Trial Outcomes & Findings for The Assessment of Copper Parameters in Wilson Disease Participants on Standard of Care Treatment (NCT NCT02763215)

NCT ID: NCT02763215

Last Updated: 2020-12-01

Results Overview

To achieve a normalized NCC concentration, participants must have had 2 consecutive measures within (or below) the normal concentration range (0.8 to 2.3 micromolar \[μM\]). 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.

• Recruitment status: COMPLETED
• Target enrollment: 64 participants
• Primary outcome timeframe: Baseline through Month 6
• Results posted on: 2020-12-01

Participant Flow

Participant milestones

Measure	Total Participants in this study were not treated with any investigational products, but with standard of care (SoC) medications, which could include penicillamine, trientine, zinc, or a combination of a copper chelator and zinc.
Overall Study STARTED	64
Overall Study COMPLETED	57
Overall Study NOT COMPLETED	7

Reasons for withdrawal

Measure	Total Participants in this study were not treated with any investigational products, but with standard of care (SoC) medications, which could include penicillamine, trientine, zinc, or a combination of a copper chelator and zinc.
Overall Study Participants withdrew from study	3
Overall Study Missed visit	1
Overall Study Visit outside visit-window	1
Overall Study Death	1
Overall Study Lost to Follow-up	1

Baseline Characteristics

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

Baseline characteristics by cohort

Measure	Total n=64 Participants Participants in this study were not treated with any investigational products, but with SoC medications, which could include penicillamine, trientine, zinc, or a combination of a copper chelator and zinc.
Age, Continuous	32.0 years STANDARD_DEVIATION 11.97 • n=5 Participants
Sex: Female, Male Female	36 Participants n=5 Participants
Sex: Female, Male Male	28 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	1 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	62 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	1 Participants n=5 Participants
Race/Ethnicity, Customized Race · Asian	2 Participants n=5 Participants
Race/Ethnicity, Customized Race · Black or African American	1 Participants n=5 Participants
Race/Ethnicity, Customized Race · White	57 Participants n=5 Participants
Race/Ethnicity, Customized Race · Other	4 Participants n=5 Participants

PRIMARY outcome

Timeframe: Baseline through Month 6

Population: Full Analysis Set: Participants enrolled in the study. To be eligibly enrolled, participants must have been receiving the 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.

To achieve a normalized NCC concentration, participants must have had 2 consecutive measures within (or below) the normal concentration range (0.8 to 2.3 micromolar [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.

Outcome measures

Measure	Total n=64 Participants Participants in this study were not treated with any investigational products, but with SoC medications, which could include penicillamine, trientine, zinc, or a combination of a copper chelator and zinc.
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	51.6 Percentage of participants Interval 51.2 to 78.8

SECONDARY outcome

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

Population: Full Analysis Set: Participants enrolled in the study. To be eligibly enrolled, participants must have been receiving the 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.

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).

Outcome measures

Measure	Total n=64 Participants Participants in this study were not treated with any investigational products, but with SoC medications, which could include penicillamine, trientine, zinc, or a combination of a copper chelator and zinc.
Percentage Of Participants Who Achieved Or Maintained Normalized Concentrations Of NCC Or Reached A Reduction Of ≥ 25% In NCC Through Last Assessment	62.5 Percentage of participants Interval 59.0 to 83.9

SECONDARY outcome

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

Population: Full Analysis Set: Enrolled participants. To be eligibly enrolled, participants must have been receiving the 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. The number of participants analyzed per row represents number of overall participants continuing in the study at each timepoint with non-missing and non-negative baseline and post-baseline values for NCC.

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).

