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

Wilson's Disease Clinical Trial

Official title:

A Phase 1 Pharmacokinetic Profiling Study in Patients Receiving Trientine Dihydrochloride for the Treatment of Wilson's Disease.

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01874028
• Other study ID #: TR-001PK
• Status: Completed
• Phase: Phase 1
• First received: June 6, 2013
• Last updated: September 18, 2014
• Start date: May 2013
• Est. completion date: March 2014

Study information

• Verified date: September 2014
• Source: Univar BV
• Contact: n/a
• Is FDA regulated: No
• Health authority: Germany: Federal Institute for Drugs and Medical Devices
• Study type: Interventional

Clinical Trial Summary

To evaluate the effects of a single dose of trientine in children ≥6 years and adult patients with Wilson's disease.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 20
• Est. completion date: March 2014
• Est. primary completion date: March 2014
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 6 Years to 90 Years

Eligibility

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

Endpoint Classification: Pharmacokinetics Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Wilson's Disease

Intervention

Drug:
• trientine dihydrochloride
Patients will take their normal prescribed dose (x1) of trientine dihydrochloride 300mg

Locations

Country	Name	City	State
Germany	Universitätsklinik Heidelberg	Heidelberg

Sponsors (2)

Lead Sponsor	Collaborator
Univar BV	Aptiv Solutions

Country where clinical trial is conducted

Germany, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Pharmacokinetic measurements	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.	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	No