This study is being conducted to evaluate the efficacy, safety, pharmacokinetics (PK), and pharmacodynamics of ALXN1840 versus standard of care in pediatric participants with Wilson disease (WD).
Participants who complete the 48 weeks of treatment in Period 1 will have the option to receive ALXN1840 for 24 weeks in Period 2 (open-label extension). Safety will be monitored throughout the study.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
SINGLE
Enrollment
40
Administered as an oral tablet.
Depending on the site/region, participants randomized to receive Standard of Care treatment will receive trientine, penicillamine, zinc, or a combination of these medicines, administered according to standard regimens.
Research Site
Parkville, Australia
Research Site
South Brisbane, Australia
Research Site
Percent Change From Baseline to Week 48 in Non-ceruloplasmin-bound Copper in Plasma
Plasma samples were planned to be collected to measure ceruloplasmin-bound copper. Due to the early termination of the study, data for this Outcome Measure were not collected for any of the cohorts.
Time frame: Baseline, Week 48
Number of Participants With Treatment-emergent Adverse Events (TEAEs) During the Primary Evaluation Period
An adverse event (AE) was any untoward medical occurrence in a participant administered the study drug and which did not necessarily have a causal relationship with this treatment. TEAEs were defined as AEs with onset after the first dose of study intervention or existing events that worsened in severity after the first dose of study intervention. A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' Section.
Time frame: Baseline up to Week 48
Area Under the Effect Versus Time Curve (AUEC) for Plasma Total Copper and Direct NCC
Time frame: Week 48
Maximum Observed Concentration (Cmax) of ALXN1840 for Plasma Total Molybdenum and Plasma Ultrafiltrate Molybdenum Concentrations
Time frame: Week 48
Area Under the Plasma Concentration Versus Time Curve From Time 0 to the End of the Dosing Interval (AUCtau) of ALXN1840 for Plasma Total Molybdenum and Plasma Ultrafiltrate Molybdenum
Time frame: Week 48
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Lille, France
Research Site
Toulouse, France
Research Site
Hanover, Germany
Research Site
Tübingen, Germany
Research Site
Kumamoto, Japan
Research Site
Kurume-shi, Japan
Research Site
Meguro-ku, Japan
Research Site
Sapporo, Japan
...and 10 more locations