Outcome measures

Measure	Total n=64 Participants Participants in this study were not treated with any investigational products, but with SoC medications, which could include penicillamine, trientine, zinc, or a combination of a copper chelator and zinc.
Change From Baseline In NCC Concentrations At Month 6, Month 24, And Last Assessment Month 6	-0.60 μmol/L Standard Deviation 1.441
Change From Baseline In NCC Concentrations At Month 6, Month 24, And Last Assessment Month 24	-1.24 μmol/L Standard Deviation 1.905
Change From Baseline In NCC Concentrations At Month 6, Month 24, And Last Assessment Last Assessment	-0.94 μmol/L Standard Deviation 1.811

SECONDARY outcome

Timeframe: Baseline, up to 24 months

Population: Full Analysis Set with an elevated NCC at Baseline.

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

Outcome measures

Measure	Total n=12 Participants Participants in this study were not treated with any investigational products, but with SoC medications, which could include penicillamine, trientine, zinc, or a combination of a copper chelator and zinc.
Time To Normalization Of NCC If Above The Reference Range At The Time Of Enrollment	139.5 Days Interval 62.0 to 545.0

SECONDARY outcome

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

Population: Full Analysis Set: Enrolled participants. To be eligibly enrolled, participants must have been receiving the 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. The number of participants analyzed per row represents number of overall participants continuing in the study at each timepoint with non-missing baseline and post-baseline values for each outcome measure.

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).

Outcome measures

Measure	Total n=64 Participants Participants in this study were not treated with any investigational products, but with SoC medications, which could include penicillamine, trientine, zinc, or a combination of a copper chelator and zinc.
Change From Baseline In Exchangeable Copper At Month 6, Month 24, And Last Assessment Month 6	-7.9 ng/mL Standard Deviation 26.02
Change From Baseline In Exchangeable Copper At Month 6, Month 24, And Last Assessment Month 24	-9.5 ng/mL Standard Deviation 27.61
Change From Baseline In Exchangeable Copper At Month 6, Month 24, And Last Assessment Last Assessment	-8.3 ng/mL Standard Deviation 27.25

SECONDARY outcome

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

Population: Full Analysis Set: Enrolled participants. To be eligibly enrolled, participants must have been receiving the 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. The number of participants analyzed per row represents number of overall participants continuing in the study at each timepoint with non-missing baseline and post-baseline values for each outcome measure.

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).

Outcome measures

Measure	Total n=64 Participants Participants in this study were not treated with any investigational products, but with SoC medications, which could include penicillamine, trientine, zinc, or a combination of a copper chelator and zinc.
Change From Baseline In Copper Plasma Ultrafiltrate At Month 6, Month 24, And Last Assessment Month 6	-3.96 ng/mL Standard Deviation 13.082
Change From Baseline In Copper Plasma Ultrafiltrate At Month 6, Month 24, And Last Assessment Month 24	0.00 ng/mL Standard Deviation 30.295
Change From Baseline In Copper Plasma Ultrafiltrate At Month 6, Month 24, And Last Assessment Last Assessment	-0.68 ng/mL Standard Deviation 28.470

SECONDARY outcome

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

Population: Full Analysis Set: Enrolled participants. To be eligibly enrolled, participants must have been receiving the 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. The number of participants analyzed per row represents number of overall participants continuing in the study at each timepoint with non-missing baseline and post-baseline values for each outcome measure.

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).

Outcome measures

Measure	Total n=64 Participants Participants in this study were not treated with any investigational products, but with SoC medications, which could include penicillamine, trientine, zinc, or a combination of a copper chelator and zinc.
Change From Baseline In Plasma Total Copper Used To Calculate NCC At Month 6, Month 24, And Last Assessment Month 6	0.0 ng/mL Interval -48.0 to 33.0
Change From Baseline In Plasma Total Copper Used To Calculate NCC At Month 6, Month 24, And Last Assessment Month 24	0.0 ng/mL Interval -76.0 to 19.0
Change From Baseline In Plasma Total Copper Used To Calculate NCC At Month 6, Month 24, And Last Assessment Last Assessment	0.0 ng/mL Interval -76.0 to 36.0

SECONDARY outcome

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

Population: Full Analysis Set: Enrolled participants. To be eligibly enrolled, participants must have been receiving the 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. The number of participants analyzed per row represents number of overall participants continuing in the study at each timepoint with non-missing baseline and post-baseline values for each outcome measure.

