Orphalan has launched the TRADITiONAL Study, a global Phase III clinical trial of an investigational once-daily formulation of trientine tetrahydrochloride for individuals with Wilson disease, a rare genetic disorder of copper metabolism.
The randomised, multi-centre, open-label, parallel-group trial will compare the efficacy and safety of the experimental trientine regimen with D-penicillamine, a currently used copper-chelating agent, as first-line therapy.
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It involves 48 weeks of treatment following randomisation.
The study is recruiting both symptomatic and asymptomatic patients aged eight years and above who are either new to all Wilson disease therapies or have not previously received chelator treatment.
Patients who have used zinc salts for up to 28 days may also be eligible. After a screening period of around four weeks, participants will be monitored for efficacy, safety, tolerability and satisfaction with the treatment over the course of the study.
Initial clinical sites in the US include University of Colorado Anschutz School of Medicine, University of Michigan Medical Center, and Yale University School of Medicine.
Additional sites are expected to join as enrolment expands to Saudi Arabia, China and Pakistan later in the year.
Orphalan chief medical officer Omar Kamlin said: “Successful management of Wilson disease requires lifelong therapy, and current treatment regimens can be complex and burdensome for many patients, posing significant challenges with adherence.
“The initiation of the global TRADITiONAL Study in the US reflects Orphalan’s commitment to addressing the unmet needs of Wilson disease patients by investigating a therapeutic approach which may simplify the treatment burden.”
Wilson disease is caused by mutations in the ATP7B gene and hinders the body’s ability to eliminate excess copper, leading to toxic build-up in organs, particularly the liver and brain.
Orphalan is focused on developing treatments for rare diseases such as Wilson disease and, more recently, has expanded its portfolio to include therapies for infantile epileptic spasms syndrome and neuro-oncology conditions.
