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.
Information provided by (Responsible Party):
First received: June 6, 2013
Last updated: September 18, 2014
Last verified: September 2014
To evaluate the effects of a single dose of trientine in children ≥6 years and adult patients with Wilson's disease.
Drug: trientine dihydrochloride
||Endpoint Classification: Pharmacokinetics Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
||A Phase 1 Pharmacokinetic Profiling Study in Patients Receiving Trientine Dihydrochloride for the Treatment of Wilson's Disease.
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.
| Study Start Date:
| Study Completion Date:
| Primary Completion Date:
||March 2014 (Final data collection date for primary outcome measure)
Experimental: Trientine dihydrochloride
Drug: trientine dihydrochloride
Patients will take their normal prescribed dose (x1) of trientine dihydrochloride 300mg
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.
|Ages Eligible for Study:
||6 Years to 90 Years
|Genders Eligible for Study:
|Accepts Healthy Volunteers:
- 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.
- 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.
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Please refer to this study by its ClinicalTrials.gov identifier: NCT01874028
|Heidelberg, Germany, 69120 |
History of Changes
|Other Study ID Numbers:
|Study First Received:
||June 6, 2013
||September 18, 2014
||Germany: Federal Institute for Drugs and Medical Devices
Keywords provided by Univar BV:
Additional relevant MeSH terms:
ClinicalTrials.gov processed this record on April 27, 2016
Basal Ganglia Diseases
Brain Diseases, Metabolic
Brain Diseases, Metabolic, Inborn
Central Nervous System Diseases
Digestive System Diseases
Genetic Diseases, Inborn
Heredodegenerative Disorders, Nervous System
Metabolism, Inborn Errors
Metal Metabolism, Inborn Errors
Nervous System Diseases
Molecular Mechanisms of Pharmacological Action