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).

Outcome measures

Measure	Total n=64 Participants Participants in this study were not treated with any investigational products, but with SoC medications, which could include penicillamine, trientine, zinc, or a combination of a copper chelator and zinc.
Change From Baseline In Serum Total Ceruloplasmin (Nephelometry) Used To Calculate NCC At Month 6, Month 24, And Last Assessment Month 6	0.0 mg/L Interval -4.0 to 14.0
Change From Baseline In Serum Total Ceruloplasmin (Nephelometry) Used To Calculate NCC At Month 6, Month 24, And Last Assessment Month 24	0.0 mg/L Interval -4.5 to 20.5
Change From Baseline In Serum Total Ceruloplasmin (Nephelometry) Used To Calculate NCC At Month 6, Month 24, And Last Assessment Last Assessment	0.0 mg/L Interval -4.5 to 20.0

SECONDARY outcome

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

Population: Full Analysis Set: Enrolled participants. To be eligibly enrolled, participants must have been receiving the 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. The number of participants analyzed per row represents number of overall participants continuing in the study at each timepoint with non-missing baseline and post-baseline values for each outcome measure.

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).

Outcome measures

Measure	Total n=64 Participants Participants in this study were not treated with any investigational products, but with SoC medications, which could include penicillamine, trientine, zinc, or a combination of a copper chelator and zinc.
Change From Baseline In 24-Hour Urinary Copper At Month 6, Month 24, And Last Assessment Month 6	-64.7 μg/day Standard Deviation 353.63
Change From Baseline In 24-Hour Urinary Copper At Month 6, Month 24, And Last Assessment Month 24	-113.4 μg/day Standard Deviation 295.76
Change From Baseline In 24-Hour Urinary Copper At Month 6, Month 24, And Last Assessment Last Assessment	-100.2 μg/day Standard Deviation 333.70

SECONDARY outcome

Timeframe: Baseline, Month 6, Month 24

Population: Full Analysis Set: Enrolled participants. To be eligibly enrolled, participants must have been receiving the 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. The number of participants analyzed per row represents number of overall participants continuing in the study at each timepoint with non-missing baseline and post-baseline values for each outcome measure.

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

Outcome measures

Measure	Total n=64 Participants Participants in this study were not treated with any investigational products, but with SoC medications, which could include penicillamine, trientine, zinc, or a combination of a copper chelator and zinc.
Change From Baseline In Plasma Total Molybdenum At Month 6 And Month 24 Month 6	-4.9 ng/mL Standard Deviation 25.60
Change From Baseline In Plasma Total Molybdenum At Month 6 And Month 24 Month 24	-5.2 ng/mL Standard Deviation 26.53

SECONDARY outcome

Timeframe: Baseline, Month 6, Month 24

Population: Full Analysis Set: Enrolled participants. To be eligibly enrolled, participants must have been receiving the 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. The number of participants analyzed per row represents number of overall participants continuing in the study at each timepoint with non-missing baseline and post-baseline values for each outcome measure.

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

Outcome measures

Measure	Total n=64 Participants Participants in this study were not treated with any investigational products, but with SoC medications, which could include penicillamine, trientine, zinc, or a combination of a copper chelator and zinc.
Change From Baseline In Molybdenum Plasma Ultrafiltrate At Month 6 And Month 24 Month 6	-2.50 ng/mL Standard Deviation 10.963
Change From Baseline In Molybdenum Plasma Ultrafiltrate At Month 6 And Month 24 Month 24	-2.89 ng/mL Standard Deviation 11.163

SECONDARY outcome

Timeframe: Baseline, Month 6, Month 24

Population: Full Analysis Set: Enrolled participants. To be eligibly enrolled, participants must have been receiving the 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. The number of participants analyzed per row represents number of overall participants continuing in the study at each timepoint with non-missing baseline and post-baseline values for each outcome measure.

