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.
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 Phase
Phase 1
Study Type
Interventional
Primary Outcome
Pharmacokinetic measurements
Condition
Wilson's Disease
Intervention
trientine dihydrochloride
Study Arms / Comparison Groups
Trientine dihydrochloride
Description:
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Recruitment Status
Drug
Estimated Enrollment
20
Start Date
May 2013
Completion Date
March 2014
Primary Completion Date
March 2014
Eligibility Criteria
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.
Gender
All
Ages
6 Years - 90 Years
Accepts Healthy Volunteers
No
Contacts
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Location Countries
Germany
Location Countries
Germany
Administrative Informations
NCT ID
NCT01874028
Organization ID
TR-001PK
Responsible Party
Sponsor
Study Sponsor
Univar BV
Collaborators
Aptiv Solutions
Study Sponsor
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Verification Date
September 2014