Trientine Tetrahydrochloride Administered Once a Day for the First Line Treatment of Wilson's Disease Patients.
Eligible age
8+ yrs
Accepts
All genders
Locations
3 states
Healthy volunteers
No
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About this study
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.
Sponsor: Orphalan
You may qualify if…
- ✓ 1. Participant is aged 8 years or older and is willing and able to give informed consent for participation in the study, or by a parent/legally authorized representative (LAR) and assent obtained (in accordance with local regulations) for any participant less than the age of majority (e.g. less than 18 years of age, depending on local requirements).
- ✓ 2. Participant has a body weight of at least 25 kg at screening.
- ✓ 3. Participant has a diagnosis of WD, as defined by a Leipzig score of greater than or equal to 4. Note that historical test results can be used for the diagnosis.
- ✓ 4. Participant has either:
- ✓ 1. Received no prior prescribed therapy \[a\] for the treatment of WD (treatment-naïve), or
- ✓ 2. Received no prescribed chelator therapy \[a\] for the treatment of WD (chelator-naïve); zinc salts are permitted for no more than 28 days prior to the start of screening assessments, and these participants must be symptomatic.
- ✓ \[a\] prescribed therapy for WD refers to the authorized chelator treatments of trientine (TETA 2HCl or TETA 4HCl) and DPA, or zinc salts.
- ✓ 5. Able and willing to comply with study procedures and requirements, as described in the informed consent.
You may not qualify if…
- ✕ 1. Any known contraindications for treatment with DPA.
- ✕ 2. Any known contraindications for treatment with TETA 4HCl.
- ✕ 3. Unable to swallow tablets/capsules independently or considered high risk for aspiration, in the opinion of the Investigator
- ✕ 4. Acute liver failure (ALF) or at high risk of ALF, in the opinion of the Investigator.
- ✕ 5. Decompensated hepatic cirrhosis, in the opinion of the Investigator.
- ✕ 6. Participants 12 years or older at screening, Model for End stage Liver Disease (MELD) score of greater than or equal to 12.
- ✕ 7. Participants 8 to 11 years at screening, Model for Pediatric End stage Liver Disease (PELD) of greater than or equal to 10
- ✕ 8. Hemoglobin of less than or equal to 9 g/dL.
Where it's recruiting
Source: ClinicalTrials.gov · NCT07465718 · last updated 2026-09-24