French pharmaceutical company Orphalan Inc announced on Tuesday the launch of the TRADITiONAL Study, a global, multicentre Phase 3 clinical trial evaluating an investigational once-daily formulation of trientine tetrahydrochloride for the first-line treatment of individuals with Wilson disease.

Wilson disease is a rare inherited disorder of copper metabolism caused by mutations in the ATP7B gene. The disease prevents the body from eliminating excess copper, leading to toxic accumulation in tissues and organs, primarily the liver and brain. Without lifelong treatment, Wilson disease can give rise to serious hepatic, neurologic, and psychiatric complications. If untreated, Wilson disease can be fatal.

The new study will evaluate safety and efficacy of the investigational once-daily formulation of trientine tetrahydrochloride compared with D-penicillamine (DPA), an established copper-chelating treatment, as first-line treatment in patients with Wilson disease, over 48 weeks following randomisation.

US study sites include University of Colorado Anschutz School of Medicine, Yale University School of Medicine, and University of Michigan Medical Center. Additional sites are expected to join this year as global enrolment expands to China, Pakistan and Saudi Arabia.