Wilson's DiseasePhase 2NCT07010575
Patient Preference Study: Standard of Care Versus Once-daily Trientine Tetrahydrochloride
A Phase 2 study testing New TETA 4HCl Formulation in people with Wilson's Disease, run by Orphalan at 1 site in United Kingdom.
Trial summary
Decentralized study to assess patient reported treatment satisfaction comparing their current standard-of-care Wilson's Disease (WD) treatment with a new once-daily Trientine (TETA) 4HCl formulation. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Wilson's Disease
- What is being tested?
- New TETA 4HCl Formulation
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 1 siteUnited Kingdom
- Study dates
- 15 Jul 2025to 18 Dec 2025
- Phase
- Phase 2
- Are there results?
- None posted in the registry
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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Willing and able to give informed consent for participation in the study.
- Proficient and fluent in English language speaker, writer and reader.
- Patients of any gender, aged 18 years or older as of signing the Informed Consent Form (ICF).
- Patients on current SOC WD maintenance treatment prescribed twice daily (or more frequently) and dose has been unchanged for at least 3-months.
- Women of childbearing potential and sexually active males must agree to adhere to a contraceptive method.
Who cannot join
- 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.
- Patients with severe anaemia (e.g., Haemoglobin <10 g/dL).
- Female participants who are pregnant (including a positive pregnancy test at Screening and on Day-1) or breastfeeding.
- Any contraindications as described in the current Investigator Brochure for TETA 4HCl.
- Subject receiving total daily dose of chelator as SOC greater or equal to 1200mg (trientine base or d-penicillamine).
- In the opinion of the investigator, the patient is likely to be a non-attender or uncooperative for routine clinical visits during the study.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Decentralized Study to Assess Patient Treatment Preference Comparing Their Current Standard-of-care Wilson's Disease (WD) Treatment to a New Trientine (TETA) 4HCl Formulation.
Decentralized study to assess patient reported treatment satisfaction comparing their current standard-of-care Wilson's Disease (WD) treatment with a new once-daily Trientine (TETA) 4HCl formulation.
Read the detailed description
This is a single arm study where patients on Standard of Care maintenance therapy with a prescribed approved Wilson's Disease therapy administered at least twice daily will be screened for eligibility by the clinical research site either following referral from a participant identification centre (PIC) or following advertisements. An initial screening Patient Reported Outcome (PRO) assessment including the Treatment Satisfaction Questionnaire for Medication-9 (TSQM-9) and Morisky Medication Adherence Scale-8 (MMAS-8) will also be collected.
Patients who meet all the study entry criteria will be switched to a new TETA 4HCl formulation for 28 days and will be monitored using Patient Reported Outcomes and specific posology questions held within a patient questionnaire pack and blood investigations. During this treatment phase (between Day 14 and Day 28 of dosing), each participant will be interviewed to collect qualitative data on disease and therapy. Patients will then be returned to their Standard of Care treatment and followed for a further 28 days continuing to be assessed using Patient Reported Outcomes and repeat blood investigations. The safety period will be finalised with an End of Study Assessment.
- Conditions
- Wilson's Disease
- Being tested
- New TETA 4HCl Formulation, Standard of Care
- Medical subject headings
- Hepatolenticular Degeneration
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
New TETA 4HCl FormulationDrug
Individual patient doses will depend on the Standard of Care (SOC) therapy at study entry and guided by recommended dosing switch schedule outlined in the study protocol. The dose may subsequently be titrated based on clinical response per the investigator's judgement.
Standard of CareDrug
Patients will be returned to their approved Wilson's Disease SOC therapy (dose and frequency) at study entry as prescribed by their treating Wilson's Disease physician.
| Study group | Role | What they receive |
|---|---|---|
| Once Daily Administration of new TETA 4HCl followed by return to standard of care | Experimental | The new formulation of TETA 4HCl will be administered once a day for 28 days. Each film-coated tablet contains 300 mg of trientine base.
Once completed patients will return to their standard of care Wilson's Disease treatment and be followed for a further 28 days. Receives: Drug: New TETA 4HCl Formulation; Drug: Standard of Care |
What is being measured
The primary outcome is the main question the study is designed to answer.
Assess and compare patient preference, convenience and satisfaction between current standard of care treatments for Wilson's Disease and the new TETA 4HCl formulation using patient reported outcome questionnaires.
Mean Treatment Satisfaction Questionnaire for Medication (TSQM-9) score over time including change from baseline by domain
Measured: From the screening assessment (-28 days to Day 1) to end of study at Week 8
Assess and compare patient preference, convenience and satisfaction between current standard of care treatments for Wilson's Disease and the new TETA 4HCl formulation using patient reported outcome questionnaires.
Incidence of categorical posology questions over time
Measured: From the screening assessment (-28 days to Day 1) to end of study at Week 8
Secondary outcomes (3)
Assess treatment adherence and tolerability of a new TETA 4HCl formulation.
Mean Morisky Medication Adherence Scale-8 (MMAS-8) score over time including change from baseline
Measured: From the screening assessment (-28 days to Day 1) to end of study at Week 8
Assess treatment adherence and tolerability of a new TETA 4HCl formulation.
Incidence of categorical laboratory safety data and adverse events (AE) assessments during the study.
Measured: From the screening assessment (-28 days to Day 1) to end of study at Week 8
Assess treatment adherence and tolerability of a new TETA 4HCl formulation.
