Prospective Study to Characterize the Pharmacokinetics and Pharmacodynamics of Cufence (Trientine Dihydrochloride) in Wilson Disease Patients

Ki vehet részt

• 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

Ki nem vehet részt

• Uncontrolled neurological disease according to the judgement of the physician
• Patient has a known intolerance, allergy or sensitivity to trientine dihydrochloride, including any component of the study medication
• 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
• Patient has severe anaemia defined as hemoglobin of < 9 g/dL
• Female patient is pregnant or lactating
• Any patients who lack the capacity to consent, including the parent(s) of a paediatric patient