Study to Assess Long-Term Outcomes of Trientine in Wilson Disease Patients Withdrawn From Therapy With d-Penicillamine

Univar BV90 enrolled

Overview

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.

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.

Study Type

INTERVENTIONAL

Allocation

NON_RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Wilson Disease

Interventions

trientine dihydrochlorideDRUG

A retrospective review of patients' medical records

Eligibility

Sex: ALLMin age: 1 YearMax age: 90 Years

Medical Language ↔ Plain English

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.

Locations (4)

Universitätsklinik Heidelberg

Heidelberg, Germany

"Aghia Sophia" Children's Hospital

Goudi, Greece

San Paolo Hospital UOC

Milan, Italy

Kings College Hospital

London, United Kingdom

Outcomes

Primary Outcomes

Clinical outcome specific to the retrospective part of the study

The clinical course of neurological and hepatic disease for each available time point after initiation of treatment (6, 12, 24, 36, and 48 months, and at the last available time point while taking second line trientine) will be scored (Investigator's score) based on neurological and hepatic status at the time of initiating trientine as: 1 = Unchanged 2 = Improved but not normal 3 = Improved to normal 4 = Asymptomatic over duration of therapy 5 = Worsened.

Time frame: 48 months

Clinical outcome specific to the prospective part of the study

The clinical course of neurological and hepatic disease will be scored (Investigator's score) based on the status at 6 and 12 months after Baseline as: 1 = Unchanged 2 = Improved but not normal 3 = Improved to normal 4 = Asymptomatic over duration of therapy 5 = Worsened A patient will be counted as a responder if they have a rating of ≤4 at the 12 month visit for both the neurological and hepatic Investigator's score. They will be counted as a non-responder if they have a rating = 5 for one or both scores at the 12 month visit or if they were discontinued from the study for any reason prior to the 12 month visit.

Time frame: 12 months

Secondary Outcomes

Safety Endpoint Applicable to both the Retrospective and Prospective Parts of the Study

All AEs related to trientine treatment, and AEs leading to discontinuation of trientine will be assessed at each available study time point.

Time frame: Up to 60 months

Quality of Life Endpoints for the Prospective Part of the Study

The QoL questionnaires will be completed for each time point and data will be compared to baseline (prospective part) after 6 and 12 months

Time frame: 12 months

Data from ClinicalTrials.gov

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