Study of ALXN1840 Versus Standard of Care in Pediatric Participants With Wilson Disease (NCT05047523) | Clinical Trial Compass
TerminatedPhase 3
Study of ALXN1840 Versus Standard of Care in Pediatric Participants With Wilson Disease
Stopped: Sponsor decision to terminate the program
Australia, France, Germany40 participantsStarted 2021-09-13
Plain-language summary
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).
Who can participate
Age range
3 Years – 17 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
. Diagnosis of Wilson Disease by Leipzig Score ≥ 4.
. Adequate venous access to allow collection of required blood samples.
. Able to swallow intact ALXN1840 tablets or mini-tablets.
. Willing to avoid intake of foods and drinks with high contents of copper.
. Willing and able to follow protocol-specified contraception requirements.
Exclusion criteria
. Decompensated hepatic cirrhosis or MELD score \> 13 (ages 12 to \<18) or PELD score \> 13 (ages 3 to \< 12).
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Percent Change From Baseline to Week 48 in Non-ceruloplasmin-bound Copper in Plasma
. Clinically significant gastrointestinal bleed within past 3 months.
. Alanine aminotransferase (ALT) \> 2 × upper limit of normal (ULN) for participants treated for \> 28 days with WD therapy or ALT \> 5 × ULN for treatment-naïve participants or participants who have been treated for ≤ 28 days.
. Marked neurological disease requiring either nasogastric feeding tube or intensive inpatient medical care.
. Hemoglobin less than lower limit of the reference range for age and sex.
. History of seizure activity within 6 months prior to informed consent/assent.
. Participants in renal failure, defined as in end-stage renal disease on dialysis (chronic kidney disease stage 5) or estimated glomerular filtration rate \< 30 milliliters/minute/1.73 meter squared.