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

Univar

Status and phase: Completed; Phase 1

Conditions: Wilson's Disease

Treatments: Drug: trientine dihydrochloride

Study type: Interventional

Funder types: Industry

Identifiers

NCT01874028

TR-001PK

Details and patient eligibility

About

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

Full 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.

Enrollment: 20 patients

Sex: All

Ages: 6 to 90 years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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20 participants in 1 patient group

Trientine dihydrochloride

Experimental group

Treatment:

Drug: trientine dihydrochloride