Trial Outcomes & Findings for Copper and Molybdenum Balance in Participants With Wilson Disease Treated With ALXN1840 (NCT NCT04573309)

Last Updated: 2024-06-24

Results Overview

Copper balance is defined as the difference between the measured copper input in food and drink, and the measured copper elimination in urine and feces, and was calculated as the average daily copper balance over the collection period.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

9 participants

Primary outcome timeframe

Accumulation: Day 1 through Day 8 (ALXN1840 15 mg)

Results posted on

2024-06-24

Participant Flow

Participant milestones

Participant milestones
Measure	Cohort 1 Participants who had received Wilson disease therapy for \>28 days prior to enrollment were administered ALXN1840 at a dose of 15 milligrams (mg)/day on Day 1 through Day 28 and then increased to 30 mg/day on Day 29 through Day 39.	Cohort 2 Participants who had received Wilson disease therapy for ≤28 days were administered ALXN1840 at a dose of 15 milligrams/day on Day 1 through Day 28 and then increased to 30 mg/day on Day 29 through Day 39.
Overall Study STARTED	8	1
Overall Study Received at Least 1 Dose of Study Drug	8	1
Overall Study COMPLETED	7	1
Overall Study NOT COMPLETED	1	0

Reasons for withdrawal

Reasons for withdrawal
Measure	Cohort 1 Participants who had received Wilson disease therapy for \>28 days prior to enrollment were administered ALXN1840 at a dose of 15 milligrams (mg)/day on Day 1 through Day 28 and then increased to 30 mg/day on Day 29 through Day 39.	Cohort 2 Participants who had received Wilson disease therapy for ≤28 days were administered ALXN1840 at a dose of 15 milligrams/day on Day 1 through Day 28 and then increased to 30 mg/day on Day 29 through Day 39.
Overall Study Protocol Violation	1	0

Baseline Characteristics

Copper and Molybdenum Balance in Participants With Wilson Disease Treated With ALXN1840

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Cohort 1 n=8 Participants Participants who had received Wilson disease therapy for \>28 days prior to enrollment were administered ALXN1840 at a dose of 15 mg/day on Day 1 through Day 28 and then increased to 30 mg/day on Day 29 through Day 39.	Cohort 2 n=1 Participants Participants who had received Wilson disease therapy for ≤28 days were administered ALXN1840 at a dose of 15 mg/day on Day 1 through Day 28 and then increased to 30 mg/day on Day 29 through Day 39.	Total n=9 Participants Total of all reporting groups
Age, Continuous	35.1 years STANDARD_DEVIATION 12.41 • n=99 Participants	26.0 years STANDARD_DEVIATION NA • n=107 Participants	34.1 years STANDARD_DEVIATION 12.00 • n=206 Participants
Sex: Female, Male Female	1 Participants n=99 Participants	1 Participants n=107 Participants	2 Participants n=206 Participants
Sex: Female, Male Male	7 Participants n=99 Participants	0 Participants n=107 Participants	7 Participants n=206 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=99 Participants	0 Participants n=107 Participants	0 Participants n=206 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	8 Participants n=99 Participants	1 Participants n=107 Participants	9 Participants n=206 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=99 Participants	0 Participants n=107 Participants	0 Participants n=206 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=99 Participants	0 Participants n=107 Participants	0 Participants n=206 Participants
Race (NIH/OMB) Asian	1 Participants n=99 Participants	0 Participants n=107 Participants	1 Participants n=206 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=99 Participants	0 Participants n=107 Participants	0 Participants n=206 Participants
Race (NIH/OMB) Black or African American	0 Participants n=99 Participants	0 Participants n=107 Participants	0 Participants n=206 Participants
Race (NIH/OMB) White	7 Participants n=99 Participants	1 Participants n=107 Participants	8 Participants n=206 Participants
Race (NIH/OMB) More than one race	0 Participants n=99 Participants	0 Participants n=107 Participants	0 Participants n=206 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=99 Participants	0 Participants n=107 Participants	0 Participants n=206 Participants

PRIMARY outcome

Timeframe: Accumulation: Day 1 through Day 8 (ALXN1840 15 mg)

Population: Full analysis set included all participants who received at least 1 dose of ALXN1840 treatment. Here, Number of Participants analyzed signifies those who were evaluable for this outcome measure.

