Efficacy and Safety of WTX101 Administered for 48 Weeks Versus Standard of Care in Wilson Disease Subjects
Genetics - Adult
What is the purpose of this trial?
Wilson Disease (WD) is an autosomal recessive disorder of impaired copper (CU) transport caused by mutations in the ATP7B gene. WTX101 (bis-choline tetrathiomolybdate) is a first-in-class copper-protein-binding agent with a unique mechanism of action, under investigation as a novel therapy for WD. It is formulated as an enteric coated tablet (15 mg strength) for oral administration. The purpose of this study is to evaluate the efficacy of WTX101 administered for 48 weeks compared to standard of care (SOC) in WD subjects aged 18 and older.
- Ages12 years and older
- Trial withAlexion Pharmaceuticals, Inc.
- Start Date04/01/2018
- End Date02/27/2021
- Last Updated12/10/2019
- Study HIC#2000022372