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Prospective Study to Characterize the Pharmacokinetics and Pharmacodynamics of Cufence (Trientine Dihydrochloride) in Wilson Disease Patients
EU CTIS ID: 2024-516431-27-00
What this study is testing
To characterize the Cufence dose – exposure – copper markers (24-hr urinary copper excretion (UCE), total serum copper (Cu), ceruloplasmin, non-ceruloplasmin bound copper (NCC)) relationships through population pharmacokinetic-pharmacodynamic (PKPD) modelling in Wilson’s disease patients and to evaluate the influence of patient characteristics on relevant model parameters, such as apparent clearance, apparent volume of distribution and drug potency.
- Therapeutic use (Phase IV)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- Patient (or a representative) must provide written, informed consent before the start of any study procedures.
- Male and female patient aged ≥ 5 years at time of consent.
- Diagnosis of WD previously determined by the physician based on a Leipzig score ≥ 4.
- Patient (≥ 18 years) has previously been treated with D-penicillamine for WD.
- Patient (< 18 years) has previously been treated with D-penicillamine or zinc for WD.
- For female patients of childbearing potential, a negative pregnancy test at the Screening visit and the Baseline visit is required. In addition, a highly effective method (failure rate <1%) of birth control must be used during the study which includes (but is not limited to) the following: - vasectomized partner (at least 6 months prior to dosing); - oral, patch, or injected contraceptives, or vaginal hormonal device (i.e. NuvaRing®), in use for at least 3 consecutive months prior to study dosing and throughout the study duration; - implanted or intrauterine contraceptives in use for at least 6 consecutive months prior to study dosing and throughout the study duration; - abstinence (must agree to use a highly effective method if they become sexually active during the study).
You likely can't join if
- Patient has evidence of uncontrolled liver disease, including but not limited to: a. New Wilson index (Dhawan Index) > 10 b. Alanine aminotransferase (ALT) > 5x upper limit of normal (ULN) c. Aspartate aminotransferase (AST) > 5x ULN d. Model for End-Stage Liver Disease (MELD) score > 13 (only applicable for patients that are ≥ 12 years of age) e. Acute liver failure f. Hepatic malignancy
- Uncontrolled neurological disease according to the judgement of the physician.
- Patient has severe anaemia defined as hemoglobin of < 9 g/dL.
- Patient has a known intolerance, allergy or sensitivity to trientine dihydrochloride, including any component of the study medication.
- Female patient is pregnant or lactating.
- Any patients who lack the capacity to consent, including the parent(s) of a paediatric patient.
See the full eligibility criteria
- Patient (or a representative) must provide written, informed consent before the start of any study procedures.
- Male and female patient aged ≥ 5 years at time of consent.
- Diagnosis of WD previously determined by the physician based on a Leipzig score ≥ 4.
- Patient (≥ 18 years) has previously been treated with D-penicillamine for WD.
- Patient (< 18 years) has previously been treated with D-penicillamine or zinc for WD.
- For female patients of childbearing potential, a negative pregnancy test at the Screening visit and the Baseline visit is required. In addition, a highly effective method (failure rate <1%) of birth control must be used during the study which includes (but is not limited to) the following: - vasectomized partner (at least 6 months prior to dosing); - oral, patch, or injected contraceptives, or vaginal hormonal device (i.e. NuvaRing®), in use for at least 3 consecutive months prior to study dosing and throughout the study duration; - implanted or intrauterine contraceptives in use for at least 6 consecutive months prior to study dosing and throughout the study duration; - abstinence (must agree to use a highly effective method if they become sexually active during the study).
- Patient is considered to be able to complete study requirements and attend the study visits, in the opinion of the investigator.
- Patient has evidence of uncontrolled liver disease, including but not limited to: a. New Wilson index (Dhawan Index) > 10 b. Alanine aminotransferase (ALT) > 5x upper limit of normal (ULN) c. Aspartate aminotransferase (AST) > 5x ULN d. Model for End-Stage Liver Disease (MELD) score > 13 (only applicable for patients that are ≥ 12 years of age) e. Acute liver failure f. Hepatic malignancy
- Uncontrolled neurological disease according to the judgement of the physician.
- Patient has severe anaemia defined as hemoglobin of < 9 g/dL.
- Patient has a known intolerance, allergy or sensitivity to trientine dihydrochloride, including any component of the study medication.
- Female patient is pregnant or lactating.
- Any patients who lack the capacity to consent, including the parent(s) of a paediatric patient.
The study team makes the final eligibility decision.
Where it's taking place
- United Kingdom
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 0-17 years, 65+ years, 18-64 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include United Kingdom. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.