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NCT07465718From ClinicalTrials.govRecruiting

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

  • Wilson's Disease

At a glance

Phase
Phase 3
Study type
Interventional
Sponsor
Orphalan
Enrolment target
38
Started
3 September 2026
Main results due
February 2028
Study sites
10
Registry updated
24 September 2026

Can you take part?

  • Aged 8 years and over.
  • Open to any sex.
  • You need the condition being studied — healthy volunteers are not accepted.

These are the headline rules only. Every study has a longer list, and whether you are eligible is decided by the research team at the site — never by this page.

Read the full eligibility criteria
Inclusion Criteria:

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.
6. Adequate venous access to allow collection of required blood samples.
7. Willing to comply with low copper diet for the duration of the study.
8. Participant requires treatment for WD, in the opinion of the Investigator.
9. Participant is able to take the study medication as prescribed, in the opinion of the Investigator.

Exclusion Criteria:

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.
9. Estimated glomerular filtration rate (eGFR) of less than 30 mL/min/1.73m²
10. Nephritis or nephrotic syndrome, in the opinion of the Investigator.
11. Alanine aminotransferase greater than 5 times upper limit of normal (ULN).
12. Severe pulmonary disease requiring home nebulization and/or home oxygen therapy.
13. Clinically significant gastrointestinal bleed within past 6-months.
14. Neurological disease requiring either nasogastric feeding or intensive inpatient medical care.
15. Active or history of seizures requiring anti-epileptics within 6 months prior to informed consent.
16. Active infection with hepatitis B virus (positive hepatitis B surface antigen) or C virus or seropositivity for human immunodeficiency virus (HIV).
17. 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.
18. Female participants of childbearing potential, currently pregnant, currently nursing, or planning a pregnancy during study period.
19. Female participants of childbearing potential, unable or unwilling to use a reliable form of contraceptive throughout the study.
20. Male participants, unable or unwilling to use a reliable form of contraceptive throughout the study.
21. Participant is not willing to comply with the prohibited medication requirements for the study.
22. 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.

What this study is about

In the sponsor’s own words, from the registry.

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.

Study sites(10)

  • University of Colorado Anschutz School of Medicine

    Denver, Colorado, United States

    Recruiting

  • Yale University School of Medicine

    New Haven, Connecticut, United States

    Not yet recruiting

  • University of Michigan Medical Centre

    Ann Arbor, Michigan, United States

    Not yet recruiting

  • Beijing YouAn Hospital, Capital Medical University

    Beijing, Beijing Municipality, China

    Not yet recruiting

  • Beijing Friendship Hospital,Capital Medical University

    Beijing, Beijing Municipality, China

    Not yet recruiting

  • The Second Affiliated Hospital Zhejiang University School of Medicine

    Hangzhou, Zhengjiang Province, China

    Not yet recruiting

  • PIMS Hospital

    Islamabad, Pakistan

    Not yet recruiting

  • Shifa International Hospital

    Islamabad, Pakistan

    Not yet recruiting

  • Children's Hospital & Institute of Child Health

    Lahore, Pakistan

    Not yet recruiting

  • King Faisal Specialist Hospital and Research Center

    Riyadh, Saudi Arabia

    Not yet recruiting

Showing 10 of 10 sites. 1 of the 10 sites on this study is recruiting right now — a site can stop enrolling while the study as a whole is still open.

Source: ClinicalTrials.gov record NCT07465718. Trialion does not run this study, is not paid to refer anyone to it, and cannot enrol you. Eligibility is always decided by the research team at the site.

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