ClinicalTrials.gg
RecruitingNCT07465718TRADITiONALUpdated Oct 15, 2026

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

A Phase 3 interventional study of TETA 4HCl formulation and D-Penicillamine in Wilson's Disease, sponsored by Orphalan. Recruiting at 10 sites in 4 countries. Open to participants aged 8 Years and older. Per ClinicalTrials.gov, last updated 2026-10-15.

Sponsored by Orphalan · Phase 3, Interventional, and Treatment

From the registry’s dates

  • Started Sep 2026; still recruiting 2 months later.
Updated Oct 15, 2026Site recruiting status changedGo to Updates ↓
Phase
Phase 3
Study type
Interventional
Enrollment
38
Allocation
Randomized
Ages
8 Years and older
Sex
All
01

Study 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.

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.

02

Conditions studied

  • Wilson's Disease

Keywords

  • trientine
  • randomized controlled trial
  • d-Penicillamine
  • Phase 3
03

In context

Hepatolenticular Degeneration

80 studies on the registry are indexed under Hepatolenticular Degeneration; 31 are open to participants now.

This study's planned enrollment of 38 is above the median of 30 across 44 interventional studies indexed under Hepatolenticular Degeneration.

Browse Hepatolenticular Degeneration studies →

Lead sponsor

Orphalan is the lead sponsor of 6 studies on the registry; 2 are open to participants now.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
8 Years and older
Sexes eligible
All
Accepts healthy volunteers
No

Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.

Inclusion criteria

  1. Participant is aged 8 years or older and is willing and able to give 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

Exclusion Criteria:

  1. Any known 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. of less than or equal to 9 g/dL.
  9. Estimated (eGFR) of less than 30 mL/min/1.73m²
  10. Nephritis or nephrotic syndrome, in the opinion of the Investigator.
  11. greater than 5 times (ULN).
  12. Severe pulmonary disease requiring home nebulization and/or home oxygen therapy.
  13. 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 .
  16. Active infection with virus (positive hepatitis B surface antigen) or C virus or seropositivity for (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 , 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 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.
Terms in these criteria, in plain language(11)

Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.

Informed consent

Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.

You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.

Contraindication

A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.

Hemoglobin (Hb)

The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).

GFR and eGFR (kidney filtering)

GFR (glomerular filtration rate) is how much blood the kidneys filter each minute. It is usually estimated from a blood test — eGFR, by formulas such as CKD-EPI or MDRD — and some studies ask for it to be measured directly instead. Usually given in mL/min/1.73 m².

Check the method, the units and the minimum the study states. A value of 90 or above is in the normal-or-high range, but kidney damage can show in other tests too.

ALT and AST (liver enzymes)

Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.

Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.

ULN (upper limit of normal)

The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.

“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.

Clinically significant

Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.

Hepatitis B tests

Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.

Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.

HIV

The human immunodeficiency virus, which weakens the immune system.

Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.

Childbearing potential

Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).

Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.

Contraception

Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.

Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.

05

Study design

Phase
Phase 3
Primary purpose
Treatment
Allocation
Randomized
Intervention model
Parallel assignment
Masking
None (open label)
Enrollment
38 participants (estimated)

Study arms

  • Experimental
    TETA 4HCl formulation

    Participants are planned to receive TETA 4HCl for the 48-week post-randomization period.

    Drug: TETA 4HCl formulation

  • Active comparator
    Standard of care d-Penicillamine (DPA)

    Participants are planned to receive DPA for the 48-week post-randomization period.

    Drug: D-Penicillamine

Interventions

  • DrugTETA 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.

  • 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.

06

What researchers measure

Primary outcomes

  1. Absolute value of serum NCC at Week 48 assessed using the NCC-speciation assay (serum NCC-Sp)

    Time frame: Week 48

Secondary outcomes

  1. Serum NCC-Sp

    Change from Baseline, within specified thresholds \[\>200, ≤ 200, ≤ 150, ≤ 100, ≤ 80, ≤ 50 μg/L and 50 to 80 μg/L\]

    Time frame: Week 48

  2. 24-hour UCE

    Absolute value, change from Baseline, within specified thresholds \[\< 100, \<200, \> 500, 150 to 500, and 200 to 500 μg/24h\]

    Time frame: Week 48

  3. 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.

