Clinical trial NCT02426905: Study to Assess Long-Term Outcomes of Trientine in Wilson Disease Patients Withdrawn From Therapy With d-Penicillamine
Official title: Multicentre, Retrospective and Prospective Study to Assess Long-Term Outcomes of Chelator-Based Treatment With Trientine in Wilson Disease Patients Withdrawn From Therapy With d-Penicillamine
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE4
Conditions: Wilson Disease
Lead sponsor: Univar BV
Sponsor class: INDUSTRY
First posted: 2015-04-27
Enrollment: 90
Sex: ALL
Minimum age (years): 1
Maximum age (years): 90
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: 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.

Detailed 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.

Eligibility criteria: 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.

Primary outcomes:
measure: Clinical outcome specific to the retrospective part of the study
timeFrame: 48 months
measure: Clinical outcome specific to the prospective part of the study
timeFrame: 12 months
Secondary outcomes:
measure: Safety Endpoint Applicable to both the Retrospective and Prospective Parts of the Study
timeFrame: Up to 60 months
measure: Quality of Life Endpoints for the Prospective Part of the Study
timeFrame: 12 months
Other outcomes:
Locations:
city: Heidelberg
country: Germany
facility: Universitätsklinik Heidelberg
city: Goudi
country: Greece
facility: "Aghia Sophia" Children's Hospital
city: Milan
country: Italy
facility: San Paolo Hospital UOC
city: London
country: United Kingdom
facility: Kings College Hospital
Study officials:
affiliation: Universitätsklinik Heidelberg
name: Karl-Heinz Weiss, MD
role: PRINCIPAL_INVESTIGATOR
References:
citation: Weiss KH, Kruse C, Manolaki N, Zuin M, Ferenci P, van Scheppingen D, Wijnberg L, de Koning CE, Dhawan A. Multicentre, retrospective study to assess long-term outcomes of chelator based treatment with trientine in Wilson disease patients withdrawn from therapy with d -penicillamine. Eur J Gastroenterol Hepatol. 2022 Sep 1;34(9):940-947. doi: 10.1097/MEG.0000000000002387. Epub 2022 Apr 29.