Wilson DiseasePhase 4NCT02426905
Study to Assess Long-Term Outcomes of Trientine in Wilson Disease Patients Withdrawn From Therapy With d-Penicillamine
A Phase 4 study testing trientine dihydrochloride in people with Wilson Disease, run by Univar BV at 4 sites in 4 countries.
Trial summary
A study to review Wilson disease patients who have previously been prescribed d- Penicillamine but were changed to trientine as treatment for their disease, and to follow them for a further 12 months. Read more about this study
- Who is it for?
- Ages 1 year to 90 years
- Conditions the study is for
- Wilson Disease
- What is being tested?
- trientine dihydrochloride
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 90 participants
- Where
- 4 sitesin 4 countries
- Study dates
- Jan 2016to Jul 2018
- Phase
- Phase 4
- 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 1 year to 90 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Patients aged 1 year to 90 years of age.
- Physician established diagnosis of Wilson disease based on a Ferenci score > 3.
- Documented treatment with d-Penicillamine, withdrawal of treatment with d- Penicillamine, followed by treatment with trientine for at least 6 months at date of informed consent.
- Able/willing to provide written informed consent.
- For enrolment in the prospective part, enrolment in the retrospective part of the study is required.
Who cannot join
- Incomplete history of medication use for trientine from initial diagnosis to latest follow up.
- Unavailable outcome data for hepatic and neurological course of disease at assessment time points.
- Patients with acute liver failure and fulminant hepatic disease with fatal outcome.
- Hypersensitivity to trientine and severe anaemia.
Trial locations and contacts
4 sites in 4 countries.
4 sites in 4 countries
About this study
Official title: Multicentre, Retrospective and Prospective Study to Assess Long-Term Outcomes of Chelator-Based Treatment With Trientine in Wilson Disease Patients Withdrawn From Therapy With d-Penicillamine
A study to review Wilson disease patients who have previously been prescribed d- Penicillamine but were changed to trientine as treatment for their disease, and to follow them for a further 12 months.
Read the detailed description
A retrospective study to review Wilson disease patients who have previously been prescribed d- Penicillamine but were changed to trientine as treatment for their disease, and to follow them prospectively for a further 12 months.
- Conditions
- Wilson Disease
- Being tested
- trientine dihydrochloride
- Medical subject headings
- Hepatolenticular Degeneration
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
trientine dihydrochlorideDrug
A retrospective review of patients' medical records
| Study group | Role | What they receive |
|---|---|---|
| Retrospective | No intervention | Not described |
| Prospective | Other | Not described Receives: Drug: trientine dihydrochloride |
What is being measured
The primary outcome is the main question the study is designed to answer.
Clinical outcome specific to the retrospective part of the study
The clinical course of neurological and hepatic disease for each available time point after initiation of treatment (6, 12, 24, 36, and 48 months, and at the last available time point while taking second line trientine) will be scored (Investigator's score) based on neurological and hepatic status at the time of initiating trientine as: 1 = Unchanged 2 = Improved but not normal 3 = Improved to normal 4 = Asymptomatic over duration of therapy 5 = Worsened.
Measured: 48 months
Clinical outcome specific to the prospective part of the study
The clinical course of neurological and hepatic disease will be scored (Investigator's score) based on the status at 6 and 12 months after Baseline as: 1 = Unchanged 2 = Improved but not normal 3 = Improved to normal 4 = Asymptomatic over duration of therapy 5 = Worsened A patient will be counted as a responder if they have a rating of ≤4 at the 12 month visit for both the neurological and hepatic Investigator's score. They will be counted as a non-responder if they have a rating = 5 for one or both scores at the 12 month visit or if they were discontinued from the study for any reason prior to the 12 month visit.
Measured: 12 months
Secondary outcomes (2)
Safety Endpoint Applicable to both the Retrospective and Prospective Parts of the Study
All AEs related to trientine treatment, and AEs leading to discontinuation of trientine will be assessed at each available study time point.
Measured: Up to 60 months
Quality of Life Endpoints for the Prospective Part of the Study
The QoL questionnaires will be completed for each time point and data will be compared to baseline (prospective part) after 6 and 12 months
Measured: 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Apr 2015 (estimated)
- Study startJan 2016
- Primary completionMay 2018 (estimated)
- Study completionJul 2018 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Wilson 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 4
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 90 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Univar BV
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Karl-Heinz Weiss, MD, Principal investigator, Universitätsklinik Heidelberg
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02426905
- Sponsor's protocol ID
- UNV-TRI-002
- Status last verified
- Aug 2017
- First submitted
- 18 Sep 2014
- Last updated
- 23 Aug 2017
- Results posted
- No
- Data monitoring committee
- No
Publications
Mohr I, Kruse C, Aliane V, Weiss KH. Prospective Study to Assess Long-Term Outcomes of Chelator-Based Treatment With Trientine Dihydrochloride in Patients With Wilson Disease. JGH Open. 2025 Mar 17;9(3):e70114. doi: 10.1002/jgh3.70114. eCollection 2025 Mar.