Outcome measures

Outcome measures
Measure	Cohort 1 n=7 Participants Participants who had received Wilson disease therapy for \>28 days prior to enrollment were administered ALXN1840 at a dose of 15 mg/day on Day 1 through Day 28 and then increased to 30 mg/day on Day 29 through Day 39.	Cohort 2 n=1 Participants Participants who had received Wilson disease therapy for ≤28 days were administered ALXN1840 at a dose of 15 mg/day on Day 1 through Day 28 and then increased to 30 mg/day on Day 29 through Day 39.
Mean Daily Copper Balance: Day 1 Through Day 8	0.8025 milligrams/day Standard Deviation 0.26860	0.3062 milligrams/day Standard Deviation NA NA signfies that standard deviation could not be calculated as there was only 1 evaluable participant.

PRIMARY outcome

Timeframe: Accumulation: Day 31 through Day 35 (ALXN1840 30 mg)

Population: Full analysis set included all participants who received at least 1 dose of ALXN1840 treatment.

Outcome measures

Outcome measures
Measure	Cohort 1 n=8 Participants Participants who had received Wilson disease therapy for \>28 days prior to enrollment were administered ALXN1840 at a dose of 15 mg/day on Day 1 through Day 28 and then increased to 30 mg/day on Day 29 through Day 39.	Cohort 2 n=1 Participants Participants who had received Wilson disease therapy for ≤28 days were administered ALXN1840 at a dose of 15 mg/day on Day 1 through Day 28 and then increased to 30 mg/day on Day 29 through Day 39.
Mean Daily Copper Balance: Day 31 Through Day 35	0.9156 milligrams/day Standard Deviation 0.37982	0.6613 milligrams/day Standard Deviation NA NA signfies that standard deviation could not be calculated as there was only 1 evaluable participant.

PRIMARY outcome

Timeframe: Accumulation: Day 25 through Day 28 (ALXN1840 15 mg)

Population: Full analysis set included all participants who received at least 1 dose of ALXN1840 treatment.

Outcome measures

Outcome measures
Measure	Cohort 1 n=8 Participants Participants who had received Wilson disease therapy for \>28 days prior to enrollment were administered ALXN1840 at a dose of 15 mg/day on Day 1 through Day 28 and then increased to 30 mg/day on Day 29 through Day 39.	Cohort 2 n=1 Participants Participants who had received Wilson disease therapy for ≤28 days were administered ALXN1840 at a dose of 15 mg/day on Day 1 through Day 28 and then increased to 30 mg/day on Day 29 through Day 39.
Mean Daily Copper Balance: Day 25 Through Day 28	0.7109 milligrams/day Standard Deviation 0.67915	0.7072 milligrams/day Standard Deviation NA NA signfies that standard deviation could not be calculated as there was only 1 evaluable participant.

PRIMARY outcome

Timeframe: Accumulation: Day 36 through Day 39 (ALXN1840 30 mg)

Population: Full analysis set included all participants who received at least 1 dose of ALXN1840 treatment.

Outcome measures

Outcome measures
Measure	Cohort 1 n=8 Participants Participants who had received Wilson disease therapy for \>28 days prior to enrollment were administered ALXN1840 at a dose of 15 mg/day on Day 1 through Day 28 and then increased to 30 mg/day on Day 29 through Day 39.	Cohort 2 n=1 Participants Participants who had received Wilson disease therapy for ≤28 days were administered ALXN1840 at a dose of 15 mg/day on Day 1 through Day 28 and then increased to 30 mg/day on Day 29 through Day 39.
Mean Daily Copper Balance: Day 36 Through Day 39	0.9975 milligrams/day Standard Deviation 0.26543	0.5662 milligrams/day Standard Deviation NA NA signfies that standard deviation could not be calculated as there was only 1 evaluable participant.

SECONDARY outcome

Timeframe: Accumulation: Baseline, Day 1 through Day 8 (ALXN1840 15 mg) and Day 31 through Day 35 (ALXN1840 30 mg); Steady State: Baseline, Day 25 through Day 28 (ALXN1840 15 mg) and Day 36 through Day 39 (ALXN1840 30 mg)

Outcome measures

Outcome measures
Measure	Cohort 1 n=7 Participants Participants who had received Wilson disease therapy for \>28 days prior to enrollment were administered ALXN1840 at a dose of 15 mg/day on Day 1 through Day 28 and then increased to 30 mg/day on Day 29 through Day 39.	Cohort 2 n=1 Participants Participants who had received Wilson disease therapy for ≤28 days were administered ALXN1840 at a dose of 15 mg/day on Day 1 through Day 28 and then increased to 30 mg/day on Day 29 through Day 39.
Change From Baseline In Mean Daily Copper Balance Day 1 through Day 8	-0.3780 milligrams/day Standard Deviation 0.14822	-0.2060 milligrams/day Standard Deviation NA NA signifies that standard deviation could not be calculated as there was only 1 evaluable participant.
Change From Baseline In Mean Daily Copper Balance Day 25 through Day 28	-0.4697 milligrams/day Standard Deviation 0.55770	0.1950 milligrams/day Standard Deviation NA NA signifies that standard deviation could not be calculated as there was only 1 evaluable participant.
Change From Baseline In Mean Daily Copper Balance Day 31 through Day 35	-0.2650 milligrams/day Standard Deviation 0.47135	0.1492 milligrams/day Standard Deviation NA NA signifies that standard deviation could not be calculated as there was only 1 evaluable participant.
Change From Baseline In Mean Daily Copper Balance Day 36 through Day 39	-0.1831 milligrams/day Standard Deviation 0.44589	0.0540 milligrams/day Standard Deviation NA NA signifies that standard deviation could not be calculated as there was only 1 evaluable participant.

SECONDARY outcome

Timeframe: Accumulation: Day 1 through Day 8 for 15 mg and Day 31 through Day 35 for 30 mg; Steady state: Day 25 through Day 28 for ALXN1840 15 mg and Day 36 through Day 39 for ALXN1840 30 mg

Copper was assessed through measurement of copper intake (in food and drink), and copper output (in feces and urine) as well as plasma total and labile bound copper.

Outcome measures

Outcome measures
Measure	Cohort 1 n=7 Participants Participants who had received Wilson disease therapy for \>28 days prior to enrollment were administered ALXN1840 at a dose of 15 mg/day on Day 1 through Day 28 and then increased to 30 mg/day on Day 29 through Day 39.	Cohort 2 n=1 Participants Participants who had received Wilson disease therapy for ≤28 days were administered ALXN1840 at a dose of 15 mg/day on Day 1 through Day 28 and then increased to 30 mg/day on Day 29 through Day 39.
Copper Quantified In Food, Drink, Feces, And Urine, Including Plasma Total And Labile Bound Copper (LBC) Copper Food Content: Day 1 through Day 8	1.6423 milligrams Standard Deviation 0.15638	1.3523 milligrams Standard Deviation NA NA signifies that standard deviation could not be calculated as there was only 1 evaluable participant.
Copper Quantified In Food, Drink, Feces, And Urine, Including Plasma Total And Labile Bound Copper (LBC) Copper Food Content: Day 25 through Day 28	1.8269 milligrams Standard Deviation 0.42485	1.5637 milligrams Standard Deviation NA NA signifies that standard deviation could not be calculated as there was only 1 evaluable participant.
Copper Quantified In Food, Drink, Feces, And Urine, Including Plasma Total And Labile Bound Copper (LBC) Copper Food Content: Day 31 through Day 35	1.8938 milligrams Standard Deviation 0.21846	1.6022 milligrams Standard Deviation NA NA signifies that standard deviation could not be calculated as there was only 1 evaluable participant.
Copper Quantified In Food, Drink, Feces, And Urine, Including Plasma Total And Labile Bound Copper (LBC) Copper Food Content: Day 36 through Day 39	1.7850 milligrams Standard Deviation 0.20107	1.4397 milligrams Standard Deviation NA NA signifies that standard deviation could not be calculated as there was only 1 evaluable participant.
Copper Quantified In Food, Drink, Feces, And Urine, Including Plasma Total And Labile Bound Copper (LBC) Copper Drink Content: Day 1 through Day 8	0.0061 milligrams Standard Deviation 0.00159	0.0055 milligrams Standard Deviation NA NA signifies that standard deviation could not be calculated as there was only 1 evaluable participant.
Copper Quantified In Food, Drink, Feces, And Urine, Including Plasma Total And Labile Bound Copper (LBC) Copper Drink Content: Day 25 through Day 28	0.0066 milligrams Standard Deviation 0.00180	0.0080 milligrams Standard Deviation NA NA signifies that standard deviation could not be calculated as there was only 1 evaluable participant.
Copper Quantified In Food, Drink, Feces, And Urine, Including Plasma Total And Labile Bound Copper (LBC) Copper Drink Content: Day 31 through Day 35	0.0071 milligrams Standard Deviation 0.00184	0.0044 milligrams Standard Deviation NA NA signifies that standard deviation could not be calculated as there was only 1 evaluable participant.
Copper Quantified In Food, Drink, Feces, And Urine, Including Plasma Total And Labile Bound Copper (LBC) Copper Drink Content: Day 36 through Day 39	0.0118 milligrams Standard Deviation 0.00920	0.0045 milligrams Standard Deviation NA NA signifies that standard deviation could not be calculated as there was only 1 evaluable participant.
Copper Quantified In Food, Drink, Feces, And Urine, Including Plasma Total And Labile Bound Copper (LBC) Copper Feces Content: Day 1 through Day 8	0.7594 milligrams Standard Deviation 0.21993	0.7718 milligrams Standard Deviation NA NA signifies that standard deviation could not be calculated as there was only 1 evaluable participant.
Copper Quantified In Food, Drink, Feces, And Urine, Including Plasma Total And Labile Bound Copper (LBC) Copper Feces Content: Day 25 through Day 28	0.9373 milligrams Standard Deviation 0.30688	0.7238 milligrams Standard Deviation NA NA signifies that standard deviation could not be calculated as there was only 1 evaluable participant.
Copper Quantified In Food, Drink, Feces, And Urine, Including Plasma Total And Labile Bound Copper (LBC) Copper Feces Content: Day 31 through Day 35	0.8270 milligrams Standard Deviation 0.33746	0.8720 milligrams Standard Deviation NA NA signifies that standard deviation could not be calculated as there was only 1 evaluable participant.
Copper Quantified In Food, Drink, Feces, And Urine, Including Plasma Total And Labile Bound Copper (LBC) Copper Feces Content: Day 36 through Day 39	0.6136 milligrams Standard Deviation 0.27213	0.7787 milligrams Standard Deviation NA NA signifies that standard deviation could not be calculated as there was only 1 evaluable participant.
Copper Quantified In Food, Drink, Feces, And Urine, Including Plasma Total And Labile Bound Copper (LBC) Copper Urine Content: Day 1 through Day 8	0.0865 milligrams Standard Deviation 0.05689	0.2798 milligrams Standard Deviation NA NA signifies that standard deviation could not be calculated as there was only 1 evaluable participant.
Copper Quantified In Food, Drink, Feces, And Urine, Including Plasma Total And Labile Bound Copper (LBC) Copper Urine Content: Day 25 through Day 28	0.1852 milligrams Standard Deviation 0.09079	0.1408 milligrams Standard Deviation NA NA signifies that standard deviation could not be calculated as there was only 1 evaluable participant.
Copper Quantified In Food, Drink, Feces, And Urine, Including Plasma Total And Labile Bound Copper (LBC) Copper Urine Content: Day 31 through Day 35	0.1584 milligrams Standard Deviation 0.07823	0.0732 milligrams Standard Deviation NA NA signifies that standard deviation could not be calculated as there was only 1 evaluable participant.
Copper Quantified In Food, Drink, Feces, And Urine, Including Plasma Total And Labile Bound Copper (LBC) Copper Urine Content: Day 36 through Day 39	0.1857 milligrams Standard Deviation 0.09506	0.0994 milligrams Standard Deviation NA NA signifies that standard deviation could not be calculated as there was only 1 evaluable participant.

SECONDARY outcome

Timeframe: Day 25 through Day 28 (ALXN1840 15 mg) and Day 36 through Day 39 (ALXN1840 30 mg)

The amount of molybdenum in food, drink, feces and urine is reported in this outcome measure.

