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

Phase 1

Completed

• Conditions: Wilson's Disease
• Interventions: Drug: trientine dihydrochloride

• Registration Number: NCT01874028
• Lead Sponsor: Univar BV

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.

Study Timeline

• Study Start: 2013-05
• Study First Submitted: 2013-06-06
• Study First Submitted QC: 2013-06-06
• Study First Posted: 2013-06-10
• Primary Completion: 2014-03
• Study Completion: 2014-03
• Status Verified: 2014-09
• Last Update Submitted: 2014-09-18
• Last Update Posted: 2014-09-19

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 20

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Trientine dihydrochloride	trientine dihydrochloride	-

Primary Outcome Measures

Name	Time	Method
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.

Trial Locations

Locations (1)

Universitätsklinik Heidelberg; 🇩🇪 Heidelberg, Germany