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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

This study has been completed.
Sponsor:
ClinicalTrials.gov Identifier:
NCT01874028
First Posted: June 10, 2013
Last Update Posted: September 19, 2014
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
Collaborator:
Aptiv Solutions
Information provided by (Responsible Party):
Univar BV
  Purpose
To evaluate the effects of a single dose of trientine in children ≥6 years and adult patients with Wilson's disease.

Condition Intervention Phase
Wilson's Disease Drug: trientine dihydrochloride Phase 1

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase 1 Pharmacokinetic Profiling Study in Patients Receiving Trientine Dihydrochloride for the Treatment of Wilson's Disease.

Resource links provided by NLM:


Further study details as provided by Univar BV:

Primary Outcome Measures:
  • Pharmacokinetic measurements [ 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 ]

    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.



Enrollment: 20
Study Start Date: May 2013
Study Completion Date: March 2014
Primary Completion Date: March 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Trientine dihydrochloride Drug: trientine dihydrochloride
Patients will take their normal prescribed dose (x1) of trientine dihydrochloride 300mg

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.
  Eligibility

Information from the National Library of Medicine

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Ages Eligible for Study:   6 Years to 90 Years   (Child, Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.
  Contacts and Locations
Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01874028


Locations
Germany
Universitätsklinik Heidelberg
Heidelberg, Germany, 69120
Sponsors and Collaborators
Univar BV
Aptiv Solutions
  More Information

Responsible Party: Univar BV
ClinicalTrials.gov Identifier: NCT01874028     History of Changes
Other Study ID Numbers: TR-001PK
First Submitted: June 6, 2013
First Posted: June 10, 2013
Last Update Posted: September 19, 2014
Last Verified: September 2014

Keywords provided by Univar BV:
Trientine
Pharmacokinetics

Additional relevant MeSH terms:
Hepatolenticular Degeneration
Liver Diseases
Digestive System Diseases
Basal Ganglia Diseases
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Movement Disorders
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Genetic Diseases, Inborn
Metabolic Diseases
Metabolism, Inborn Errors
Metal Metabolism, Inborn Errors
Trientine
Chelating Agents
Sequestering Agents
Molecular Mechanisms of Pharmacological Action