Wilson's DiseasePhase 3TRADITiONALNCT07465718
Trientine Tetrahydrochloride Administered Once a Day for the First Line Treatment of Wilson's Disease Patients.
A Phase 3 study testing TETA 4HCl formulation and D-Penicillamine in people with Wilson's Disease, run by Orphalan at 10 sites in 4 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 8 years and older
- Conditions the study is for
- Wilson's Disease
- What is being tested?
- TETA 4HCl formulation and D-Penicillamine
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 38 participants
- Where
- 10 sitesin 4 countries
- Study dates
- 3 Sep 2026to Feb 2028
- Phase
- Phase 3
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 8 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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.
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- [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.
Who cannot join
- 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.
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- 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.
Trial locations and contacts
10 sites in 4 countries. Contact the study team or a site near you to ask about taking part.
Omar Kamlin contact
10 sites in 4 countries
United States3 sites, 3 recruiting
- Recruiting
University of Michigan Medical Centre
Ann Arbor, Michigan, 48109-2029
Fred Askari
Ashley Page, 734-998-9966, ashpage@med.umich.edu
- Recruiting
University of Colorado Anschutz School of Medicine
Denver, Colorado, 80045
Jon Rice
Mallory Truelson, 303-724-9143, Mallory.truelson@cuanschutz.edu
- Not yet recruiting
Yale University School of Medicine
New Haven, Connecticut, 06519
Michael Schilsky
Cameron Groshek, MS, CCRP, 203-737-7683, Cameron.groshek@yale.edu
China3 sites, 3 recruiting
- Recruiting
Beijing YouAn Hospital, Capital Medical University
Beijing, Beijing Municipality, 100000
Sujun Zheng, M.D., +86 15810716239, zhengsujun@ccmu.edu.cn
Shan Tang, M.D., +86 15711309515, tangshanccmu@163.com
- Recruiting
Beijing Friendship Hospital,Capital Medical University
Beijing, Beijing Municipality, 100050
Jidong Jia, M.D., +8613501378269, Jiabfhtrial@163.com
Hong Ma, M.D., +86 13651096128, mahongmd@aliyun.com
- Recruiting
The Second Affiliated Hospital Zhejiang University School of Medicine
Hangzhou, Zhengjiang Province, 310009
Zhiying Wu, M.D., +86 13646715353, zhiyingwu@zju.edu.cn
Roumin Wang, M.D., +86 15158723915, rouminwang@zju.edu.cn
Pakistan3 sites, 3 recruiting
- Not yet recruiting
PIMS Hospital
Islamabad, 44000
Nadia Waheed, drnadiasalman@gmail.com
- Not yet recruiting
Shifa International Hospital
Islamabad
Mohammad Salih, mohammad.salih@gmail.com
- Not yet recruiting
Children's Hospital & Institute of Child Health
Lahore, 54000
Huma Cheema, pedgilahore@gmail.com
Saudi Arabia1 site, 1 recruiting
- Not yet recruiting
King Faisal Specialist Hospital and Research Center
Riyadh, 11211
Saad AlGhamdi, mdisaad@kfshrc.edu.sa
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.
Read the 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
- Being tested
- TETA 4HCl formulation, D-Penicillamine
- Keywords
- trientine, randomized controlled trial, d-Penicillamine, Phase 3
- Medical subject headings
- Hepatolenticular Degeneration
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
TETA 4HCl formulationDrug
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.
D-PenicillamineDrug
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.
| Study group | Role | What they receive |
|---|---|---|
| TETA 4HCl formulation | Experimental | Participants are planned to receive TETA 4HCl for the 48-week post-randomization period. Receives: Drug: TETA 4HCl formulation |
| Standard of care d-Penicillamine (DPA) | Active comparator | Participants are planned to receive DPA for the 48-week post-randomization period. Receives: Drug: D-Penicillamine |
What is being measured
The primary outcome is the main question the study is designed to answer.
Absolute value of serum NCC at Week 48 assessed using the NCC-speciation assay (serum NCC-Sp)
Measured: Week 48
Secondary outcomes (5)
Serum NCC-Sp
Change from Baseline, within specified thresholds [>200, ≤ 200, ≤ 150, ≤ 100, ≤ 80, ≤ 50 μg/L and 50 to 80 μg/L]
Measured: Week 48
24-hour UCE
Absolute value, change from Baseline, within specified thresholds [< 100, <200, > 500, 150 to 500, and 200 to 500 μg/24h]
Measured: Week 48
Investigator's assessment of signs and symptoms
Assessment of of the signs and symptoms of WD using GAS for WD, comprising a 4-item Global Disability (Tier 1) and 10-item Neurological Assessment (Tier 2). Total score, Tier 1 score, Tier 2 score, each item score.
Measured: Week 48
Clinical Global Impression of Change
Investigator rating of change in the patients WD relating to the prior clinic visit on a 7-point scale by responding to the following statement "Please rate the change in the overall severity of the patients Wilson's disease compared to the previous clinic visit".
Measured: Week 48
Clinical stability
As assessed by Independent Adjudication Committee, including final determination of the endpoint of clinical stability (yes/no), including successful de-coppering (as applicable).
Measured: Week 48
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Mar 2026
- Study start3 Sep 2026
- Primary completionFeb 2028 (estimated)
- Study completionFeb 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Wilson's DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- Randomised, parallel group, open label.
- Masking
- None (open label)
- Enrolment
- 38 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Orphalan
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07465718
- Sponsor's protocol ID
- ORPH-131-017
- Acronym
- TRADITiONAL
- Status last verified
- Oct 2026
- First submitted
- 6 Mar 2026
- Last updated
- 15 Oct 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No