Outcome measures

Outcome measures
Measure	Cohort 1 n=7 Participants Participants who had received Wilson disease therapy for \>28 days prior to enrollment were administered ALXN1840 at a dose of 15 mg/day on Day 1 through Day 28 and then increased to 30 mg/day on Day 29 through Day 39.	Cohort 2 n=1 Participants Participants who had received Wilson disease therapy for ≤28 days were administered ALXN1840 at a dose of 15 mg/day on Day 1 through Day 28 and then increased to 30 mg/day on Day 29 through Day 39.
Molybdenum Specified In ALXN1840 Doses Given And Quantified In Food, Drink, Feces, And Urine, Including Plasma At Steady State Molybdenum Food Content : Days 25 through 28	0.3324 milligrams Standard Deviation 0.18039	0.3905 milligrams Standard Deviation NA NA signifies that standard deviation could not be calculated as there was only 1 evaluable participant.
Molybdenum Specified In ALXN1840 Doses Given And Quantified In Food, Drink, Feces, And Urine, Including Plasma At Steady State Molybdenum Food Content : Days 36 through 39	0.1923 milligrams Standard Deviation 0.02354	0.1724 milligrams Standard Deviation NA NA signifies that standard deviation could not be calculated as there was only 1 evaluable participant.
Molybdenum Specified In ALXN1840 Doses Given And Quantified In Food, Drink, Feces, And Urine, Including Plasma At Steady State Molybdenum Drink Content : Days 25 through 28	0.0006 milligrams Standard Deviation 0.00012	0.0018 milligrams Standard Deviation NA NA signifies that standard deviation could not be calculated as there was only 1 evaluable participant.
Molybdenum Specified In ALXN1840 Doses Given And Quantified In Food, Drink, Feces, And Urine, Including Plasma At Steady State Molybdenum Drink Content : Days 36 through 39	0.0007 milligrams Standard Deviation 0.00024	0.0004 milligrams Standard Deviation NA NA signifies that standard deviation could not be calculated as there was only 1 evaluable participant.
Molybdenum Specified In ALXN1840 Doses Given And Quantified In Food, Drink, Feces, And Urine, Including Plasma At Steady State Molybdenum Feces Content : Days 25 through 28	1.1004 milligrams Standard Deviation 0.38506	1.0656 milligrams Standard Deviation NA NA signifies that standard deviation could not be calculated as there was only 1 evaluable participant.
Molybdenum Specified In ALXN1840 Doses Given And Quantified In Food, Drink, Feces, And Urine, Including Plasma At Steady State Molybdenum Feces Content : Days 36 through 39	1.0541 milligrams Standard Deviation 0.83207	2.1352 milligrams Standard Deviation NA NA signifies that standard deviation could not be calculated as there was only 1 evaluable participant.
Molybdenum Specified In ALXN1840 Doses Given And Quantified In Food, Drink, Feces, And Urine, Including Plasma At Steady State Molybdenum Urine Content : Days 25 through 28	0.7250 milligrams Standard Deviation 0.32736	0.5480 milligrams Standard Deviation NA NA signifies that standard deviation could not be calculated as there was only 1 evaluable participant.
Molybdenum Specified In ALXN1840 Doses Given And Quantified In Food, Drink, Feces, And Urine, Including Plasma At Steady State Molybdenum Urine Content : Days 36 through 39	0.8984 milligrams Standard Deviation 0.62633	1.0395 milligrams Standard Deviation NA NA signifies that standard deviation could not be calculated as there was only 1 evaluable participant.

SECONDARY outcome

Outcome measures

Outcome measures
Measure	Cohort 1 n=7 Participants Participants who had received Wilson disease therapy for \>28 days prior to enrollment were administered ALXN1840 at a dose of 15 mg/day on Day 1 through Day 28 and then increased to 30 mg/day on Day 29 through Day 39.	Cohort 2 n=1 Participants Participants who had received Wilson disease therapy for ≤28 days were administered ALXN1840 at a dose of 15 mg/day on Day 1 through Day 28 and then increased to 30 mg/day on Day 29 through Day 39.
Change From Baseline In Total Molybdenum Excretion In Urine And Feces Molybdenum Feces Content : Day 1 through 8	0.7102 milligrams Standard Deviation 0.22072	0.9510 milligrams Standard Deviation NA NA signifies that standard deviation could not be calculated as there was only 1 evaluable participant.
Change From Baseline In Total Molybdenum Excretion In Urine And Feces Molybdenum Feces Content : Days 25 through 28	1.0146 milligrams Standard Deviation 0.42658	0.9736 milligrams Standard Deviation NA NA signifies that standard deviation could not be calculated as there was only 1 evaluable participant.
Change From Baseline In Total Molybdenum Excretion In Urine And Feces Molybdenum Feces Content : Days 31 through 35	1.0484 milligrams Standard Deviation 0.62291	1.7188 milligrams Standard Deviation NA NA signifies that standard deviation could not be calculated as there was only 1 evaluable participant.
Change From Baseline In Total Molybdenum Excretion In Urine And Feces Molybdenum Feces Content : Days 36 through 39	0.9683 milligrams Standard Deviation 0.88027	2.0432 milligrams Standard Deviation NA NA signifies that standard deviation could not be calculated as there was only 1 evaluable participant.
Change From Baseline In Total Molybdenum Excretion In Urine And Feces Molybdenum Urine Content : Day 1 through 8	0.3972 milligrams Standard Deviation 0.15972	0.1725 milligrams Standard Deviation NA NA signifies that standard deviation could not be calculated as there was only 1 evaluable participant.
Change From Baseline In Total Molybdenum Excretion In Urine And Feces Molybdenum Urine Content : Days 25 through 28	0.6122 milligrams Standard Deviation 0.28154	0.4010 milligrams Standard Deviation NA NA signifies that standard deviation could not be calculated as there was only 1 evaluable participant.
Change From Baseline In Total Molybdenum Excretion In Urine And Feces Molybdenum Urine Content : Days 31 through 35	0.7974 milligrams Standard Deviation 0.47607	0.8085 milligrams Standard Deviation NA NA signifies that standard deviation could not be calculated as there was only 1 evaluable participant.
Change From Baseline In Total Molybdenum Excretion In Urine And Feces Molybdenum Urine Content : Days 36 through 39	0.7855 milligrams Standard Deviation 0.58820	0.8926 milligrams Standard Deviation NA NA signifies that standard deviation could not be calculated as there was only 1 evaluable participant.

