Study to Assess Long-Term Outcomes of Trientine in Wilson Disease Patients Withdrawn From Therapy With d-Penicillamine

Univar

Status and phase

Unknown

Phase 4

Conditions

Wilson Disease

Treatments

Drug: trientine dihydrochloride

Study type

Interventional

Funder types

Industry

Identifiers

NCT02426905

UNV-TRI-002

Details and patient eligibility

About

A study to review Wilson disease patients who have previously been prescribed d- Penicillamine but were changed to trientine as treatment for their disease, and to follow them for a further 12 months.

Full description

A retrospective study to review Wilson disease patients who have previously been prescribed d- Penicillamine but were changed to trientine as treatment for their disease, and to follow them prospectively for a further 12 months.

Enrollment

90 estimated patients

Sex

All

Ages

1 to 90 years old

Volunteers

No Healthy Volunteers

Inclusion criteria

Patients aged 1 year to 90 years of age.
Physician established diagnosis of Wilson disease based on a Ferenci score > 3.
Documented treatment with d-Penicillamine, withdrawal of treatment with d- Penicillamine, followed by treatment with trientine for at least 6 months at date of informed consent.
Able/willing to provide written informed consent.
For enrolment in the prospective part, enrolment in the retrospective part of the study is required.

Exclusion criteria

Incomplete history of medication use for trientine from initial diagnosis to latest follow up.
Unavailable outcome data for hepatic and neurological course of disease at assessment time points.
Patients with acute liver failure and fulminant hepatic disease with fatal outcome.
Hypersensitivity to trientine and severe anaemia.