Wilson's Disease
Conditions
Keywords
Trientine, Pharmacokinetics
Brief summary
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.
Interventions
Patients will take their normal prescribed dose (x1) of trientine dihydrochloride 300mg
Sponsors
Aptiv Solutions
Univar BV
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
6 Years to 90 Years
Healthy volunteers
No
Inclusion criteria
1. Children ≥ 6 years and adult patients 2. Confirmed diagnosis of Wilson's disease by Leipzig-Score \>3 (Ferenci et al 2003) 3. Current treatment with trientine dihydrochloride 4. Signed informed consent including parental consent in patients ≤ 18 years 5. Agree to remain in the study site1 for the PK measurements period.
Exclusion criteria
1. Known clinically significant allergy or hypersensitivity to drugs that, in the opinion of the investigator, may affect the patient's safety 2. Have any clinically significant conditions that would interfere with the collection or interpretation of the study results or would compromise the patient's health 3. Women of child bearing potential who do not use contraceptives, breastfeeding, or pregnant women 4. Severe anaemia (haemoglobin \<9 mg/dL) 5. 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 6. Participation in any interventional clinical study at the same time or within the 4 weeks prior his study.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Pharmacokinetic measurements | 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. |
Countries
Germany
Outcome results
None listed