Wilson's DiseasePhase 1NCT01874028
A Phase 1 Study to Assess the Effects in the Body of a Single Dose of Trientine Dihydrochloride in Wilson's Disease Patients
A Phase 1 study testing trientine dihydrochloride in people with Wilson's Disease, run by Univar BV at 1 site in Germany.
Trial summary
To evaluate the effects of a single dose of trientine in children ≥6 years and adult patients with Wilson's disease. Read more about this study
- Who is it for?
- Ages 6 to 90 years
- Conditions the study is for
- Wilson's Disease
- What is being tested?
- trientine dihydrochloride
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- 1 siteGermany
- Study dates
- May 2013to Mar 2014
- Phase
- Phase 1
- 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 6 to 90 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Children ≥ 6 years and adult patients
- Confirmed diagnosis of Wilson's disease by Leipzig-Score >3 (Ferenci et al 2003)
- Current treatment with trientine dihydrochloride
- Signed informed consent including parental consent in patients ≤ 18 years
- Agree to remain in the study site1 for the PK measurements period.
Who cannot join
- Known clinically significant allergy or hypersensitivity to drugs that, in the opinion of the investigator, may affect the patient's safety
- Have any clinically significant conditions that would interfere with the collection or interpretation of the study results or would compromise the patient's health
- Women of child bearing potential who do not use contraceptives, breastfeeding, or pregnant women
- Severe anaemia (haemoglobin <9 mg/dL)
- In the judgment of the Investigator, is likely to be noncompliant or uncooperative during the study, or unable to cooperate because of a language problem or poor mental development
- Participation in any interventional clinical study at the same time or within the 4 weeks prior his study.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Phase 1 Pharmacokinetic Profiling Study in Patients Receiving Trientine Dihydrochloride for the Treatment of Wilson's Disease.
To evaluate the effects of a single dose of trientine in children ≥6 years and adult patients with Wilson's disease.
Read the detailed description
An open, single-dose study to determine the pharmacokinetics, and safety of oral administration of trientine dihydrochloride in both children and adult patients with Wilson's Disease. Blood samples will be taken pre-dose and at 10 time-points post-dose to investigate the pharmacokinetic profile of trientine dihydrochloride up to 12 hours after intake of study medication in 20 patients.
- Conditions
- Wilson's Disease
- Being tested
- trientine dihydrochloride
- Keywords
- Trientine, Pharmacokinetics
- 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
Patients will take their normal prescribed dose (x1) of trientine dihydrochloride 300mg
| Study group | Role | What they receive |
|---|---|---|
| Trientine dihydrochloride | Experimental | Not described Receives: Drug: trientine dihydrochloride |
What is being measured
The primary outcome is the main question the study is designed to answer.
Pharmacokinetic measurements
The main objective of the study is to evaluate the pharmacokinetics of a single dose of trientine dihydrochloride in children ≥6 years and adult patients with Wilson's disease by PK analysis.
Non-compartmental or model-independent methods will be used to derive pharmacokinetic parameters using individual patient plasma concentration-time data. Pharmacokinetic parameters will include the Cmax, Tmax, T1/2, and the concentration-time curved (AUC0-t) and AUCinf.
Measured: Evaluation of PK parameters will occur at 11 time points:Pre-dose), and at 30 minutes, 1, 1.5, 2, 3, 4, 5, 6, 8, and 12 hours post-dose
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Jun 2013 (estimated)
- Study startMay 2013
- Primary completionMar 2014
- Study completionMar 2014
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 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 20 participants (actual)
Sponsor and investigators
- Lead sponsor
- Univar BV
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Aptiv Solutions
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01874028
- Sponsor's protocol ID
- TR-001PK
- Status last verified
- Sep 2014
- First submitted
- 6 Jun 2013
- Last updated
- 19 Sep 2014
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 19 Sep 2014. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1 Pharmacokinetic Profiling Study in Patients Receiving Trientine Dihydrochloride for the Treatment of Wilson's Disease.
- ClinicalTrials.gov ID
- NCT01874028
- Acronym / sponsor ID
- TR-001PK
- Conditions
- Wilson's Disease
- Keywords
- Trientine, Pharmacokinetics
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 20 participants (actual)
- Lead sponsor
- Univar BVIndustry (pharma, biotech, devices)
- Collaborators
- Aptiv Solutions
- Study start
- May 2013
- Primary completion
- Mar 2014 (actual)
- Study completion
- Mar 2014 (actual)
- First posted
- 10 Jun 2013 (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.
To evaluate the effects of a single dose of trientine in children ≥6 years and adult patients with Wilson's disease.
Detailed description
An open, single-dose study to determine the pharmacokinetics, and safety of oral administration of trientine dihydrochloride in both children and adult patients with Wilson's Disease. Blood samples will be taken pre-dose and at 10 time-points post-dose to investigate the pharmacokinetic profile of trientine dihydrochloride up to 12 hours after intake of study medication in 20 patients.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Trientine dihydrochloride | Experimental | Drug: trientine dihydrochloride |
Who may qualify
- Ages
- 6 to 90 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (5 listed)
- Children ≥ 6 years and adult patients
- Confirmed diagnosis of Wilson's disease by Leipzig-Score >3 (Ferenci et al 2003)
- Current treatment with trientine dihydrochloride
- Signed informed consent including parental consent in patients ≤ 18 years
- Agree to remain in the study site1 for the PK measurements period.
Key exclusion criteria (6 listed)
- Known clinically significant allergy or hypersensitivity to drugs that, in the opinion of the investigator, may affect the patient's safety
- Have any clinically significant conditions that would interfere with the collection or interpretation of the study results or would compromise the patient's health
- Women of child bearing potential who do not use contraceptives, breastfeeding, or pregnant women
- Severe anaemia (haemoglobin <9 mg/dL)
- In the judgment of the Investigator, is likely to be noncompliant or uncooperative during the study, or unable to cooperate because of a language problem or poor mental development
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 dihydrochloridePatients will take their normal prescribed dose (x1) of trientine dihydrochloride 300mg | Drug | Trientine dihydrochloride |
Primary outcome
Pharmacokinetic measurements
Time frame: Evaluation of PK parameters will occur at 11 time points:Pre-dose), and at 30 minutes, 1, 1.5, 2, 3, 4, 5, 6, 8, and 12 hours post-dose
The main objective of the study is to evaluate the pharmacokinetics of a single dose of trientine dihydrochloride in children ≥6 years and adult patients with Wilson's disease by PK analysis. Non-compartmental or model-independent methods will be used to derive pharmacokinetic parameters using individual patient plasma concentration-time data. Pharmacokinetic parameters will include the Cmax, Tmax, T1/2, and the concentration-time curved (AUC0-t) and AUCinf.
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 19 Sep 2014.
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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.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing trientine dihydrochloride in people with Wilson's Disease, run by Univar BV at 1 site in Germany.
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 19 Sep 2014. 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 Germany. 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
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?
Pharmacokinetic measurements (time frame: Evaluation of PK parameters will occur at 11 time points:Pre-dose), and at 30 minutes, 1, 1.5, 2, 3, 4, 5, 6, 8, and 12 hours post-dose). 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 19 Sep 2014; AzTrials last refreshed its copy on 31 Oct 2026.