY
First posted: 2016-05-05
Enrollment: 64
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: PROBABILITY_SAMPLE
Study population: Wilson disease participants receiving SoC medications for the treatment of WD. Standard of care medications could include penicillamine, trientine, zinc, or a combination of a copper chelator and zinc.

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.


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.

Primary outcomes:
measure: 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
timeFrame: Baseline through Month 6
Secondary outcomes:
measure: Percentage Of Participants Who Achieved Or Maintained Normalized Concentrations Of NCC Or Reached A Reduction Of ≥ 25% In NCC Through Last Assessment
timeFrame: Baseline through Last Assessment (ranging from 1 to 24 months)
measure: Change From Baseline In NCC Concentrations At Month 6, Month 24, And Last Assessment
timeFrame: Baseline through Last Assessment (ranging from 1 to 24 months)
measure: Time To Normalization Of NCC If Above The Reference Range At The Time Of Enrollment
timeFrame: Baseline, up to 24 months
measure: Change From Baseline In Exchangeable Copper At Month 6, Month 24, And Last Assessment
timeFrame: Baseline through Last Assessment (ranging from 1 to 24 months)
measure: Change From Baseline In Copper Plasma Ultrafiltrate At Month 6, Month 24, And Last Assessment
timeFrame: Baseline through Last Assessment (ranging from 1 to 24 months)
measure: Change From Baseline In Plasma Total Copper Used To Calculate NCC At Month 6, Month 24, And Last Assessment
timeFrame: Baseline through Last Assessment (ranging from 1 to 24 months)
measure: Change From Baseline In Serum Total Ceruloplasmin (Nephelometry) Used To Calculate NCC At Month 6, Month 24, And Last Assessment
timeFrame: Baseline through Last Assessment (ranging from 1 to 24 months)
measure: Change From Baseline In 24-Hour Urinary Copper At Month 6, Month 24, And Last Assessment
timeFrame: Baseline through Last Assessment (ranging from 1 to 24 months)
measure: Change From Baseline In Plasma Total Molybdenum At Month 6 And Month 24
timeFrame: Baseline, Month 6, Month 24
measure: Change From Baseline In Molybdenum Plasma Ultrafiltrate At Month 6 And Month 24
timeFrame: Baseline, Month 6, Month 24
measure: Change From Baseline In 24-Hour Urinary Molybdenum At Month 6 And Month 24
timeFrame: Baseline, Month 6, Month 24
measure: Change From Baseline In Hepatic Laboratory Measures For Alanine Aminotransferase (ALT) At Month 6, Month 24, And Last Assessment
timeFrame: Baseline through Last Assessment (ranging from 1 to 24 months)
measure: Change From Baseline In Hepatic Laboratory Measures For Aspartate Aminotransferase (AST) At Month 6, Month 24, And Last Assessment
timeFrame: Baseline through Last Assessment (ranging from 1 to 24 months)
measure: Change From Baseline In Hepatic Laboratory Measures For Bilirubin At Month 6, Month 24, And Last Assessment
timeFrame: Baseline through Last Assessment (ranging from 1 to 24 months)
measure: Change From Baseline In Hepatic Laboratory Measures For International Normalized Ratio (INR) At Month 6, Month 24, And Last Assessment
timeFrame: Baseline through Last Assessment (ranging from 1 to 24 months)
measure: Change From Baseline In Clinical Global Impression (CGI) Scale Item 1 (Severity Of Illness) At Month 6, Month 24, And Last Assessment
timeFrame: Baseline through Last Assessment (ranging from 1 to 24 months)
measure: CGI Scale Item 2 (Global Improvement) At Month 6, Month 24, And Last Assessment
timeFrame: Baseline through Last Assessment (ranging from 1 to 24 months)
Other outcomes:
Locations:
city: New Haven
country: United States
facility: Clinical Trial Site
state: Connecticut
city: Chicago
country: United States
facility: Clinical Trial Site
state: Illinois
city: Ann Arbor
country: United States
facility: Clinical Trial Site
state: Michigan
city: Nashville
country: United States
facility: Clinical Trial Site
state: Tennessee
city: Seattle
country: United States
facility: Clinical Trial Site
state: Washington
city: Vienna
country: Austria
facility: Clinical Trial Site
city: Heidelberg
country: Germany
facility: Clinical Trial Site
city: Warszawa
country: Poland
facility: Clinical Trial Site
city: Birmingham
country: United Kingdom
facility: Clinical Trial Site
city: Guildford
country: United Kingdom
facility: Clinical Trial Site
Study officials:
affiliation: Alexion Pharmaceuticals, Inc.
name: Alexion Pharmaceuticals, Inc.
role: STUDY_DIRECTOR
References: