This exploratory study will investigate the effects of ALXN1840 on copper balance in participants with Wilson disease (WD).
Participants who are treatment experienced (which includes standard-of-care therapies or ALXN1840) and treatment naïve are eligible for this study.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
9
Administered orally as tablets.
Research Site
New Haven, Connecticut, United States
Research Site
Grafton, New Zealand
Research Site
London, United Kingdom
Mean Daily Copper Balance: Day 1 Through Day 8
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.
Time frame: Accumulation: Day 1 through Day 8 (ALXN1840 15 mg)
Mean Daily Copper Balance: Day 31 Through Day 35
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.
Time frame: Accumulation: Day 31 through Day 35 (ALXN1840 30 mg)
Mean Daily Copper Balance: Day 25 Through Day 28
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.
Time frame: Accumulation: Day 25 through Day 28 (ALXN1840 15 mg)
Mean Daily Copper Balance: Day 36 Through Day 39
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.
Time frame: Accumulation: Day 36 through Day 39 (ALXN1840 30 mg)
Change From Baseline In Mean Daily Copper Balance
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.
Time frame: 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)
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
Copper Quantified In Food, Drink, Feces, And Urine, Including Plasma Total And Labile Bound Copper (LBC)
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.
Time frame: 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
Molybdenum Specified In ALXN1840 Doses Given And Quantified In Food, Drink, Feces, And Urine, Including Plasma At Steady State
The amount of molybdenum in food, drink, feces and urine is reported in this outcome measure.
Time frame: Day 25 through Day 28 (ALXN1840 15 mg) and Day 36 through Day 39 (ALXN1840 30 mg)
Change From Baseline In Total Molybdenum Excretion In Urine And Feces
Time frame: 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)
Mean Daily Molybdenum Balance At ALXN1840 Steady State
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).
Time frame: Steady state: Day 25 through Day 28 (ALXN1840 15 mg) and Day 36 through Day 39 (ALXN1840 30 mg)
Accumulation Of Molybdenum As Determined By Molybdenum Balance
Molybdenum balance was assessed through measurement of molybdenum intake (in food, drink, and ALXN1840), and molybdenum output (in feces and urine).
Time frame: Accumulation: Day 1 through Day 8 (ALXN1840 15 mg) and Day 31 through Day 35 ((ALXN1840 30 mg)
Maximum Observed Plasma Concentration (Cmax) of Total Molybdenum and Plasma Ultrafiltrate (PUF) Molybdenum
Time frame: Day 1 up to Day 39
Area Under The Concentration Time Curve (AUC0-inf) of Total Molybdenum and Plasma Ultrafiltrate (PUF) Molybdenum
Time frame: Day 39