#China #Hangzhou #Orphalan #Wilson Disease #TRADITiONAL Study

Orphalan Expands TRADITiONAL Study into China

Orphalan SA has recently announced the significant expansion of its pioneering TRADITiONAL Study into China, with the first participant officially randomised in this global effort. This Phase 3 clinical trial investigates an innovative once-daily formulation of trientine tetrahydrochloride as a first-line treatment for Wilson disease. The TRADITiONAL Study seeks to compare this investigational remedy against D-penicillamine, an established copper-chelating therapy, over the course of 48 weeks following randomisation.

The global study initially launched in the United States in August 2026 and progressively activated sites across the country, as well as at the King Faisal Specialist Hospital in Riyadh, Saudi Arabia. With the expansion to China, Orphalan aims to enhance their global presence in clinical research, addressing the unmet medical needs of people diagnosed with Wilson disease.

A crucial step in this expansion is the formal randomisation of the first participant at The Second Affiliated Hospital of Zhejiang University School of Medicine. Moreover, patient recruitment is currently underway at three major hospitals in China, where Beijing Friendship Hospital serves as the coordinating lead site. The activated study sites include:

• - Beijing Friendship Hospital, Capital Medical University (lead site)
• - The Second Affiliated Hospital of Zhejiang University School of Medicine (first randomised participant site)
• - Beijing You'an Hospital, Capital Medical University (active clinical site)

Omar Kamlin, Chief Medical Officer of Orphalan, expressed the importance of this milestone, stating that the activation of Chinese clinical centers and the first participant's randomisation indicate a major advancement in developing innovative therapies aimed at easing the treatment burden on those suffering from Wilson disease. Currently available therapies can often be complicated, and Orphalan's goal is to provide therapeutic options that better support daily management of this condition while effectively controlling copper levels in the body.

The TRADITiONAL Study is designed as a global, multicentre, and randomised trial for both symptomatic and asymptomatic Wilson disease patients aged eight and older. Participants may either be treatment-naïve or have previously been given zinc salt therapies for up to 28 days. Following an initial screening period of approximately four weeks, participants will undergo 48 weeks of treatment aimed at assessing efficacy, safety, tolerability, and patient-reported satisfaction regarding their treatment experiences.

Currently, the global enrolment for this study is active in the United States and China, with more international sites expected to join later this year. Interested individuals can find more detailed information about the TRADITiONAL Study on ClinicalTrials.gov under the identifier NCT07465718.

About Wilson Disease

Wilson disease is a rare inherited disorder resulting from mutations in the ATP7B gene, which disrupt the body's ability to eliminate excess copper. Consequently, copper accumulates in various organs and tissues—primarily the liver and brain—leading to potentially fatal hepatic, neurological, and psychiatric complications if left untreated. Lifelong management and treatment are crucial to prevent severe ramifications associated with the disease.

About Orphalan

Orphalan is a global pharmaceutical firm committed to innovating and commercialising treatments for rare diseases, including Wilson disease. The company's portfolio has recently broadened to encompass therapies for infantile epileptic spasms syndrome (IESS), a rare form of paediatric epilepsy, and they have established a focus on neuro-oncological conditions where significant patient needs are often unmet. By combining a rigorous scientific approach with a patient-centered strategy, Orphalan works closely with healthcare professionals alongside patient communities to enhance the quality of care provided to individuals living with rare disorders. To learn more, please visit www.orphalan.com.