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

Starting soon · Phase 3

Conditions studied: Wilson's Disease

In brief

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.

Key facts

Study ID: NCT07465718
Run by: Orphalan
People needed: 38
Starts: 2026-08-01
Expected to finish: 2028-02-01
Last updated by the study team: 2026-08-05

Who can join

Age: 8 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

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).
Participant has a body weight of at least 25 kg at screening.
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.
Participant has either:
Received no prior prescribed therapy [a] for the treatment of WD (treatment-naïve), or
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.
Able and willing to comply with study procedures and requirements, as described in the informed consent.
Adequate venous access to allow collection of required blood samples.
Willing to comply with low copper diet for the duration of the study.
Participant requires treatment for WD, in the opinion of the Investigator.
Participant is able to take the study medication as prescribed, in the opinion of the Investigator.

You may not qualify if…

Any known contraindications for treatment with DPA.
Any known contraindications for treatment with TETA 4HCl.
Unable to swallow tablets/capsules independently or considered high risk for aspiration, in the opinion of the Investigator
Acute liver failure (ALF) or at high risk of ALF, in the opinion of the Investigator.
Decompensated hepatic cirrhosis, in the opinion of the Investigator.
Participants 12 years or older at screening, Model for End stage Liver Disease (MELD) score of greater than or equal to 12.
Participants 8 to 11 years at screening, Model for Pediatric End stage Liver Disease (PELD) of greater than or equal to 10
Hemoglobin of less than or equal to 9 g/dL.
Estimated glomerular filtration rate (eGFR) of less than 30 mL/min/1.73m²
Nephritis or nephrotic syndrome, in the opinion of the Investigator.
Alanine aminotransferase greater than 5 times upper limit of normal (ULN).
Severe pulmonary disease requiring home nebulization and/or home oxygen therapy.
Clinically significant gastrointestinal bleed within past 6-months.
Neurological disease requiring either nasogastric feeding or intensive inpatient medical care.
Active or history of seizures requiring anti-epileptics within 6 months prior to informed consent.
Active infection with hepatitis B virus (positive hepatitis B surface antigen) or C virus or seropositivity for human immunodeficiency virus (HIV).
Major systemic disease or other illness that would, in the opinion of the Investigator, compromise patient safety or interfere with the collection or interpretation of the study results.
Female participants of childbearing potential, currently pregnant, currently nursing, or planning a pregnancy during study period.
Female participants of childbearing potential, unable or unwilling to use a reliable form of contraceptive throughout the study.
Male participants, unable or unwilling to use a reliable form of contraceptive throughout the study.
Participant is not willing to comply with the prohibited medication requirements for the study.
In the opinion of the Investigator, the participant is likely to be non-compliant or uncooperative for the required study visits or study assessments, or has any disease, disability, illness or abnormal laboratory values that could compromise patient safety or interfere with the collection or interpretation of study results.

Where it is running

University of Colorado Anschutz School of Medicine — Denver, Colorado, United States
Yale University School of Medicine — New Haven, Connecticut, United States
University of Michigan Medical Centre — Ann Arbor, Michigan, United States
Beijing YouAn Hospital, Capital Medical University — Beijing, Beijing Municipality, China
Beijing Friendship Hospital,Capital Medical University — Beijing, Beijing Municipality, China
The Second Affiliated Hospital Zhejiang University School of Medicine — Hangzhou, Zhengjiang Province, China
PIMS Hospital — Islamabad, Pakistan
Shifa International Hospital — Islamabad, Pakistan
Children's Hospital & Institute of Child Health — Lahore, Pakistan
King Faisal Specialist Hospital and Research Center — Riyadh, Saudi Arabia

Full record on ClinicalTrials.gov

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