Mean of continuous laboratory safety data during the study
Measured: From the screening assessment (-28 days to Day 1) to end of study at Week 8
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Jun 2025
- Study start15 Jul 2025
- Primary completion18 Dec 2025
- Study completion18 Dec 2025
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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 10 participants (actual)
Sponsor and investigators
- Lead sponsor
- Orphalan
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Anna Davidsson, Principal investigator, VCTC
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07010575
- Sponsor's protocol ID
- ORPH-131-015
- Status last verified
- Jan 2026
- First submitted
- 12 May 2025
- Last updated
- 6 Apr 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 6 Apr 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Decentralized Study to Assess Patient Treatment Preference Comparing Their Current Standard-of-care Wilson's Disease (WD) Treatment to a New Trientine (TETA) 4HCl Formulation.
- ClinicalTrials.gov ID
- NCT07010575
- Acronym / sponsor ID
- ORPH-131-015
- Conditions
- Wilson's Disease
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 10 participants (actual)
- Lead sponsor
- OrphalanIndustry (pharma, biotech, devices)
- Study start
- 15 Jul 2025 (actual)
- Primary completion
- 18 Dec 2025 (actual)
- Study completion
- 18 Dec 2025 (actual)
- First posted
- 8 Jun 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Decentralized study to assess patient reported treatment satisfaction comparing their current standard-of-care Wilson's Disease (WD) treatment with a new once-daily Trientine (TETA) 4HCl formulation.
Detailed description
This is a single arm study where patients on Standard of Care maintenance therapy with a prescribed approved Wilson's Disease therapy administered at least twice daily will be screened for eligibility by the clinical research site either following referral from a participant identification centre (PIC) or following advertisements. An initial screening Patient Reported Outcome (PRO) assessment including the Treatment Satisfaction Questionnaire for Medication-9 (TSQM-9) and Morisky Medication Adherence Scale-8 (MMAS-8) will also be collected.
Patients who meet all the study entry criteria will be switched to a new TETA 4HCl formulation for 28 days and will be monitored using Patient Reported Outcomes and specific posology questions held within a patient questionnaire pack and blood investigations. During this treatment phase (between Day 14 and Day 28 of dosing), each participant will be interviewed to collect qualitative data on disease and therapy. Patients will then be returned to their Standard of Care treatment and followed for a further 28 days continuing to be assessed using Patient Reported Outcomes and repeat blood investigations. The safety period will be finalised with an End of Study Assessment.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Once Daily Administration of new TETA 4HCl followed by return to standard of care | Experimental | Drug: New TETA 4HCl Formulation, Drug: Standard of CareThe new formulation of TETA 4HCl will be administered once a day for 28 days. Each film-coated tablet contains 300 mg of trientine base. Once completed patients will return to their standard of care Wilson's Disease treatment and be followed for a further 28 days. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- Willing and able to give informed consent for participation in the study.
- Proficient and fluent in English language speaker, writer and reader.
- Patients of any gender, aged 18 years or older as of signing the Informed Consent Form (ICF).
- Patients on current SOC WD maintenance treatment prescribed twice daily (or more frequently) and dose has been unchanged for at least 3-months.
- Women of childbearing potential and sexually active males must agree to adhere to a contraceptive method.
Key exclusion criteria (6 listed)
- 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.
- Patients with severe anaemia (e.g., Haemoglobin <10 g/dL).
- Female participants who are pregnant (including a positive pregnancy test at Screening and on Day-1) or breastfeeding.
- Any contraindications as described in the current Investigator Brochure for TETA 4HCl.
- Subject receiving total daily dose of chelator as SOC greater or equal to 1200mg (trientine base or d-penicillamine).
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| New TETA 4HCl FormulationIndividual patient doses will depend on the Standard of Care (SOC) therapy at study entry and guided by recommended dosing switch schedule outlined in the study protocol. The dose may subsequently be titrated based on clinical response per the investigator's judgement. | Drug | Once Daily Administration of new TETA 4HCl followed by return to standard of care |
| Standard of CarePatients will be returned to their approved Wilson's Disease SOC therapy (dose and frequency) at study entry as prescribed by their treating Wilson's Disease physician. | Drug | Once Daily Administration of new TETA 4HCl followed by return to standard of care |
Primary outcome
Assess and compare patient preference, convenience and satisfaction between current standard of care treatments for Wilson's Disease and the new TETA 4HCl formulation using patient reported outcome questionnaires.
Time frame: From the screening assessment (-28 days to Day 1) to end of study at Week 8
Mean Treatment Satisfaction Questionnaire for Medication (TSQM-9) score over time including change from baseline by domain
Assess and compare patient preference, convenience and satisfaction between current standard of care treatments for Wilson's Disease and the new TETA 4HCl formulation using patient reported outcome questionnaires.
Time frame: From the screening assessment (-28 days to Day 1) to end of study at Week 8
Incidence of categorical posology questions over time
3 secondary outcomes
Assess treatment adherence and tolerability of a new TETA 4HCl formulation.
Time frame: From the screening assessment (-28 days to Day 1) to end of study at Week 8
Assess treatment adherence and tolerability of a new TETA 4HCl formulation.
Time frame: From the screening assessment (-28 days to Day 1) to end of study at Week 8
Assess treatment adherence and tolerability of a new TETA 4HCl formulation.
Time frame: From the screening assessment (-28 days to Day 1) to end of study at Week 8
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 Apr 2026.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Anna Davidsson principal investigator
VCTC
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing New TETA 4HCl Formulation in people with Wilson's Disease, run by Orphalan at 1 site in United Kingdom.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 6 Apr 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Assess and compare patient preference, convenience and satisfaction between current standard of care treatments for Wilson's Disease and the new TETA 4HCl formulation using patient reported outcome questionnaires. (time frame: From the screening assessment (-28 days to Day 1) to end of study at Week 8). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 6 Apr 2026.