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

Outcome measures

Measure	Total n=64 Participants Participants in this study were not treated with any investigational products, but with SoC medications, which could include penicillamine, trientine, zinc, or a combination of a copper chelator and zinc.
Change From Baseline In 24-Hour Urinary Molybdenum At Month 6 And Month 24 Month 24	2.0 μg/day Interval -18.5 to 21.0
Change From Baseline In 24-Hour Urinary Molybdenum At Month 6 And Month 24 Month 6	2.5 μg/day Interval -11.5 to 25.0

SECONDARY outcome

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

Population: Full Analysis Set: Enrolled participants. To be eligibly enrolled, participants must have been receiving the 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. The number of participants analyzed per row represents number of overall participants continuing in the study at each timepoint with non-missing baseline and post-baseline values for each outcome measure.

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).

Outcome measures

Measure	Total n=64 Participants Participants in this study were not treated with any investigational products, but with SoC medications, which could include penicillamine, trientine, zinc, or a combination of a copper chelator and zinc.
Change From Baseline In Hepatic Laboratory Measures For Alanine Aminotransferase (ALT) At Month 6, Month 24, And Last Assessment Month 6	0.8 U/L Standard Deviation 29.53
Change From Baseline In Hepatic Laboratory Measures For Alanine Aminotransferase (ALT) At Month 6, Month 24, And Last Assessment Month 24	-1.6 U/L Standard Deviation 28.09
Change From Baseline In Hepatic Laboratory Measures For Alanine Aminotransferase (ALT) At Month 6, Month 24, And Last Assessment Last Assessment	-1.5 U/L Standard Deviation 28.55

SECONDARY outcome

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

Population: Full Analysis Set: Enrolled participants. To be eligibly enrolled, participants must have been receiving the 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. The number of participants analyzed per row represents number of overall participants continuing in the study at each timepoint with non-missing baseline and post-baseline values for each outcome measure.

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).

Outcome measures

Measure	Total n=64 Participants Participants in this study were not treated with any investigational products, but with SoC medications, which could include penicillamine, trientine, zinc, or a combination of a copper chelator and zinc.
Change From Baseline In Hepatic Laboratory Measures For Aspartate Aminotransferase (AST) At Month 6, Month 24, And Last Assessment Month 6	0.3 U/L Standard Deviation 12.69
Change From Baseline In Hepatic Laboratory Measures For Aspartate Aminotransferase (AST) At Month 6, Month 24, And Last Assessment Month 24	-0.7 U/L Standard Deviation 11.86
Change From Baseline In Hepatic Laboratory Measures For Aspartate Aminotransferase (AST) At Month 6, Month 24, And Last Assessment Last Assessment	-0.6 U/L Standard Deviation 12.54

SECONDARY outcome

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

Population: Full Analysis Set: Enrolled participants. To be eligibly enrolled, participants must have been receiving the 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. The number of participants analyzed per row represents number of overall participants continuing in the study at each timepoint with non-missing baseline and post-baseline values for each outcome measure.

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).

Outcome measures

Measure	Total n=64 Participants Participants in this study were not treated with any investigational products, but with SoC medications, which could include penicillamine, trientine, zinc, or a combination of a copper chelator and zinc.
Change From Baseline In Hepatic Laboratory Measures For Bilirubin At Month 6, Month 24, And Last Assessment Month 6	0.2 μmol/L Standard Deviation 6.10
Change From Baseline In Hepatic Laboratory Measures For Bilirubin At Month 6, Month 24, And Last Assessment Month 24	0.5 μmol/L Standard Deviation 7.11
Change From Baseline In Hepatic Laboratory Measures For Bilirubin At Month 6, Month 24, And Last Assessment Last Assessment	0.5 μmol/L Standard Deviation 6.88

SECONDARY outcome

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

Population: Full Analysis Set: Enrolled participants. To be eligibly enrolled, participants must have been receiving the 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. The number of participants analyzed per row represents number of overall participants continuing in the study at each timepoint with non-missing baseline and post-baseline values for each outcome measure.

