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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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

, ,

Verification Date

September 2014