A Phase 1 Study to Assess the Effects in the Body of a Single Dose of Trientine Dihydrochloride in Wilson's Disease Patients

NCT01874028 | Completed Phase 1

• 1 other identifier: TR-001PK
• Study Type: interventional
• Target: 20
• Locations: 1 country
• Sites: 1

Brief Summary

To evaluate the effects of a single dose of trientine in children ≥6 years and adult patients with Wilson's disease.

Conditions

Wilson's Disease

Keywords

Trientine; Pharmacokinetics

Outcome Measures

Primary Outcomes (1)

• Pharmacokinetic measurements

  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.

  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

Study Arms (1)

Trientine dihydrochloride

EXPERIMENTAL

Drug: trientine dihydrochloride

Interventions

trientine dihydrochloride DRUG

Patients will take their normal prescribed dose (x1) of trientine dihydrochloride 300mg

Trientine dihydrochloride

Eligibility Criteria

• Age: 6 Years - 90 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Univar BV: lead
• Aptiv Solutions: collaborator

Study Sites (1)

Universitätsklinik Heidelberg

Heidelberg, 69120, Germany

Location

MeSH Terms

Conditions

Hepatolenticular Degeneration

Interventions

Trientine

Condition Hierarchy (Ancestors)

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; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Metabolism, Inborn Errors; Metal Metabolism, Inborn Errors; Metabolic Diseases; Nutritional and Metabolic Diseases

Intervention Hierarchy (Ancestors)

Ethylenediamines; Diamines; Polyamines; Amines; Organic Chemicals

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 6, 2013
• First Posted: June 10, 2013
• Study Start: May 1, 2013
• Primary Completion: March 1, 2014
• Study Completion: March 1, 2014
• Last Updated: September 19, 2014

Record last verified: 2014-09

Locations

DE (1)