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

Outcome measures

Measure	Total n=64 Participants Participants in this study were not treated with any investigational products, but with SoC medications, which could include penicillamine, trientine, zinc, or a combination of a copper chelator and zinc.
Change From Baseline In Hepatic Laboratory Measures For International Normalized Ratio (INR) At Month 6, Month 24, And Last Assessment Month 6	-0.02 Ratio Standard Deviation 0.116
Change From Baseline In Hepatic Laboratory Measures For International Normalized Ratio (INR) At Month 6, Month 24, And Last Assessment Month 24	-0.01 Ratio Standard Deviation 0.114
Change From Baseline In Hepatic Laboratory Measures For International Normalized Ratio (INR) At Month 6, Month 24, And Last Assessment Last Assessment	0.00 Ratio Standard Deviation 0.114

SECONDARY outcome

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

Population: Full Analysis Set: Enrolled participants. To be eligibly enrolled, participants must have been receiving the 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. The number of participants analyzed per row represents number of overall participants continuing in the study at each timepoint with non-missing baseline and post-baseline values for each outcome measure.

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).

Outcome measures

Measure	Total n=64 Participants Participants in this study were not treated with any investigational products, but with SoC medications, which could include penicillamine, trientine, zinc, or a combination of a copper chelator and zinc.
Change From Baseline In Clinical Global Impression (CGI) Scale Item 1 (Severity Of Illness) At Month 6, Month 24, And Last Assessment Month 6	-0.3 score on a scale Standard Deviation 0.89
Change From Baseline In Clinical Global Impression (CGI) Scale Item 1 (Severity Of Illness) At Month 6, Month 24, And Last Assessment Month 24	-0.6 score on a scale Standard Deviation 0.97
Change From Baseline In Clinical Global Impression (CGI) Scale Item 1 (Severity Of Illness) At Month 6, Month 24, And Last Assessment Last Assessment	-0.6 score on a scale Standard Deviation 0.94

SECONDARY outcome

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

Population: Full Analysis Set: Enrolled participants. To be eligibly enrolled, participants must have been receiving the 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. The number of participants analyzed per row represents number of overall participants continuing in the study at each timepoint with non-missing baseline and post-baseline values for each outcome measure.

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).

Outcome measures

Measure	Total n=64 Participants Participants in this study were not treated with any investigational products, but with SoC medications, which could include penicillamine, trientine, zinc, or a combination of a copper chelator and zinc.
CGI Scale Item 2 (Global Improvement) At Month 6, Month 24, And Last Assessment Month 6	3.3 score on a scale Standard Deviation 1.08
CGI Scale Item 2 (Global Improvement) At Month 6, Month 24, And Last Assessment Month 24	3.3 score on a scale Standard Deviation 1.01
CGI Scale Item 2 (Global Improvement) At Month 6, Month 24, And Last Assessment Last Assessment	3.4 score on a scale Standard Deviation 0.98

Adverse Events

Total

Serious events: 0 serious events

Other events: 1 other events

Deaths: 1 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Measure

Total n=64 participants at risk Participants in this study were not treated with any investigational products, but with SoC medications, which could include penicillamine, trientine, zinc, or a combination of a copper chelator and zinc.

Vascular disorders Haematoma

1.6% 1/64 • From baseline up to 30 months. Only adverse events (AEs) associated with study-related interventional testing or assessments were collected and summarized by onset, duration, intensity, seriousness, and outcome. AEs not collected included AEs that might have been associated with the specimen collection procedure but resulted only in local, mild or transient discomforts, redness, slight discomfort and AEs related to WD or treatment, or any other medical condition or events experienced during the study, including death.

Additional Information

Alexion Pharmaceuticals, Inc.

Alexion Pharmaceuticals, Inc.

Phone: 855-752-2356

Email: clinicaltrials@alexion.com

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place