NCT07010575

Patient Preference Study: Standard of Care Versus Once-daily Trientine Tetrahydrochloride

Decentralized Study to Assess Patient Treatment Preference Comparing Their Current Standard-of-care Wilson's Disease (WD) Treatment to a New Trientine (TETA) 4HCl Formulation.

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 10 (actual)
• Sponsor: Orphalan · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

Decentralized study to assess patient reported treatment satisfaction comparing their current standard-of-care Wilson's Disease (WD) treatment with a new once-daily Trientine (TETA) 4HCl formulation.

Detailed description

This is a single arm study where patients on Standard of Care maintenance therapy with a prescribed approved Wilson's Disease therapy administered at least twice daily will be screened for eligibility by the clinical research site either following referral from a participant identification centre (PIC) or following advertisements. An initial screening Patient Reported Outcome (PRO) assessment including the Treatment Satisfaction Questionnaire for Medication-9 (TSQM-9) and Morisky Medication Adherence Scale-8 (MMAS-8) will also be collected. Patients who meet all the study entry criteria will be switched to a new TETA 4HCl formulation for 28 days and will be monitored using Patient Reported Outcomes and specific posology questions held within a patient questionnaire pack and blood investigations. During this treatment phase (between Day 14 and Day 28 of dosing), each participant will be interviewed to collect qualitative data on disease and therapy. Patients will then be returned to their Standard of Care treatment and followed for a further 28 days continuing to be assessed using Patient Reported Outcomes and repeat blood investigations. The safety period will be finalised with an End of Study Assessment.

Conditions

• Wilson's Disease

Interventions

Type	Name	Description
DRUG	New TETA 4HCl Formulation	Individual patient doses will depend on the Standard of Care (SOC) therapy at study entry and guided by recommended dosing switch schedule outlined in the study protocol. The dose may subsequently be titrated based on clinical response per the investigator's judgement.
DRUG	Standard of Care	Patients will be returned to their approved Wilson's Disease SOC therapy (dose and frequency) at study entry as prescribed by their treating Wilson's Disease physician.

Timeline

• Start date: 2025-07-15
• Primary completion: 2025-12-18
• Completion: 2025-12-18
• First posted: 2025-06-08
• Last updated: 2026-04-06

Locations

1 site across 1 country: United Kingdom