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

Primary Purpose

Wilson's Disease

Status
Completed
Phase
Phase 1
Locations
Germany
Study Type
Interventional
Intervention
trientine dihydrochloride
Sponsored by
Univar BV
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Wilson's Disease focused on measuring Trientine, Pharmacokinetics

Eligibility Criteria

6 Years - 90 Years (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

  • Universitätsklinik Heidelberg

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Trientine dihydrochloride

Arm Description

Outcomes

Primary Outcome Measures

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.

Secondary Outcome Measures

Full Information

First Posted
June 6, 2013
Last Updated
September 18, 2014
Sponsor
Univar BV
Collaborators
Aptiv Solutions
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1. Study Identification

Unique Protocol Identification Number
NCT01874028
Brief Title
A Phase 1 Study to Assess the Effects in the Body of a Single Dose of Trientine Dihydrochloride in Wilson's Disease Patients
Official Title
A Phase 1 Pharmacokinetic Profiling Study in Patients Receiving Trientine Dihydrochloride for the Treatment of Wilson's Disease.
Study Type
Interventional

2. Study Status

Record Verification Date
September 2014
Overall Recruitment Status
Completed
Study Start Date
May 2013 (undefined)
Primary Completion Date
March 2014 (Actual)
Study Completion Date
March 2014 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Univar BV
Collaborators
Aptiv Solutions

4. Oversight

Data Monitoring Committee
No

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Wilson's Disease
Keywords
Trientine, Pharmacokinetics

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 1
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
20 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Trientine dihydrochloride
Arm Type
Experimental
Intervention Type
Drug
Intervention Name(s)
trientine dihydrochloride
Intervention Description
Patients will take their normal prescribed dose (x1) of trientine dihydrochloride 300mg
Primary Outcome Measure Information:
Title
Pharmacokinetic measurements
Description
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.
Time Frame
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

10. Eligibility

Sex
All
Minimum Age & Unit of Time
6 Years
Maximum Age & Unit of Time
90 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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.
Facility Information:
Facility Name
Universitätsklinik Heidelberg
City
Heidelberg
ZIP/Postal Code
69120
Country
Germany

12. IPD Sharing Statement

Learn more about this trial

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

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