Study to Assess Long-Term Outcomes of Trientine in Wilson Disease Patients Withdrawn From Therapy With d-Penicillamine

Wilson Disease Clinical Trial

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Prospective Study to Assess Long-Term Outcomes of Chelator-Based Treatment With Trientine in Wilson Disease Patients Withdrawn From Therapy With d-Penicillamine

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT02426905
• Other study ID #: UNV-TRI-002
• Status: Active, not recruiting
• Phase: Phase 4
• First received: September 18, 2014
• Last updated: August 22, 2017
• Start date: January 2016
• Est. completion date: July 2018

Study information

• Verified date: August 2017
• Source: Univar BV
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 90
• Est. completion date: July 2018
• Est. primary completion date: May 2018
• Accepts healthy volunteers: No
• Gender: All
• Age group: 1 Year to 90 Years

Eligibility

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.

Related Conditions & MeSH terms

• Wilson Disease

Intervention

Drug:
• trientine dihydrochloride
A retrospective review of patients' medical records

Locations

Country	Name	City	State
Germany	Universitätsklinik Heidelberg	Heidelberg
Greece	"Aghia Sophia" Children's Hospital	Goudi
Italy	San Paolo Hospital UOC	Milan
United Kingdom	Kings College Hospital	London

Sponsors (1)

Lead Sponsor	Collaborator
Univar BV

Countries where clinical trial is conducted

Germany,  Greece,  Italy,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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.	48 months
Primary	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.	12 months
Secondary	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.	Up to 60 months
Secondary	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	12 months