SECONDARY outcome

Timeframe: Steady state: Day 25 through Day 28 (ALXN1840 15 mg) and Day 36 through Day 39 (ALXN1840 30 mg)

Molybdenum balance at steady state was assessed through measurement of molybdenum intake (in food, drink, and ALXN1840), and molybdenum output (in feces and urine). Steady state is defined as molybdenum (out) equal to molybdenum (in).

Outcome measures

Outcome measures
Measure	Cohort 1 n=7 Participants Participants who had received Wilson disease therapy for \>28 days prior to enrollment were administered ALXN1840 at a dose of 15 mg/day on Day 1 through Day 28 and then increased to 30 mg/day on Day 29 through Day 39.	Cohort 2 n=1 Participants Participants who had received Wilson disease therapy for ≤28 days were administered ALXN1840 at a dose of 15 mg/day on Day 1 through Day 28 and then increased to 30 mg/day on Day 29 through Day 39.
Mean Daily Molybdenum Balance At ALXN1840 Steady State Days 25 through 28	1.3618 milligrams/day Standard Deviation 0.44254	2.1087 milligrams/day Standard Deviation NA NA signifies that standard deviation could not be calculated as there was only 1 evaluable participant.
Mean Daily Molybdenum Balance At ALXN1840 Steady State Days 36 through 39	2.7599 milligrams/day Standard Deviation 1.68008	3.6581 milligrams/day Standard Deviation NA NA signifies that standard deviation could not be calculated as there was only 1 evaluable participant.

SECONDARY outcome

Timeframe: Accumulation: Day 1 through Day 8 (ALXN1840 15 mg) and Day 31 through Day 35 ((ALXN1840 30 mg)

Molybdenum balance was assessed through measurement of molybdenum intake (in food, drink, and ALXN1840), and molybdenum output (in feces and urine).

Outcome measures

Outcome measures
Measure	Cohort 1 n=7 Participants Participants who had received Wilson disease therapy for \>28 days prior to enrollment were administered ALXN1840 at a dose of 15 mg/day on Day 1 through Day 28 and then increased to 30 mg/day on Day 29 through Day 39.	Cohort 2 n=1 Participants Participants who had received Wilson disease therapy for ≤28 days were administered ALXN1840 at a dose of 15 mg/day on Day 1 through Day 28 and then increased to 30 mg/day on Day 29 through Day 39.
Accumulation Of Molybdenum As Determined By Molybdenum Balance Days 1 through 8	2.2458 milligrams Standard Deviation 0.23232	2.0876 milligrams Standard Deviation NA NA signifies that standard deviation could not be calculated as there was only 1 evaluable participant.
Accumulation Of Molybdenum As Determined By Molybdenum Balance Days 31 through 35	2.6265 milligrams Standard Deviation 1.96365	4.0775 milligrams Standard Deviation NA NA signifies that standard deviation could not be calculated as there was only 1 evaluable participant.

SECONDARY outcome

Timeframe: Day 1 up to Day 39

Population: Pharmacokinetic (PK) analysis set included all participants who had sufficient plasma samples to enable the calculation of PK parameters and provide PK profiles.

