NCT07465718

Trientine Tetrahydrochloride Administered Once a Day for the First Line Treatment of Wilson's Disease Patients.

• Status: Not Yet Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 38 (estimated)
• Sponsor: Orphalan · Industry
• Sex: All
• Age: 8 Years
• Healthy volunteers: Not accepted

Summary

The goal of this clinical trial is to learn if a new trientine tetrahydrochloride (TETA 4HCl) formulation administered once a day compared to d-Penicillamine (DPA) as a first line treatment for people living with Wilson's disease (WD) is effective and safe. The study is enrolling children aged 8 years and older weighing at least 55 lb (25 kg) and adults with a recent diagnosis of WD. People recently diagnosed with WD, may be eligible for the study if they have either not started copper chelating treatment (such as DPA or trientine) or have been taking zinc salts for less than 28 days. Participants will be randomly allocated (like tossing a coin) to receive either DPA or TETA 4HCL for 48 weeks. During this time period participants will have up to 12 visits for health checks and assessments including blood and urine testing. In addition, at some visits participants may be asked to complete questionnaires on treatment satisfaction, and overall well-being.

Detailed description

Wilson's disease (WD) is a rare, autosomal recessive genetic disorder of copper metabolism leading to progressive copper accumulation primarily in the liver and brain. Chelators are drugs that bind and remove copper from the body in the urine. d-Penicillamine (DPA) is currently the only approved first line chelator for the treatment of WD with trientine, an alternative copper chelator, only indicated for second line use. DPA is associated with numerous side effects which may lead to drug discontinuation in approximately 30% of people living with WD. Trientine is used following intolerance to DPA. All current WD therapies have to be taken multiple times a day. This can be challenging for people living with WD who have to take treatment every day and lifelong. A new formulation of trientine tetrahydrochloride (TETA 4HCl) has been developed to be administered once a day. Recently diagnosed consenting people with WD will enter a 28-day screening period (as required for confirmation of WD diagnosis, detailed neurological evaluation, and results of tests for eligibility) and a 48-week follow-up post-randomization. Symptomatic and asymptomatic WD patients 8 years of age and older with a body weight of at least 25 kg who are either naïve to all WD therapies (treatment-naïve) or naïve to chelator WD therapy (chelator-naïve) will be enrolled. Participants will receive treatment with either DPA or TETA 4HCl for the 48 week post-randomization period.

Conditions

• Wilson's Disease

Interventions

Type	Name	Description
DRUG	TETA 4HCl formulation	The new formulation of TETA 4HCl will be administered once a day. Each film-coated tablet is scored to enable halving, if required. Randomized participants are planned to receive TETA 4HCl for the 48-week post-randomization period.
DRUG	D-Penicillamine	Standard of care DPA is to be used, per the sites and treating physician's usual practice. To be administered in accordance with the product labelling and/or the institutions treatment practice guidelines. Randomised participants are planned to receive DPA for the 48-week post-randomization period.

Timeline

• Start date: 2026-05-01
• Primary completion: 2028-01-01
• Completion: 2028-01-01
• First posted: 2026-03-12
• Last updated: 2026-03-12

Regulatory

• FDA-regulated drug study