    Time frame: Week 48

  4. 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".

    Time frame: Week 48

  5. 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).

    Time frame: Week 48

07

Study locations

5 of 10 sites recruiting
08

References and documents

Individual participant data

Plan to share: No

No publications or documents are linked to this record.

09

Timeline

3 registry updates since Sep 25, 2026
ElapsedEstimatedEstimated, not yet reached

Started Sep 2026 (actual). Primary completion is estimated for Feb 2028, 17 months after the start.

How these figures are worked out, and which studies are left out of them.

Registered
Mar 6, 2026
First posted
Mar 12, 2026
StartActual
Sep 3, 2026
Primary completionEst.
Feb 2028
CompletionEst.
Feb 2028
Last update
Oct 15, 2026
What these dates mean
Start.
When the first participant enrolled; while estimated, when the study expects to open to recruitment.
Primary completion.
When the last participant was examined or treated to collect data for the study's main question, its primary outcome. A study that stopped early gives the date its data collection stopped.
Completion.
When the last participant's last visit took place, for every outcome, secondary ones and side effects included: the end of the study. Often later than primary completion, sometimes the same day.
Actual and Est.
Actual means the registry says the date has happened. Est. (estimated) is a date the sponsor plans or expects. It can still move, and the sponsor is expected to change it to actual once it has passed.
Sites
4 sites changed recruiting status (4 now recruiting)
Show 4 sites
  • University of Michigan Medical Centre · Ann Arbor, United StatesNot yet recruiting→Recruiting
  • Beijing YouAn Hospital, Capital Medical University · Beijing, ChinaNot yet recruiting→Recruiting
  • Beijing Friendship Hospital,Capital Medical University · Beijing, ChinaNot yet recruiting→Recruiting
  • The Second Affiliated Hospital Zhejiang University School of Medicine · Hangzhou, ChinaNot yet recruiting→Recruiting
across 2 updates, Oct 13, 2026 – Oct 15, 2026
Site contacts
Changed at 1 site. See the sites
Show the site
  • University of Colorado Anschutz School of Medicine · Denver, United StatesContact M•••@cuanschutz.edu · •••9143→M•••@cuanschutz.edu · •••9143
Oct 2, 2026
Show all 3 updates
  1. Oct 15, 2026
    3 sites changed recruiting status (3 now recruiting)
    Show 3 sites
    • Beijing YouAn Hospital, Capital Medical University · Beijing, ChinaNot yet recruiting→Recruiting
    • Beijing Friendship Hospital,Capital Medical University · Beijing, ChinaNot yet recruiting→Recruiting
    • The Second Affiliated Hospital Zhejiang University School of Medicine · Hangzhou, ChinaNot yet recruiting→Recruiting
    + 1 other change: verification date
  2. Oct 13, 2026
    University of Michigan Medical Centre is now Recruiting
  3. Oct 2, 2026
    Site contacts Changed at 1 site. See the sites
    Show the site
    • University of Colorado Anschutz School of Medicine · Denver, United StatesContact M•••@cuanschutz.edu · •••9143→M•••@cuanschutz.edu · •••9143

From the registry record's own update history. This site started tracking changes on Sep 25, 2026; for anything earlier, see the record history on ClinicalTrials.gov ↗

10

Registry details

Key details

Study ID
NCT07465718
Lead sponsor
Orphalan
Responsible party
Sponsor
First posted
Mar 12, 2026
Start date
Sep 3, 2026
Primary completion
Feb 2028 (estimated)
Completion
Feb 2028 (estimated)
Last update
Oct 15, 2026

Study contacts

Carla Bennett, Bsc. Hons
Contact
clinicaloperations@orphalan.com
+44 (0) 7918380893

Oversight

Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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