PubMed 40104015Weiss KH, Kruse C, Manolaki N, Zuin M, Ferenci P, van Scheppingen D, Wijnberg L, de Koning CE, Dhawan A. Multicentre, retrospective study to assess long-term outcomes of chelator based treatment with trientine in Wilson disease patients withdrawn from therapy with d -penicillamine. Eur J Gastroenterol Hepatol. 2022 Sep 1;34(9):940-947. doi: 10.1097/MEG.0000000000002387. Epub 2022 Apr 29.
PubMed 35482910
Study at a glance
From the registry record, last updated by the sponsor on 23 Aug 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Multicentre, Retrospective and Prospective Study to Assess Long-Term Outcomes of Chelator-Based Treatment With Trientine in Wilson Disease Patients Withdrawn From Therapy With d-Penicillamine
- ClinicalTrials.gov ID
- NCT02426905
- Acronym / sponsor ID
- UNV-TRI-002
- Conditions
- Wilson Disease
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 90 participants (estimated)
- Lead sponsor
- Univar BVIndustry (pharma, biotech, devices)
- Study start
- Jan 2016
- Primary completion
- May 2018 (estimated)
- Study completion
- Jul 2018 (estimated)
- First posted
- 27 Apr 2015 (estimated)
- 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.
A study to review Wilson disease patients who have previously been prescribed d- Penicillamine but were changed to trientine as treatment for their disease, and to follow them for a further 12 months.
Detailed description
A retrospective study to review Wilson disease patients who have previously been prescribed d- Penicillamine but were changed to trientine as treatment for their disease, and to follow them prospectively for a further 12 months.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Retrospective | No intervention | – |
| Prospective | Other | Drug: trientine dihydrochloride |
Who may qualify
- Ages
- 1 year to 90 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (5 listed)
- Patients aged 1 year to 90 years of age.
- Physician established diagnosis of Wilson disease based on a Ferenci score > 3.
- Documented treatment with d-Penicillamine, withdrawal of treatment with d- Penicillamine, followed by treatment with trientine for at least 6 months at date of informed consent.
- Able/willing to provide written informed consent.
- For enrolment in the prospective part, enrolment in the retrospective part of the study is required.
Key exclusion criteria (4 listed)
- Incomplete history of medication use for trientine from initial diagnosis to latest follow up.
- Unavailable outcome data for hepatic and neurological course of disease at assessment time points.
- Patients with acute liver failure and fulminant hepatic disease with fatal outcome.
- Hypersensitivity to trientine and severe anaemia.
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 |
|---|---|---|
| trientine dihydrochlorideA retrospective review of patients' medical records | Drug | Prospective |
Primary outcome
Clinical outcome specific to the retrospective part of the study
Time frame: 48 months
The clinical course of neurological and hepatic disease for each available time point after initiation of treatment (6, 12, 24, 36, and 48 months, and at the last available time point while taking second line trientine) will be scored (Investigator's score) based on neurological and hepatic status at the time of initiating trientine as: 1 = Unchanged 2 = Improved but not normal 3 = Improved to normal 4 = Asymptomatic over duration of therapy 5 = Worsened.
Clinical outcome specific to the prospective part of the study
Time frame: 12 months
The clinical course of neurological and hepatic disease will be scored (Investigator's score) based on the status at 6 and 12 months after Baseline as: 1 = Unchanged 2 = Improved but not normal 3 = Improved to normal 4 = Asymptomatic over duration of therapy 5 = Worsened A patient will be counted as a responder if they have a rating of ≤4 at the 12 month visit for both the neurological and hepatic Investigator's score. They will be counted as a non-responder if they have a rating = 5 for one or both scores at the 12 month visit or if they were discontinued from the study for any reason prior to the 12 month visit.
2 secondary outcomes
Safety Endpoint Applicable to both the Retrospective and Prospective Parts of the Study
Time frame: Up to 60 months
Quality of Life Endpoints for the Prospective Part of the Study
Time frame: 12 months
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Aug 2017.
- Derived from this study: PubMed 40104015Mohr I, Kruse C, Aliane V, Weiss KH. Prospective Study to Assess Long-Term Outcomes of Chelator-Based Treatment With Trientine Dihydrochloride in Patients With Wilson Disease. JGH Open. 2025 Mar 17;9(3):e70114. doi: 10.1002/jgh3.70114. eCollection 2025 Mar.
- Derived from this study: PubMed 35482910Weiss KH, Kruse C, Manolaki N, Zuin M, Ferenci P, van Scheppingen D, Wijnberg L, de Koning CE, Dhawan A. Multicentre, retrospective study to assess long-term outcomes of chelator based treatment with trientine in Wilson disease patients withdrawn from therapy with d -penicillamine. Eur J Gastroenterol Hepatol. 2022 Sep 1;34(9):940-947. doi: 10.1097/MEG.0000000000002387. Epub 2022 Apr 29.
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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.
Karl-Heinz Weiss, MD principal investigator
Universitätsklinik Heidelberg
4 sites in 4 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing trientine dihydrochloride in people with Wilson Disease, run by Univar BV at 4 sites in 4 countries.
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 4 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: status not confirmed, as of the sponsor's last update on 23 Aug 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites in Germany, Greece, Italy, 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 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
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?
Clinical outcome specific to the retrospective part of the study (time frame: 48 months). 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?
On the ClinicalTrials.gov record for NCT02426905, which also lists 2 linked publications.
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 23 Aug 2017.