Orphalan Initiates Global Phase 3 TRADITiONAL Study of Once-Daily Trientine in Wilson Disease
核心洞察
Orphalan announced the launch of the TRADITiONAL Study, a global, multicenter Phase 3 trial evaluating an investigational once-daily formulation of trientine tetrahydrochloride for first-line Wilson disease (搜索) treatment.
The open-label study will compare the once-daily trientine formulation against D-penicillamine over 48 weeks in patients aged 8 years and older who are treatment-naïve or chelator-naïve.
The trial aims to address adherence challenges posed by complex, burdensome lifelong regimens, with U.S. sites including the University of Colorado, Yale, and the University of Michigan.
Orphalan Inc. has announced the launch of the TRADITiONAL Study, a global, multicenter Phase 3 clinical trial evaluating an investigational once-daily formulation of trientine tetrahydrochloride for the first-line treatment of individuals with Wilson disease (搜索). The study will assess the safety and efficacy of the investigational once-daily formulation compared with D-penicillamine (DPA), an established copper-chelating treatment, as first-line therapy in patients with Wilson disease over 48 weeks following randomization.
"Successful management of Wilson disease (搜索) requires lifelong therapy, and current treatment regimens can be complex and burdensome for many patients, posing significant challenges with adherence," said Omar Kamlin, Chief Medical Officer, Orphalan. "The initiation of the global TRADITiONAL Study in the USA reflects Orphalan's commitment in addressing the unmet needs of Wilson disease patients by investigating a therapeutic approach which may simplify the treatment burden."
Study Design and Patient Population
TRADITiONAL is a global, multicenter, randomized, parallel-group, open-label Phase 3 study in symptomatic and asymptomatic Wilson disease (搜索) patients aged 8 years and older who are either naïve to all Wilson disease therapies (treatment-naïve) or naïve to chelator therapy. Symptomatic patients treated with zinc salts for 28 days or fewer may also be eligible. Following approximately four weeks of screening, participants will receive 48 weeks of treatment. The study will evaluate efficacy, safety, tolerability, and patient-reported treatment satisfaction.
Trial Sites and Global Expansion
U.S. study sites include the University of Colorado Anschutz School of Medicine, Yale University School of Medicine, and the University of Michigan Medical Center. Additional sites are expected to join this year as global enrollment expands to China, Pakistan, and Saudi Arabia. Further information is available on ClinicalTrials.gov under identifier NCT07465718.
Wilson Disease Background
Wilson disease (搜索) is a rare inherited disorder of copper metabolism caused by mutations in the ATP7B (搜索) 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, and importantly, untreated Wilson disease can be fatal.
About Orphalan
Orphalan is a pharmaceutical company dedicated to developing and commercializing treatments for rare diseases, including Wilson disease (搜索). The company's portfolio has recently expanded to include infantile epileptic spasms syndrome (IESS), a rare form of paediatric epilepsy, as well as neuro-oncology, focusing on conditions with significant unmet medical need. By combining scientific rigor with a patient-centered mindset, Orphalan works closely with healthcare professionals and patient communities to improve standards of care.