Outcome measures

Outcome measures
Measure	Cohort 1 n=7 Participants Participants who had received Wilson disease therapy for \>28 days prior to enrollment were administered ALXN1840 at a dose of 15 mg/day on Day 1 through Day 28 and then increased to 30 mg/day on Day 29 through Day 39.	Cohort 2 n=1 Participants Participants who had received Wilson disease therapy for ≤28 days were administered ALXN1840 at a dose of 15 mg/day on Day 1 through Day 28 and then increased to 30 mg/day on Day 29 through Day 39.
Maximum Observed Plasma Concentration (Cmax) of Total Molybdenum and Plasma Ultrafiltrate (PUF) Molybdenum Total Molydenum	430.451 nanograms/milliliters Geometric Coefficient of Variation 46.6	1271.551 nanograms/milliliters Geometric Coefficient of Variation NA NA signifies that Geometric Coefficient of Variation (CV) could not be calculated as there was only 1 evaluable participant.
Maximum Observed Plasma Concentration (Cmax) of Total Molybdenum and Plasma Ultrafiltrate (PUF) Molybdenum Plasma ultrafiltrate molybdenum	13.546 nanograms/milliliters Geometric Coefficient of Variation 43.5	17.823 nanograms/milliliters Geometric Coefficient of Variation NA NA signifies that geometric CV could not be calculated as there was only 1 evaluable participant.

SECONDARY outcome

Timeframe: Day 39

Population: Pharmacokinetic analysis set included all participants who had sufficient plasma samples to enable the calculation of PK parameters and provide PK profiles. Here, Number of participants analyzed signifies those who were evaluable for this outcome measure and number analyzed signifies those who were evaluable at specified time points.

Outcome measures

Outcome measures
Measure	Cohort 1 n=6 Participants Participants who had received Wilson disease therapy for \>28 days prior to enrollment were administered ALXN1840 at a dose of 15 mg/day on Day 1 through Day 28 and then increased to 30 mg/day on Day 29 through Day 39.	Cohort 2 n=1 Participants Participants who had received Wilson disease therapy for ≤28 days were administered ALXN1840 at a dose of 15 mg/day on Day 1 through Day 28 and then increased to 30 mg/day on Day 29 through Day 39.
Area Under The Concentration Time Curve (AUC0-inf) of Total Molybdenum and Plasma Ultrafiltrate (PUF) Molybdenum Total Molydenum	13532.591 hours*nanograms/milliliters Geometric Coefficient of Variation 49.3	65694.679 hours*nanograms/milliliters Geometric Coefficient of Variation NA NA signifies that geometric CV could not be calculated as there was only 1 evaluable participant.
Area Under The Concentration Time Curve (AUC0-inf) of Total Molybdenum and Plasma Ultrafiltrate (PUF) Molybdenum Plasma ultrafiltrate molybdenum	292.737 hours*nanograms/milliliters Geometric Coefficient of Variation 11.9	476.960 hours*nanograms/milliliters Geometric Coefficient of Variation NA NA signifies that geometric CV could not be calculated as there was only 1 evaluable participant.

Adverse Events

Cohort 1

Serious events: 0 serious events

Other events: 6 other events

Deaths: 0 deaths

Cohort 2

Serious events: 0 serious events

Other events: 1 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Additional Information

Alexion Pharmaceuticals, Inc.

