A Phase 1 Study to Assess the Effects in the Body of a Single Dose of Trientine Dihydrochloride in Wilson's Disease Patients

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.

Inclusion criteria

• 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.

Exclusion criteria

• 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.