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

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

To achieve a normalized NCC concentration, participants must have 2 consecutive measures within (or below) the normal range (0.8 to 2.3 μM). Two consecutive measurements required that the measurements occurred on separate dates and were assigned to 2 different visits. Non-ceruloplasmin-bound copper was calculated by subtracting the amount of copper bound to the ceruloplasmin from the total plasma copper concentration. Last Assessment was the last available post-baseline result for each participant (ranging from 1 to 24 months).

Time frame: Baseline through Last Assessment (ranging from 1 to 24 months)

Change From Baseline In NCC Concentrations At Month 6, Month 24, And Last Assessment

Evaluation of NCC concentrations over time are reported as micromoles/liter (μmol/L). Last Assessment was the last available post-baseline result for each participant (ranging from 1 to 24 months).

Time frame: Baseline through Last Assessment (ranging from 1 to 24 months)

Time To Normalization Of NCC If Above The Reference Range At The Time Of Enrollment

The time to normalization of NCC was measured by Kaplan-Meier analysis.

Time frame: Baseline, up to 24 months

Change From Baseline In Exchangeable Copper At Month 6, Month 24, And Last Assessment

The change in exchangeable copper was evaluated over time and is reported as nanograms/milliliter (ng/mL). Last Assessment was the last available post-baseline result for each participant (ranging from 1 to 24 months).

Time frame: Baseline through Last Assessment (ranging from 1 to 24 months)

Change From Baseline In Copper Plasma Ultrafiltrate At Month 6, Month 24, And Last Assessment

Change in copper plasma ultrafiltrate was measured over time and is reported as ng/mL. Last Assessment was the last available post-baseline result for each participant (ranging from 1 to 24 months).

Time frame: Baseline through Last Assessment (ranging from 1 to 24 months)

Change From Baseline In Plasma Total Copper Used To Calculate NCC At Month 6, Month 24, And Last Assessment

Plasma total copper was measured over time and is reported as ng/mL. Last Assessment was the last available post-baseline result for each participant (ranging from 1 to 24 months).

Time frame: Baseline through Last Assessment (ranging from 1 to 24 months)

Change From Baseline In Serum Total Ceruloplasmin (Nephelometry) Used To Calculate NCC At Month 6, Month 24, And Last Assessment

The total serum (nephelometry) was measured over time and is reported as milligrams (mg)/L. Last Assessment was the last available post-baseline result for each participant (ranging from 1 to 24 months).

Time frame: Baseline through Last Assessment (ranging from 1 to 24 months)

Change From Baseline In 24-Hour Urinary Copper At Month 6, Month 24, And Last Assessment

Evaluation of 24-hour urinary copper over time is reported as micrograms (μg)/day. Last Assessment was the last available post-baseline result for each participant (ranging from 1 to 24 months).

Time frame: Baseline through Last Assessment (ranging from 1 to 24 months)

Change From Baseline In Plasma Total Molybdenum At Month 6 And Month 24

The plasma total molybdenum was measured over time and is reported in ng/mL.

Time frame: Baseline, Month 6, Month 24

Change From Baseline In Molybdenum Plasma Ultrafiltrate At Month 6 And Month 24

The molybdenum plasma ultrafiltrate was measured over time and is reported as ng/mL.

Time frame: Baseline, Month 6, Month 24

Change From Baseline In 24-Hour Urinary Molybdenum At Month 6 And Month 24

Evaluation of 24-hour urinary molybdenum over time is reported as μg/day.

Time frame: Baseline, Month 6, Month 24

Change From Baseline In Hepatic Laboratory Measures For Alanine Aminotransferase (ALT) At Month 6, Month 24, And Last Assessment

The ALT levels were measured over time and are reported as units (U)/L. Last Assessment was the last available post-baseline result for each participant (ranging from 1 to 24 months).

Time frame: Baseline through Last Assessment (ranging from 1 to 24 months)

Change From Baseline In Hepatic Laboratory Measures For Aspartate Aminotransferase (AST) At Month 6, Month 24, And Last Assessment

The AST levels were measured over time and are reported as U/L. Last Assessment was the last available post-baseline result for each participant (ranging from 1 to 24 months).

Time frame: Baseline through Last Assessment (ranging from 1 to 24 months)

Change From Baseline In Hepatic Laboratory Measures For Bilirubin At Month 6, Month 24, And Last Assessment

Bilirubin was measured over time and is reported as μmol/L. Last Assessment was the last available post-baseline result for each participant (ranging from 1 to 24 months).

Time frame: Baseline through Last Assessment (ranging from 1 to 24 months)

Change From Baseline In Hepatic Laboratory Measures For International Normalized Ratio (INR) At Month 6, Month 24, And Last Assessment

The INR was measured over time. Last Assessment was the last available post-baseline result for each participant (ranging from 1 to 24 months).

Time frame: Baseline through Last Assessment (ranging from 1 to 24 months)

Change From Baseline In Clinical Global Impression (CGI) Scale Item 1 (Severity Of Illness) At Month 6, Month 24, And Last Assessment

The CGI scale item 1 (severity of illness) rating scale is a commonly used measure of symptom severity, treatment response, and the efficacy of treatments in treatment studies of participants with mental disorders. It uses the Clinical Global Impression - Severity Scale (CGI-S), a 7-point scale that requires the Investigator to rate the severity of the participant's illness at the time of assessment, relative to the Investigator's past experience with participants who had the same diagnosis. Considering total clinical experience, a participant was assessed on severity of illness at the time of rating as: 1, normal, not at all ill; 2, borderline ill; 3, mildly ill; 4, moderately ill; 5, markedly ill; 6, severely ill; or 7, extremely ill. A decrease in score indicates improvement in disease severity. Last Assessment was the last available post-baseline result for each participant (ranging from 1 to 24 months).

Time frame: Baseline through Last Assessment (ranging from 1 to 24 months)

CGI Scale Item 2 (Global Improvement) At Month 6, Month 24, And Last Assessment

The CGI scale item 2 (global improvement) is a rating scale commonly used measure of symptom severity, treatment response, and the efficacy of treatments in treatment studies of participants with mental disorders. It uses the Clinical Global Impression - Improvement Scale (CGI-I), a 7-point scale that requires the Investigator to assess how much the participant's illness has improved or worsened relative to the Baseline state at the beginning of the study and rate as: 1, very much improved; 2, much improved; 3, minimally improved; 4, no change; 5, minimally worse; 6, much worse; or 7, very much worse. A decrease in score indicates improvement in disease severity. Last Assessment was the last available post-baseline result for each participant (ranging from 1 to 24 months).

Time frame: Baseline through Last Assessment (ranging from 1 to 24 months)