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 is currently recruiting participants. (see Contacts and Locations)
Verified December 2013 by Univar BV
Sponsor:
Collaborator:
Aptiv Solutions
Information provided by (Responsible Party):
Univar BV
ClinicalTrials.gov Identifier:
NCT01874028
First received: June 6, 2013
Last updated: December 10, 2013
Last verified: December 2013
  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: Endpoint Classification: Pharmacokinetics Study
Intervention Model: Single Group Assignment
Masking: 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 ] [ Designated as safety issue: No ]

    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.



Estimated Enrollment: 20
Study Start Date: May 2013
Estimated Study Completion Date: May 2014
Estimated Primary Completion Date: May 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

Ages Eligible for Study:   6 Years to 90 Years
Genders Eligible for Study:   Both
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
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01874028

Locations
Germany
Universitätsklinik Heidelberg Recruiting
Heidelberg, Germany, 69120
Contact: Karl-Heinz Weiss, MD    +49 6221 56 8700    Karl-Heinz.Weiss@med.uni-heidelberg.de   
Principal Investigator: Karl-Heinz Weiss, MD         
Sponsors and Collaborators
Univar BV
Aptiv Solutions
  More Information

No publications provided

Responsible Party: Univar BV
ClinicalTrials.gov Identifier: NCT01874028     History of Changes
Other Study ID Numbers: TR-001PK
Study First Received: June 6, 2013
Last Updated: December 10, 2013
Health Authority: Germany: Federal Institute for Drugs and Medical Devices

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
Metabolism, Inborn Errors
Metal Metabolism, Inborn Errors
Metabolic Diseases
Trientine
Chelating Agents
Molecular Mechanisms of Pharmacological Action
Pharmacologic Actions

ClinicalTrials.gov processed this record on July 29, 2014