NCT03403205

Efficacy and Safety of ALXN1840 Administered for 48 Weeks Versus Standard of Care in Participants With Wilson Disease

A Phase 3, Randomized, Rater-Blinded, Multi-Center Study to Evaluate the Efficacy and Safety of ALXN1840 Administered for 48 Weeks Versus Standard of Care in Patients With Wilson Disease Aged 12 Years and Older, With an Extension Period of up to 60 Months

• Status: Terminated
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 214 (actual)
• Sponsor: Alexion Pharmaceuticals, Inc. · Industry
• Sex: All
• Age: 12 Years
• Healthy volunteers: Not accepted

Summary

The study will evaluate the efficacy and safety of ALXN1840 (formerly called WTX101) administered for 48 weeks compared to standard of care (SoC) in Wilson Disease (WD) participants aged 12 and older in the Primary Evaluation Period. In addition, efficacy and safety will be evaluated during an optional 60-month Extension Period.

Detailed description

The study consists of 2 cohorts. Cohort 1: Participants who have received SoC therapy for \> 28 days and Cohort 2: Participants who are treatment-naïve or who have received SoC therapy for ≤ 28 days. All enrolled participants were randomized by cohort in a 2:1 ratio to treatment with ALXN1840 or SoC (either as continued therapy in Cohort 1 or as continued or initial therapy in Cohort 2).

Conditions

• Wilson Disease

Interventions

Type	Name	Description
DRUG	ALXN1840	ALXN1840 administered orally in 15 mg tablets
DRUG	SoC Therapy	Depending on the site/region, participants randomized to receive SoC treatment will receive trientine, penicillamine, Zinc, or a combination of these medicines, administered according to standard regimens.

Timeline

• Start date: 2018-02-22
• Primary completion: 2023-06-30
• Completion: 2023-06-30
• First posted: 2018-01-18
• Last updated: 2024-06-17
• Results posted: 2023-10-10

Locations

56 sites across 22 countries: United States, Australia, Austria, Canada, Czechia, Denmark, France, Germany, Hong Kong, Hungary, Israel, Japan, New Zealand, Poland, Russia, Serbia, Singapore, South Korea, Spain, Taiwan, Turkey (Türkiye), United Kingdom

Regulatory

• FDA-regulated drug study