A Phase 3 Study of ALXN1840 vs SoC in Paediatric Participants With WD
Research type
Research Study
Full title
A multicenter, randomized, controlled, open-label, rater-blinded study to evaluate efficacy, safety, pharmacokinetics, and pharmacodynamics of ALXN1840 versus standard of care in pediatric participants with Wilson disease.
IRAS ID
1003831
Contact name
Zeeshan Khawaja
Contact email
Sponsor organisation
Alexion Pharmaceuticals, Inc.
Eudract number
2021-001015-82
Clinicaltrials.gov Identifier
Research summary
Wilson disease (WD) is a genetic disorder which leads to people having higher levels of copper in their body than normal, which can be toxic and can cause damage to the liver, brain and other organs. WD is most commonly diagnosed in teenagers and young adults but presentation of the disease generally occurs over 5 years of age. There are treatments approved for children and adolescents (penicillamine, trientine and zinc) but they can have problems with tolerability and a dosing of 2 – 4 times daily is inconvenient. There is an unmet need in this patient population for more effective and convenient treatment.
This randomised study aims to determine whether treatment with ALXN1840 is safe and effective in treating paediatric patients with WD, aged 3 to 17 years old, versus standard of care (SoC) therapy.
Participants who meet all eligibility criteria will be randomised in a 1:1 ratio to receive ALXN1840 or SoC for approximately 12 months (48 weeks). ALXN1840 will be taken orally once per day and SoC therapy administered as normal.
The study includes screening assessments which must be completed within 28 days of signing the consent form, a 48 week treatment period (13 study visits) and a 24 week follow-up period (9 study visits). Assessments include physical examinations, vital signs, an ECG, blood and urine sample collection for testing, an optional stool sample and completion of quality of life questionnaires. If participants wish to continue in the study after 48 weeks they may participate in Period 2 where they will receive ALXN1840 for 24 weeks. The total study duration, including period 2, will be up approximately 1.5 years.
Approximately 48 participants will participate worldwide. Recruitment is competitive but we anticipate approximately 4 patients from up to 3 hospitals in the UK.
REC name
East of England - Essex Research Ethics Committee
REC reference
21/EE/0215
Date of REC Opinion
8 Oct 2021
REC opinion
Further Information Favourable Opinion