NCT05047523

Study of ALXN1840 Versus Standard of Care in Pediatric Participants With Wilson Disease

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

• Status: Terminated
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 40 (actual)
• Sponsor: Alexion Pharmaceuticals, Inc. · Industry
• Sex: All
• Age: 3 Years – 17 Years
• Healthy volunteers: Not accepted

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).

Detailed description

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.

Conditions

• Wilson Disease

Interventions

Type	Name	Description
DRUG	ALXN1840	Administered as an oral tablet.
DRUG	Standard of Care	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.

Timeline

• Start date: 2021-09-13
• Primary completion: 2023-06-26
• Completion: 2023-06-26
• First posted: 2021-09-17
• Last updated: 2024-10-23
• Results posted: 2024-10-15

Locations

20 sites across 7 countries: Australia, France, Germany, Japan, Poland, South Korea, Spain

Regulatory

• FDA-regulated drug study