Phone: 855-752-2356

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Measure	Cohort 1 n=8 participants at risk Participants who had received Wilson disease therapy for \>28 days prior to enrollment were administered ALXN1840 at a dose of 15 mg/day on Day 1 through Day 28 and then increased to 30 mg/day on Day 29 through Day 39.	Cohort 2 n=1 participants at risk Participants who had received Wilson disease therapy for ≤28 days were administered ALXN1840 at a dose of 15 mg/day on Day 1 through Day 28 and then increased to 30 mg/day on Day 29 through Day 39.
Gastrointestinal disorders Constipation	25.0% 2/8 • Number of events 2 • Baseline up to Day 56 Safety set included all participants who received at least 1 dose of ALXN1840.	100.0% 1/1 • Number of events 1 • Baseline up to Day 56 Safety set included all participants who received at least 1 dose of ALXN1840.
Gastrointestinal disorders Abdominal distension	12.5% 1/8 • Number of events 1 • Baseline up to Day 56 Safety set included all participants who received at least 1 dose of ALXN1840.	0.00% 0/1 • Baseline up to Day 56 Safety set included all participants who received at least 1 dose of ALXN1840.
Infections and infestations Hordeolum	12.5% 1/8 • Number of events 1 • Baseline up to Day 56 Safety set included all participants who received at least 1 dose of ALXN1840.	0.00% 0/1 • Baseline up to Day 56 Safety set included all participants who received at least 1 dose of ALXN1840.
Infections and infestations Periodontitis	12.5% 1/8 • Number of events 1 • Baseline up to Day 56 Safety set included all participants who received at least 1 dose of ALXN1840.	0.00% 0/1 • Baseline up to Day 56 Safety set included all participants who received at least 1 dose of ALXN1840.
Infections and infestations Pulpitis dental	12.5% 1/8 • Number of events 1 • Baseline up to Day 56 Safety set included all participants who received at least 1 dose of ALXN1840.	0.00% 0/1 • Baseline up to Day 56 Safety set included all participants who received at least 1 dose of ALXN1840.
Infections and infestations Viral upper respiratory tract infection	12.5% 1/8 • Number of events 1 • Baseline up to Day 56 Safety set included all participants who received at least 1 dose of ALXN1840.	0.00% 0/1 • Baseline up to Day 56 Safety set included all participants who received at least 1 dose of ALXN1840.
Injury, poisoning and procedural complications Contusion	12.5% 1/8 • Number of events 1 • Baseline up to Day 56 Safety set included all participants who received at least 1 dose of ALXN1840.	0.00% 0/1 • Baseline up to Day 56 Safety set included all participants who received at least 1 dose of ALXN1840.
Injury, poisoning and procedural complications Nail injury	12.5% 1/8 • Number of events 1 • Baseline up to Day 56 Safety set included all participants who received at least 1 dose of ALXN1840.	0.00% 0/1 • Baseline up to Day 56 Safety set included all participants who received at least 1 dose of ALXN1840.
Investigations Liver function test abnormal	12.5% 1/8 • Number of events 1 • Baseline up to Day 56 Safety set included all participants who received at least 1 dose of ALXN1840.	0.00% 0/1 • Baseline up to Day 56 Safety set included all participants who received at least 1 dose of ALXN1840.
Investigations Transaminases increased	12.5% 1/8 • Number of events 1 • Baseline up to Day 56 Safety set included all participants who received at least 1 dose of ALXN1840.	0.00% 0/1 • Baseline up to Day 56 Safety set included all participants who received at least 1 dose of ALXN1840.
Nervous system disorders Dizziness	12.5% 1/8 • Number of events 1 • Baseline up to Day 56 Safety set included all participants who received at least 1 dose of ALXN1840.	0.00% 0/1 • Baseline up to Day 56 Safety set included all participants who received at least 1 dose of ALXN1840.
Nervous system disorders Headache	0.00% 0/8 • Baseline up to Day 56 Safety set included all participants who received at least 1 dose of ALXN1840.	100.0% 1/1 • Number of events 2 • Baseline up to Day 56 Safety set included all participants who received at least 1 dose of ALXN1840.
Nervous system disorders Migraine	12.5% 1/8 • Number of events 1 • Baseline up to Day 56 Safety set included all participants who received at least 1 dose of ALXN1840.	0.00% 0/1 • Baseline up to Day 56 Safety set included all participants who received at least 1 dose of ALXN1840.
Psychiatric disorders Sleep disorder	12.5% 1/8 • Number of events 1 • Baseline up to Day 56 Safety set included all participants who received at least 1 dose of ALXN1840.	0.00% 0/1 • Baseline up to Day 56 Safety set included all participants who received at least 1 dose of ALXN1840.
Reproductive system and breast disorders Breast tenderness	12.5% 1/8 • Number of events 1 • Baseline up to Day 56 Safety set included all participants who received at least 1 dose of ALXN1840.	0.00% 0/1 • Baseline up to Day 56 Safety set included all participants who received at least 1 dose of ALXN1840.
Skin and subcutaneous tissue disorders Acne	12.5% 1/8 • Number of events 1 • Baseline up to Day 56 Safety set included all participants who received at least 1 dose of ALXN1840.	0.00% 0/1 • Baseline up to Day 56 Safety set included all participants who received at least 1 dose of ALXN1840.
Skin and subcutaneous tissue disorders Dry skin	12.5% 1/8 • Number of events 1 • Baseline up to Day 56 Safety set included all participants who received at least 1 dose of ALXN1840.	0.00% 0/1 • Baseline up to Day 56 Safety set included all participants who received at least 1 dose of ALXN1840.
Skin and subcutaneous tissue disorders Rash	12.5% 1/8 • Number of events 1 • Baseline up to Day 56 Safety set included all participants who received at least 1 dose of ALXN1840.	0.00% 0/1 • Baseline up to Day 56 Safety set included all participants who received at least 1 dose of ALXN1840.
Vascular disorders Phlebitis superficial	0.00% 0/8 • Baseline up to Day 56 Safety set included all participants who received at least 1 dose of ALXN1840.	100.0% 1/1 • Number of events 1 • Baseline up to Day 56 Safety set included all participants who received at least 1 dose of ALXN1840.