Wilson Disease Standard of Care Treatment Study
Research type
Research Study
Full title
Multi-Center Study for the Assessment of Copper Parameters in Wilson Disease Subjects on Standard of Care Treatment
IRAS ID
200993
Contact name
Aftab Ala
Contact email
Sponsor organisation
Alexion Pharmaceuticals, Inc.
Duration of Study in the UK
2 years, 8 months, 1 days
Research summary
Wilson Disease is a genetic disorder that causes copper (a mineral in the body) to accumulate in the liver and other tissues in the body, which can cause organs to not work properly. The purpose of this study is to learn more about copper in blood and urine from subjects with Wilson Disease while receiving their regular medication for the condition. This information will be used to help the Sponsor Company, Wilson Therapeutics, to monitor copper levels, and may help in the future management of patients with this condition. Wilson Therapeutics will also use the information to help them evaluate the safety and effectiveness of a possible future drug that the company is working on to treat Wilson Disease. The data will be collected during routinely scheduled Wilson Disease clinic visits at approximately 6- month intervals. Data collected will include; relevant medical history and Wilson Disease medication history (including all Copper measurements and relevant clinical laboratory results for approximately one year prior to study enrollment), blood samples for assessment of plasma Copper parameters and biochemistry, hematology and coagulation measures, urine from the routine 24h-urine collection for assessment of Copper parameters, Wilson Disease medications, and subjects overall clinical status assessed by Clinical Global Impression (CGI) scale items 1 and 2. In addition to blood samples collected as Standard Of Care for analysis at the local laboratory, study blood samples of up to 30 mL (2 tablespoons) will be collected for analysis at the central laboratory at 8 time points (Enrollment, 1, 2, 3, 6, 12, 18, and 24 Months), i.e. at 5 routine clinic WD visits and at 3 additional time points.
REC name
South Central - Hampshire A Research Ethics Committee
REC reference
16/SC/0121
Date of REC Opinion
22 Feb 2016
REC opinion
Favourable Opinion